Discovering, Developing and Delivering Gene Therapies. Transforming Lives.
GeneVentiv Therapeutics is a pre-clinical gene therapy company focused on blood disorders. Our lead program, GENV-HEM (AAV8.FVa), is the only single infusion, universal, AAV-based gene therapy able to treat all types of hemophilia. Unlike other AAV-based hemophilia gene therapies, GENV-HEM is the only gene therapy able to treat the 33% of hemophilia patients with neutralizing antibodies (inhibitors) 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. GENV-HEM has received Orphan Drug Designation from the FDA for Hemophilia A and B with or without inhibitors and a Letter of Support from the National Bleeding Disorders Foundation.
Advances in Medicine Driven by Innovation
Challenge:
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.
Solution:
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.
