[{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Partnership","leadProduct":"RP-L201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Lonza","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Lonza"},{"orgOrder":0,"company":"Ideaya Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"IDE196","moa":"PKC","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ideaya Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Ideaya Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Ideaya Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Olipudase alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Vaccine","year":"2020","type":"Not Applicable","leadProduct":"INO-3106","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Vaccine","year":"2020","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder for Concentrate for Solution for Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanifit","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"SNF472","moa":"Calcification","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Sanifit","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sanifit \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanifit \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"N-Acetyl-L-Leucine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"IntraBio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"LifeMax Laboratories","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"LM-030","moa":"KLK7\/ELA2","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"LifeMax Laboratories","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"LifeMax Laboratories \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"LifeMax Laboratories \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Azitra","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"ATR-12","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Azitra \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ Not Applicable"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Series B Financing","leadProduct":"OMNI nuclease","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Emendo Biotherapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"1","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-L201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"AlgoTherapeutix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Amitriptyline","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AlgoTherapeutix","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"AlgoTherapeutix \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"AlgoTherapeutix \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder for Concentrate for Solution for Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution For Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Canakinumab","moa":"IL-1 beta","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Carmine Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"RBCEV-based gene therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Takeda Pharmaceutical","amount2":0.90000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.90000000000000002,"dosageForm":"","sponsorNew":"Takeda Pharmaceutical \/ Carmine Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Takeda Pharmaceutical \/ Carmine Therapeutics"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Public Offering","leadProduct":"INZ-701","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.089999999999999997,"dosageForm":"","sponsorNew":"Inozyme Pharma \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-L301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Vaccine","year":"2020","type":"Not Applicable","leadProduct":"INO-3107","moa":"HPV6","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cerecor","sponsor":"Calidi Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"CERC-006","moa":"mTOR 1\/2 complex","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Cerecor","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Cerecor \/ Calidi Biotherapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Cerecor \/ Calidi Biotherapeutics"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Satralizumab-mwge","moa":"IL-6R alpha","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Oncopeptides","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Melphalan Flufenamide","moa":"DNA cross-linking","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Oncopeptides","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Oncopeptides \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Oncopeptides \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Novit LP","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Private Placement","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Novit LP","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Novit LP"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-L401","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Episalvan","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Raziel Therapeutics","sponsor":"Fosun Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Licensing Agreement","leadProduct":"RZL 012","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Raziel Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Injection","sponsorNew":"Raziel Therapeutics \/ Fosun Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Raziel Therapeutics \/ Fosun Pharma"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Cowen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Public Offering","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Cowen","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Cowen"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Gavorestat","moa":"Aldose reductase","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Applied Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Applied Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution For Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Avalglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution For Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TSHA-102","moa":"MECP2 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Xenon Pharmaceuticals Inc","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"NBI-921352","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Neurocrine Biosciences \/ Xenon Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Neurocrine Biosciences \/ Xenon Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-L201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"N-Acetyl-L-Leucine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"IntraBio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanifit","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"SNF472","moa":"Calcification","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Sanifit","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sanifit \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanifit \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Episalvan","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Mepolizumab","moa":"IL-5","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"GSK \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"GSK \/ Not Applicable"},{"orgOrder":0,"company":"XOMA","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Enzyme replacement therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"XOMA","amount2":0.14999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"","sponsorNew":"XOMA \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"XOMA \/ Chiesi Group"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Roth Capital Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Public Offering","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Roth Capital Partners","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Roth Capital Partners"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"RP-L401","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Rocket Pharmaceuticals \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ California Institute for Regenerative Medicine"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"intravenous infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"University of Oxford","sponsor":"Evox Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Exosome-based therapeutics","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"University of Oxford","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"University of Oxford \/ Evox Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"University of Oxford \/ Evox Therapeutics"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Tesofensine","moa":"Triple monoamine reuptake","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Saniona \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Series A Financing","leadProduct":"SAR439483","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Sofinnova Investments","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Sofinnova Investments"},{"orgOrder":0,"company":"Adrestia Therapeutics","sponsor":"GSK","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Adrestia Therapeutics","amount2":1.1499999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":1.1499999999999999,"dosageForm":"","sponsorNew":"Adrestia Therapeutics \/ GlaxoSmithKline","highestDevelopmentStatusID":"3","companyTruncated":"Adrestia Therapeutics \/ GlaxoSmithKline"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Longitude Capital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Series A Financing","leadProduct":"LX1004","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.089999999999999997,"dosageForm":"","sponsorNew":"Lexeo Therapeutics \/ Longitude Capital","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Longitude Capital"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2021","type":"Not Applicable","leadProduct":"Elamipretide","moa":"Microtubule associated protein 2","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2021","type":"Public Offering","leadProduct":"Elamipretide","moa":"Microtubule associated protein 2","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"Ensoma","sponsor":"5AM Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Series A Financing","leadProduct":"Engenious vectors","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ensoma","amount2":0.070000000000000007,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"Ensoma \/ 5AM Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Ensoma \/ 5AM Ventures"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Private Placement","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Novadip Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"NVD-003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Novadip Biosciences","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Novadip Biosciences \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Novadip Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Precigen","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Vaccine","year":"2021","type":"Not Applicable","leadProduct":"PRGN-2012","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Precigen","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Precigen \/ National Institutes of Health","highestDevelopmentStatusID":"6","companyTruncated":"Precigen \/ National Institutes of Health"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Anakinra","moa":"IL-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Swedish Orphan Biovitrum AB \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Antisense oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Vilobelimab","moa":"Complement 5a receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"InflaRx \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Abingworth","pharmaFlowCategory":"D","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2021","type":"Private Placement","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Abingworth","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Abingworth"},{"orgOrder":0,"company":"AGC Biologics","sponsor":"Rocket Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Partnership","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AGC Biologics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous infusion","sponsorNew":"AGC Biologics \/ Rocket Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"AGC Biologics \/ Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Allen Institute","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"AAV-based gene therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Allen Institute","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Allen Institute \/ BioMarin","highestDevelopmentStatusID":"1","companyTruncated":"Allen Institute \/ BioMarin"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Satralizumab-mwge","moa":"IL-6R alpha","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Canakinumab","moa":"IL-1 beta","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Series B Financing","leadProduct":"AAV-based gene therapies","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"","sponsorNew":"Affinia Therapeutics \/ EcoR1 