[{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2017","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Arup Laboratories","sponsor":"Medicover","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Partnership","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Arup Laboratories","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Arup Laboratories \/ Medicover","highestDevelopmentStatusID":"15","companyTruncated":"Arup Laboratories \/ Medicover"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin 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Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Coagulation factor 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IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"BioMarin Pharmaceutical \/ Undisclosed"}]

Find Clinical Drug Pipeline Developments & Deals for Valoctocogene Roxaparvovec

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                          01

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          August 05, 2024

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Valoctocogene Roxaparvovec is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Hemophilia A.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          January 25, 2024

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Under the partnership, ARUP Laboratories developed AAV5 DetectCDx™, a companion diagnostic that aids in the selection of adult patients with severe hemophilia A eligible for treatment with Roctavian (valoctocogene roxaparvovec-rvox), a new gene therapy...

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          December 06, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Medicover

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          04

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          July 11, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : BioMarin Pharmaceutical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          June 29, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          March 06, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          January 09, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          November 23, 2022

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) also received orphan drug designation from the EMA and FDA for severe hemophilia A. Phase 1/2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Fact...

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          November 09, 2022

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : The GENEr8-1 Phase 3 study for valoctocogene roxaparvovec demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate.

                          Product Name : Roctavian

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          October 12, 2022

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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