Product
Valoctocogene Roxaparvovec
Aliases
BMN 270
2 clinical trials
4 indications
Indication
Hemophilia AIndication
Gene TherapyIndication
CoagulopathyIndication
Blood DisorderClinical trial
A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII InfusionsStatus: Completed, Estimated PCD: 2019-05-22
Clinical trial
A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5Status: Active (not recruiting), Estimated PCD: 2024-11-01