JCR Pharmaceuticals and Modalis Therapeutics have agreed to proceed to the next phase of their existing research partnership with a new joint research agreement.
In December 2023, Modalis and JCR entered a joint research collaboration to evaluate the delivery of gene therapy to the central nervous system (CNS).
The two companies have validated the initial proof of concept in the joint research programme for the development of a novel gene therapy.
Under the expanded partnership, the two companies will jointly develop a novel gene therapy to improve efficacy, safety, and reduce burden through intravenous injection (IV).
Modalis and JCR will conduct pre-clinical studies for the development of a new gene therapy for CNS disease using JCR’s J-Brain Cargo technology.
The J-Brain Cargo technology helps gene therapy to cross the blood-brain barrier (BBB).
The two companies will also develop a gene therapy payload based on Modalis’ Guide Nucleotide-Directed Modulation (CRISPR-GNDM) technology.
JCR director, senior managing executive officer and research division executive director Hiroyuki Sonoda said: “I am pleased to share the progress we’ve achieved in our collaboration with Modalis.
“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.”
JCR is a Japan-based pharmaceutical company focused on advancing treatments for rare and genetic diseases, expanding operations to the US, Europe, and Latin America.
Established in 2016, Modalis develops therapeutics for patients suffering from serious genetic disorders such as neuromuscular diseases, CNS diseases, and cardiomyopathies.
The company carries out research and development in the field of epigenetic medicine.
Its CRISPR-GNDM is a unique epigenome modulation technology that can specifically modulate the expression of disease-relevant genes without breaking double-strand DNA.
Modalis CEO Haruhiko Morita said: “We are very happy to move the joint research collaboration with JCR to the next phase. This is the result of hard work on both companies and an important strategic step for Modalis’ research activities.
“As a pioneer in this technology, we have demonstrated promising long-term drug efficacy in mouse disease model studies, including demonstration of target engagement and safety in non-human primates, exhibiting strong biodistribution for our lead program in neuromuscular disorders. We believe that CRISPR-GNDM has huge potential in the field of CNS diseases.
“So, the combination with J-Brain Cargo technology could be a very significant breakthrough to maximise the potential and value of our proprietary epigenome editing technology (CRISPR-GNDM) in CNS diseases.”
