[{"orgOrder":0,"company":"Fenix Innovation Group","sponsor":"Neurotech International","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"AUSTRALIA","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"NTI164","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Fenix Innovation Group","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Fenix Innovation Group \/ Neurotech International","highestDevelopmentStatusID":"7","companyTruncated":"Fenix Innovation Group \/ Neurotech International"},{"orgOrder":0,"company":"Tern Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TTX-381","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Tern Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Tern Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Tern Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sixera Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWEDEN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"SXR1096","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Sixera Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"Sixera Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sixera Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Great Point Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Great Point Partners","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Great Point Partners"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular","sponsorNew":"Myrtelle \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Myrtelle \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Myrtelle \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Levetiracetam","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Myrtelle \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"MYR-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Myrtelle \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"MYR-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Myrtelle \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Inapplicable"},{"orgOrder":0,"company":"Myrtelle","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"rAAV-Olig001-ASPA","moa":"Aspartoacylase gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Myrtelle","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Myrtelle \/ Pfizer Inc","highestDevelopmentStatusID":"7","companyTruncated":"Myrtelle \/ Pfizer Inc"},{"orgOrder":0,"company":"Neoleukin Therapeutics","sponsor":"Neurogene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Merger","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neoleukin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neoleukin Therapeutics \/ Neurogene","highestDevelopmentStatusID":"7","companyTruncated":"Neoleukin Therapeutics \/ Neurogene"},{"orgOrder":0,"company":"Neoleukin Therapeutics","sponsor":"Neurogene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Merger","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neoleukin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neoleukin Therapeutics \/ Neurogene","highestDevelopmentStatusID":"7","companyTruncated":"Neoleukin Therapeutics \/ Neurogene"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"NGN-401","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Citizens JMP","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Private Placement","leadProduct":"BB-301","moa":"PABPN1","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":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Citizens JMP","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Citizens JMP"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Leerink Partners"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"BB-301","moa":"PABPN1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Benitec Biopharma","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"Intramuscular Injection","sponsorNew":"Benitec Biopharma \/ JMP Securities","highestDevelopmentStatusID":"7","companyTruncated":"Benitec Biopharma \/ JMP Securities"},{"orgOrder":0,"company":"Anupam Sehgal","sponsor":"Taysha Gene Therapies | GlycoNet","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"TSHA-101","moa":"Beta-hexosaminidase subunit alpha (HEXA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Anupam Sehgal","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Anupam Sehgal \/ Taysha Gene Therapies | GlycoNet","highestDevelopmentStatusID":"7","companyTruncated":"Anupam Sehgal \/ Taysha Gene Therapies | GlycoNet"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"GeneDx","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ GeneDx","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ GeneDx"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Silicon Valley Bank","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Financing","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Silicon Valley Bank","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Silicon Valley Bank"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Undisclosed"},{"orgOrder":0,"company":"Viralgen","sponsor":"Elpida","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Cell and Gene therapy","year":"2023","type":"Partnership","leadProduct":"Melpida","moa":"AP4M1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Viralgen","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Viralgen \/ Elpida","highestDevelopmentStatusID":"7","companyTruncated":"Viralgen \/ Elpida"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Financing","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.050000000000000003,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Astellas Pharma","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Astellas Pharma"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"GordonMD Global Investments","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ GordonMD Global Investments","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ GordonMD Global Investments"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ RA Capital Management","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ RA Capital Management"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Nationwide Children\u2019s Hospital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Financing","leadProduct":"CLN-301","moa":"CLN3","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Alcyone Therapeutics \/ Nationwide Children\u2019s Hospital","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Nationwide Children\u2019s Hospital"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"TSHA-102","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Taysha Gene Therapies","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Jefferies"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Acquisition","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2021","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Berge Minassian","sponsor":"Elpida Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"ION283","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Berge Minassian","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Berge Minassian \/ Elpida Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Berge Minassian \/ Elpida Therapeutics"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"TSHA-120","moa":"Gigaxonin (GAN)","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2020","type":"Inapplicable","leadProduct":"Betamethasone","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"GeneTx Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"TSHA-102","moa":"MECP2","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":"Intrathecal Injection","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"MyoPax","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Satori-01","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraurethral Injection","sponsorNew":"MyoPax \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Inapplicable"},{"orgOrder":0,"company":"MyoPax","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Satori-01","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intraurethral Injection","sponsorNew":"MyoPax \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Inapplicable"},{"orgOrder":0,"company":"Kanglin Biotechnology","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"KL003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Kanglin Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Kanglin Biotechnology \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Kanglin Biotechnology \/ Inapplicable"},{"orgOrder":0,"company":"Kanglin