Highly competitive NIH program validates science, funds costly toxicology work, and accelerates progress toward IND for a one-time gene therapy for hemophilia A, with or without inhibitors
RALEIGH, NC, Oct. 7, 2025 /PRNewswire/ — GeneVentiv Therapeutics, a biotechnology company pioneering next-generation gene therapies for rare diseases, today announced that it has been selected into the Catalyze Program of the National Heart, Lung, and Blood Institute (NHLBI). Through this program, NIH will fund and provide preclinical services with leading national providers for the GLP toxicology study required for the company’s IND submission of GENV-HEM, its investigational one-time gene therapy for hemophilia A, including patients with inhibitors.
The NHLBI Catalyze Program is an ultra-selective initiative that supports only the most promising medical innovations with the potential for transformative patient impact. By covering the most resource-intensive portion of IND-enabling toxicology, Catalyze support significantly reduces capital requirements and accelerates the company’s timeline to clinical entry.
“Selection into the NHLBI Catalyze Program represents strong validation of our science and regulatory strategy,” said Damon Race, Chief Executive Officer of GeneVentiv Therapeutics. “It removes a significant cost barrier in gene therapy development and directs investor capital to higher-return activities to advance GENV-HEM into the clinic. This also frees capital for further development of GENV-002, our gene editing therapy for both Infantile and Late Onset Pompe disease.”
This Catalyze support complements GeneVentiv’s ongoing NHLBI Small Business Innovation Research (SBIR) award, which funds development of critical IND-enabling assays and a confirmatory large-animal (canine) efficacy and safety study. Together, these programs provide broad-based, non-dilutive support across efficacy, safety, and regulatory preparation, substantially reducing costs and de-risking the path to IND.
“Having merit-based NHLBI awards for both efficacy and toxicology underscores the strength and broad applicability of our approach,” said Dr. Paris Margaritis, Chief Scientific Officer of GeneVentiv Therapeutics. “GENV-HEM is designed to be the first gene therapy to treat all hemophilia A patients, including those with inhibitors, using a single product. With NHLBI support for critical preclinical studies, we are uniquely positioned to bring forward a one-time therapy with transformative potential for the hemophilia community.”
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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