Pharmaceuticals company JCR Pharmaceuticals Co, Ltd (TOKYO: 4552) and Modalis Therapeutics Corporation (TOKYO: 4883), a research and development company, on Monday announced the successful validation of the initial proof of concept in their joint research for a novel gene therapy targeting a central nervous system (CNS) disease.

Based on the success to date, both companies have agreed to proceed to the next phase, entering into a new joint research agreement to conduct pre-clinical studies.

This collaboration leverages JCR's proprietary J-Brain Cargo technology, designed to cross the blood-brain barrier, alongside Modalis' CRISPR-GNDM platform, which modulates gene expression without altering DNA sequences. This phase aims to develop a gene therapy that offers improved efficacy, safety and a less invasive treatment option via intravenous injection.

The partnership, which began in December 2023, represents a significant step forward in gene therapy development. While the transition to the next phase is positive news, it is expected to have only a minor impact on both companies' consolidated financial results for their respective fiscal years (JCR's fiscal year ends March 2025, Modalis' ends December 2025).