GNSC-001 is first gene therapy for prevalent musculoskeletal diseases like OA

GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection into the affected joint; IL-1 is a key mediator involved in pathogenesis of OA

OA affects more than 30 million Americans and is the leading cause of disability; GNSC-001 may have a role to play in helping these patients in a safe and efficacious manner

PALO ALTO, Calif., Nov. 12, 2024 /PRNewswire/ -- Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation (FTD) for GNSC-001, a potential first-in-class gene therapy for the treatment of patients with osteoarthritis (OA) of the knee.

GNSC-001 is a genetic medicine – a recombinant adeno-associated vector (AAV) expressing an optimized form of interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks interleukin-1 (IL-1) signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, as well as cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection into the affected joint.

"Fast Track designation from the FDA underscores the serious unmet medical need in patients with widespread, debilitating diseases like OA," said Thomas Chalberg, Ph.D., founder and CEO of Genascence. "Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease. Current treatment options are short-term, and while they can provide temporary relief of symptoms, they do not slow down or reverse disease progression. We believe GNSC-001 has potential to deliver transformative results and we are excited to advance our clinical program so we can help patients suffering from this disabling disease."

Fast Track designation is designed to help drugs reach patients faster by facilitating the development and expediting the review of drugs with the potential to fill an unmet medical need and treat serious or life-threatening conditions. Programs receiving FTD benefit from early and frequent interactions with the FDA during the clinical development process and, if relevant criteria are met, the FDA may consider reviewing portions of a marketing application before the sponsor submits the complete application.

Data from the Phase 1 clinical trial of GNSC-001 for the treatment of OA demonstrated that it was well tolerated. The study findings also showed that a single intra-articular injection of GNSC resulted in elevated IL-1Ra expression over baseline in synovial fluid for the length of the 12-month study. Additionally, treatment with GNSC-001 showed a trend in improvement of pain and function scores in all study participants, with a limited amount of disease progression in this first-in-human study.

About Osteoarthritis (OA) of the Knee
Osteoarthritis (OA) is a degenerative joint disease that is the leading cause of disability. It is characterized by destruction of cartilage and structural changes in bone within the joint, which contribute to pain and loss of joint function. Osteoarthritis affects more than 30 million Americans and is increasing as a result of the aging population and increasing prevalence of obesity. Osteoarthritis represents a major economic burden, owing to direct medical costs and loss of productivity. Each year, millions of patients are treated for knee OA with NSAIDs, opioids, and steroid injections into the knee to manage their knee pain. There are no currently available therapies known to alter or slow down OA progression.

About Genascence Corporation
Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence's founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit www.genascence.com.

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SOURCE Genascence