GeneVentiv Therapeutics has achieved a significant regulatory milestone with positive FDA feedback on its lead gene therapy program for hemophilia A. The privately held biotechnology company announced that its INTERACT meeting with the U.S. Food and Drug Administration confirmed alignment on the IND-enabling development plan for GENV-HEM, a novel gene therapy designed to treat hemophilia A patients with or without inhibitors.
Regulatory Milestone Reduces Development Risk
The FDA meeting validated GeneVentiv's comprehensive development strategy, including both the IND-enabling study design and chemistry, manufacturing, and controls (CMC) approach. According to Damon Race, Chief Executive Officer of GeneVentiv Therapeutics, "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."
Race emphasized that the regulatory alignment substantially reduces risk for the program, allowing the company to focus on advancing GENV-HEM toward IND submission readiness.
Novel AAV-Based Approach Targets Factor V Expression
GENV-HEM represents an innovative approach to hemophilia A treatment through AAV-based gene therapy that expresses activated Factor V. The therapy is designed as a one-time treatment for patients with hemophilia A, including those with inhibitors—antibodies to Factor VIII that complicate traditional treatment approaches.
Dr. Paris Margaritis, Chief Scientific Officer, highlighted the therapy's potential clinical impact: "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."
Development Timeline and Next Steps
Following the successful INTERACT meeting, GeneVentiv plans to continue regulatory engagement as it advances through IND-enabling studies. The company has outlined specific upcoming milestones, with a Pre-IND Meeting and IND submission planned for 2026.
The regulatory feedback provides GeneVentiv with a clear development pathway for bringing this potential one-time gene therapy to hemophilia A patients who currently face limited treatment options, particularly those with inhibitors that render conventional Factor VIII replacement therapy less effective.
Broader Pipeline Development
Beyond GENV-HEM, GeneVentiv is developing GENV-002, a dual-vector CRISPR gene-editing therapy for both Infantile and Late Onset Pompe disease. This program utilizes a liver-depot strategy to secrete GAA systemically, demonstrating the company's commitment to addressing rare and serious diseases through innovative gene therapy approaches.
The company's mission focuses on developing therapies that can reach every patient within a disease area, rather than serving only select patient populations—a particularly relevant goal for rare disease treatment where patient access remains a significant challenge.
