Benefits of Roctavian, Hem A Gene Therapy, Holding for 2 or More Years

Benefits of Roctavian, Hem A Gene Therapy, Holding for 2 or More Years

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Benefits of Roctavian, Hem A Gene Therapy, Holding for 2 or More Years

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for preventive treatment, or prophylaxis, over at least two years in men with severe hemophilia A, according to updated data from the Phase 3 GENEr8-1 trial. Notably, the activity of factor VIII (FVIII), the missing clotting protein in hemophilia A, remained in the therapeutic range through two years post-dosing. “A potential single treatment that provides a durable…

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