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JCR & Modalis: Unlocking the Next Phase of Gene Therapy

Marcus LeeSunday, Jan 5, 2025 10:10 pm ET
4min read



TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) and Modalis Therapeutics Corporation (Modalis) (Tokyo Stock Exchange: 4883) today announced their transition to the next phase of their joint research agreement, marking a significant milestone in the development of novel gene therapies for undisclosed central nervous system (CNS) diseases. This collaboration, which combines the strengths of both companies, aims to establish new gene therapies by leveraging JCR Pharmaceuticals' proprietary technology for crossing the blood-brain barrier (BBB), J-Brain Cargo®, and Modalis' proprietary epigenome modulation technology, CRISPR-GNDM®.



J-Brain Cargo®, a proprietary technology of JCR Pharmaceuticals, has successfully and efficiently delivered biopharmaceutical candidates of various modalities to the CNS. This technology enables the crossing of the BBB, which is a significant challenge in delivering drugs to the brain. By utilizing J-Brain Cargo®, Modalis and JCR Pharmaceuticals aim to establish new gene therapies for undisclosed CNS diseases. Modalis CEO Haru Morita stated, "JCR Pharmaceuticals' proprietary BBB penetrating technology, J-Brain Cargo®, has successfully and efficiently delivered biopharmaceutical candidates of various modalities to the CNS."

CRISPR-GNDM®, a proprietary epigenome modulation technology developed by Modalis, has demonstrated long-term drug efficacy and functional improvement in animal disease model studies. This technology, which does not require DNA cleavage, has been shown to achieve target engagement and safety in non-human primates (NHPs). By leveraging this technology, Modalis aims to develop innovative new gene therapeutics in collaboration with JCR Pharmaceuticals.



The combination of these technologies addresses the challenges of target engagement and safety in NHPs by providing efficient delivery to the CNS and ensuring the safety of the therapeutic agents, respectively. This combination of technologies is expected to lead to the development of innovative new gene therapeutics, with the potential to revolutionize the treatment of serious genetic disorders such as muscle diseases, CNS diseases, and cardiomyopathies.

Modalis was founded in 2016 based on technology from the University 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, the industry trend is shifting towards minimally invasive administration methods such as intravenous injection (IV) to deliver the drug to a wider area of the 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®.

JCR Pharmaceuticals, founded in 1986, is a leading Japanese pharmaceutical company specializing in the development and commercialization of innovative drugs. The Company has a strong track record in the development of novel therapeutics, including the first-in-class drug, JCR1134, for the treatment of amyotrophic lateral sclerosis (ALS). By combining the strengths of both companies, Modalis and JCR Pharmaceuticals aim to advance the development of innovative gene therapeutics, with the potential to transform the treatment landscape for patients with serious genetic disorders.

In conclusion, the transition to the next phase of the joint research agreement between JCR Pharmaceuticals and Modalis marks a significant milestone in the development of novel gene therapies for undisclosed CNS diseases. By leveraging the complementary strengths of both companies, this collaboration aims to establish new gene therapies that address the challenges of target engagement and safety in NHPs, ultimately revolutionizing the treatment of serious genetic disorders. As the field of gene therapy continues to evolve, the potential for innovative solutions such as those being developed by Modalis and JCR Pharmaceuticals remains vast and exciting.
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