Biotechnology","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2024","type":"Series A Financing","leadProduct":"KL003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Kanglin Biotechnology","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Intravenous Injection","sponsorNew":"Kanglin Biotechnology \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Kanglin Biotechnology \/ Undisclosed"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Aeglea BioTherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Aeglea BioTherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Aeglea BioTherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Aeglea BioTherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"||KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Jasper Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Briquilimab","moa":"||KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Jasper Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Jasper Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Jasper Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Briquilimab","moa":"KIT","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Jasper Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Jasper Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Jasper Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"CARE Pharma Shanghai Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ CARE Pharma Shanghai Ltd.","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ CARE Pharma Shanghai Ltd."},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-301","moa":"CD34+","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Arbor Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"BBP-812","moa":"Aspartoacylase (ASPA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Resilience","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"BBP-812","moa":"Aspartoacylase (ASPA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Resilience \/ BridgeBio Pharma","highestDevelopmentStatusID":"7","companyTruncated":"Resilience \/ BridgeBio Pharma"},{"orgOrder":0,"company":"Genethon","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GNT-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Genethon \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Inapplicable"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Elixirgen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Partnership","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Lonza Group","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Lonza Group"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0.58999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.58999999999999997,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Public Offering","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Jefferies","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Jefferies"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Series C Financing","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0.070000000000000007,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Intravenous Infusion","sponsorNew":"Arbor Biotechnologies \/ Arch Ventures","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Arch Ventures"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Arbor Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Inapplicable"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Elixirgen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Elixirgen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"mRNA-3704","moa":"Methylmalonyl-CoA mutase (MMUT)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ImmuneCare Biopharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"IMC-002","moa":"CD20\/CD47","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ImmuneCare Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"ImmuneCare Biopharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ImmuneCare Biopharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"hLB-001","moa":"G6PC","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"BBP-812","moa":"Aspartoacylase (ASPA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Arbor Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"ABO-101","moa":"HS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Arbor Biotechnologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Arbor Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Arbor Biotechnologies \/ Inapplicable"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Pegtarviliase","moa":"Homocysteine","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Aeglea BioTherapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"LB-001","moa":"Methylmalonyl-CoA mutase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"LogicBio Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"LogicBio Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Omoprubart","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CANbridge Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"7","companyTruncated":"CANbridge Pharmaceuticals \/ WuXi Biologics"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EXG-001","moa":"||ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Elixirgen Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Wacker Chemie AG","sponsor":"Caeregen Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Other Large Molecule","year":"2023","type":"Collaboration","leadProduct":"CTR-107","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Wacker Chemie AG","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Wacker Chemie AG \/ Caeregen Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Wacker Chemie AG \/ Caeregen Therapeutics"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"HG004","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Ophthalmic Injection","sponsorNew":"HuidaGene Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"HuidaGene Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"HuidaGene Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"HG004","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"HuidaGene Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Ophthalmic Injection","sponsorNew":"HuidaGene Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"HuidaGene Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"FP-045","moa":"Mitochondrial aldehyde dehydrogenase (ALDH2)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Foresee Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Foresee Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Foresee Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"GRIN Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"TRPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Collaboration","leadProduct":"FP-045","moa":"Mitochondrial aldehyde dehydrogenase (ALDH2)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Foresee Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Foresee Pharmaceuticals \/ TRPharm","highestDevelopmentStatusID":"7","companyTruncated":"Foresee Pharmaceuticals \/ TRPharm"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"GRIN Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Hemab Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Hemab Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"AnnJi Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AnnJi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Liquid","sponsorNew":"AnnJi Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"AnnJi Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"AnnJi Pharmaceutical","sponsor":"Avenue Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Other Small Molecule","year":"2023","type":"Licensing Agreement","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AnnJi Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.25,"dosageForm":"Oral Liquid","sponsorNew":"AnnJi Pharmaceutical \/ Avenue Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"AnnJi Pharmaceutical \/ Avenue Therapeutics"},{"orgOrder":0,"company":"AnnJi Pharmaceutical","sponsor":"Avenue Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TAIWAN","productType":"Other Small Molecule","year":"2023","type":"Licensing Agreement","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AnnJi Pharmaceutical","amount2":0.25,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.25,"dosageForm":"Oral Liquid","sponsorNew":"AnnJi Pharmaceutical \/ Avenue Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"AnnJi Pharmaceutical \/ Avenue