One-Time gene therapy could free hemophilia patients from frequent infusions
Disease control
Completed
This study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The goal was to help their bodies produce their own clotting factor VIII, reducing or stopping the need for regular factor infusions. Participants also received steroids …
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 13, 2026 16:03 UTC