Clinical Trial Update: Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 [Phase 1, Phase 2]

View source ↗

Summary

BioMarin's Phase 1/2 gene therapy trial in severe Hemophilia A patients with antibodies against AAV5 was terminated and results have been posted.

Upgrade for the full analysis, importance score, and drug timeline.

Diseases / indications

• Hemophilia

Drugs mentioned

• Valrox (valoctocogene roxaparvovec)

Snippet

Sponsor: BioMarin Pharmaceutical | Conditions: Hemophilia A, Gene Therapy | Phase 1, Phase 2 | Status: TERMINATED | Results posted: 2025-08-22