Modalis, JCR Pharma renew CNS gene therapy pact

Following the validation of the initial proof of concept, JCR Pharmaceuticals and Modalis Therapeutics are advancing to the next phase of research for development of a novel gene therapy for a central nervous system disease with a new agreement.

Japanese rare disease specialist JCR Pharma signed an initial joint research collab with CRISPR technology company Modalis Therapeutics in December 2023 to combine JCR’s proprietary J-Brain Cargo technology for blood-brain barrier penetration with Modalis’ proprietary CRISPR-GNDM platform for epigenome editing. The CRISPR-GNDM technology aims to control gene expressions by modulating the switch. It uses an AAV vector to transduce target cells with GNDM DNA, which binds to a target gene to turn on or off to control insufficient or excessive amount of mRNA expressed from a gene.

Now, proof of concept in hand, the partners will move to the next phase, which involves jointly developing a novel gene therapy for the undisclosed CNS disease to provide patients with improved efficacy, safety, and less burden via IV injection in a minimally invasive and efficient manner.

Back in September, Modalis, headquartered in Tokyo with R&D operations in Massachusetts, was granted FDA Rare Pediatric Disease designation for its lead candidate for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), MDL-101. The treatment, which utilizes the company’s CRISPR-GNDM technology, targets the LAMA1 gene to compensate for LAMA2 protein deficiencies in patients with LAMA2-CMD.