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Modalis and JCR Pharmaceuticals Establish Collaborative Research Agreement

Wednesday, December 13, 2023

Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) has recently announced a collaborative effort with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) through a joint research agreement. The collaboration focuses on jointly evaluating gene therapy drug delivery technology targeting the central nervous system (CNS) for potential applications in treating undisclosed CNS diseases. The partnership aims to leverage JCR Pharmaceuticals' proprietary J-Brain Cargo® technology, designed for efficiently crossing the blood-brain barrier (BBB), and Modalis' own CRISPR-GNDM® (Guide Nucleotide-Directed Modulation), a proprietary epigenome modulation technology that operates without the need for DNA cleavage.

Founded in 2016 with technology originating from the University of Tokyo, Modalis conducts its research and development activities in Massachusetts, USA. The company specializes in developing therapeutics for individuals facing serious genetic disorders, including muscle diseases, CNS diseases, and cardiomyopathies.

In the evolving landscape of gene therapy, there is a concerted effort to develop novel therapeutics with enhanced efficacy and safety, particularly through the use of tissue-tropic drug delivery technology. While Modalis has previously concentrated on intra-cisterna magna (ICM: intracranial) administration to deliver drugs to the CNS region, the industry trend is shifting towards minimally invasive administration methods like intravenous injection (IV). This joint research initiative signifies a crucial step towards exploring the application of J-Brain Cargo® with the goal of establishing new gene therapies in conjunction with CRISPR-GNDM®.

Haru Morita, CEO of Modalis, expressed confidence in the collaboration, stating, “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 the 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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