JST decides to invest in two university ventures based on "SUpport Program of Capital Contribution to Early-stage Companies (SUCCESS)."

The Japan Science and Technology Agency, JST, has invested in C4U (standing for "CRISPR for you") Corporation, a venture company from Osaka University, and FerroptoCure Inc., a venture company from Keio University, under JST's SUCCESS program.

C4U Corporation, a venture company from Osaka University

C4U is a bio-business venture started by Osaka University. Its aims are to develop CRISPR-Cas3, a genome-editing technology, for use in industry. The company was established in March 2018 based on the research and development results of the research project, "Development of novel genome editing technology in mice and rats," which was part of the JST Program on Open Innovation Platform with Enterprises, Research Institute and Academia (OPERA).

CRISPR-Cas3, developed in 2018 as a domestic genome editing technology, has been shown to be safe for fewer off-target mutations, thanks to the longer spacer sequence, which may increase the specificity for target recognition. It also has the feature of being able to cleave a wide range of genes and their surrounding sequences. Furthermore, it is not hampered by the complicated patent conflicts currently faced by CRISPR-Cas9, which has been the global forerunner in this research field. As such, CRISPR-Cas3 has attracted attention as a promising genome editing technology to compete with CRISPR-Cas9.

C4U aims to develop novel gene therapies for genetic diseases using CRISPR-Cas3 technology. Additionally, it aims to deploy a platform for this technology that enables collaborative research with leading researchers affiliated with various research organizations.

FerroptoCure Inc., a venture company from Keio University

FerroptoCure is a start-up that develops anticancer drugs based on cell death—called ferroptosis—induced by oxidative stress. The company was established in May 2010 based on the results of the research project, "Analysis of abnormal regulation of cell differentiation and drug development research by using induced cancer stem cells" under JST's program, Core Research for Evolutionary Science and Technology (CREST) and that of the research project, "Development of novel anticancer drugs using oxidative stress" under JST's Program of Start-up incubation from COre REsearch (SCORE): University promotion type.

When reactive oxygen species accumulate in cancer cells due to anticancer drug therapy or as byproducts of the metabolism, unsaturated fatty acids in the cells are oxidized and lipid peroxide accumulates in the cells, inducing cell death (i.e., ferroptosis). On the other hand, cancer cells utilize reduced glutathione (GSH), an antioxidant, to reduce lipid peroxides generated by reactive oxygen species and suppress ferroptosis. Proteins and enzymes, such as cystine/glutamate transporter (xCT) and glutathione peroxidase 4 (GPX4), are involved in this suppression. Ferroptosis-inducing therapies targeting these antioxidant mechanisms are attracting attention as a new cancer treatment strategy. Cancer cells resistant to current approved anticancer drugs are known to be highly susceptible to ferroptosis, and a method of inducing ferroptosis is strongly expected as a next-generation cancer treatment strategy for cancers that have not been treated successfully to date.

To commercialize the drug, the company is converting an approved existing drug and developing it to treat a new disease (drug repositioning). For the treatment of triple negative breast cancer (refractory breast cancer), the company plans to start clinical trials of the drug in 2023. It is expected to be developed safely and quickly.