JCR Pharmaceuticals and Modalis Therapeutics have validated the initial proof of concept in a joint research program for the development of a novel gene therapy for a central nervous system (CNS) disease.

Due to the success of the partnership thus far, Modalis and JCR have agreed to proceed to the next phase of their research by entering into a new joint research agreement.

The purpose of this joint agreement is to conduct pre-clinical studies for the development of a new gene therapy for the undisclosed CNS disease by applying J-Brain Cargo®, JCR’s proprietary technology that is able to cross the blood-brain barrier (BBB), and a gene therapy payload based on CRISPR-GNDM® (Guide Nucleotide-Directed Modulation), Modalis’ proprietary epigenome modulation technology which does not cleave or alter DNA sequences.

The two companies began a joint research collaboration in December of 2023 to evaluate the drug delivery technology of the gene therapy to the CNS. As a result of this research, the initial proof of concept has been validated. The next phase of the agreement is to jointly develop a novel and innovative gene therapy to provide patients with improved efficacy, safety, and less burden via intravenous injection (IV) in a minimally invasive and efficient manner.

“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.

“We are very happy to move the joint research collaboration with JCR to the next phase. This is the result of hard work on both companies and an important strategic step for Modalis’ research activities. As a pioneer in this technology, we have demonstrated promising long-term drug efficacy in mouse disease model studies, including demonstration of target engagement and safety in non-human primates, 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 very significant breakthrough to maximize the potential and value of our proprietary epigenome editing technology (CRISPR-GNDM®) in CNS diseases,” said Haruhiko Morita, CEO of Modalis.