One-Shot gene therapy aims to free patients from lifelong injections
Disease control
Completed
This study tested a single-dose gene therapy, called valoctocogene roxaparvovec, in 22 adult men with severe hemophilia A. The goal was to see if the therapy could safely help the liver produce its own blood-clotting factor (Factor VIII), reducing or eliminating the need for regu…
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC