FDA Grants RMAT Designation to Genascence's GNSC-001 for Knee OA

Genascence Corporation, a pioneering clinical-stage biotechnology company, has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting the Regenerative Medicine Advanced Therapy (RMAT) designation to its innovative gene therapy, GNSC-001, for knee osteoarthritis (OA). This potential first-in-class therapy targets interleukin 1 (IL-1), a key mediator in OA's pathogenesis, offering sustained inhibition through a single injection. GNSC-001, a recombinant adeno-associated viral vector, expresses an optimized human interleukin-1 receptor antagonist (IL-1Ra), aiming to transform OA treatment by addressing the root cause of inflammation and joint damage. Thomas Chalberg, Ph.D., Genascence's founder and CEO, highlights the therapy's promise and the FDA's recognition of its potential to revolutionize OA care. With successful early trials and a $12 million award from the California Institute for Regenerative Medicine supporting its development, GNSC-001 is on a fast track to late-stage clinical trials, aiming to provide a much-needed solution for millions suffering from this debilitating condition.

Osteoarthritis, affecting over 30 million Americans, represents a significant health and economic burden, with current treatments only managing symptoms without altering disease progression. Genascence's approach, leveraging gene therapy to target OA's underlying mechanisms, could mark a turning point in musculoskeletal disease treatment. The company's collaboration with leading research institutions and its focus on prevalent conditions underscore the potential impact of GNSC-001. For more information on this groundbreaking development, visit Genascence Corporation.