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GENEr8-JPN: Phase 3 single-arm gene therapy study of BMN 270 in Japanese adults with severe hemophilia A
jRCT2033230441Phase 3Active, not recruiting
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Jan 2024
- 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 hFVIII activity from baseline to Weeks 49–52. Started Jan 2024; status: active, not recruiting.
02
Drugs
valoctocogene roxaparvovec
03
Conditions
hemophilia A
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