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

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.

From PR News:

PackGene is a world-leading AAV vector CRO and CDMO company. We provides economical, reliable, and scalable plasmid DNA and AAV viral vector production for early-stage drug discovery, preclinical development and clinical trials for Cell and Gene Therapy (CGT). If you want more information about our AAV services, please contact us.