Capital","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ EcoR1 Capital"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"SAN711","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Saniona \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Not Applicable"},{"orgOrder":0,"company":"Strongbridge Biopharma","sponsor":"Xeris Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Acquisition","leadProduct":"Dichlorphenamide","moa":"CA","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Strongbridge Biopharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Strongbridge Biopharma \/ Xeris Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Strongbridge Biopharma \/ Xeris Pharmaceuticals"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Disc medicine","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"F. Hoffmann-La Roche","amount2":0.20000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Oral","sponsorNew":"F. Hoffmann-La Roche \/ Disc Medicine","highestDevelopmentStatusID":"4","companyTruncated":"F. Hoffmann-La Roche \/ Disc Medicine"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"SAN711","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Saniona \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Oleogel-S10","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Episalvan","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Oleogel-S10","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Mitapivat","moa":"Pyruvate kinase","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Baymedica","sponsor":"InMed Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Acquisition","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Baymedica","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"Baymedica \/ InMed Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Baymedica \/ InMed Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Private Placement","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ H.C. Wainwright & Co.","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ H.C. Wainwright & Co."},{"orgOrder":0,"company":"Glycomine","sponsor":"ImmuneID","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Series B Financing","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Glycomine \/ ImmuneID","highestDevelopmentStatusID":"4","companyTruncated":"Glycomine \/ ImmuneID"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"SAN711","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Saniona \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"mRNA-based Molecular Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"LX1004","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Lexeo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Beckley Psytech","sponsor":"Integrated","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Series B Financing","leadProduct":"Psilocybine","moa":"5-HT2A receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Beckley Psytech","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Capsule","sponsorNew":"Beckley Psytech \/ Integrated","highestDevelopmentStatusID":"6","companyTruncated":"Beckley Psytech \/ Integrated"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2021","type":"Not Applicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"OrbiMed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Series B Financing","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.089999999999999997,"dosageForm":"Oral","sponsorNew":"Disc medicine \/ OrbiMed","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ OrbiMed"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Institute for Life Changing Medicines","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"mRNA-3351","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Moderna Therapeutics \/ Institute for Life Changing Medicines","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Institute for Life Changing Medicines"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"RD Fund","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Financing","leadProduct":"OPGx-001","moa":"LCA5 gene mutation","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ RD Fund","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ RD Fund"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Marzeptacog Alfa","moa":"Factor VIII","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Catalyst Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Catalyst Biosciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Catalyst Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Eladocagene Exuparvovec","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Liquid Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Retrotope","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Linoleic Acid","moa":"Lipid peroxidation","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Retrotope","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Retrotope \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Retrotope \/ Not Applicable"},{"orgOrder":0,"company":"Enzyvant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Allogeneic Processed Thymus Tissue-agdc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Enzyvant","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Enzyvant \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Enzyvant \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Acquisition","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Kedrion","amount2":0.02,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Lyophilized Powder For Injection","sponsorNew":"Kedrion \/ Kedrion Biopharma","highestDevelopmentStatusID":"12","companyTruncated":"Kedrion \/ Kedrion Biopharma"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Acquisition","leadProduct":"Plasminogen","moa":"Plasminogen","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Kedrion","amount2":0.02,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Lyophilized Powder for Injection","sponsorNew":"Kedrion \/ Kedrion","highestDevelopmentStatusID":"12","companyTruncated":"Kedrion \/ Kedrion"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Nicotinamide Riboside","moa":"NAD","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"ChromaDex, Inc","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ChromaDex, Inc \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"ChromaDex, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"AFT Pharmaceuticals","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AFT Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"AFT Pharmaceuticals \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"AFT Pharmaceuticals \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Genpharm","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Genpharm","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Genpharm \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Genpharm \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"InveniAI","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"InveniAI","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"InveniAI \/ Bristol Myers Squibb","highestDevelopmentStatusID":"2","companyTruncated":"InveniAI \/ Bristol Myers Squibb"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Mepolizumab","moa":"DNA synthesis","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"GSK","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"GSK \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"GSK \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Orpharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Orpharm","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Orpharm"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Matrix Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Financing","leadProduct":"MZE001","moa":"GYS1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0.19,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Matrix Capital Management","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Matrix Capital Management"},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV.7m8-L-opsin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Adverum Biotechnologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal","sponsorNew":"Adverum Biotechnologies \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Adverum Biotechnologies \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Oleogel-S10","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"MZE001","moa":"GYS1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Argonaut Manufacturing Services","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotides","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Argonaut Manufacturing Services","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Argonaut Manufacturing Services \/ n-Lorem Foundation","highestDevelopmentStatusID":"1","companyTruncated":"Argonaut Manufacturing Services \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"NHS England","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ NHS England","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ NHS England"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Liquid","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Haemnet","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Hemab Therapeutics \/ Haemnet","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Haemnet"},{"orgOrder":0,"company":"Akouos","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAVAnc80-antiVEGF Vector","moa":"VEGF","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Akouos","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracochlear","sponsorNew":"Akouos \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Akouos \/ Not Applicable"},{"orgOrder":0,"company":"Cytiva","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Cytiva","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cytiva \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Cytiva \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Erkim","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Erkim","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Erkim \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Erkim \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"MZE001","moa":"GYS1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"SAN711","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Saniona \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Saniona \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Neopharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Neopharm","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Neopharm"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"TargED Biopharmaceuticals","sponsor":"Andera Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Series A Financing","leadProduct":"Microlyse","moa":"ULVWF","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"TargED Biopharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"TargED Biopharmaceuticals \/ Andera Partners","highestDevelopmentStatusID":"4","companyTruncated":"TargED Biopharmaceuticals \/ Andera Partners"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"TRPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"FP-045","moa":"ALDH2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Foresee Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Foresee Pharmaceuticals \/ TRPharm","highestDevelopmentStatusID":"7","companyTruncated":"Foresee Pharmaceuticals \/ TRPharm"},{"orgOrder":0,"company":"Parexel Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Parexel Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Parexel Biotech \/ n-Lorem","highestDevelopmentStatusID":"2","companyTruncated":"Parexel Biotech \/ n-Lorem"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Oleogel-S10","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Erkim","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Erkim","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Erkim \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Erkim \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"InnoCare Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Orelabrutinib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"InnoCare Pharma","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"InnoCare Pharma \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"InnoCare Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"ANX005","moa":"C1q","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Annexon Biosciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Annexon Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Creyon Bio","sponsor":"DCVC Bio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Series A Financing","leadProduct":"Oligonucleotide-based Medicine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Creyon Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Creyon Bio \/ DCVC Bio","highestDevelopmentStatusID":"2","companyTruncated":"Creyon Bio \/ DCVC Bio"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"Fosdenopterin","moa":"Molybdenum cofactor