C4U

C4U Corporation, established in March 2018 as a spin-off from Osaka University, is a biotechnology firm centered on the commercialization of its proprietary CRISPR-Cas3 genome editing platform. The company was founded by Professor Tomoji Mashimo, who invented the CRISPR-Cas3 technology and now serves as the Chief Scientific Officer. The founding vision, encapsulated in the name C4U (CRISPR for you), is to make this advanced technology broadly accessible to researchers and various industries to foster societal benefits. Akimitsu Hirai, an expert in law and intellectual property with a background in the Human Genome Project, was appointed President and CEO in August 2020 to steer the company's business strategy.

The core of C4U's business is the CRISPR-Cas3 technology, which offers distinct advantages over the more widely known CRISPR-Cas9 system. This includes a superior safety profile due to a highly selective targeting mechanism that minimizes off-target mutations, the capability to induce large-scale gene deletions, and freedom to operate under an independent patent portfolio. C4U holds an exclusive worldwide license from Osaka University for the foundational Cas3 patent, which has been granted in major markets including Japan, the U.S., China, and Europe. This strong intellectual property position allows for simplified sublicensing, a key element of the company's revenue model.

C4U's business model is twofold, focusing on both an internal therapeutic pipeline and external partnerships. The company generates revenue by licensing its platform technology to partners in diverse sectors. Collaborations are active in areas such as diagnostics development with TAUNS Laboratories, genetic modification services with iPS Portal, and agricultural applications with Inplanta Innovations. The company also pursues its own drug discovery programs, targeting conditions with high unmet medical needs like severe congenital neutropenia, Duchenne muscular dystrophy, and solid tumors. These efforts are supported by joint research with institutions like Hiroshima University and Noile-Immune Biotech. The company operates across a range of industries, including medicine, agriculture, fisheries, and industrial biotechnology, aiming to provide solutions for gene therapy, food security, and renewable energy.

Since its inception, C4U has secured significant funding to advance its research and development. Following initial seed funding from Osaka University Venture Capital in 2018 and 2019, the company raised approximately $8.5 million in a Series A round in 2021 and $11 million in a Series B round in 2023. More recently, in June 2025, the company secured a substantial non-dilutive grant of up to $14 million from the Japanese Agency for Medical Research and Development (AMED) to advance its therapies into clinical trials. Keywords: CRISPR-Cas3, genome editing, gene therapy, C4U Corporation, Tomoji Mashimo, Akimitsu Hirai, Osaka University spin-off, biotechnology, genetic diseases, DNA editing, large-scale gene deletion, off-target mutations, drug discovery platform, licensing model, cell therapy, agricultural biotechnology, aquaculture genetics, industrial biotechnology, severe congenital neutropenia, Duchenne muscular dystrophy, solid tumors, allogeneic CAR-T, regenerative medicine, intellectual property licensing, freedom to operate (FTO), venture capital-backed, AMED grant