Genascence’s AAV Gene Therapy for Knee OA Earns FDA RMAT Designation

PALO ALTO, Calif. – July 17, 2025 – Genascence Corporation, a clinical-stage biotechnology company, announced today that its investigational AAV gene therapy, GNSC-001, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). GNSC-001 is a potential first-in-class AAV-based treatment for knee osteoarthritis (OA) designed to offer long-term, sustained inhibition of IL-1 after a single intra-articular injection.

The RMAT designation, an FDA program expediting promising cell and gene therapies for serious conditions, was granted based on positive safety and efficacy data from two clinical trials, including the DONATELLO Phase 1b trial.

Genascence Founder and CEO, Thomas Chalberg, Ph.D., highlighted GNSC-001 as the first IL-1 inhibitor for OA to show sustained therapeutic IL-1Ra expression. This designation will enable closer collaboration with the FDA to accelerate late-stage development.

Having previously received FDA Fast Track designation in Q4 2024, Genascence plans to initiate its Phase 2b/3 study in 2026. Knee OA affects over 30 million Americans and currently lacks therapies to alter or slow its progression, representing a significant unmet medical need.