synthesis","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Xentria","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"XTMAB-16","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Xentria","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Xentria \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Xentria \/ Not Applicable"},{"orgOrder":0,"company":"InflaRx","sponsor":"Innovaderm Research","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Vilobelimab","moa":"Complement 5a receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"InflaRx \/ Innovaderm Research","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Innovaderm Research"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Funding","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"n-Lorem Foundation \/ The Conrad Prebys Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ The Conrad Prebys Foundation"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"University of the Witwatersrand","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cyclo Therapeutics \/ University of the Witwatersrand","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ University of the Witwatersrand"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"AZ-3102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Azafaros \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Episalvan","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Abionyx Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Dipalmitoylphosphatidylglycerol","moa":"Apolipoprotein A-I","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Abionyx Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Abionyx Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Abionyx Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"TSHA-102","moa":"MECP2 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"INZ-701","moa":"PPi\/Adenosine","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Alpelisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Italian Medicines Agency","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orchard Therapeutics \/ Italian Medicines Agency","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Italian Medicines Agency"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"Aquaporin-4 protein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Aeterna Zentaris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Aeterna Zentaris \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Aeterna Zentaris \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Eladocagene Exuparvovec","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraputaminal Infusion","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Octantbio","sponsor":"Catalio Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Series B Financing","leadProduct":"Chaperon Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Octantbio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Octantbio \/ Catalio Capital Management","highestDevelopmentStatusID":"2","companyTruncated":"Octantbio \/ Catalio Capital Management"},{"orgOrder":0,"company":"Savara","sponsor":"Silicon Valley Bank","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Financing","leadProduct":"Molgramostim","moa":"GM-CSF","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation Spray","sponsorNew":"Savara \/ Silicon Valley Bank","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Silicon Valley Bank"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"MDL-101","moa":"Laminin alpha-1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Modalis Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Modalis Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Episalvan","moa":"Transient receptor potential channel stimulant","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"University of L\u2019Aquila","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ University of L\u2019Aquila","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ University of L\u2019Aquila"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"Sigma-1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Liquid","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"mRNA-based Therapeutic","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"C5 complement","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"TARA-002","moa":"Th1","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Acquisition","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ KemPharm","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ KemPharm"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ASC Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ASC Therapeutics \/ UMass Chan Medical School","highestDevelopmentStatusID":"4","companyTruncated":"ASC Therapeutics \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Affinia Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Capsida","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV9-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Capsida \/ Not Applicable","highestDevelopmentStatusID":"3","companyTruncated":"Capsida \/ Not Applicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Eladocagene Exuparvovec","moa":"AADC gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Marnetegragene Autotemcel","moa":"Gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"Enzyme","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"DepYmed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"DPM-1001","moa":"PTP1B","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"DepYmed","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"DepYmed \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"DepYmed \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Hong Kong Winhealth Pharma Group","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Licensing Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Hong Kong Winhealth Pharma Group","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Hong Kong Winhealth Pharma Group \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"5","companyTruncated":"Hong Kong Winhealth Pharma Group \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Cresence","sponsor":"Bioasis","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Acquisition","leadProduct":"CRES101","moa":"M2 polarization","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Cresence","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cresence \/ Bioasis","highestDevelopmentStatusID":"5","companyTruncated":"Cresence \/ Bioasis"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"RVB-001","moa":"Proteolysis","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"ResVita Bio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ResVita Bio \/ Not Applicable","highestDevelopmentStatusID":"1","companyTruncated":"ResVita Bio \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Venthera","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"BBP-681","moa":"PI3K","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"BridgeBio Pharma \/ Venthera","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Venthera"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Autoimmunity Modifying Biologicals","moa":"Alpha synuclein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Aeterna Zentaris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Aeterna Zentaris \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Aeterna Zentaris \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Savara \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Not Applicable"},{"orgOrder":0,"company":"Zenyaku Kogyo","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Rituximab","moa":"CD20","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Zenyaku Kogyo","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Zenyaku Kogyo \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"12","companyTruncated":"Zenyaku Kogyo \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"Generian Pharmaceuticals","sponsor":"Mitobridge","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Generian Pharmaceuticals","amount2":0.17999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0.17999999999999999,"dosageForm":"","sponsorNew":"Generian Pharmaceuticals \/ Mitobridge","highestDevelopmentStatusID":"3","companyTruncated":"Generian Pharmaceuticals \/ Mitobridge"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Leaps by Bayer","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Series B Financing","leadProduct":"SORT LNP-based DNAI1 mRNA Therapeutic","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.20000000000000001,"dosageForm":"Aerosol","sponsorNew":"ReCode Therapeutics \/ Leaps by Bayer","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Leaps by Bayer"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"New Enterprise Associates","pharmaFlowCategory":"D","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Private Placement","leadProduct":"Mavorixafor","moa":"CXCR4 ant","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ New Enterprise Associates","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ New Enterprise Associates"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Birch Bark Extract","moa":"Keratinocytes","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa","moa":"SMPD1","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"CY6463","moa":"Soluble guanylyl cyclase","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Vilobelimab","moa":"C5a","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"InflaRx \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Vilobelimab","moa":"Complement 5a receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"InflaRx \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Birch Bark Extract","moa":"Transient receptor potential channel","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Divestment","leadProduct":"Burosumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Ultragenyx Pharmaceutical","amount2":0.5,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.5,"dosageForm":"Injectable\/Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Kyowa Kirin","highestDevelopmentStatusID":"12","companyTruncated":"Ultragenyx Pharmaceutical \/ Kyowa Kirin"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2 Gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Endo Ventures Limited","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Endo Ventures Limited","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Endo Ventures Limited"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Eladocagene Exuparvovec","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Strm.Bio","sponsor":"National Center for Advancing Translational Sciences","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Funding","leadProduct":"EV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Strm.Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Strm.Bio \/ National Center for Advancing Translational Sciences","highestDevelopmentStatusID":"4","companyTruncated":"Strm.Bio \/ National Center for Advancing Translational Sciences"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"InMed Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"KP104","moa":"Complement system","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Kira Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kira Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Kira Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Acer Therapeutics \/ Relief Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Acer Therapeutics \/ Relief Therapeutics"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"OMS906","moa":"MASP-3","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Omeros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Omeros \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Omeros \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Divestment","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Novo Nordisk","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Novo Nordisk"},{"orgOrder":0,"company":"Epigenic Therapeutics","sponsor":"Morningside Venture Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Financing","leadProduct":"Epigenetic Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Epigenic Therapeutics","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"","sponsorNew":"Epigenic Therapeutics \/ Morningside Venture Capital","highestDevelopmentStatusID":"4","companyTruncated":"Epigenic Therapeutics \/ Morningside Venture Capital"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"ImmunAbs","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"IM-101","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"ImmunAbs","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ImmunAbs \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"ImmunAbs \/ Not Applicable"},{"orgOrder":0,"company":"Resilience","sponsor":"Mayo Clinic","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Resilience \/ Mayo Clinic","highestDevelopmentStatusID":"4","companyTruncated":"Resilience \/ Mayo Clinic"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutic","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutic","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutic"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Relief Therapeutics \/ Acer Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Relief Therapeutics \/ Acer Therapeutics"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Crovalimab","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Chugai Pharmaceutical \/ Roche","highestDevelopmentStatusID":"10","companyTruncated":"Chugai Pharmaceutical \/ Roche"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Equecabtagene Autoleucel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"IASO Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"IASO Bio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"IASO Bio \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"Splicing","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Eculizumab","moa":"Complement C5","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amgen Inc \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amgen Inc \/ Not Applicable"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Perelman School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"KP104","moa":"Complement system","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Kira Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kira Pharmaceuticals \/ Perelman School of Medicine","highestDevelopmentStatusID":"8","companyTruncated":"Kira Pharmaceuticals \/ Perelman School of Medicine"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Olipudase Alfa-rpcp","moa":"Acid sphingomyelinase","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"MZE001","moa":"GYS1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation Solution","sponsorNew":"Savara \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Private Placement","leadProduct":"Cannabinol","moa":"CB2\/CB1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Cream","sponsorNew":"InMed Pharmaceuticals \/ H.C. Wainwright & Co.","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ H.C. Wainwright & Co."