Advances in Medicine Driven by Innovation
None of the AAV-based, hemophilia gene therapies approved or in development can treat the 33% of hemophilia patients with inhibitors (neutralizing antibodies) to their missing clotting factor. There are 50,000 inhibitor patients in the developed world. Approved, single infusion, gene therapies for non-inhibitor patients are priced between $2.9M and $3.5M. In addition, the only approved gene therapy for hemophilia A without inhibitors, which expresses FVIII, has limited durability. This is likely due to FVIII production in cells that do not naturally produce it. Non-native expression can lead to accumulation of misfolded protein, cell death and loss of FVIII expression.
Our lead program GENV-HEM (AAV8.FVa) expresses FVa that can treat all types of hemophilia, including inhibitor patients, in a single infusion.
GENV-HEM directs expression of FVa in its native cell, addressing the durability challenge facing existing FVIII AAV gene therapies.