Modalis and JCR Pharma Ink Research Pact on Drug Delivery

Modalis Therapeutics Corporation (Modalis) announced a joint research agreement with JCR Pharmaceuticals Co., Ltd. (JCR Pharmaceuticals) on the joint evaluation of gene therapy drug delivery technology to the central nervous system (CNS) with CNS diseases as a target. Taking advantage of this agreement, Modalis and JCR Pharmaceuticals intend to develop new gene therapies for unspecified CNS diseases using 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 that does not require DNA cleavage.

Modalis was founded in 2016 based on technology from 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, as per pharmabiz.

In recent years, the field of gene therapy has actively explored the creation of innovative medicines with increased efficacy and safety by leveraging tissue tropic drug delivery technology. Modalis has previously focused on intracranial (ICM) administration to deliver medications to the CNS region. However, minimally invasive administration methods like as intravenous injection (IV) are expected to become more prevalent in the future in order to distribute the medicine to a larger area of the brain while putting less strain on the patient. 

Haru Morita, CEO of Modalis, said: “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.”