TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)-- Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) today announced that it has entered into a joint research agreement with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) on joint evaluation of the drug delivery technology of the gene therapy to the central nervous system (CNS) assuming CNS diseases as a target. Taking this agreement as an opportunity, Modalis and JCR Pharmaceuticals aim to establish the new gene therapies for undisclosed CNS diseases by applying J-Brain Cargo^®, JCR Pharmaceuticals’ proprietary technology for crossing the blood-brain barrier (BBB), and CRISPR-GNDM^® (Guide Nucleotide-Directed Modulation), Modalis proprietary epigenome modulation technology which does not require DNA cleavage.

Modalis was founded in 2016 based on technology from Univ. of Tokyo and conducts its research and development in Massachusetts, USA. The Company develops therapeutics for patients suffering from serious genetic disorders such as muscle diseases, CNS diseases, and cardiomyopathies.

In recent years, the development of novel therapeutics with improved efficacy and safety has been actively pursued in the field of gene therapy by utilizing tissue tropic drug delivery technology. In the past, Modalis has focused on intra-cisterna magna (ICM: intracranial) administration to deliver drugs to the CNS region. However, minimally invasive administration methods such as intravenous injection (IV) are expected to become the focus in the future in order to deliver the drug to a wider area of brain with less burden on the patient. Therefore, this joint research is an important step to explore the application of J-Brain Cargo^®, with the aim of establishing new gene therapies in combination with CRISPR-GNDM^®.

Haru Morita, CEO of Modalis, stated: “JCR Pharmaceuticals’ proprietary BBB penetrating technology, J-Brain Cargo^®, has successfully and efficiently delivered biopharmaceutical candidates of various modalities to the CNS. As a pioneer in developing CRISPR based epigenome editing technology (CRISPR-GNDM^®), we have demonstrated long-term drug efficacy and functional improvement in animal disease model studies, including demonstration of target engagement and safety in NHPs. We believe that leveraging these complementary technologies and strengths of our two companies will lead to the development of innovative new gene therapeutics.”

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