[{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"DiNAQOR AG","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"DiNA-001","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical ","highestDevelopmentStatusID":"5","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical "},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"BMN 307","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"BMN 307","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"BMN 307","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2021","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"AAV-based gene therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2021","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2021","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Entos Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"Fusogenix-formulated Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin","highestDevelopmentStatusID":"1","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2021","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"BMN 307","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2022","type":"Divestment","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0.11,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Injectable\/Injection","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Vosoritide","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Orsini Specialty Pharmacy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec-rvox","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Vosoritide","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"Vosoritide","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"CAMP4 Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Collaboration","leadProduct":"regRNA-based Therapy","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"14","companyTruncated":"BioMarin Pharmaceutical \/ BioMarin Pharmaceutical"}]

Find Clinical Drug Pipeline Developments & Deals by BioMarin 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

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : The collaboration combines BioMarin’s expertise in drug development with CAMP4’s platform-based target discovery capabilities to advance novel regRNA-targeting medicines for rare genetic conditions.

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          October 01, 2024

                          Lead Product(s) : regRNA-based Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Recipient : CAMP4 Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

                          blank

                          02

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy approved for severe hemophilia A, that uses an adeno-associated virus 5, codes for human Factor VIII.

                          Brand Name : Roctavian

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 05, 2024

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          03

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

                          Brand Name : Voxzogo

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          October 20, 2023

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          04

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

                          Brand Name : Voxzogo

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          September 15, 2023

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          05

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Brand Name : Roctavian

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 11, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec-rvox

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Recipient : Orsini Specialty Pharmacy

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          06

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Brand Name : Roctavian

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          June 29, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          07

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

                          Brand Name : Voxzogo

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          March 07, 2023

                          Lead Product(s) : Vosoritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          08

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Brand Name : Roctavian

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 06, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          09

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Brand Name : Roctavian

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          January 09, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank

                          10

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions.

                          Brand Name : Roctavian

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 23, 2022

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          blank