JCR Pharmaceuticals Presents Preclinical Gene Therapy Data that Demonstrate Promising Central Nervous System Uptake at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting

JCR Pharmaceuticals Presents Preclinical Gene Therapy Data that Demonstrate Promising Central Nervous System Uptake at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting

- Research Describes the Potential of JCR’s Platform Technologies, Including JUST-AAV Gene Therapy, to Facilitate Delivery of Therapies to the Central Nervous System -

JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced today that it presented preclinical data from its novel platform technologies, including JUST-AAV gene therapy, in oral and poster sessions at the American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting, held May 11-15, 2026, in Boston, Massachusetts. Additionally, Alexion, AstraZeneca Rare Disease, presented preclinical data from a research collaboration that applies JCR’s JUST-AAV technology in an oral session.

JUST-AAV is JCR’s novel adeno-associated vector (AAV) vector platform technology, which is under preclinical investigation, and is designed to enhance targeted delivery to the central nervous system (CNS) and/or muscle and reduce liver tropism, aiming to improve safety and efficacy of AAV-based gene delivery technologies. JUST-AAV encompasses a range of vector types optimized for various target tissues, including liver-sparing, muscle-targeting, and brain-targeting variants. This proprietary technology holds promise for advancing the field of AAV-based gene therapy.