The FDA awarded Regenerative Medicine Advanced Therapy (RMAT) designation to GNSC-001, a potential gene therapy from Genascence that blocks interleukin 1 (IL-1) for the treatment of knee osteoarthritis (OA). The product, which received FDA Fast Track designation last year, is a recombinant adeno-associated viral vector expressing an optimized human interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks IL-1 signaling.
IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, and cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single intra-articular injection into the affected joint.
“GNSC-001 is the first IL-1 inhibitor for OA that has been shown to generate IL-1Ra expression levels that maintain therapeutic thresholds long-term following a single administration to inhibit pathogenic IL-1. The FDA RMAT designation for GNSC-001 underscores the strength of the clinical data to date, recognizing its potential to transform the treatment paradigm for OA,” said Thomas Chalberg, PhD, founder and CEO of Genascence. “With the RMAT designation for GNSC-001, we look forward to working closely with the FDA as we seek to accelerate late-stage clinical development of GNSC-001 so we can bring a new treatment option to people suffering from this incapacitating, disabling disease.”
To date, GNSC-001 has been studied in two human clinical trials. The Phase I study was a single-center, open-label, dose-escalation design in nine participants with knee OA. The DONATELLO Phase Ib clinical trial is a double-blind, placebo-controlled dose-ranging study designed to evaluate the safety, tolerability, and pharmacodynamics of a single intra-articular injection of GNSC-001 in patients with OA of the knee that enrolled 67 participants at 10 centers across the U.S.
The RMAT designation is a special FDA program aimed at speeding up the development and review of promising cell and gene therapies under the 21st Century Cures Act. The FDA grants RMAT designation to drug candidates that show potential to treat, modify, reverse, or cure serious or life-threatening diseases and address unmet medical needs. The FDA’s RMAT designation offers sponsor companies the benefits of the fast track and breakthrough therapy designation programs.
RMAT also designation provides accelerated development and review pathways for regenerative medicine products, including early and intensive FDA guidance, potential eligibility for accelerated approval and priority review, and the possibility of rolling review.
There are no currently available therapies known to alter or slow down OA progression, according to Genascence.
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