},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Arctic Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Licensing Agreement","leadProduct":"N-acetylcysteine Amide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Arctic Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Nacuity Pharmaceuticals \/ Arctic Therapeutics"},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Monoclonal Antibody-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ ZabBio","highestDevelopmentStatusID":"2","companyTruncated":"KBio \/ ZabBio"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Baymedica","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Cannabinol","moa":"CB2\/CB1 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"InMed Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"InMed Pharmaceuticals \/ BayMedica","highestDevelopmentStatusID":"8","companyTruncated":"InMed Pharmaceuticals \/ BayMedica"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Danicopan","moa":"Factor D","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Danicopan","moa":"Factor D","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Mitapivat","moa":"PKR","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"APR-TD011","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Spray","sponsorNew":"Relief Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"KBio","sponsor":"Leaf Expression Systems","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"Monoclonal Antibody","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ Leaf Expression Systems","highestDevelopmentStatusID":"4","companyTruncated":"KBio \/ Leaf Expression Systems"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"BTIG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ BTIG","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ BTIG"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"ATSN-101","moa":"LCA1 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Saol therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Dichloroacetic Acid","moa":"PDK","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Saol therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Saol therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Saol therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"ARSA gene","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"WuXi Biologics","sponsor":"Toregem BioPharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"TRG035","moa":"USAG1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"WuXi Biologics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"WuXi Biologics \/ Toregem BioPharma","highestDevelopmentStatusID":"4","companyTruncated":"WuXi Biologics \/ Toregem BioPharma"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"Complement 5a receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"Complement 5a receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"INO-3107","moa":"HPV6\/HPV11","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avilar Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"LX1004","moa":"TPP1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Lexeo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"Complement 5a receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Alexion Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Financing","leadProduct":"TSHA-102","moa":"MECP2 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.050000000000000003,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ Astellas","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Astellas"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Taysha Gene Therapies","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Goldman Sachs & Co","highestDevelopmentStatusID":"8","companyTruncated":"Taysha Gene Therapies \/ Goldman Sachs & Co"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Sagard Healthcare Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Financing","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Sagard Healthcare Partners","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Sagard Healthcare Partners"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Oleogel-S10","moa":"Transient receptor potential channel","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"Factor B","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"C5 complement","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Disc medicine \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Not Applicable"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avilar Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"SVB Securities","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Public Offering","leadProduct":"Ganaxolone","moa":"GABA A receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0.059999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ SVB Securities","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ SVB Securities"},{"orgOrder":0,"company":"Rallybio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"RLYB116","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rallybio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Rallybio \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rallybio \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Eculizumab","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Tenacia Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Ganaxolone","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0.27000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.27000000000000002,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Tenacia Biotechnology","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Tenacia Biotechnology"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"EDIT-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Not Applicable"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"N-Acetyl-L-Leucine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Granules for Oral Suspension","sponsorNew":"IntraBio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"AbCellera","sponsor":"Rallybio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"Antibody-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"AbCellera","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AbCellera \/ Rallybio","highestDevelopmentStatusID":"3","companyTruncated":"AbCellera \/ Rallybio"},{"orgOrder":0,"company":"Altavant Sciences","sponsor":"Enzyvant Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Merger","leadProduct":"Allogeneic Processed Thymus Tissue-agdc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Altavant Sciences","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Altavant Sciences \/ Enzyvant Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Altavant Sciences \/ Enzyvant Therapeutics"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"KP104","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Kira Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Kira Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Kira Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV8.hLCA5","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Volanesorsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Genomic Biopharma","sponsor":"POIC e dintorni","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"RNA-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Genomic Biopharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Genomic Biopharma \/ POIC e dintorni","highestDevelopmentStatusID":"2","companyTruncated":"Genomic Biopharma \/ POIC e dintorni"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"N-Acetyl-L-Leucine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Granules for Oral Suspension","sponsorNew":"IntraBio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Not Applicable"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"NM3086","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"ANI Pharmaceuticals Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Levocarnitine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"ANI Pharmaceuticals Inc","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ANI Pharmaceuticals Inc \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"ANI Pharmaceuticals Inc \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Ethris","sponsor":"Cipla","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"ETH45","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ethris","amount2":0.02,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Inhalation","sponsorNew":"Ethris \/ Cipla","highestDevelopmentStatusID":"2","companyTruncated":"Ethris \/ Cipla"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Public Offering","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ H.C. Wainwright & Co.","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ H.C. Wainwright & Co."},{"orgOrder":0,"company":"Disc medicine","sponsor":"Gemini Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Merger","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.17999999999999999,"dosageForm":"","sponsorNew":"Disc medicine \/ Gemini Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Gemini Therapeutics"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Disc medicine \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Not Applicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Medable","sponsor":"Every Cure","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Medable","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Medable \/ Every Cure","highestDevelopmentStatusID":"1","companyTruncated":"Medable \/ Every Cure"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Public Offering","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ H.C. Wainwright & Co.","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ H.C. Wainwright & Co."