Product
Valoctocogene
Aliases
BMN 270, valoctocogene roxaparvovec
2 clinical trials
1 indication
Indication
Hemophilia AClinical 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 in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII InfusionsStatus: Active (not recruiting), Estimated PCD: 2020-11-16
Clinical trial
A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia AStatus: Active (not recruiting), Estimated PCD: 2024-03-01