Clinical Trial Update: Gene Therapy Study in Severe Haemophilia A Patients (270-201) [Phase 1, Phase 2]

View source ↗

Summary

A Phase 1/2 gene therapy trial (NCT02576795) evaluating valoctocogene roxaparvovec for severe haemophilia A has completed, with results posted on 2025-04-10.

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

Diseases / indications

• Haemophilia

Drugs mentioned

• Roctavian (valoctocogene roxaparvovec)

Snippet

Sponsor: BioMarin Pharmaceutical | Conditions: Severe Haemophilia A | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2025-04-10