Expansion into ASO, mRNA therapeutics, and ncRNA-targeted therapeutics

「Research toward the creation of nucleic acid drugs with Mitsubishi Gas Chemical Company, Inc. (Pipeline 2) is also progressing smoothly, and in July 2026 we filed a composition-of-matter patent application for a drug candidate obtained through the research.」

「The nucleic-acid drug projects comprise an ASO targeting the p53 gene for acute kidney injury caused by ischemia, and an ASO for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), created through joint research with Professor Naohisa Okano James of The Jikei University School of Medicine. Both projects have produced drug-candidate compounds and involved applications for substance patents, and the p53 ASO has been granted a patent in Japan.」

「In November, we began joint research with Shimane University, a national university corporation, on an ASO designed to suppress primary graft dysfunction, a condition in which a transplanted lung has difficulty functioning properly from the outset after lung transplantation.」

「This is a joint drug-discovery research project for a nucleic-acid drug ASO targeting long RNA. It aims to obtain an ASO compound as a development candidate based on QbD and establish a manufacturing method. The company will primarily own the results obtained through the joint research, while Mitsubishi Gas Chemical Company, Inc. will own part of them. <Research period> 30 June 2025 for 3 years」

「In the development of nucleic-acid drugs, we have already identified an antisense oligonucleotide (ASO), a type of nucleic-acid drug that reduces the amount of p53 gene mRNA and suppresses protein expression. Along with obtaining a patent in Japan, we are continuing our own research to obtain ASOs with higher activity more efficiently. Since December 2023, we have also been considering the possibility of collaborating with Mitsubishi Gas Chemical Company, Inc. on the research, development and manufacture of ASOs.」

「In December 2023, we began considering the conclusion of a joint research agreement with Mitsubishi Gas Chemical Company, Inc. for the research, development and manufacture of ASOs. During the cumulative first-quarter period, we held repeated discussions on details of the joint research, including the gene candidates to be targeted by the ASOs. Before beginning the joint research with Mitsubishi Gas Chemical Company, Inc., we also began our own research, drawing on our existing ASO expertise to obtain highly active ASOs more efficiently.」

「To ensure business stability, we are preparing future businesses to follow our core business of mRNA-targeted small-molecule drug discovery. Specifically, we are applying in silico RNA structural analysis to the sequence design of antisense oligonucleotides (ASOs), a type of nucleic-acid drug previously considered difficult to design theoretically. We believe that our in silico RNA structural analysis can enable us to obtain drug-candidate compounds in a short period, as quickly as 8 months in our case, thereby reducing research and development costs.」