NEW YORK – JCR Pharmaceuticals and Modalis Therapeutics are advancing a gene therapy for an undisclosed central nervous system disease, the companies said this weekend.
Ashiya, Japan-based JCR and Tokyo-based Modalis in late 2023 entered into a joint research collaboration to evaluate JCR's J-Brain Cargo, which is designed to cross the blood-brain barrier and deliver gene therapies to the central nervous system. The companies said they have validated an initial proof of concept for this drug delivery technology.
The companies are now entering into a new joint research agreement under which they will conduct preclinical development of a novel gene therapy delivered through a minimally invasive intravenous injection. The gene therapy involves epigenome editing using Modalis' epigenome modulation platform CRISPR-GNDM, which stands for Guide Nucleotide-Directed Modulation.
The companies both expect the announcement of this research agreement to have a minor impact on their respective consolidated financial results for the current fiscal year, which for JCR ends in March 2025 and for Modalis ends in December 2025.