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 presented promising preclinical data on its JUST-AAV gene therapy platform at the ASGCT 29th Annual Meeting. The technology aims to enhance delivery to the central nervous system while reducing liver tropism. Results showed improved CNS uptake and therapeutic efficacy in models of GM1 gangliosidosis and neuronal ceroid lipofuscinosis. The findings support the potential for new treatment options for challenging CNS diseases, with plans for first-in-human clinical trials.