GeneVentiv Therapeutics, Inc., a privately held biotechnology company developing one-time gene therapies for rare and inherited diseases, announced that it has held a successful INTERACT meeting with the U.S. Food and Drug Administration (FDA) for its lead program, GENV-HEM, a novel gene therapy candidate for Hemophilia A, including patients with or without inhibitors to Factor VIII.
The meeting confirmed alignment on GeneVentiv’s IND-enabling development plan, encompassing both preclinical studies and CMC (Chemistry, Manufacturing, and Controls) strategy.
“This positive and constructive meeting with the FDA represents a key regulatory milestone for GeneVentiv,” said Damon Race, Chief Executive Officer of GeneVentiv Therapeutics.
“The Agency’s confirmation of our IND-enabling development plan validates the rigor of our scientific and manufacturing approach and provides a clear path toward first-in-human studies. With regulatory risk now substantially reduced, we are focused on advancing GENV-HEM to IND submission readiness.”
Dr. Paris Margaritis, Chief Scientific Officer, added, “We appreciate the FDA’s thoughtful engagement and early alignment on our development strategy. GENV-HEM has shown robust preclinical activity and the potential to meet the unmet needs of hemophilia patients through durable endogenous Factor Va expression.”
GeneVentiv plans to continue regulatory engagement as it progresses GENV-HEM through IND-enabling studies, with a Pre-IND meeting and IND submission targeted for 2026.
GeneVentiv Therapeutics is a biotechnology company developing transformative gene therapies for rare and serious diseases. Its lead candidate, GENV-HEM, is an AAV-based gene therapy expressing activated Factor V, designed as a one-time treatment for patients with Hemophilia A, including those with inhibitors. The company’s second program, GENV-002, is a dual-vector CRISPR gene-editing therapy for Pompe disease (Infantile and Late Onset forms), using a liver-depot strategy to enable systemic secretion of GAA.
GeneVentiv’s mission is to pioneer gene therapies that can reach all patients within a disease population, not just a limited subset.
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