Discovering, Developing and Delivering Gene Therapies. Transforming Lives.
GeneVentiv Therapeutics is a pre-clinical gene therapy company focused on blood diseases and disorders. Our lead program, GENV-HEM, is the first durable and universal gene therapy treatment for Hemophilia A and B with or without inhibitors. GENV-HEM has received Orphan Drug Designation from the FDA and a Letter of Support from the National Hemophilia Foundation. GeneVentiv is advancing an additional gene therapy for Hemophilia Arthropathy, a degenerative joint disease afflicting roughly 50% of patients with hemophilia.
Advances in Medicine Driven by Innovation
Challenge:
Competitor’s AAV based gene therapies for hemophilia A have failed to demonstrate the lifetime durability and none of the hemophilia A or B gene therapies can treat patients with inhibitors (up to 30% of patients). Gene therapies in development to treat hemophilia A express clotting Factor VIII, which is not natively expressed in hepatocytes targeted by gene therapy. This non-native expression can lead to accumulation of misfolded protein, cell death and loss of expression.
Solution:
Native expression of FVa gives us durability vs. FVIII, making us the first durable, universal gene therapy for all types of hemophilia (hemophilia A and B) and the first AAV based gene therapy that can treat patients with inhibitors.
