MILPITAS, Calif.– ASC Therapeutics, a privately-held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies, today announced a partnership with the University of Massachusetts Medical School (UMMS) to co-develop a gene therapy for Maple Syrup Urine Disease (MSUD).
Under the agreement, the research group at UMMS, led by Guangping Gao, PhD, director of the Horae Gene Therapy Center at UMMS, and Dan Wang, PhD, assistant professor of RNA therapeutics, will develop multiple animal models for MSUD, as well as conduct pre-clinical testing using such animal models and proprietary AAV constructs. ASC Therapeutics receives an exclusive option on the selected constructs and intellectual property resulting from this partnership. ASC Therapeutics and UMMS will jointly conduct further IND-enabling studies, regulatory and manufacturing activities to obtain IND clearance and advance this program into clinical stage.
Dr. Ruhong Jiang, PhD, ASC Therapeutics’ CEO, said, “We look forward to working with the UMMS team led by Dr. Gao and Dr. Wang, leading experts for the development and application of AAV technology. This partnership solidifies our gene therapy pipeline and will provide novel genetic therapies to patients with severe unmet medical needs worldwide.”
Dr. Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, Professor of Microbiology & Physiological Systems, and Co-Director of the Li Weibo Institute for Rare Diseases Research, at the University of Massachusetts Medical School, stated, “Our significant progress developing an AAV-based therapy for MSUD will strongly benefit from this partnership by accelerating the progression of this much-needed treatment into the clinic. ASC Therapeutics’ IND clearance by the FDA of a novel gene therapy for hemophilia A confirms their excellence, commitment and expertise in translating preclinical discoveries into clinical programs.”
Dr. Kevin Strauss, MD, Medical Director at the Clinic for Special Children in Pennsylvania, a collaborating clinical expert, added, “We are excited to see a novel treatment being developed for MSUD. Lifelong dietary therapy and episodic hospital management are tedious and difficult, and do not uniformly protect affected individuals from the most life-threatening complications of the disease. Currently, liver transplantation is the only alternative treatment, but is not available in all clinical contexts and entails additional risks of surgery and chronic immune suppression. There is a pressing unmet need for safer, more effective, precision therapies to treat MSUD. At the CSC, we look forward to collaborating with the UMMS and ASC Therapeutics teams to move MSUD gene therapy into clinical trials, creating new hope for patients and their families.”
