April 12, 2023
Damon R. Race, President & CEO
RALEIGH, NC – GeneVentiv Therapeutics, a pre-clinical gene therapy company focused on blood diseases and disorders, today announced it has been invited to present as part of Labcorp’s symposium at the 26th Annual Meeting of the American Society of Cell and Gene Therapy (ASGCT). GeneVentiv will give a presentation on engaging with CDMOs and CROs during pre-clinical development to accelerate timelines to clinical trials. GeneVentiv will present during the symposium to be held on May 19th, 2023 from 12-1:30 PM PST.
“We’ve benefited from early engagement with CDMOs and CROs by improving our understanding of timelines and costs, IND capabilities, regulatory requirements and clinical trial design,” said Damon Race, President and CEO of GeneVentiv Therapeutics. The Company looks forward to sharing information on how engagement with CDMOs and CROs has benefited the development of GENV-HEM, which is GeneVentiv’s lead AAV-based gene therapy which has an FDA Orphan Drug Designation for hemophilia A or B with or without inhibitors.
GENV-HEM: AAV8.FVa Gene Therapy for Hemophilia A or B with or without Inhibitors
GeneVentiv’s most advanced candidate is designed to bypass missing or deficient factor VIII (FVIII, hemophilia A) or factor IX (FIX, hemophilia B) and any neutralizing antibodies (inhibitors). Activated factor V (FVa) forms the prothrombinase complex with activated factor X and drives thrombin generation in response to injury. Interestingly, hemophilic patients who also harbor a procoagulant mutation in factor V (FV Leiden) show improved clinical outcomes. These data suggest that enhancing FVa function may bypass the deficiencies in hemophilia and, therefore, be unaffected by inhibitors to FVIII or FIX. This approach is embodied in the GENV-HEM program that delivers a human FVa transgene for increased liver expression, aiming to enhance thrombin generation and restore hemostasis in all hemophilia patients.
About GeneVentiv Therapeutics:
GeneVentiv Therapeutics is a pre-clinical gene therapy company focused on blood diseases and disorders. Our lead program, GENV-HEM, is the first durable, 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.