RALEIGH, NC – GeneVentiv Therapeutics, a pre-clinical gene therapy company developing the first universal gene therapy for all types of hemophilia and the first for patients with inhibitors, today was announced winner of the Startup Spotlight pitch competition at BioPharm USA 2023. This year, the BioPharm USA event was held in Raleigh, NC from September 13-14, 2023.
“I am extremely proud to have won the Startup Spotlight pitch competition, especially considering the high-caliber of the companies that participated. The judges recognized the tremendous unmet need for inhibitor patients that our gene therapy addresses,” commented Damon Race, CEO of GeneVentiv Therapeutics.
GeneVentiv’s lead program, GENV-HEM (AAV8.FVa), is the only single infusion, universal, AAV-based gene therapy able to treat all types of hemophilia. Unlike other AAV-based hemophilia gene therapies, GENV-HEM is the only gene therapy able to treat the 33% of hemophilia patients with neutralizing antibodies (inhibitors) to their missing clotting factor. There are 50,000 inhibitor patients in the developed world.
Approved, single infusion, gene therapies for non-inhibitor patients are priced between $2.9M and $3.5M. GENV-HEM has received Orphan Drug Designation from the FDA for Hemophilia A and B with or without inhibitors and a Letter of Support from the National Bleeding Disorders Foundation.
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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