Genascence Announces First Close in Series A Financing led by Pacira BioSciences and Expands Board of Directors and Scientific Advisory Board

• Proceeds will be used to advance clinical program of GNSC-001, company's lead candidate in osteoarthritis (OA)
• Brian Kotzin, M.D., and Jackson Streeter, M.D., join Genascence's board of directors
• Company expands Scientific Advisory Board of internationally recognized leaders in musculoskeletal diseases and medicine, gene therapy, molecular physiology, and molecular biology
• Additional data from Phase 1 clinical trial of GNSC-001 in OA to be presented at upcoming American Society of Gene & Cell Therapy annual meeting

PALO ALTO, Calif., May 10, 2022 /PRNewswire/ -- Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced it has completed the first closing in a Series A financing led by Pacira BioSciences, Inc., totaling $10.5 million. Additional investors included Polymerase Capital, DeepWork Capital, and University of Florida Research Foundation.

Proceeds of the financing will be used to accelerate clinical development of GNSC-001, the company's lead program in osteoarthritis (OA). GNSC-001 is a genetic medicine – a recombinant 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.

"We are thrilled to have support from this outstanding syndicate led by Pacira BioSciences, one of the world's leading non-opioid pain management companies," said Thomas Chalberg, Ph.D., founder and CEO of Genascence. "Osteoarthritis affects more than 30 million Americans and is the leading cause of disability and chronic pain, as well as a significant risk factor for opioid addiction. Patients have limited treatment options, and nothing is currently available that is able to slow down progression of this disabling disease. This financing will position us to progress the GNSC-001 program and build out our pipeline. I look forward to working with our tremendous co-founders and board of directors, world-renowned scientific advisory board, and leadership team to advance our pipeline so that we can deliver transformative results for patients suffering from musculoskeletal conditions like OA as quickly as possible."

"We are extremely excited to back the Genascence team and their groundbreaking gene therapy candidate as they take on osteoarthritis,  one of the largest problems in all of medicine and the leading cause of disability and chronic pain," said Ron Ellis, DO, senior vice president of business development, Pacira Biosciences. "We believe Genascence could transform the treatment paradigm for prevalent musculoskeletal diseases like OA and help the many patients around the world who are most in need."

As part of the financing, Brian Kotzin, M.D., senior vice president and chief medical officer at Nektar Therapeutics, and Jackson Streeter, M.D., partner, DeepWork Capital and director, University of Florida Ventures, join Genascence's board of directors. Dr. Chalberg was elected chairman of the board.

Genascence announced the expansion of its Scientific Advisory Board of internationally recognized leaders in musculoskeletal diseases and medicine, gene therapy, molecular physiology, and molecular biology. These experts join Genascence's original Scientific Advisory Board members, Drs. Attur, Conaghan, and Wright, and scientific founders in advising the company's research and development.

• Mukundan Attur, Ph.D. – associate professor, department of medicine, NYU Grossman School of Medicine
• Philip Conaghan, M.D. – professor of musculoskeletal medicine, University of Leeds, U.K.
• Marc C. Hochberg, M.D., MPH, MACP, MACR – professor of medicine and epidemiology & public health, head, division of rheumatology and clinical immunology, and vice chair, department of medicine, University of Maryland School of Medicine, and director, Medical Care Clinical Center, Veteran Affairs Maryland Health Care System
• Virginia Byers Kraus, M.D, Ph.D. – professor of medicine, pathology, and orthopaedic surgery, and faculty member, Duke Molecular Physiology Institute, Duke University School of Medicine
• Nancy E. Lane, M.D. – endowed professor of medicine, rheumatology, and aging research, director of the Center for Musculoskeletal Health, 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
• Thomas J. Schnitzer, M.D., Ph.D. – professor of physical medicine and rehabilitation, anesthesiology, and medicine (rheumatology), Northwestern University Feinberg School of Medicine
• John Fraser Wright, Ph.D. – professor of pediatrics in the Center for Definitive and Curative Medicine, Stanford University; scientific co-founder of Kriya Therapeutics; co-founder and former chief technology officer, Spark Therapeutics

Genascence was founded in 2017, in Palo Alto, California, by a team of experts and industry leaders with deep experience and proven track record in the design, development, and manufacturing of successful gene therapies and biological medicines, and company building.

• Thomas Chalberg, Ph.D. – founder and CEO; managing director, Polymerase Capital; founder and CEO, SightGlass Vision; chief operating officer, Oncorus, Inc.; founder and CEO, Avalanche Biotechnologies
• Christopher H. Evans, Ph.D. – founder; John and Posy Krehbiel Professor of Orthopedics and director of the Musculoskeletal Gene Therapy Research Laboratory, Mayo Clinic
• Steven Ghivizzani, Ph.D. – founder; professor and director of orthopedic gene therapy, University of Florida College of Medicine
• Annahita Keravala, Ph.D. – founder; senior vice president and head of gene therapy, CODA Biotherapeutics; associate vice president and head of AAV platform, Rocket Pharmaceuticals; director of research, Adverum Biotechnologies
• Paul Robbins, Ph.D. – founder; professor of biochemistry, molecular biology, and biophysics, University of Minnesota

The company completed a seed round of financing in 2019 led by Polymerase Capital.

In addition, the company announced that additional data from the Phase 1 clinical trial of GNSC-001 for the treatment of osteoarthritis (OA), including 12-month follow up on all subjects, will be presented in a poster session at the upcoming American Society of Gene & Cell Therapy's (ASGCT) 25^th Annual Meeting being held virtually and in-person May 16-19, 2022, in Washington, D.C. Presentation details are as follows:

Title: A Phase I Trial of Osteoarthritis Gene Therapy (NCT02790723) 
Date: May 17, 2022 5:30-6:30 PM ET 
Session: Gene and Cell Therapy Trials in Progress 
Abstract Number: 799 
Location: Hall D 
Presenter: Christopher H. Evans, Ph.D.

In this investigator-sponsored Phase 1 single-arm, open-label, dose-escalation clinical trial of GNSC-001, a total of nine subjects with knee OA were enrolled. Three subjects were treated in each of three cohorts, receiving either 1x10¹¹ vg, 1x10¹² vg, or 1x10¹³ 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.

Abstracts can be accessed via the conference website at annualmeeting.asgct.org.

Osteoarthritis (OA), or degenerative joint disease, is the leading cause of disability and chronic pain, as well as a significant risk factor for opioid addiction. 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.

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