[{"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":"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":"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":"Azitra","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"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":"U.S.A","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":"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-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":"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":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Precigen","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","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":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","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":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","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":"Beckley Psytech","sponsor":"Integrated","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","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":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Matrix Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"DENMARK","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":"Xentria","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","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":"Rallybio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Frontera Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","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":"U.S.A","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":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"BPGbio","sponsor":"Debra of America","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Calico Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"TWi Biotechnology","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","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":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"Satellos Bioscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"SAT-3247","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Satellos Bioscience \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Satellos Bioscience \/ Not Applicable"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"CHINA","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":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Aeovian Pharmaceuticals","sponsor":"Hevolution","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"AskBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Maze Therapeutics","sponsor":"Shionogi","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"AprilBio","sponsor":"Evommune","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","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":"KBio","sponsor":"National Institute of Allergy and Infectious Diseases","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","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":"Relief Therapeutics","sponsor":"SWK Funding LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"Hypochlorous Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"","sponsorNew":"Relief Therapeutics \/ SWK Funding LLC","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ SWK Funding LLC"},{"orgOrder":0,"company":"ImmuneSensor Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"IMSB301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ImmuneSensor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ImmuneSensor Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ImmuneSensor Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Azitra","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","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":"Azitra","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","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":"Relief Therapeutics","sponsor":"SWK Funding LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"Hypochlorous Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"","sponsorNew":"Relief Therapeutics \/ SWK Funding LLC","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ SWK Funding LLC"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"BCX17725","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Hypochlorous Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Relief Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ImmuneSensor Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"IMSB301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ImmuneSensor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ImmuneSensor Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ImmuneSensor Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ImmuneSensor Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"IMSB301","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ImmuneSensor Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ImmuneSensor Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ImmuneSensor Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Hypochlorous Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Relief Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Relief Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Relief Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"Dystrophin protein","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ABX1100","moa":"GYS1 enzyme","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Satellos Bioscience","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"SAT-3247","moa":"AAK1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Satellos Bioscience","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Satellos Bioscience \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Satellos Bioscience \/ Inapplicable"},{"orgOrder":0,"company":"Ribomic","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"RBM-007","moa":"FGFR 2\/3","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Ribomic","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"RBM-007","moa":"FGFR 2\/3","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Janus Henderson Investors","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Janus Henderson Investors","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Janus Henderson Investors"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ADX-324","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"ADARx Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ADARx Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series C Financing","leadProduct":"ADX-324","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"ADARx Pharmaceuticals \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"6","companyTruncated":"ADARx Pharmaceuticals \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Ascenta Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"ADX-324","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"ADARx Pharmaceuticals \/ Ascenta Capital","highestDevelopmentStatusID":"6","companyTruncated":"ADARx Pharmaceuticals \/ Ascenta Capital"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Johnson & Johnson Innovative Medicine","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","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 \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","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 \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Bioluminescence Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Series B Financing","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 \/ Bioluminescence Ventures","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Bioluminescence Ventures"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","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 \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aro Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ABX1100","moa":"Gys1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Aro Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aro Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Aro Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Goldman Sachs & Co.","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Public Offering","leadProduct":"ENTR-601-44","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Entrada Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Entrada Therapeutics \/ Goldman Sachs & Co.","highestDevelopmentStatusID":"6","companyTruncated":"Entrada Therapeutics \/ Goldman Sachs & Co."}]
Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Reset All