},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"TAK-755","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Vilobelimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"InflaRx \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Not Applicable"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Chiesi Farmaceutici","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Oleogel-S10","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":1.48,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":1.48,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Chiesi Farmaceutici","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Chiesi Farmaceutici"},{"orgOrder":0,"company":"Orbus Therapeutics","sponsor":"Michigan State University","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Licensing Agreement","leadProduct":"Eflornithine Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Orbus Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Orbus Therapeutics \/ Michigan State University","highestDevelopmentStatusID":"1","companyTruncated":"Orbus Therapeutics \/ Michigan State University"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"FT-001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Frontera Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Frontera Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Frontera Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ANX005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Annexon Biosciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Annexon Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"GLM101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"GNT-003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"GenKOre","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"GenKOre","amount2":0.29999999999999999,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.29999999999999999,"dosageForm":"","sponsorNew":"GenKOre \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"GenKOre \/ Undisclosed"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"NM8074","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Precision Genetic Medicine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Beam Therapeutics","amount2":1.3500000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":1.3500000000000001,"dosageForm":"","sponsorNew":"Beam Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"Beam Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Code Bio","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Code Bio","amount2":2,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":2,"dosageForm":"","sponsorNew":"Code Bio \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Code Bio \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Amgen Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"IRELAND","productType":"Large molecule","year":"2022","type":"Acquisition","leadProduct":"Teprotumumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Horizon Therapeutics","amount2":27.800000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":27.800000000000001,"dosageForm":"Injectable\/Injection","sponsorNew":"Horizon Therapeutics \/ Amgen Inc","highestDevelopmentStatusID":"12","companyTruncated":"Horizon Therapeutics \/ Amgen Inc"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"NICHD","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Scenic Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Scenic Biotech \/ NICHD","highestDevelopmentStatusID":"2","companyTruncated":"Scenic Biotech \/ NICHD"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Octapharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Human Pooled Plasma","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Octapharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Octapharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Octapharma \/ Not Applicable"},{"orgOrder":0,"company":"Roche Diagnostics GmbH","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Roche Diagnostics GmbH","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Roche Diagnostics GmbH \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Roche Diagnostics GmbH \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ATSN-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Evozyne","amount2":0.40000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.40000000000000002,"dosageForm":"","sponsorNew":"Evozyne \/ Takeda","highestDevelopmentStatusID":"2","companyTruncated":"Evozyne \/ Takeda"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Private Placement","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Disc medicine \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"RLF-TD011","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Spray","sponsorNew":"Relief Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"MZE001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"New Therapies Council","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ New Therapies Council","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ New Therapies Council"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"MZE001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Endo International","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Collagenase Clostridium Histolyticum","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Endo International","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Endo International \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Endo International \/ Not Applicable"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Nobelpharma America","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Nobelpharma America \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Nobelpharma America \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Collaboration","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Disc medicine \/ National Institutes of Health","highestDevelopmentStatusID":"7","companyTruncated":"Disc medicine \/ National Institutes of Health"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"MZE001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"BPGbio","sponsor":"Debra of America","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Partnership","leadProduct":"BPM31510","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"BPGbio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"BPGbio \/ Debra of America","highestDevelopmentStatusID":"6","companyTruncated":"BPGbio \/ Debra of America"},{"orgOrder":0,"company":"Incyte Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Pemigatinib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Incyte Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Incyte Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Incyte Corporation \/ Not Applicable"},{"orgOrder":0,"company":"CSL Vifor","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Avacopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"CSL Vifor","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"CSL Vifor \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"CSL Vifor \/ Not Applicable"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Levothyroxine Sodium","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Zydus Lifesciences \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Zydus Lifesciences \/ Not Applicable"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"PANTHERx Rare Pharmacy \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"AB-1005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AskBio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Not Applicable"},{"orgOrder":0,"company":"Samsung Bioepis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Eculizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Samsung Bioepis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Samsung Bioepis \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Samsung Bioepis \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"RespireRx Pharmaceuticals","sponsor":"University College London","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Collaboration","leadProduct":"Ampakine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"RespireRx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"RespireRx Pharmaceuticals \/ University College London","highestDevelopmentStatusID":"4","companyTruncated":"RespireRx Pharmaceuticals \/ University College London"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Oleogel-S10","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amryt Pharma","amount2":1.48,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":1.48,"dosageForm":"Gel","sponsorNew":"Amryt Pharma \/ Chiesi Group","highestDevelopmentStatusID":"12","companyTruncated":"Amryt Pharma \/ Chiesi Group"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Amylyx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Amylyx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Amylyx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ATSN-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"ABBV-CLS-7262","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Calico Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Calico Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Calico Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Silk Road Therapies","sponsor":"Soligenix","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Agreement","leadProduct":"Pentoxifylline","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Silk Road Therapies","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Silk Road Therapies \/ Soligenix","highestDevelopmentStatusID":"8","companyTruncated":"Silk Road Therapies \/ Soligenix"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Licensing Agreement","leadProduct":"MZE001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0.75,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.75,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Sanofi","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Sanofi"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Divestment","leadProduct":"Zagociguat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"British Patient Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Series B Financing","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"","sponsorNew":"OMass Therapeutics \/ British Patient Capital","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ British Patient Capital"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Private Placement","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.070000000000000007,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Piper Sandler","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Piper Sandler"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Apadamtase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Life Edit Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Life Edit Therapeutics","amount2":1.9199999999999999,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":1.9199999999999999,"dosageForm":"","sponsorNew":"Life Edit Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Life Edit Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RP-L301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Innoviva","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Vaccine","year":"2023","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Innoviva","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Innoviva \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Innoviva \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Govorestat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Applied Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Applied Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Ganaxolone","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Marinus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Pharmanovia","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2023","type":"Licensing Agreement","leadProduct":"Elamipretide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Stealth Biotherapeutics \/ Pharmanovia","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Pharmanovia"},{"orgOrder":0,"company":"Pharming","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Divestment","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0.02,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Tablet","sponsorNew":"Pharming \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"TWi Biotechnology","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Tofacitinib Citrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"TWi Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"TWi Biotechnology \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"TWi Biotechnology \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Rafael Holdings","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Private Placement","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Rafael Holdings","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Rafael Holdings"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"NM3086","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Atlas Venture","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Series A Financing","leadProduct":"Usher1B-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AAVantgarde Bio","amount2":0.070000000000000007,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"AAVantgarde Bio \/ Atlas Venture","highestDevelopmentStatusID":"4","companyTruncated":"AAVantgarde Bio \/ Atlas Venture"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Public Offering","leadProduct":"Strain of Staphylococcus Epidermidis","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Ointment","sponsorNew":"Azitra \/ ThinkEquity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ ThinkEquity"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Mandos Health","sponsor":"INPDR","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Collaboration","leadProduct":"Adrabetadex","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Mandos Health","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mandos Health \/ INPDR","highestDevelopmentStatusID":"9","companyTruncated":"Mandos Health \/ INPDR"},{"orgOrder":0,"company":"Greenwich Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Greenwich Biosciences","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Greenwich Biosciences \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Greenwich Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Jazz Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Jazz Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Jazz Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Public Offering","leadProduct":"ATR-12","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"","sponsorNew":"Azitra \/ ThinkEquity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ ThinkEquity"},{"orgOrder":0,"company":"NephroDI Therapeutics","sponsor":"McQuade Center for Strategic Research and Development","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Agreement","leadProduct":"NDI-5001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"NephroDI Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NephroDI Therapeutics \/ McQuade Center for Strategic Research and Development","highestDevelopmentStatusID":"4","companyTruncated":"NephroDI Therapeutics \/ McQuade Center for Strategic Research and Development"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.16,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"CARE Pharma Shanghai Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Omoprubart","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ CARE Pharma Shanghai Ltd.","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ CARE Pharma Shanghai Ltd."