JCR Pharmaceuticals Co., Ltd. (TOKYO:4552; “JCR”) and Modalis Therapeutics Corporation (TOKYO:4883; “Modalis”) today announced that they have validated the initial proof of concept in a joint research program for the development of a novel gene therapy for a central nervous system (CNS) disease. Due to the success of the partnership thus far, Modalis and JCR have agreed to proceed to the next phase of their research by entering into a new joint research agreement.

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The purpose of this joint agreement is to conduct pre-clinical studies for the development of a new gene therapy for the undisclosed CNS disease by applying J-Brain Cargo^®, JCR’s proprietary technology that is able to cross the blood-brain barrier (BBB), and a gene therapy payload based on CRISPR-GNDM^® (Guide Nucleotide-Directed Modulation), Modalis’ proprietary epigenome modulation technology which does not cleave or alter DNA sequences.

The two companies began a joint research collaboration in December of 2023 to evaluate the drug delivery technology of the gene therapy to the CNS. As a result of this research, the initial proof of concept has been validated. The next phase of the agreement is to jointly develop a novel and innovative gene therapy to provide patients with improved efficacy, safety, and less burden via intravenous injection (IV) in a minimally invasive and efficient manner.

“I am pleased to share the progress we’ve achieved in our collaboration with Modalis,” said Hiroyuki Sonoda, Ph.D., Director, Senior Managing Executive Officer, and Executive Director, Research Division at JCR. “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.”

“We are very happy to move the joint research collaboration with JCR to the next phase,” said Haruhiko Morita, CEO of Modalis. “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 maximize the potential and value of our proprietary epigenome editing technology (CRISPR-GNDM^®) in CNS diseases.”

This announcement is expected to have a minor impact on both companies’ consolidated financial results for this fiscal year (JCR: ending March 2025, Modalis: ending December 2025).

About JCR Pharmaceuticals Co., Ltd.

JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, we are expanding to the US, Europe, and Latin America. Our innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. We are also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. Our core values of reliability, confidence, and persistence drive our mission to enhance global medical progress. For more information, visit https://www.jcrpharm.co.jp/en/site/en/.

About Modalis Therapeutics Corporation

Modalis was founded in 2016 and conducts its research and development in Massachusetts, USA. Modalis is a forerunner in the field of epigenetic medicine. Modalis develops therapeutics for patients suffering from serious genetic disorders such as neuromuscular diseases, CNS diseases, and cardiomyopathies. Modalis’ proprietary CRISPR-GNDM^® technology is capable of specifically modulating the expression of disease-relevant genes without introducing double-strand DNA breaks. For more information, visit https://www.modalistx.com/en/.

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