GeneVentiv Invited to Present at The National Hemophilia Foundation’s 17th Workshop

February 21, 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 new data from pre-clinical studies of its lead gene therapy for hemophilia A or B with or without inhibitors at the National Hemophilia Foundation’s 17th Workshop on Novel Technologies and Gene Transfer for Hemophilia.

“We’ve made significant progress in our pre-clinical development of GENV-HEM and have benefited from the input of R&D teams at multiple strategics.  We look forward to presenting new data confirming in vivo durability, efficacy and safety in challenging and widely accepted functional assays. We expect these exciting results to inform our progression to IND work. More importantly, to bring our program one step closer to providing a durable gene therapy for all hemophilia patients, especially those with inhibitors, who are unable to benefit from any of the current gene-based therapeutics,” said Damon Race, President and CEO of GeneVentiv Therapeutics.

“The GENV-HEM program represents a novel adeno-associated virus (AAV) approach for hemophilia inhibitor patients. The in vivo data define key components of a gene-based hemophilia therapeutic: transgene expression threshold, AAV vector dose required, hemostatic efficacy and associated safety. Having worked on gene therapies for inhibitor patients for over 20 years, I am truly excited about this data,” said Paris Margaritis, consulting CSO of GeneVentiv Therapeutics.

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 of new pre-clinical data

Title: Gene-based, therapeutic and long-term expression of activated Factor V for hemophilia complicated by inhibitors

Date: Friday, March 24, 2023, 3:10 pm EST
Abstract Summary: GeneVentiv is conducting pre-clinical trials to inform IND studies. The abstract describes the scientific rationale, rational design of the gene therapy, existing and new pre-clinical data.  Collectively, the pre-clinical data demonstrates the in vivo efficacy, safety and therapeutic window of AAV8-FVa gene therapy for hemophilias irrespective of inhibitors.

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.