Gene Therapy: JCR Pharmaceuticals and Modalis Advance Research
JCR Pharmaceuticals Co., Ltd. (TOKYO:4552) and Modalis Therapeutics Corporation (TOKYO:4883) have announced a major advancement in their joint research program aimed at developing a novel gene therapy for central nervous system (CNS) diseases. The companies have successfully validated the initial proof of concept, and due to the positive results so far, they are transitioning to the next phase of their collaboration with a new joint research agreement.
This new phase of the partnership focuses on conducting pre-clinical studies for a new gene therapy targeting an undisclosed CNS disease. The research will leverage JCR’s proprietary J-Brain Cargo® technology, which allows for the delivery of therapeutic agents across the blood-brain barrier (BBB), combined with Modalis’ innovative CRISPR-GNDM® technology. CRISPR-GNDM® is an epigenome modulation platform that can precisely adjust gene expression without altering or cleaving DNA sequences.
The collaboration between JCR and Modalis began in December 2023, with an initial focus on evaluating the efficacy of gene therapy drug delivery to the CNS. This early-stage research has led to the successful validation of the proof of concept. The companies will now focus on developing a gene therapy that aims to provide patients with improved efficacy, safety, and a less invasive treatment option through intravenous (IV) injection.
“We are excited to share the progress we’ve made in our collaboration with Modalis,” said Hiroyuki Sonoda, Ph.D., Director and Senior Managing Executive Officer at JCR. “By combining our J-Brain Cargo® technology for BBB penetration with Modalis’ CRISPR-GNDM® platform for epigenome editing, we are opening up new possibilities for innovative therapies that could make a significant impact in the treatment of CNS diseases.”
Haruhiko Morita, CEO of Modalis, expressed his enthusiasm about the next phase of the joint research: “This marks an important milestone for Modalis, and we are thrilled to continue our collaboration with JCR. Our CRISPR-GNDM® technology has shown promising results in preclinical studies, demonstrating strong drug efficacy and safety. We believe that combining this technology with JCR’s J-Brain Cargo® platform offers a unique opportunity to unlock the full potential of CRISPR-GNDM® in treating CNS diseases.”
CRISPR-GNDM® is a technology developed by Modalis that enables the modulation of disease-related genes by adjusting their expression without making permanent alterations to the DNA sequence. This innovative approach has already demonstrated efficacy in treating neuromuscular disorders and other diseases in animal models. The combination of this technology with JCR’s J-Brain Cargo® system, which facilitates the crossing of the BBB, has the potential to create a breakthrough in the treatment of CNS diseases, which are notoriously difficult to target due to the protective nature of the BBB.
The companies expect this collaboration to significantly advance the development of gene therapies for CNS diseases, with the goal of providing patients with more effective, safer, and minimally invasive treatment options. While the agreement is expected to have a minor financial impact on both companies for the current fiscal year—ending March 2025 for JCR and December 2025 for Modalis—the long-term potential for this research is substantial.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals is a global leader in specialty pharmaceuticals, with nearly 50 years of expertise in the treatment of rare and genetic diseases. The company’s Gene Therapy address a range of conditions, including growth disorders, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also actively developing treatments for other rare diseases, such as MPS I, MPS IIIA and B, and more. Headquartered in Japan, JCR is expanding its operations internationally, including in the United States, Europe, and Latin America. The company’s commitment to reliability, confidence, and persistence drives its mission to improve global medical care.
About Modalis Therapeutics Corporation
Founded in 2016, Modalis Therapeutics is a leader in the field of epigenetic medicine, focused on developing therapies for serious genetic disorders such as neuromuscular diseases, CNS diseases, and cardiomyopathies. Modalis’ proprietary CRISPR-GNDM® technology enables precise modulation of disease-relevant gene expression without the need for DNA cleavage or alteration. The company conducts its research and development in Massachusetts, USA, and is pioneering new approaches to gene therapy for patients with genetically driven diseases. For more information, visit Modalis Therapeutics’ website.
Through this collaboration, JCR and Modalis are poised to significantly impact the field of gene therapy, bringing new hope to patients suffering from challenging CNS diseases.
Please find the related post
