Genascence Corp.

—Accomplished physician-scientist with more than 15 years' biopharmaceutical industry experience in medical affairs, clinical development, and drug development —Visionary leader skilled at leveraging scientific and clinical expertise to build innovative first-in-class therapeutic programs in areas of osteoarthritis, immunology, oncology, and neuroscience PALO ALTO, Calif., Oct. 29, 2024 /PRNewswire/ -- Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced the appointment of Jeymi Tambiah, MBChB, FRCS, MS, FAPCR, MFPM(Dis) as its chief medical officer. Dr. Tambiah is an accomplished physician-scientist with more than 15 years' biopharmaceutical industry experience creating and leading strategies for medical affairs, clinical development, and drug development programs. He also brings deep experience in translational medicine, commercialization, and health outcomes research to Genascence. Continue Reading Jeymi Tambiah, Chief Medical Officer, Genascence "Jeymi is a visionary leader skilled at leveraging scientific and clinical expertise to build innovative first-in-class therapeutic programs in the areas of osteoarthritis, immunology, oncology, and neuroscience and I am excited to welcome him to Genascence as our chief medical offer," said Thomas Chalberg, Ph.D., founder and CEO of Genascence. "His experience includes all phases of drug development from bench through Phase 1-4 clinical studies and building elite high performance medical affairs teams. Jeymi's development and research expertise, particularly within the osteoarthritis research community, will be invaluable as we execute on our strategy to bring gene therapy to people suffering from prevalent musculoskeletal diseases." "Genascence is a company that is transforming how we treat debilitating musculoskeletal diseases like osteoarthritis by pioneering the development of gene therapy," said Dr. Tambiah. "I am thrilled to join this outstanding team under Tom's leadership and apply my knowledge and experience to advance our clinical program so that we can bring potentially curative therapies to patients in desperate need of better treatments." Before joining Genascence, Jeymi was senior vice president of clinical development and external innovation at Biosplice Therapeutics, a start-up biotech developing molecules modulating mRNA splicing in oncology, arthritis, and neurodegenerative diseases, where he was primarily responsible for clinical development, medical affairs, and external thoughtleader and scientific partnerships. Previously, Jeymi worked for UCB Pharma in various roles of increasing leadership within medical affairs, launching the TNF-inhibitor certolizumab pegol within the European Union, U.K., and U.S. across multiple immunology indications. Before his move into pharma, Jeymi trained and practiced in cardiothoracic surgery at Guys' and St. Thomas' Hospitals in London. Jeymi is very active in the osteoarthritis research community, with numerous research awards and publications, holding many committee appointments, and regularly presenting at international conferences. He is the North American representative for the Faculty of Pharmaceutical Medicine UK, and in this role is dedicated to supporting the education and mentoring of physicians in drug development. Jeymi earned his medical degree from the University of Manchester, UK, a doctorate degree in vascular biology from Imperial College London where he was a Wellcome Research Fellow, and a BSc (Hons) in medical science from the University of St. Andrews, UK. He is a fellow of the Royal College of Surgeons of England and Edinburgh, a member of Faculty of Pharmaceutical Physicians UK by Distinction, and a fellow of the Academy of Physicians in Clinical Research. 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 . SOURCE Genascence WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ROSEMONT, Ill. , Feb. 2, 2024 /PRNewswire/ -- The 2024 Orthopaedic Research and Education Foundation (OREF) Clinical Research Award was presented to Christopher H. Evans, PhD (Mayo Clinic), Steven C. Ghivizzani, PhD (University of Florida), and Paul D. Robbins, PhD (University of Minnesota), for their research on local gene therapy for osteoarthritis (OA). Together, the team spearheaded the research from a laboratory concept to human clinical trials over the course of 30 years. The OREF Award recognizes outstanding clinical research related to musculoskeletal disease or injury. Continue Reading The 2024 Orthopaedic Research and Education Foundation (OREF) Clinical Research Award was presented to Christopher H. Evans, PhD (Mayo Clinic), Steven C. Ghivizzani, PhD (University of Florida), and Paul D. Robbins, PhD (University of Minnesota), for their research on local gene therapy for osteoarthritis (OA). To read more about the award, please click here. According to the Centers for Disease Control and Prevention, OA is the most common form of arthritis, affecting 32.5 million Americans and inflicting a significant economic burden on patients and the economy.i OA, which is caused by the breakdown of joint cartilage between bones, can cause pain, stiffness and swelling, potentially leading to reduced function or even disability.i There is no cure for OA and treatment options are limited. Many patients eventually receive a total joint replacement. When the researchers began to study the use of gene therapy—which modifies a person's genes to treat or cure diseaseii—for arthritis and other non-lethal diseases, gene therapy clinical trials were in the early stages and had only involved cancer and rare genetic diseases. "Gene therapy was focused on curing genetic diseases when we entered the field," said Dr. Evans, John and Posy Krehbiel Professor of Orthopedics, Mayo Clinic, and Professor of Molecular Medicine, Mayo Clinic Alix School of Medicine in Rochester, Minn. "At the time, studying gene therapy for arthritis was radically different because, instead of treating a genetic disease, we were looking at treating a non-genetic one, albeit one of the most common diseases on the planet. This was not a genetic fix, but we wanted to explore a sophisticated way of delivering anti-arthritic gene products to those who need therapy as there were not many treatment advances for OA." Evolution from Concept to Clinical Trials Dr. Evans and his colleagues