Bleeding, FVIII activity, and safety three years after gene transfer with valoctocogene roxaparvovec: results from GENEr8-1

Johnny Mahlangu, M.D., M.Med.
University of the Witwatersrand
Johannesburg, South Africa

Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is known to provide endogenous factor VIII (FVIII) production to prevent bleeding in people with severe hemophilia A. The open-label, multicenter phase three GENEr8-1 trial evaluates the use of AAV5-hFVIII-SQ in comparison to baseline on FVIII prophylaxis. Mahlangu, M.D., M.Med., through this presentation, will present results that conclude hemostatic efficacy delivered by AAV5-hFVIII-SQ in comparison to FVIII prophylaxis.