Modalis Therapeutics has entered into a collaborative research agreement with JCR Pharmaceuticals, focusing on the joint evaluation of gene therapy drug delivery technology targeting the central nervous system (CNS).

The collaboration is aimed at developing new gene therapies for undisclosed CNS diseases by leveraging JCR Pharmaceuticals’ proprietary technology, J-Brain Cargo, designed for crossing the blood-brain barrier (BBB).

Modalis will contribute its proprietary epigenome modulation technology, CRISPR-GNDM (Guide Nucleotide-Directed Modulation), which does not require DNA cleavage.

The strategic collaboration, prompted by the research agreement, aims to harness the strengths of both organisations to pioneer innovative gene therapies.

J-Brain Cargo’s capability to traverse the BBB is a crucial aspect, coupled with Modalis’ CRISPR-GNDM technology, offering a unique approach that eliminates the need for DNA cleavage.

Modalis, founded in 2016 and rooted in technology originating from the University of Tokyo, conducts its research and development activities in Massachusetts, US.

The company is dedicated to developing therapeutics for individuals grappling with severe genetic disorders, including muscle diseases, CNS diseases, and cardiomyopathies.

The gene therapy landscape has seen a surge in efforts to develop novel therapeutics with enhanced efficacy and safety, especially in utilising tissue tropic drug delivery technology. While Modalis has historically concentrated on intra-cisterna magna (ICM: intracranial) administration for CNS drug delivery, the industry trend is shifting towards minimally invasive methods like intravenous injection (IV).

The joint research between Modalis and JCR Pharmaceuticals represents a pivotal step towards exploring the application of J-Brain Cargo, to establish new gene therapies, particularly in conjunction with the less invasive CRISPR-GNDM approach.

Modalis CEO Haru Morita said: “JCR Pharmaceuticals’ proprietary BBB penetrating technology, J-Brain Cargo, has successfully and efficiently delivered biopharmaceutical candidates of various modalities to the CNS.

“As a pioneer in developing CRISPR-based epigenome editing technology (CRISPR-GNDM), we have demonstrated long-term drug efficacy and functional improvement in animal disease model studies, including demonstration of target engagement and safety in NHPs.

“We believe that leveraging these complementary technologies and strengths of our two companies will lead to the development of innovative new gene therapeutics.”