BCKDHB

First in-vivo gene replacement therapy program for MSUD MSUD is a severe genetic disease with liver transplantation as the only treatment currently available MILPITAS, Calif.--(BUSINESS WIRE)-- 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.” About Maple Syrup Urine Disease Maple syrup urine disease (MSUD) is a rare genetic disorder affecting degradation of the branched-chain amino acids (BCAA) leucine, isoleucine, and valine and their ketoacid derivatives. MSUD is caused by biallelic mutations in one of three genes that encode subunits of the branched-chain ketoacid dehydrogenase complex (BCKDC), namely BCKDHA, BCKDHB, and DBT. Dietary BCAA restriction is the mainstay of treatment but has insufficient efficacy, and affords no protection against episodic and life-threatening encephalopathic crises. Severe (‘classic’) MSUD is fatal without treatment. MSUD affects approximately 1 per 185,000 births worldwide. About ASC Therapeutics ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in-vivo gene editing and allogeneic cell therapies for hematological and other rare diseases. Led by a management team of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing multiple therapeutic programs based on three technology platforms: 1) In-vivo gene therapies, initially focusing on ASC618 for hemophilia A, for which U.S. FDA IND clearance was recently obtained; 2) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 3) Allogeneic cell therapy, with the first indication on Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease. To learn more please visit . About the University of Massachusetts Medical School The University of Massachusetts Medical School (UMMS), one of five campuses of the University system, is comprised of the School of Medicine, the Graduate School of Biomedical Sciences, the Graduate School of Nursing, a thriving research enterprise and an innovative public service initiative, Commonwealth Medicine. Its mission is to advance the health of the people of the Commonwealth through pioneering education, research, public service and health care delivery with its clinical partner, UMass Memorial Health Care. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education. The Medical School attracts more than $266 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2006, UMMS’s Craig C. Mello, PhD, Howard Hughes Medical Institute Investigator and the Blais University Chair in Molecular Medicine, was awarded the Nobel Prize in Physiology or Medicine, along with colleague Andrew Z. Fire, PhD, of Stanford University, for their discoveries related to RNA interference (RNAi). The 2013 opening of the Albert Sherman Center ushered in a new era of biomedical research and education on campus. Designed to maximize collaboration across fields, the Sherman Center is home to scientists pursuing novel research in emerging scientific fields with the goal of translating new discoveries into innovative therapies for human diseases. About the Clinic for Special Children in Pennsylvania The Clinic for Special Children is a non-profit organization located in Strasburg, Pennsylvania, which provides comprehensive primary care and advanced laboratory services to those who live with genetic or other complex medical disorders. Founded in 1989, the organization provides services to over 1,150 individuals annually and is recognized as a world-leader in translational and precision medicine. The organization is primarily supported through community fundraising events and donations. For more information, please visit .