GeneVentiv Therapeutics is developing GENV-HEM (AAV8.FVa), a single-infusion, AAV-based gene therapy that treats all types of hemophilia, including patients with neutralizing antibodies to their missing clotting factor. This therapy addresses the unmet need of the 50,000 inhibitor patients in the developed world, offering a durable solution that overcomes the limitations of existing hemophilia treatments.
Funding
$6.7M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Existing AAV-based gene therapies for hemophilia cannot treat the 33% of hemophilia patients who have developed neutralizing antibodies (inhibitors) against their missing clotting factor. This leaves a significant portion of hemophilia patients, approximately 50,000 in the developed world, without access to potentially curative gene therapy treatments. Furthermore, some existing gene therapies exhibit limited durability due to non-native expression of the clotting factor.
Solution
GeneVentiv Therapeutics is developing GENV-HEM (AAV8.FVa), a single-infusion, AAV-based gene therapy designed to treat all types of hemophilia, including patients with inhibitors. GENV-HEM utilizes Factor Va (FVa) expression to bypass the need for Factor VIII or IX, thus circumventing the issue of neutralizing antibodies. The therapy delivers the FVa gene to its native cell type, potentially leading to more durable expression and long-term therapeutic benefit compared to existing FVIII-based gene therapies. By addressing both inhibitor and durability challenges, GENV-HEM aims to provide a universal and lasting solution for hemophilia patients.
Target Audience
The primary target audience includes hemophilia A and B patients, including the subgroup with neutralizing antibodies (inhibitors) to Factor VIII or Factor IX, and the physicians who treat them.
Features
- AAV8 vector for efficient gene delivery.
- Factor Va (FVa) expression to treat all hemophilia types, including those with inhibitors.
- Single-infusion administration.
- Native cell expression of FVa for potentially improved durability.
- Orphan Drug Designation from the FDA for Hemophilia A and B with or without inhibitors.