Gene therapy for hemophilia targets the genetic defect responsible for the body’s inability to produce clotting factors—factor VIII (for hemophilia A) or factor IX (for hemophilia B). The treatment introduces functional copies of the F8 or F9 genes into liver cells using viral vectors, such as AAV (adeno-associated virus). These functional genes enable the production of the missing clotting factors, significantly reducing or even eliminating the need for routine injections of clotting factor concentrates. A notable example is Roctavian (valoctocogene roxaparvovec), which has demonstrated remarkable success in lowering bleeding episodes for hemophilia A patients, offering a more sustainable treatment option.
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