RALEIGH, NC – GeneVentiv Therapeutics, a pre-clinical gene therapy company focused on blood diseases and disorders, today announced the award of a $250,000 Strategic Research Loan (SRL) from the North Carolina Biotechnology Center (NCBC).
GeneVentiv’s lead program, GENV-HEM, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Hemophilia A or B patients with or without inhibitors. “The Strategic Research Loan from the North Carolina Biotechnology Center enables GeneVentiv to complete significant pre-clinical studies in preparation for an investigational new drug (IND) application and human clinical trials,” commented Damon Race, CEO of GeneVentiv Therapeutics.
“This funding from the NC Biotechnology Center adds to our momentum. In the past six months we’ve received Orphan Drug Designation, added a second gene therapy for hemophilia arthropathy, earned the support of the National Hemophilia Foundation and won a UNC KickStart Commercialization grant,” said Damon Race. “The North Carolina Biotechnology Center conducts rigorous due diligence and receiving this award represents another point of external validation for our lead program.”
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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