GENV-HEM

GENV-HEM is an investigational, single-infusion gene therapy designed to treat all types of hemophilia—including both hemophilia A and B, with or without clotting factor inhibitors. It uses a serotype 8 adeno-associated virus (AAV8) vector to deliver a bioengineered gene encoding the activated form of coagulation Factor V (FVa) directly to liver cells, which are the primary producers of clotting factors in the body. By increasing endogenous FVa production, GENV-HEM aims to restore normal blood clotting through the common pathway of the coagulation cascade, functioning downstream of Factor VIII and IX. This approach allows for effective treatment even in patients who have developed inhibitors against standard replacement therapies. Preclinical studies have shown that a single dose can prevent bleeding episodes in animal models regardless of inhibitor status. The drug has received orphan drug designation from the US FDA for both hemophilia A and B[1][3][4][5][8].

Other names

AAV8-hFVaAAV-8-hFVaAAV 8-hFVaAAV8-FVaAAV-8-FVaAAV 8-FVa