},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"INZ-701","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Wacker Chemie AG","sponsor":"Caeregen Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"CTR-107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Wacker Chemie AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Wacker Chemie AG \/ Caeregen Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Wacker Chemie AG \/ Caeregen Therapeutics"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Voyager Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Voyager Therapeutics, Inc \/ Sangamo Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Voyager Therapeutics, Inc \/ Sangamo Therapeutics"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"N-Acetyl-L-Leucine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Granules for Oral Suspension","sponsorNew":"IntraBio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Guggenheim Securities, LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0.19,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Guggenheim Securities, LLC","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Guggenheim Securities, LLC"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Savara \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Not Applicable"},{"orgOrder":0,"company":"EDIRNA","sponsor":"Sirnaomics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Partnership","leadProduct":"RNA-editing Therapeutic","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"EDIRNA","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"EDIRNA \/ Sirnaomics","highestDevelopmentStatusID":"3","companyTruncated":"EDIRNA \/ Sirnaomics"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Fulcrum Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"CAMP4 Therapeutics","amount2":0.070000000000000007,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"CAMP4 Therapeutics \/ Fulcrum Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"CAMP4 Therapeutics \/ Fulcrum Therapeutics"},{"orgOrder":0,"company":"MyoPax","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Human Muscle Stem Cell Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"MyoPax \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Not Applicable"},{"orgOrder":0,"company":"Burjeel Holdings","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.A.E","productType":"Small molecule","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Burjeel Holdings","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Burjeel Holdings \/ BridgeBio Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Burjeel Holdings \/ BridgeBio Pharma"},{"orgOrder":0,"company":"Caeregen Therapeutics","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Funding","leadProduct":"CTR-107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"Caeregen Therapeutics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Caeregen Therapeutics \/ National Institutes of Health","highestDevelopmentStatusID":"5","companyTruncated":"Caeregen Therapeutics \/ National Institutes of Health"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"INZ-701","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ Not Applicable"},{"orgOrder":0,"company":"MyoPax","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Human Muscle Stem Cell Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"MyoPax \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Not Applicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Pfizer Inc","amount2":1,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":1,"dosageForm":"","sponsorNew":"Pfizer Inc \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Pfizer Inc \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"BofA Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Public Offering","leadProduct":"rhENPP1-Fc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Injectable\/Injection","sponsorNew":"Inozyme Pharma \/ BofA Securities","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ BofA Securities"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Disc medicine \/ Not Applicable"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"AnnJi Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"AJ201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avenue Therapeutics \/ AnnJi Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ AnnJi Pharmaceutical"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Invus","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Series A Financing","leadProduct":"Zagociguat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Invus","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Invus"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Tisento Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Divestment","leadProduct":"Zagociguat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Cyclerion Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Tablet","sponsorNew":"Cyclerion Therapeutics \/ Tisento Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Cyclerion Therapeutics \/ Tisento Therapeutics"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Divestment","leadProduct":"Sepofarsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0.16,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"Injectable\/Injection","sponsorNew":"ProQR Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"BofA Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Public Offering","leadProduct":"rhENPP1-Fc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inozyme Pharma","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Injectable\/Injection","sponsorNew":"Inozyme Pharma \/ BofA Securities","highestDevelopmentStatusID":"7","companyTruncated":"Inozyme Pharma \/ BofA Securities"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Rafael Holdings","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Private Placement","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Rafael Holdings","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Rafael Holdings"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Hercules Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Financing","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Hercules Capital","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Hercules Capital"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Public Offering","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Public Offering","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Genosera","sponsor":"Solve GNE","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Agreement","leadProduct":"GA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Genosera","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Genosera \/ Solve GNE","highestDevelopmentStatusID":"4","companyTruncated":"Genosera \/ Solve GNE"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ RA Capital Management","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ RA Capital Management"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Pozelimab-bbfg","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regeneron Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Regeneron Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"GordonMD Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ GordonMD Global Investments","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ GordonMD Global Investments"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Agreement","leadProduct":"Pozelimab-bbfg","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Orsini Specialty Pharmacy \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Daewoong Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Botulinum toxin type A","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Daewoong Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Daewoong Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Daewoong Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Genentech \/ Chugai Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Genentech \/ Chugai Pharmaceutical"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"OPGx-LCA5","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Public Offering","leadProduct":"RP-A501","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.17999999999999999,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ J.P. Morgan","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ J.P. Morgan"},{"orgOrder":0,"company":"AlgoTherapeutix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Amitriptyline Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AlgoTherapeutix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"AlgoTherapeutix \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"AlgoTherapeutix \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RP-A501","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Bioluminescence Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Series B Financing","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0.26000000000000001,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.26000000000000001,"dosageForm":"Solution for Nebulizer","sponsorNew":"ReCode Therapeutics \/ Bioluminescence Ventures","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Bioluminescence Ventures"},{"orgOrder":0,"company":"Airna","sponsor":"ARCH Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Financing","leadProduct":"RNA Editing Therapeutic","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Airna","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Airna \/ ARCH Venture Partners","highestDevelopmentStatusID":"3","companyTruncated":"Airna \/ ARCH Venture Partners"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Pfizer Inc","amount2":1,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":1,"dosageForm":"","sponsorNew":"Pfizer Inc \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Pfizer Inc \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Applied Molecular Transport","sponsor":"Cyclo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Merger","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Applied Molecular Transport","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Applied Molecular Transport \/ Cyclo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Applied Molecular Transport \/ Cyclo Therapeutics"},{"orgOrder":0,"company":"Viralgen","sponsor":"Elpida","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Partnership","leadProduct":"Serotype 9 AAV Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Viralgen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Viralgen \/ Elpida","highestDevelopmentStatusID":"7","companyTruncated":"Viralgen \/ Elpida"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"INZ-701","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Vigil Neuroscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"VGL101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Vigil Neuroscience","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Vigil Neuroscience \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Vigil Neuroscience \/ Not Applicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Chenodeoxycholic Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Satralizumab-mwge","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Marnetegragene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"St. Jude Children\u2019s Research Hospital","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Collaboration","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"St. Jude Children\u2019s Research Hospital","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"St. Jude Children\u2019s Research Hospital \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"St. Jude Children\u2019s Research Hospital \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Acquisition","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0.39000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.47999999999999998,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Kyowa