[{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Divestment","leadProduct":"Burosumab","moa":"FGF23","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Kyowa Kirin","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Kyowa Kirin"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Burosumab","moa":"FGF23","graph1":"Musculoskeletal","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Evinacumab","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Evinacumab","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Evinacumab","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Evinacumab","moa":"ANGPTL3","graph1":"Cardiology\/Vascular Diseases","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"GeneTx Biotherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2022","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ GeneTx Biotherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ GeneTx Biotherapeutics"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2023","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2024","type":"Inapplicable","leadProduct":"Betamethasone","moa":"UBE3A-AS","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"DTX401","moa":"||Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"UX053","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"ATP7B copper transport","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"UX701","moa":"ATP7B copper transport","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"UX701","moa":"ATP7B copper transport","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"UX701","moa":"ATP7B copper transport","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"UX701","moa":"ATP7B copper transport","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Rebisufligene Etisparvovec","moa":"AAV9","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"OTC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"OTC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"OTC","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Mereo BioPharma Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Public Offering","leadProduct":"Setrusumab","moa":"Sclerostin","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ J.P. Morgan","highestDevelopmentStatusID":"10","companyTruncated":"Ultragenyx Pharmaceutical \/ J.P. Morgan"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Avalotcagene Ontaparvovec","moa":"OTC","graph1":"Immunology","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Immunology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Daiichi Sankyo","pharmaFlowCategory":"D","therapeuticArea":"Technology","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Technology","graph2":"Discovery Platform","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Technology","amount2New":0,"dosageForm":"Oral","sponsorNew":"Ultragenyx Pharmaceutical \/ Daiichi Sankyo","highestDevelopmentStatusID":"3","companyTruncated":"Ultragenyx Pharmaceutical \/ Daiichi Sankyo"}]

Find Clinical Drug Pipeline Developments & Deals by Ultragenyx Pharmaceutical

Menu
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

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated for the treatment of sanfilippo syndrome type A (MPS IIIA) in children.

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 18, 2025

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          02

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated for the treatment of sanfilippo syndrome type A (MPS IIIA) in children.

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 05, 2025

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : Evkeeza (evinacumab) is the first ANGPTL3 inhibitor. It is approved for the treatment of children 6 month and older with Homozygous Familial Hypercholesterolemia (HoFH).

                          Product Name : Evkeeza

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          January 06, 2025

                          Lead Product(s) : Evinacumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          04

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : Betamethasone

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          06

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : UX111 (rebisufligene etisparvovec) is an AAV gene therapy. It is being evaluated in phase 3 clinical trials for the treatment of sanfilippo syndrome type A (MPS IIIA).

                          Product Name : UX111

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 06, 2024

                          Lead Product(s) : Rebisufligene Etisparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          07

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.

                          Product Name : UX143

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          November 06, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Mereo BioPharma Group

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.

                          Product Name : GTX-102

                          Product Type : Steroid

                          Upfront Cash : Inapplicable

                          July 17, 2024

                          Lead Product(s) : Betamethasone

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          09

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : UX143 (setrusumab), is a sclerostin inhibitor antibody drug candidate, which is currently being evaluated for the treatment of osteogenesis imperfecta.

                          Product Name : UX143

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          July 10, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          10

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : The net proceeds of the offering to continue to advance the late-stage clinical program UX143 (setrusumab), which is being evaluated for the treatment of osteogenesis imperfecta.

                          Product Name : UX143

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          June 13, 2024

                          Lead Product(s) : Setrusumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : J.P. Morgan

                          Deal Size : $350.0 million

                          Deal Type : Public Offering

                          blank