Therapeutics"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"NPI-001","moa":"Free radical","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Beyond Batten Disease Foundation","sponsor":"Theranexus","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Beyond Batten Disease Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Beyond Batten Disease Foundation \/ Theranexus","highestDevelopmentStatusID":"7","companyTruncated":"Beyond Batten Disease Foundation \/ Theranexus"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"AnnJi Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","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":"Oral Liquid","sponsorNew":"Avenue Therapeutics \/ AnnJi Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ AnnJi Pharmaceutical"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","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":"Oral Liquid","sponsorNew":"Avenue Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"AJ201","moa":"Nrf1\/2","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":"Oral Liquid","sponsorNew":"Avenue Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Maxim Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Public Offering","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Oral Liquid","sponsorNew":"Avenue Therapeutics \/ Maxim Group LLC","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Maxim Group LLC"},{"orgOrder":0,"company":"Avenue Therapeutics","sponsor":"Maxim Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Public Offering","leadProduct":"AJ201","moa":"Nrf1\/2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Avenue Therapeutics","amount2":0.01,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Oral Liquid","sponsorNew":"Avenue Therapeutics \/ Maxim Group LLC","highestDevelopmentStatusID":"7","companyTruncated":"Avenue Therapeutics \/ Maxim Group LLC"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Kabuki Syndrome Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Other Small Molecule","year":"2021","type":"Funding","leadProduct":"Vafidemstat","moa":"Lysine-specific histone demethylase 1 (LSD)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Oryzon Genomics \/ Kabuki Syndrome Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Oryzon Genomics \/ Kabuki Syndrome Foundation"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"N-Acetylcysteine Amide","moa":"Free radical","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nacuity Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Nacuity Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Nacuity Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor; GRIN1\/GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"GRIN Therapeutics \/ Avance Clinical","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Avance Clinical"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Haemnet","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"HMB-001","moa":"Factor VIIa","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Hemab Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Hemab Therapeutics \/ Haemnet","highestDevelopmentStatusID":"7","companyTruncated":"Hemab Therapeutics \/ Haemnet"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Kennedy Krieger Institute","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Other Small Molecule","year":"2022","type":"Collaboration","leadProduct":"Vafidemstat","moa":"Lysine-specific histone demethylase 1 (LSD)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Oryzon Genomics \/ Kennedy Krieger Institute","highestDevelopmentStatusID":"7","companyTruncated":"Oryzon Genomics \/ Kennedy Krieger Institute"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"DS-2325a","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Daiichi Sankyo \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Daiichi Sankyo \/ Inapplicable"},{"orgOrder":0,"company":"Nona Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Batoclimab","moa":"IgG receptor FcRn large subunit p51","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Nona Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Nona Biosciences \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Nona Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"OT-58","moa":"Cystathionine beta-synthase (CBS)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orphan Technologies \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Inapplicable"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Enzyme","year":"2020","type":"Acquisition","leadProduct":"OT-58","moa":"Cystathionine beta-synthase (CBS)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0.52000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.52000000000000002,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orphan Technologies \/ Travere Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Travere Therapeutics"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Enzyme","year":"2020","type":"Acquisition","leadProduct":"OT-58","moa":"Cystathionine beta-synthase (CBS)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0.52000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.52000000000000002,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orphan Technologies \/ Travere Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Travere Therapeutics"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Kyowa Kirin","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"KK8123","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous","sponsorNew":"Kyowa Kirin \/ Kyowa Kirin","highestDevelopmentStatusID":"7","companyTruncated":"Kyowa Kirin \/ Kyowa Kirin"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"DS-2325a","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Daiichi Sankyo \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Daiichi Sankyo \/ Inapplicable"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"DS-2325a","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Daiichi Sankyo \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Daiichi Sankyo \/ Inapplicable"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ITALY","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"AAVB-081","moa":"MYO7A","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"AAVantgarde Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"AAVantgarde Bio \/ Inapplicable"},{"orgOrder":0,"company":"OBiO Technology","sponsor":"Chinagene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"ZVS101e","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"OBiO Technology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"OBiO Technology \/ Chinagene","highestDevelopmentStatusID":"7","companyTruncated":"OBiO Technology \/ Chinagene"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"||LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"ATSN-101","moa":"||LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ATSN-101","moa":"LCA1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"SAR439483","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Series A Financing","leadProduct":"SAR439483","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Sofinnova Investments","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Sofinnova Investments"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"AbbVie Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Termination","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ AbbVie Inc","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ AbbVie Inc"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AGN-151587","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"EDIT-101","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Editas Medicine","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Editas Medicine \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Editas Medicine \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"OPGx-001","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"OPGx-LCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Perceptive Advisors","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"NGGT Suzhou Biotechnology","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"NGGT001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"NGGT Suzhou Biotechnology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"NGGT Suzhou Biotechnology \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"NGGT Suzhou Biotechnology \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"RD