Kirin","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Kyowa Kirin"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"GC1126A","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"GC Biopharma \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"GC Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"rhENPP1-Fc","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Inozyme Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Inozyme Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ANX005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Annexon Biosciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Annexon Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"American Regent","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Levocarnitine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"American Regent","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"American Regent \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"American Regent \/ Not Applicable"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"OrphanDC","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Pegcetacoplan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pint Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Pint Pharma \/ OrphanDC","highestDevelopmentStatusID":"12","companyTruncated":"Pint Pharma \/ OrphanDC"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Nedosiran","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Pre-Filled Syringe","sponsorNew":"Novo Nordisk \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novo Nordisk \/ Not Applicable"},{"orgOrder":0,"company":"AB Institut de Neuroci\u00e8ncies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AB Institut de Neuroci\u00e8ncies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AB Institut de Neuroci\u00e8ncies \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"AB Institut de Neuroci\u00e8ncies \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"UT Southwestern Medical Center \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"UT Southwestern Medical Center \/ Not Applicable"},{"orgOrder":0,"company":"Myto Therapeutics","sponsor":"University of California","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Monomethyl Fumarate Prodrug","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Myto Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Myto Therapeutics \/ University of California","highestDevelopmentStatusID":"4","companyTruncated":"Myto Therapeutics \/ University of California"},{"orgOrder":0,"company":"EA Pharma","sponsor":"Eisai","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Risedronate Sodium","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"EA Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"EA Pharma \/ Eisai","highestDevelopmentStatusID":"12","companyTruncated":"EA Pharma \/ Eisai"},{"orgOrder":0,"company":"OMERS Life Sciences","sponsor":"Xencor","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Large molecule","year":"2023","type":"Divestment","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"OMERS Life Sciences","amount2":0.22,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.22,"dosageForm":"Infusion","sponsorNew":"OMERS Life Sciences \/ Xencor","highestDevelopmentStatusID":"12","companyTruncated":"OMERS Life Sciences \/ Xencor"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Zagociguat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Tisento Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Tisento Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Tisento Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"PaxMedica","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Suramin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"IND Enabling","graph3":"PaxMedica","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"PaxMedica \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"PaxMedica \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ATSN-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Partnership","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Ligand Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Ligand Pharmaceuticals"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Divestment","leadProduct":"Sepofarsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0.19,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"ADAMTS13 Recombinant-krhn","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Vilobelimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"InflaRx","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"InflaRx \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"InflaRx \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Financing","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"AJ201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Avenue Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Bexicaserin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Longboard Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Longboard Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Longboard Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Vaccine","year":"2024","type":"Not Applicable","leadProduct":"INO-3107","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Inovio Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Inovio Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Inovio Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Mitapivat","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Agios Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Agios Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Agios Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV2-GDNF","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Bayer AG"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Ruxoprubart","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Dusquetide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Soligenix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Soligenix \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Soligenix \/ Not Applicable"},{"orgOrder":0,"company":"Drug Farm","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"DF-003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Drug Farm","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Drug Farm \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Drug Farm \/ Not Applicable"},{"orgOrder":0,"company":"LadRx","sponsor":"XOMA","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"LadRx","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"LadRx \/ XOMA","highestDevelopmentStatusID":"9","companyTruncated":"LadRx \/ XOMA"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Inhalation Solution","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"ATSN-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Blue Owl Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0.5,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.5,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Blue Owl Capital","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Blue Owl Capital"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Acquisition","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0.47999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.47999999999999998,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Kyowa Kirin","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Kyowa Kirin"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Beneluxa Initiative","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Agreement","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Beneluxa Initiative","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Beneluxa Initiative"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"NS-229","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NS Pharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Jazz Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Jazz Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Jazz Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Avicanna","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Avicanna","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"Avicanna \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avicanna \/ Undisclosed"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Avalglucosidase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Not Applicable"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Amylyx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Amylyx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Amylyx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Ruxoprubart","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ATR-12","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Ointment","sponsorNew":"Azitra \/ ThinkEquity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ ThinkEquity"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Public Offering","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0.25,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.25,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ J.P. Morgan"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Acoramidis Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"BridgeBio Pharma \/ Bayer AG","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Bayer AG"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Aeovian Pharmaceuticals","sponsor":"Hevolution","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"AV078","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aeovian Pharmaceuticals","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.050000000000000003,"dosageForm":"Solution","sponsorNew":"Aeovian Pharmaceuticals \/ Hevolution","highestDevelopmentStatusID":"6","companyTruncated":"Aeovian Pharmaceuticals \/ Hevolution"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Ravulizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"12","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"OPGx-LCA5","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Apadamtase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"N-Acetyl-L-Leucine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"IntraBio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Granules for Oral Suspension","sponsorNew":"IntraBio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"IntraBio \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Applied Molecular Transport","sponsor":"Cyclo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Merger","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Applied Molecular Transport","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Applied Molecular Transport \/ Cyclo Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Applied Molecular Transport \/ Cyclo Therapeutics"},{"orgOrder":0,"company":"Invitae","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Invitae","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Invitae \/ BridgeBio Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Invitae \/ BridgeBio Pharma"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ATR-12","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Azitra","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Ointment","sponsorNew":"Azitra \/ ThinkEquity","highestDevelopmentStatusID":"6","companyTruncated":"Azitra \/ ThinkEquity"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"HG004","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"HuidaGene Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"HuidaGene Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Praxis Precision Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Elsunersen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Praxis Precision Medicines","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Praxis Precision Medicines \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Praxis Precision Medicines \/ Not Applicable"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Palvella Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"AB-1005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"AskBio \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Atidarsagene Autotemcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"GLM101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Alembic Pharmaceuticals Limited","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Bimatoprost","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Alembic Pharmaceuticals Limited","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution\/Drops","sponsorNew":"Alembic Pharmaceuticals Limited \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alembic Pharmaceuticals Limited \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Anavex Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Anavex Life Sciences \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Dusquetide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Soligenix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Soligenix \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Soligenix \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"SKINVISIBLE