GeneVentiv Obtains $2.5 Million SBIR Grant to Develop Hemophilia Gene Therapy

GeneVentiv Therapeutics has received a $2.5 million grant to conduct preclinical tests on its one-time infusion gene therapy for hemophilia A and B, with or without clotting factor inhibitors. The therapy, GENV-HEM, will be tested in a large study with a canine model of hemophilia, following promising results in mouse models of both hemophilia types. Funds will also be used to evaluate the gene therapy's distribution in the body and associated toxicology. These studies are expected to support a future investigational new drug application for the therapy.

GeneVentiv Secures $2.5 Million SBIR Grant to Advance One-Time Infusion Gene Therapy for Hemophilia

GeneVentiv Therapeutics, a pioneering biotechnology company, has received a prestigious $2.5 million Small Business Innovation Research (SBIR) grant from the National Heart, Lung, and Blood Institute, a division of the esteemed National Institutes of Health. This grant marks a significant milestone in the development of GeneVentiv's transformative gene therapy, GENV-HEM, for the treatment of hemophilia A and B, both with and without clotting factor inhibitors.

GENV-HEM, a one-time infusion gene therapy, has demonstrated remarkable promise in treating hemophilia in preclinical mouse models. The SBIR grant will enable GeneVentiv to further evaluate the therapy's efficacy and safety in a larger study using a canine model of hemophilia. The canine model offers a more robust representation of human physiology, providing valuable insights into the therapy's performance in a setting closer to that of patients.

The grant will also support critical studies to assess the biodistribution and toxicology of GENV-HEM in the body. These comprehensive investigations are essential for establishing the safety profile of the therapy and for preparing an Investigational New Drug (IND) application, which would allow GENV-HEM to enter human clinical trials.

"This SBIR grant is a resounding endorsement of the transformative potential of GENV-HEM," said Paris Margaritis, Chief Scientific Officer of GeneVentiv. "The findings from our mouse studies have been tremendously encouraging, and this grant will enable us to validate these results in a larger animal model and conduct the rigorous safety assessments required for IND submission."

Hemophilia, a rare genetic disorder characterized by excessive and prolonged bleeding, affects approximately 1 in 5,000 males worldwide. The condition arises from deficiencies or malfunctions of clotting factors, primarily factor VIII (FVIII) in hemophilia A and factor IX (FIX) in hemophilia B.

Current treatment options for hemophilia include factor replacement therapy, which involves administering synthetic or plasma-derived clotting factors. However, this approach can be limited by the development of neutralizing antibodies (inhibitors) that prevent the clotting factors from functioning properly.

GENV-HEM employs a cutting-edge gene therapy approach that aims to overcome these challenges. The therapy utilizes a harmless adeno-associated virus (AAV) as a carrier to deliver a gene encoding activated clotting factor V (FVa). FVa interacts with another clotting protein, factor X (FX), initiating a cascade of events that lead to the formation of blood clots independently of FVIII or FIX.

"GENV-HEM has the potential to revolutionize the treatment of hemophilia by offering a single-infusion, lifetime solution for patients with and without inhibitors," said Damon Race, CEO of GeneVentiv. "This grant from the NIH is a pivotal step towards bringing this life-changing therapy to patients."

In recognition of its significant therapeutic potential, GENV-HEM has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia A and B. GeneVentiv has also forged a strategic collaboration with ReciBioPharm to ensure that GENV-HEM meets the stringent Good Manufacturing Practice (GMP) standards and Good Laboratory Practice (GLP) requirements for manufacturing and testing.

"The combined support of this grant and our manufacturing partnership with ReciBioPharm significantly reduces the financial burden on investors while maximizing resources for the critical IND-enabling studies," added Race. "This grant brings us one step closer to submitting the IND for GENV-HEM, paving the way for human clinical trials and ultimately providing a transformative treatment option for patients with hemophilia."

The SBIR grant from the NIH represents a testament to the groundbreaking nature of GeneVentiv's gene therapy approach for hemophilia. With promising preclinical results, a robust canine study, and comprehensive safety assessments underway, GENV-HEM is poised to make a profound impact on the lives of hemophilia patients worldwide.