PALO ALTO, Calif., May 5, 2021 /PRNewswire/ — Genascence Corporation, a clinical-stage biotechnology company developing life-changing gene therapy products for prevalent musculoskeletal diseases, today announced that dosing across all three cohorts has been completed in the Phase 1 clinical trial of GNSC-001 for the treatment of osteoarthritis (OA).
GNSC-001 is an adeno-associated vector (AAV) carrying a coding sequence for interleukin-1 receptor antagonist (IL-1Ra), a potent inhibitor of interleukin-1 (IL-1) signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation 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.
Initial data from the Phase 1 Study will be presented at the American Society of Gene and Cell Therapy’s (ASGCT) 24th Annual Meeting taking place May 11-14, 2021 in virtual format. Presentation details are as follows:
Presentation Title: Osteoarthritis Gene Therapy: Interim Report from a Phase I Clinical Trial (NCT02790723)
Abstract number: 595
Available online: May 11-14, 2021
Speaker: Christopher H. Evans, Ph.D.
Registration and meeting details available at www.asgct.org
Interim safety data results show that GNSC-001 was safe and well tolerated, with adverse events being absent (8/9) or mild/moderate (1/9) in all treated subjects. Blood chemistry and hematology markers have remained normal, with no evidence of the neutropenia that can accompany systemic delivery of IL-1 inhibitors. Immunology and biodistribution studies have been consistent with pre-clinical observations and as expected for local administration of AAV therapy.
“These are important clinical milestones in the development of GNSC-001 as a potential gene therapy for OA,” said Thomas Chalberg, Ph.D., Co-Founder and Chief Executive Officer of Genascence. “Osteoarthritis patients have limited treatment options and nothing is currently available that is able to slow down progression of this disabling disease. Patients endure years of pain and disability, and OA represents an enormous burden on patients and the healthcare system as a whole.”
The Phase 1 Study for GNSC-001 is an investigator-sponsored clinical trial. In this single arm, open label, dose escalation clinical trial, a total of nine subjects with knee OA were enrolled. Three subjects were treated in each of three cohorts, receiving either 1×1011 vg, 1×1012 vg, or 1×1013 vg GNSC-001 delivered by intra-articular injection. The primary endpoint is safety and tolerability. The study was supported by funding from the U.S. Department of Defense Peer Reviewed Medical Research Program (PRMRP). More information is available at https://clinicaltrials.gov/ct2/show/NCT02790723.
“The completion of enrollment and interim safety data in this clinical trial marks an important milestone in the development of potential new options for patients with osteoarthritis,” said Philip Conaghan, MD, Professor of Musculoskeletal Medicine at the University of Leeds, U.K. “The initial safety data are encouraging. Based upon our understanding of the role of IL-1 in osteoarthritis, we believe that this therapy has the potential to improve symptoms and possibly reduce structural disease progression in osteoarthritis patients.”
Following the Phase 1 trial, Genascence plans to initiate a Phase 2 trial to further evaluate the safety and efficacy of GNSC-001 in osteoarthritis. Genascence has filed a company-sponsored IND for the Phase 2 study, which has been cleared by US FDA.
Genascence was founded in 2017, with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. The company completed a seed round of financing in 2019 led by Polymerase Capital. Genascence’s founders and leadership team have deep experience in the design, development and manufacturing of successful gene therapies and biological medicines.
- Thomas Chalberg, Ph.D. – Co-Founder, Chairman, and Chief Executive Officer. Founder and Managing Director, Polymerase Capital; Founder/CEO of SightGlass Vision; Chief Operating Officer of Oncorus; Founder/CEO of Avalanche Biotechnologies
- Christopher Evans, Ph.D. – Co-Founder. John and Posy Krehbiel Professor of Orthopedics and Director of Musculoskeletal Gene Therapy Research Laboratory at Mayo Clinic
- Steven Ghivizzani, Ph.D. – Co-Founder. Professor and Director Orthopedic Gene Therapy, University of Florida College of Medicine
- Paul Robbins, Ph.D. – Co-Founder. Professor of Biochemistry, Molecular Biology, and Biophysics, University of Minnesota
- Mukundan Attur, Ph.D. – Consultant and Scientific Advisory Board. Associate Professor, Dept. of Medicine at NYU Grossman School of Medicine
- Philip Conaghan, M.D. – Consultant and Scientific Advisory Board. Professor of Musculoskeletal Medicine, University of Leeds, U.K.
- Annahita Keravala, Ph.D. – Consultant and Scientific Advisory Board. SVP and Head of Gene Therapy, CODA Biotherapeutics; Associate VP and Head of AAV, Rocket Pharmaceuticals; Director of Research, Adverum Biotechnologies
- John Fraser Wright, Ph.D. – Consultant and Scientific Advisory Board. Professor of Pediatrics in the Center for Definitive and Curative Medicine at Stanford University; Scientific Co-Founder of Kriya Therapeutics; Co-Founder and former Chief Technology Officer of Spark Therapeutics
About Osteoarthritis (OA) of the Knee
Osteoarthritis (OA), or degenerative joint disease, 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 GNSC-001
GNSC-001 is an adeno-associated vector (AAV) carrying a coding sequence for interleukin-1 receptor antagonist (IL-1Ra), a potent inhibitor of IL-1 signaling. IL-1 is a pro-inflammatory cytokine thought to contribute to inflammation and pain as well as cartilage degeneration in osteoarthritis. GNSC-001 is administered as a one-time intra-articular injection, which is a straightforward procedure performed in a physician’s office. GNSC-001 is currently being developed for osteoarthritis of the knee.
About Genascence Corporation
Genascence is a clinical-stage biotechnology company developing life-changing gene therapy products for highly prevalent musculoskeletal diseases affecting millions of patients. Genascence is headquartered in Palo Alto, California. For more information, please visit www.genascence.com.
Disclosure
Dr. Evans and Mayo Clinic have a financial interest related to this research. This research has been reviewed by the Mayo Clinic Conflict of Interest Review Board and is being conducted in compliance with Mayo Clinic Conflict of Interest policies.
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SOURCE Genascence
