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 preclinical gene therapy company focused on identifying, in-licensing, and developing transformative genetic medicines for diseases with significant unmet needs. GeneVentiv is advancing two first-in-class programs. GENV-HEM is a liver-directed gene therapy designed to provide activated Factor V (FVa) for patients with hemophilia A, with or without FVIII inhibitors. GENV-002 is an in vivo gene-editing therapy for both infantile-onset (IOPD) and late-onset (LOPD) Pompe disease, designed to stably integrate a functional GAA transgene and establish the liver as a durable source of GAA enzyme.
Together, these programs are designed to address serious diseases where significant unmet needs remain.
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