targeted rheumatoid arthritis (RA) when they began their research 30 years ago at the University of Pittsburgh as, unlike with OA, there was significant information about the biology of the rheumatoid joint. The team accomplished a major milestone, the first-in-human transfer of a gene to a joint, made possible by several articular (joint) gene transfer technologies developed by Drs. Evans, Ghivizzani and Robbins. However, when numerous successful non-genetic RA treatments were introduced, it reduced the need for gene therapy for RA. The technologies and key learnings from the RA research were redirected to a new disease indication: OA. To transfer genes, vectors are used, which are essentially vehicles that deliver therapeutic genetic materials directly into a cell.iii The research team explored using self-complementary adeno-associated virus (scAAV), a small virus that can be engineered to deliver DNA to target cells,iv as it caused no known human disease and allowed for delivery directly to the joints. Dr. Evans and his colleagues developed the scAAV.IL-1Ra vector as a therapeutic agent for OA. Following animal studies that showed the efficacy of scAAV.IL-1Ra in joints with OA, a successful Investigational New Drug application was filed with the U.S. Food and Drug Administration (FDA) in 2015. In a Phase I clinical trial, nine patients (three cohorts of three patients) were injected with scAAV.IL-1Ra in escalating doses of 1011 (low dose), 1012 (medium dose), or 1013 (high dose) by an intra-articular injection into one knee joint with OA. Eligible patients had mid-stage disease, symptomatic OA and failed at least two conservative treatments prior to the study. Patients reported pain (visual analog scale, VAS) and function (Western Ontario MacMaster University Osteoarthritis Index, WOMAC) and were followed for one year. Outcomes included: No serious adverse events were reported. Patients reported improved symptoms based on VAS and WOMAC. Patients who received the lowest dose reported mild and temporary improvement. Patients who received the medium and high doses saw sustained symptomatic improvement during the entire 12-month follow-up timeframe. Expression of IL-1Ra, a natural anti-inflammatory protein, was higher in the high-dose and medium-dose groups. This elevated expression in these groups was sustained through the 12 months of follow up, suggesting that the 1012 dose could be the most cost-effective dose, but further investigation is needed. "This was the first study that used localized gene therapy, meaning we could deliver the therapeutic target directly to the joint, which has enormous implications for safety and significantly reduces the cost associated with this procedure/therapy," said Dr. Evans. "By focusing on the joint, you don't have to treat the whole body as many of the previous vectors delivered systemically go straight to the liver, causing liver damage. The amount of vector we delivered to the knee is significantly less than what is used in systemic diseases, which cuts down on the cost dramatically." Future Studies The team is currently enrolling for a Phase Ib trial with 50 patients who have mid-stage OA. The study will include a placebo group and examine how the level and persistence of IL-1Ra expression in the joint is affected by immune suppression. It will also determine the appropriate vector dose – 1012 or 1013. If the 1012 dose is found to be the most effective, it could mean a ten-fold lower cost and less risk of adverse events for patients. Due to the expense of gene therapy trials, Drs. Evans, Ghivizzani and Robbins co-founded an arthritis gene therapy startup company, Genascence Corporation, which will help fund future clinical trials. About the OREF Clinical Research Award The OREF Clinical Research Award was established in 1995 to recognize outstanding clinical research related directly to musculoskeletal disease or injury. All submitted manuscripts are reviewed, graded and selected by the AAOS Research Development Committee. The award provides $20,000 to recipients. For more information about the manuscript submission process, please visit aaos.org/kappadelta. About the OREF The Orthopaedic Research and Education Foundation (OREF) is an independent, 501(c)3 non-profit organization that raises funds to support research on diseases and injuries of bones, nerves and muscles and to enhance clinical care leading to improved health, increased activity and a better quality of life for patients. To further its mission, OREF is committed to exploring ways to partner with others to move the field of musculoskeletal research forward. For more information, visit . About the AAOS With more than 39,000 members, the American Academy of Orthopaedic Surgeons is the world's largest medical association of musculoskeletal specialists. The AAOS is the trusted leader in advancing musculoskeletal health. It provides the highest quality, most comprehensive education to help orthopaedic surgeons and allied health professionals at every career level to best treat patients in their daily practices. The AAOS is the source for information on bone and joint conditions, treatments and related musculoskeletal healthcare issues; and it leads the healthcare discussion on advancing quality. Follow the AAOS on Facebook, X, LinkedIn and Instagram. Disclosure Funding and Conflicts of Interest Drs. Evans, Ghivizzani and Robbins are co-founders of Genascence Corp., an arthritis gene therapy company Funding Sources: NIH Grants R01 AR43623 R21 AR049606 R01 AR048566 R01 AR057422 R01AR051085 AR048566-S X01NS066865 DoD Grants W81XWH-16-1-0540 W81XWH-14-1-0498 W81XWH-19-1-0515 i Centers for Disease Control and Prevention. Osteoarthritis (OA). . Reviewed 7/27/2020. Accessed 12/20/2023. ii U.S. Food and Drug Administration. What is Gene Therapy? . Published 7/25/2018. Accessed. 12/20/2023. iii American Society of Gene +Cell Therapy. Vectors 101. . Updated 11/05/2021. Accessed 12/21/2023. iv Naso MF, Tomkowicz B, Perry WL 3rd, Strohl WR. Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. BioDrugs. 2017 Aug;31(4):317-334. SOURCE American Academy of Orthopaedic Surgeons

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 PALO ALTO, Calif., Jan. 3, 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. For more information, please visit . SOURCE Genascence