JCR Pharmaceuticals Co, Ltd (TSE 4552) presented promising preclinical data on its adeno-associated virus (AAV) gene therapy research at the European Society of Gene and Cell Therapy's 31st Annual Congress in Rome, held from 22-25 October 2024.

The research leverages JCR's proprietary J-Brain Cargo (JBC) technology, designed to enable gene therapy delivery to the central nervous system (CNS) by crossing the blood-brain barrier.

Key findings demonstrated that the JBC-modified AAV vector achieved more efficient CNS cell transduction in animal models than conventional AAV9 vectors. The application to genetic CNS disease models showed reduced toxic substances, brain improvements, better behavioral outcomes, and increased survival rates. Additional modifications to the JBC-AAV molecule significantly decreased liver transduction, underscoring its targeted delivery advantages.

The JBC-AAV platform's capsid can be customized for specific affinity and uptake, supporting flexible and scalable production. These characteristics position JCR's JBC technology as a potential breakthrough for treating genetic CNS disorders.

JCR continues to develop therapies for rare and genetic diseases worldwide, expanding its impact beyond Japan to the US, Europe and Latin America.