#USA #Raleigh #Hemophilia A #GeneVentiv #GENV-HEM

GeneVentiv Therapeutics Receives Positive FDA Feedback

On October 29, 2025, GeneVentiv Therapeutics, a privately held company focused on innovative gene therapies for rare diseases, announced a significant breakthrough regarding its GENV-HEM program. The company successfully held an INTERACT meeting with the U.S. Food and Drug Administration (FDA), a critical step for advancing its gene therapy for Hemophilia A, including cases with inhibitors.

GeneVentiv's GENV-HEM therapy represents a novel approach in treating this condition, which is caused by a deficiency of clotting Factor VIII. This meeting with the FDA confirmed alignment on several critical components of the therapy's development plan, including its Investigational New Drug (IND)-enabling studies and Chemistry, Manufacturing, and Controls (CMC) strategy.

During this pivotal meeting, GeneVentiv's executives expressed their satisfaction with the FDA's constructive feedback. Damon Race, Chief Executive Officer of the company, remarked, "This positive and constructive meeting with the FDA represents a key regulatory milestone for GeneVentiv. 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."

The importance of the FDA's support cannot be understated, as it has substantially reduced regulatory risks. With a clearer pathway to IND submission, GeneVentiv is turning its focus to preparing GENV-HEM for trials with the goal of introducing the therapy to patients who need it the most. Dr. Paris Margaritis, Chief Scientific Officer, reinforced the company's commitment to ongoing dialogue with regulatory authorities, focusing on advancing GENV-HEM through the various necessary studies. He acknowledged the important role of the FDA in guiding their development strategy, stating, “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.”

As part of their ongoing strategy, GeneVentiv plans to engage in several upcoming milestones throughout 2026, including a Pre-IND Meeting and the subsequent IND submission. This forward-looking approach highlights the company's dedication to not only advancing gene therapy for Hemophilia A but also contributing to transformative treatments for other serious rare diseases in the future.

In addition to GENV-HEM, GeneVentiv is making strides with its second program, GENV-002, which focuses on a dual-vector CRISPR gene-editing therapy designed to provide systemic secretion of GAA for both Infantile and Late Onset Pompe disease. The innovative design of GENV-002 further exemplifies GeneVentiv's mission to deliver pioneering therapies that benefit every patient within its targeted disease areas.

In summary, GeneVentiv Therapeutics is on an upward trajectory following its successful FDA INTERACT meeting, demonstrating significant progress in developing GENV-HEM for Hemophilia A. Their dedication to addressing unmet medical needs in rare diseases continues to set them apart in the biotechnology field. As regulatory hurdles are cleared, the hope remains strong that their efforts will culminate in viable treatments for patients awaiting a solution.