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Phase 3 single-arm BMN 270 gene therapy study in severe hemophilia A
2017-003573-34Phase 3Unknown
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Oct 2017
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of valoctocogene roxaparvovec in hemophilia A, sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: Change in human factor VIII activity during Weeks 49-52. Status: unknown.
02
Drugs
valoctocogene roxaparvovec
03
Conditions
hemophilia A
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