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Phase 1/2 Study of Valoctocogene roxaparvovec (BMN 270) in Hemophilia A Patients With Active or Prior Inhibitors
2019-003213-34Phase 2Unknown
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Mar 2020
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of valoctocogene roxaparvovec in hemophilia A (2 cohorts), sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: FVIII inhibitors. Status: unknown.
02
Drugs
valoctocogene roxaparvovec
03
Conditions
hemophilia A
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