Fund","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Financing","leadProduct":"OPGx-001","moa":"LCA5","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ RD Fund","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ RD Fund"},{"orgOrder":0,"company":"Aegle Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AGLE-102","moa":"COL7A1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aegle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"Aegle Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aegle Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Aegle Therapeutics","sponsor":"US Department of Defense","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"AGLE-102","moa":"COL7A1","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aegle Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"Aegle Therapeutics \/ US Department of Defense","highestDevelopmentStatusID":"7","companyTruncated":"Aegle Therapeutics \/ US Department of Defense"},{"orgOrder":0,"company":"CARE Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"CAN106","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CARE Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CARE Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CARE Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"National Human Genome Research Institute","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SEL-302","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ National Human Genome Research Institute","highestDevelopmentStatusID":"7","companyTruncated":"Selecta Biosciences \/ National Human Genome Research Institute"},{"orgOrder":0,"company":"Lantu Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Vebeglogene Autotemcel","moa":"Beta-globin","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lantu Biopharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Lantu Biopharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lantu Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Eyestem","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"INDIA","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Eyecyte-RPE","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Eyestem","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Eyestem \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Eyestem \/ Inapplicable"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"SEL-302","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Selecta Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Minovia Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"MNV-BLD","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Minovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Minovia Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Minovia Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"LX1004","moa":"Tripeptidyl-peptidase I (TPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lexeo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"LX1004","moa":"Tripeptidyl-peptidase I (TPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracisternal Injection","sponsorNew":"Lexeo Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Longitude Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Series A Financing","leadProduct":"LX1004","moa":"Tripeptidyl-peptidase I (TPP1)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Lexeo Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.089999999999999997,"dosageForm":"Intracisternal Injection","sponsorNew":"Lexeo Therapeutics \/ Longitude Capital","highestDevelopmentStatusID":"7","companyTruncated":"Lexeo Therapeutics \/ Longitude Capital"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"mRNA-3705","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"VT30","moa":"PI3K","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Phoenicia BioScience","sponsor":"National Heart, Lung, and Blood Institute","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Benserazide Hydrochloride","moa":"DOPA decarboxylase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Phoenicia BioScience","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Phoenicia BioScience \/ National Heart, Lung, and Blood Institute","highestDevelopmentStatusID":"7","companyTruncated":"Phoenicia BioScience \/ National Heart, Lung, and Blood Institute"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Venthera","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"BBP-681","moa":"PI3K","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"BridgeBio Pharma \/ Venthera","highestDevelopmentStatusID":"7","companyTruncated":"BridgeBio Pharma \/ Venthera"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Tividenofusp Alpha","moa":"IDS","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Gangnam Severance Hospital","sponsor":"Daewoong Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Human Umbilical Cord Blood Derived Mesenchymal Stem Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Gangnam Severance Hospital","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gangnam Severance Hospital \/ Daewoong Pharmaceutical","highestDevelopmentStatusID":"7","companyTruncated":"Gangnam Severance Hospital \/ Daewoong Pharmaceutical"},{"orgOrder":0,"company":"Aspa Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"AAV9 BBP-812","moa":"AAV9","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Aspa Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Aspa Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Aspa Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Vitro Biopharma Inc.","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Allorx Stem Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Vitro Biopharma Inc.","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vitro Biopharma Inc. \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Vitro Biopharma Inc. \/ Inapplicable"},{"orgOrder":0,"company":"Phoenicis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"PTW-002","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Phoenicis Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Hydrogel","sponsorNew":"Phoenicis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Phoenicis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"ADARx Pharmaceuticals | Novotech","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ADX-038","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ADARx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ADARx Pharmaceuticals \/ ADARx Pharmaceuticals | Novotech","highestDevelopmentStatusID":"7","companyTruncated":"ADARx Pharmaceuticals \/ ADARx Pharmaceuticals | Novotech"},{"orgOrder":0,"company":"CARE Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"CAN106","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"CARE Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CARE Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"CARE Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"SEL-302","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Selecta Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Argenx","sponsor":"M. Peter Marinkovich","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Efgartigimod","moa":"IgG receptor FcRn large subunit p51","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Argenx","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Argenx \/ M. Peter Marinkovich","highestDevelopmentStatusID":"7","companyTruncated":"Argenx \/ M. Peter Marinkovich"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Avance Clinical","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Radiprodil","moa":"Glutamate NMDA receptor| GRIN1|GRIN2B","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GRIN Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GRIN Therapeutics \/ Avance Clinical","highestDevelopmentStatusID":"7","companyTruncated":"GRIN Therapeutics \/ Avance Clinical"},{"orgOrder":0,"company":"Mahzi Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"MZ-1866","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Mahzi Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Mahzi Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Mahzi Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"GEMMA Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"GB221","moa":"HER2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"GEMMA Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GEMMA Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"GEMMA Biotherapeutics \/ Undisclosed"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

