Genascence Announces Initiation of Phase 1b Clinical Trial of GNSC-001 Gene Therapy for Knee Osteoarthritis (OA)

Phase 1b clinical trial in the U.S. is actively recruiting patients with OA at 10 sites

Initial six-month data expected fourth quarter of 2024

Genascence Corporation (“Genascence”), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced the initiation of its Phase 1b clinical trial of GNSC-001 for the treatment of knee osteoarthritis (OA) (DONATELLO). The study is now actively enrolling at ten clinical centers throughout the United States. A total of approximately 50 patients will participate, with trial enrollment expected to complete in the first quarter of 2024.

GNSC-001 is a genetic medicine – a recombinant adeno-associated viral vector expressing an optimized form of 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.

“Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease, and there are no currently available treatments to slow down disease progression,” said Thomas Chalberg, Ph.D., founder and CEO of Genascence. “We are excited that our Phase 1B DONATELLO clinical trial is now fully up and running at 10 clinical sites across the U.S. This is a critical next step toward the development of the first gene therapy for prevalent musculoskeletal diseases like OA.”

“Our GNSC-001 gene therapy has the potential to transform the treatment landscape for individuals suffering from OA,” said Lachy McLean, M.D., Ph.D., chief medical officer of Genascence. “We look forward to sharing initial six-month data from the DONATELLO clinical trial in the fourth quarter of 2024 and continue advancing our clinical program so we can bring a new option to patients as quickly as possible.”

The DONATELLO Phase 1b clinical trial (NCT05835895) is a randomized, double-blinded, 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. The study is targeted to enroll approximately 50 patients with OA at 10 centers across the U.S. Initial six-month data of the DONATELLO clinical trial are expected in the fourth quarter of 2024.

About Osteoarthritis (OA) of the Knee

Osteoarthritis (OA) is a progressive 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.

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