GeneVentiv Invited to Present at BioPharm America 2023

August 30, 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 at BioPharm AmericaTM, which will be held in Raleigh, NC from September 13-14th.  GeneVentiv will give a presentation on GENV-HEM, GeneVentiv’s lead AAV-based gene therapy which has an FDA Orphan Drug Designation for hemophilia A or B with or without inhibitors on September 14th from 3:45-5 PM EST.

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.

Oral presentation, corporate overview and summary of pre-clinical studies

Title: GeneVentiv’s AAV8.FVA for Hemophilia A or B with or without Inhibitors

Date: Thursday, September 14, 2023, 3:45 pm EST

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.