JCR Pharmaceuticals And Modalis Therapeutics Advance CNS Gene Therapy With Collaborative Research Agreement

JCR Pharmaceuticals Co., Ltd. and Modalis Therapeutics Corporation have announced a significant milestone in their joint effort to develop a novel gene therapy for a central nervous system (CNS) disease. The companies have successfully validated the initial proof of concept, prompting them to advance their partnership by signing a new joint research agreement.

“I am pleased to share the progress we’ve achieved in our collaboration with Modalis. By combining our proprietary J-Brain Cargo® technology for BBB penetration with Modalis’ CRISPR-GNDM® platform for epigenome editing, we are opening the door to innovative therapeutic possibilities that could make a meaningful difference,” said Hiroyuki Sonoda, PhD, Director, Senior Managing Executive Officer, and Executive Director, Research Division at JCR

This new agreement aims to conduct pre-clinical studies aimed at developing a gene therapy for the unnamed CNS condition. This project will leverage JCR’s proprietary J-Brain Cargo® technology, which enables drugs to cross the blood-brain barrier (BBB), along with Modalis’ CRISPR-GNDM® technology. This unique gene modulation platform can regulate gene expression without cutting or altering DNA sequences.

Haruhiko Morita, CEO of Modalis, said in a statement, “We are very happy to move the joint research collaboration with JCR to the next phase. This results from hard work on both companies and is an important strategic step for Modalis’ research activities. As pioneers in this technology, we have demonstrated promising long-term drug efficacy in mouse disease model studies, including target engagement and safety in non-human primates and exhibiting strong biodistribution for our lead program in neuromuscular disorders. We believe that CRISPR-GNDM® has huge potential in the field of CNS diseases. So, the combination with J-Brain Cargo® technology could be a significant breakthrough to maximise the potential and value of our proprietary epigenome editing technology (CRISPR-GNDM®) in CNS diseases.” 

The collaboration between JCR and Modalis began in December 2023, focusing on evaluating gene therapy delivery to the CNS. Thanks to promising early results, the two companies are now moving forward to co-develop a novel treatment. They aim to create a more effective, safer, and less burdensome therapy for patients by utilising intravenous (IV) delivery, offering a minimally invasive and efficient treatment option.