Genascence Receives $11.6 Million Award From California Institute for Regenerative Medicine to Help Advance its Lead Gene Therapy Program in Knee Osteoarthritis

• Funding over four years will support Phase 1b clinical trial of GNSC-001, as well as phase-appropriate manufacturing activities
• Osteoarthritis affects more than 30 million Americans, and is leading cause of disability

PALO ALTO, Calif., Feb. 23, 2023 /PRNewswire/ -- Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced it has been awarded $11.6 million over four years from the California Institute for Regenerative Medicine (CIRM). The funding will be used to support Genascence's Phase 1b clinical trial of GNSC-001 in knee osteoarthritis (OA), as well as phase-appropriate manufacturing activities.

"We applaud the Genascence team for tackling one of the most prevalent degenerative diseases. If successful, Genascence's novel gene therapy approach could usher in a step-change in how we treat OA and other musculoskeletal diseases, which are among the leading causes of disability in California and around the world," said Maria T. Millan, M.D., President and CEO of CIRM.

The Phase 1b clinical trial entitled, "A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study Evaluating GNSC-001 Safety, Pharmacodynamics, and Biomarkers in Knee Osteoarthritis," is designed to enroll 50 patients with OA. The study will assess the safety and pharmacodynamics of GNSC-001 at two dose levels, as well as evaluate the effect of GNSC-001 on symptoms and biomarkers of disease progression over time.

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.

"We are unwavering in our commitment to developing novel therapies for widespread, debilitating diseases like OA. Current treatment options are short-term, and while they can provide temporary relief of symptoms, they do not slow down or reverse disease progression," said Thomas Chalberg, Ph.D., Founder and CEO of Genascence. "We are honored to have CIRM award this funding to help advance GNSC-001, our lead clinical program in OA where we hope to demonstrate transformative results for patients."

Dr. Chalberg is serving as the program's principal investigator. Co-investigators include: Annahita Keravala, Ph.D., Founder and Chief Scientific Officer of Genascence; Christopher H. Evans, the John and Posy Krehbiel Professor of Orthopedics and Director of the Musculoskeletal Gene Therapy Research Laboratory at the Mayo Clinic, and Founder of Genascence; Philip Conaghan, M.D., Professor of Musculoskeletal Medicine, University of Leeds, U.K.; and Nancy E. Lane, M.D., Distinguished Professor of Medicine, Rheumatology, and Aging Research and Director of the K12 National Institutes of Health (NIH) Building Interdisciplinary Research Careers in Women's Health at the University of California at Davis School of Medicine.

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 the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to www.cirm.ca.gov

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.

SOURCE Genascence