Single-arm study | Two-year outcomes of Valoctocogene Roxaparvovec therapy for Hemophilia A

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A – New England Journal of Medicine (link to abstract – $ for full-text)

Commentary on Twitter

In the GENEr8-1 trial, participants with hemophilia A received B-domain–deleted factor VIII gene therapy delivered in an AAV5 vector. A decrease in annualized bleeding rates was maintained for 2 years despite declining factor VIII levels. https://t.co/hYy3pT8ssa#hematology pic.twitter.com/HuJQa0HiTR

— NEJM (@NEJM) February 23, 2023