Xls
Filters Filter
×
FILTER:
filter
Company Name
    filter

    Year

      filter

      DEALS // DEV.

        filter

        Country

          filter
          Sponsor
            filter

            Therapeutic Area

              filter

              Study Phase

                filter

                Deal Type

                  filter

                  Product Type

                    filter

                    Dosage Form

                      filter

                      Lead Product

                        filter

                        Target

                          Loading...

                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          November 17, 2014

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : SAR421869

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          January 06, 2012

                          Lead Product(s) : SAR421869

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Mahzi Therapeutics

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Mahzi Therapeutics

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Details : MZ-1866 is a Cell and Gene therapy drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Pitt-Hopkins syndrome.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 21, 2025

                          Lead Product(s) : MZ-1866

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          04

                          GEMMA Biotherapeutics

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          GEMMA Biotherapeutics

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Details : GB221 is a drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Spinal Muscular Atrophies of Childhood.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          July 17, 2025

                          Lead Product(s) : GB221

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Efgartigimod is a Antibody drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Epidermolysis Bullosa.

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          June 09, 2025

                          Lead Product(s) : Efgartigimod

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : M. Peter Marinkovich

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          06

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : The proceeds will be used to fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy, in Rett syndrome.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          May 28, 2025

                          Lead Product(s) : TSHA-102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Jefferies

                          Deal Size : Undisclosed

                          Deal Type : Public Offering

                          blank

                          07

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : The company will fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy, in Rett syndrome.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          May 28, 2025

                          Lead Product(s) : TSHA-102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Jefferies

                          Deal Size : $200.0 million

                          Deal Type : Public Offering

                          blank

                          08

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The MOA of this substance blocks the accumulation of glycosphingolipids and neuroinflammation.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 13, 2025

                          Lead Product(s) : Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Beyond Batten Disease Foundation

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : The financing aims to advance the clinical developed of company's ealry-stage product CLN-301, which is being evaluated for the treatment of CLN3 Batten disease.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 24, 2025

                          Lead Product(s) : CLN-301

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Nationwide Children’s Hospital

                          Deal Size : Undisclosed

                          Deal Type : Financing

                          blank

                          10

                          Arbor Biotechnologies

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Arbor Biotechnologies

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 21, 2025

                          Lead Product(s) : ABO-101

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank