JCR Pharmaceuticals Advances Gene Therapy Delivery to CNS and Muscle with Novel Capsid Technology
Wednesday, October 08, 2025
JCR Pharmaceuticals Co., Ltd, a global biopharmaceutical company focused on therapies for rare and genetic diseases, has presented non-clinical data showing that its proprietary JUST-AAV capsid engineering platform can deliver adeno-associated virus (AAV) gene therapies efficiently to the central nervous system (CNS) and muscle while reducing liver exposure. The findings were presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) in Seville, Spain.
The data demonstrate that the JUST-AAV platform improves distribution of gene therapy to muscle in both mice and monkeys, achieving higher transduction in muscle tissue and significantly lower liver tropism compared with conventional AAV vectors. These results highlight the platform’s potential to enhance the safety and effectiveness of AAV-based therapies for CNS and muscle disorders.
JUST-AAV includes a variety of vector types optimised for specific tissues, including liver-sparing, muscle-targeting, and brain-targeting variants. Incorporation of the J-Brain Cargo® technology further improves CNS transduction compared with standard AAV9 capsids, as shown in preclinical studies.
The findings indicate that the JUST-AAV platform could provide more precise, efficient, and safer delivery of gene therapies, advancing treatment options for complex CNS and muscle conditions.
Source: businesswire.com
