JCR Pharma & Modalis Validate Breakthrough CNS Gene Therapy Concept

JCR Pharmaceuticals Co., Ltd., a worldwide specialty pharmaceuticals firm, and Modalis Therapeutics Corporation, a leader in epigenetic medicine, have revealed that they have confirmed the preliminary proof of concept in a joint research programme aimed at creating a new gene therapy for a central nervous system (CNS) disorder. As a result of the successful collaboration so far, Modalis and JCR have decided to advance to the next stage of their research by establishing a new joint research agreement.

The aim of this collaborative agreement is to perform pre-clinical studies aimed at developing a novel gene therapy for the unspecified CNS disease using J-Brain Cargo, JCR’s exclusive technology that can traverse the blood-brain barrier (BBB), alongside a gene therapy payload utilizing CRISPR-GNDM (Guide Nucleotide-Directed Modulation), an epigenome modulation technology from Modalis that does not cut or modify DNA sequences.

The two firms initiated a joint research partnership in December 2023 to assess the gene therapy's drug delivery technology to the CNS.

“I am pleased to share the progress we’ve achieved in our collaboration with Modalis,” said Hiroyuki Sonoda, Ph.D., director, senior managing executive officer, and executive director, research division at JCR. “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.”