PHARMACEUTICALS INC","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"SKINVISIBLE PHARMACEUTICALS INC","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"SKINVISIBLE PHARMACEUTICALS INC \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"SKINVISIBLE PHARMACEUTICALS INC \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"CBL-514","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Caliway Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Caliway Biopharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Caliway Biopharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"GLM101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Glycomine \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Glycomine \/ Not Applicable"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Ersodetug","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Harrow","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Triamcinolone Acetonide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0.13,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.13,"dosageForm":"Suspension","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Harrow","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Harrow"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Xenon Pharmaceuticals Inc","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"NBI-921352","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Neurocrine Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Neurocrine Biosciences \/ Xenon Pharmaceuticals Inc","highestDevelopmentStatusID":"8","companyTruncated":"Neurocrine Biosciences \/ Xenon Pharmaceuticals Inc"},{"orgOrder":0,"company":"Medicure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Pyridoxal Phosphate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Medicure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medicure \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Medicure \/ Not Applicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"LP659","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Longboard Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Longboard Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Longboard Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Dusquetide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Soligenix","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Soligenix \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Soligenix \/ Not Applicable"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Imlifidase","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Hansa Biopharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Hansa Biopharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Hansa Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"ATSN-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Atsena Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Iptacopan Hydrochloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"HMB-001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Hemab Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RP-L102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Mina Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Collaboration","leadProduct":"RNAa-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Mina Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Mina Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"3","companyTruncated":"Mina Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Elamipretide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Galectin Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Belapectin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Galectin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Galectin Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Galectin Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Amylyx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder In Sachet","sponsorNew":"Amylyx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Amylyx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Avenacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Desmopressin Acetate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Avenacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avenacy \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Avenacy \/ Not Applicable"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"NM5072","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"NovelMed Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"NovelMed Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Not Applicable"},{"orgOrder":0,"company":"Sentynl Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Fosdenopterin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Sentynl Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sentynl Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sentynl Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Citizens JMP","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Private Placement","leadProduct":"BB-301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.040000000000000001,"dosageForm":"Injection","sponsorNew":"Benitec Biopharma \/ Citizens JMP","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Citizens JMP"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AAV2-GDNF","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AskBio \/ Bayer AG","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Bayer AG"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Ipsen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Skyhawk Therapeutics","amount2":1.8,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":1.8,"dosageForm":"","sponsorNew":"Skyhawk Therapeutics \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Skyhawk Therapeutics \/ Ipsen"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Danicopan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Medivir","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"MIV-711","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Medivir","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Medivir \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Medivir \/ Not Applicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Hongene Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Partnership","leadProduct":"ASO-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Hongene Biotech","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Hongene Biotech \/ n-Lorem Foundation","highestDevelopmentStatusID":"3","companyTruncated":"Hongene Biotech \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"TSHA-102","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"Silk Road Therapies","sponsor":"Poolbeg Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Pentoxifylline","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Silk Road Therapies","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Gel","sponsorNew":"Silk Road Therapies \/ Poolbeg Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Silk Road Therapies \/ Poolbeg Pharma"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Divestment","leadProduct":"Mavorixafor","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"X4 Pharmaceuticals","amount2":0.11,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0.11,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"12","companyTruncated":"X4 Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Shionogi","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"MZE001","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Maze Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Maze Therapeutics \/ Shionogi","highestDevelopmentStatusID":"6","companyTruncated":"Maze Therapeutics \/ Shionogi"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Elamipretide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Pegcetacoplan","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Swedish Orphan Biovitrum AB \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Swedish Orphan Biovitrum AB \/ Not Applicable"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ANX005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Annexon Biosciences","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.13,"dosageForm":"Infusion","sponsorNew":"Annexon Biosciences \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Annexon Biosciences \/ J.P. Morgan"},{"orgOrder":0,"company":"BlueSphere Bio","sponsor":"National Cancer Institute","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"TCR T-cell Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"BlueSphere Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"BlueSphere Bio \/ National Cancer Institute","highestDevelopmentStatusID":"2","companyTruncated":"BlueSphere Bio \/ National Cancer Institute"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"AJ201","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Avenue Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Crovalimab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"F. Hoffmann-La Roche \/ Not Applicable"},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Dexamethasone Sodium Phosphate","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Quince Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Quince Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Quince Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AB-1005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AskBio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"AskBio \/ Not Applicable"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"MDL-101","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Modalis Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Modalis Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Neurotech Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Revakinagene Taroretcel","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Neurotech Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Neurotech Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Neurotech Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Astellas Pharma \/ UMass Chan Medical School","highestDevelopmentStatusID":"2","companyTruncated":"Astellas Pharma \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"ANX005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Annexon Biosciences","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.13,"dosageForm":"","sponsorNew":"Annexon Biosciences \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Annexon Biosciences \/ J.P. Morgan"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"ANX005","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Annexon Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Annexon Biosciences \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Annexon Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Apadamtase Alfa","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"AprilBio","sponsor":"Evommune","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Licensing Agreement","leadProduct":"APB-R3","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"AprilBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AprilBio \/ Evommune","highestDevelopmentStatusID":"6","companyTruncated":"AprilBio \/ Evommune"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Eculizumab","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Amgen Inc \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amgen Inc \/ Not Applicable"},{"orgOrder":0,"company":"KORU Medical Systems","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Agreement","leadProduct":"Enzyme Replacement Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"KORU Medical Systems","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KORU Medical Systems \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"KORU Medical Systems \/ Undisclosed"},{"orgOrder":0,"company":"KBio","sponsor":"National Institute of Allergy and Infectious Diseases","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"EV68-228-N","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ National Institute of Allergy and Infectious Diseases","highestDevelopmentStatusID":"6","companyTruncated":"KBio \/ National Institute of Allergy and Infectious Diseases"},{"orgOrder":0,"company":"Savara","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Savara \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Jefferies"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Savara \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Chenodeoxycholic Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Nicotinamide Riboside Chloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ChromaDex, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ChromaDex, Inc \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ChromaDex, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"University College Cork","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Quoin Pharmaceuticals \/ University College Cork","highestDevelopmentStatusID":"4","companyTruncated":"Quoin Pharmaceuticals \/ University College Cork"}]
Find Clinical Drug Pipelines for Rare Diseases and Disorders
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target