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JCR Pharmaceuticals Enhances Delivery of AAV Gene Therapy to Central Nervous System and Muscle with Novel Capsid Engineering Platform Technology
HYOGO, Japan, October 07, 2025--JCR presented non-clinical data that demonstrate the ability of its proprietary JUST-AAV platform to achieve efficient delivery of AAV gene therapy.
About this update from Jcr Pharmaceuticals Co., Ltd.
[{"type":"image","alt":"","displaySize":"","headline":null,"caption":"","credit":null,"className":"","disableSlideshowImg":false,"size":{"original":{"width":480,"height":251,"url":"https://media.zenfs.com/en/business-wire.com/9fdec4b27e0c32bbd8e7e7cfb9aa04e3"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/.bQFDcP4NSg_J50yOKJAxA--/YXBwaWQ9aGlnaGxhbmRlcjt3PTk2MDtoPTUwMjtjZj13ZWJw/https://media.zenfs.com/en/business-wire.com/9fdec4b27e0c32bbd8e7e7cfb9aa04e3","width":480,"height":251}},"lazy":false},{"type":"text","content":"-Oral Presentation at ESGCT Highlights Potential of JCR’s Proprietary JUST-AAV Platform to Facilitate More Efficient Delivery While Reducing Liver Tropism-","length":155,"tagName":"p","attribs":{}},{"type":"text","content":"HYOGO, Japan, October 07, 2025--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today presented non-clinical data demonstrating the ability of its proprietary JUST-AAV capsid engineering platform to achieve efficient delivery of adeno-associated virus (AAV) gene therapy to the central nervous system (CNS) and muscle while reducing liver exposure. The data, presented at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress, in Seville, Spain, showcase the platform’s potential to overcome longstanding challenges in treating CNS and muscle disorders.","length":712,"tagName":"p"},{"type":"text","content":"In an oral presentation, JCR researchers reported that the JUST-AAV platform enables the efficient delivery of JUST-AAV into the muscle in mice and monkeys, when compared to the conventional AAV. The JUST-AAV was modified to improve the distribution into the muscle. It achieved higher transduction in muscle, while it showed reduced tropism drastically in the liver. It indicates the platform’s potential to improve the safety and efficacy of AAV-based gene delivery technologies to target tissues.","length":499,"tagName":"p"},{"type":"text","content":""Our JUST-AAV capsid engineering platform points the way toward development of new, more precisely targeted gene therapies for central nervous system diseases that have long been considered out of reach," said Hiroyuki Sonoda, Ph.D., Director, Senior Managing Executive Officer, and Executive Director of th...