GeneVentiv AAV Gene Therapy for Hemophilia A Advances After Successful FDA INTERACT Meeting

RALEIGH, N.C. — October 29, 2025 — GeneVentiv Therapeutics, Inc., a privately held biotechnology company, today announced a successful INTERACT meeting with the U.S. Food and Drug Administration (FDA) regarding its lead program, GENV-HEM. This novel, one-time gene therapy is being developed for Hemophilia A in patients with or without Factor VIII inhibitors. The FDA meeting confirmed alignment on GeneVentiv’s entire Investigational New Drug (IND)-enabling development plan, including the critical IND-enabling study and the Chemistry, Manufacturing, and Controls (CMC) strategy.

AAV-Based Approach Confirms Regulatory Path

GENV-HEM is an AAV-based gene therapy that expresses activated Factor V. The therapy is designed to provide a durable, single-dose treatment solution for all patients with Hemophilia A, addressing a significant unmet need for patients who develop inhibitors (antibodies) to standard Factor VIII replacement therapies.

“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 further 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, highlighted the preclinical success of the program: “GENV-HEM has demonstrated robust preclinical activity and the potential to address the unmet needs of hemophilia patients with or without inhibitors through durable endogenous Factor Va expression.”

The company will continue advancing its AAV-based GENV-HEM program through IND-enabling studies, with a Pre-IND Meeting and the full IND submission planned as the next major milestones for 2026. The success of this AAV delivery system is central to the company’s mission to pioneer therapies that can reach every patient within a disease area.