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 4E13 vg/kg inHemophilia A Patients with Residual FVIII Levels

Project: Research project

Description

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 4E13 vg/kg in Hemophilia A Patients with Residual FVIII Levels
StatusActive
Effective start/end date7/15/187/15/23

Funding

  • BIOMARIN PHARMACEUTICAL, INC.

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Transfer Factor
Dependovirus
Hemophilia A
Safety
Genes
human F8 protein