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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"}]

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                          01

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Nexviazyme (avalglucosidase alfa) is a USFDA approved GAA stimulator enzyme replacement therapy. It is being evaluated for the treatment of treatment-naïve Infantile-onset Pompe disease.

                          Brand Name : Nexviazyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 05, 2024

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Lead Product(s) : Olipudase Alfa-rpcp

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Xenpozyme, (olipudase Alfa-rpcp) hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase, an enzyme that allows for the breakdown of the lipid sphingomyelin.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 31, 2022

                          Lead Product(s) : Olipudase Alfa-rpcp

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Xenpozyme (Olipudase alfa), is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 28, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Recommendation based on positive results from two clinical trials in which Xenpozyme (olipudase alfa) provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 19, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Approval based on positive results of Xenpozyme® (olipudase alfa) from two separate clinical trials in children and adults demonstrating improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 28, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

                          Brand Name : GZ402665

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          December 06, 2021

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          07

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Relyvrio (taurursodiol and sodium phenylbutyrate) is a glutamine chelator small molecule drug candidate, being evaluated for the treatment of wolfram syndrome.

                          Brand Name : Relyvrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 17, 2024

                          Lead Product(s) : Sodium Phenylbutyrate,Tauroursodeoxycholic Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : MTP-131 (elamipretide) is an investigational mitochondrial protective agent , which is currently being evaluated for the treatment of patients with Barth syndrome.

                          Brand Name : MTP-131

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          October 11, 2024

                          Lead Product(s) : Elamipretide

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage clinical trial studies for the treatment of LAMA2-congenital muscular dystrophy.

                          Brand Name : MDL-101

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 30, 2024

                          Lead Product(s) : MDL-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Fi Europe 2024
                          Not Confirmed
                          Fi Europe 2024
                          Not Confirmed

                          Details : Molgramostim is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis.

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          September 27, 2024

                          Lead Product(s) : Molgramostim

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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