C4U Announces the Second License Agreement with RACTHERA for CRISPR-Cas3 Technology

C4U Corporation (“C4U”) is pleased to announce that it has entered into the second license agreement with RACTHERA Co., Ltd. (“RACTHERA”) to grant a license for patents related to CRISPR-Cas3 technology, for which C4U holds exclusive licenses including sublicensing rights.

Since 2021, C4U has been conducting collaborative research on cells and genes for regenerative medicine products utilizing CRISPR-Cas3 technology, initially with Sumitomo Pharma Co., Ltd. (“Sumitomo Pharma”) and since February 2025, with RACTHERA following its succession of Sumitomo Pharma's regenerative medicine and cell therapy business. Among these efforts, earlier collaborative research focused on cell and gene for central nervous system diseases achieved its expected results, leading to the conclusion of the first license agreement between C4U and Sumitomo Pharma in March 2024.

Following the initial project, the second collaborative research has also yielded certain results. Based on these outcomes, RACTHERA expressed an interest in obtaining a license for the CRISPR-Cas3 patents, leading to the second license agreement covering the target cell and gene in this research.

Akimitsu Hirai, President and CEO of C4U, stated: “We are very pleased to have concluded this second licensing agreement with RACTHERA. RACTHERA is deeply committed to the advancement of regenerative medicine and cell therapy, and we believe that their selection of CRISPR-Cas3 technology for multiple R&D projects will accelerate the application of novel therapies utilizing CRISPR-Cas3. We look forward to continuing our collaboration with them.”

About C4U
C4U is a privately held biotech company based in Osaka, Japan, and is focused on the development of safe and efficient gene therapies utilizing its proprietary next generation CRISPR-Cas3 gene editing platform. In comparison to the CRISPR-Cas9 platform, CRISPR-Cas3 presents the distinct benefits of: 1) no off-target by the higher selectivity of deletion site (improved safety); 2) efficient knockouts by the larger deletion of gene sequences; and 3) an entirely independent patent portfolio. C4U has been granted a worldwide exclusive license to CRISPR-Cas3 by the University of Osaka for use in eukaryotic cells thus simplifying sublicensing transactions which is in sharp contrast to the complex and heavily litigated CRISPR-Cas9 patent landscape.
URL: https://www.crispr4u.jp/en/

About RACTHERA
RACTHERA Co., Ltd. is a joint venture established in November 2024 by Sumitomo Chemical Co., Ltd. and Sumitomo Pharma Co., Ltd. with the mission of advancing research and development in regenerative and cellular medicine. On February 1, 2025, RACTHERA succeeded the regenerative medicine and cell therapy business (excluding the business related to regenerative and cellular medicine manufacturing plants) from Sumitomo Pharma. Following the establishment of RACTHERA, the agreements between C4U and Sumitomo Pharma have been transferred to RACTHERA.
URL: https://www.racthera.co.jp/english/

Glossary of Terms

Genome Editing Technology: A technique that introduces artificially designed DNA cleavage enzymes into cells to selectively cleave and modify localized parts of the genome.

CRISPR-Cas3: Similar to CRISPR-Cas9, it cleaves double-stranded DNA. It is considered a safer genome editing tool because its crRNA (guide) recognition sequence is longer (27-base guide sequence), resulting in high specificity for recognizing genomic sequences and a low risk of inducing off-target mutations (mutations at unintended sites). It is also proficient at causing large deletions, allowing for loss of gene function or the removal of large regions containing disease-causing genetic mutations, in addition to gene modification.

CRISPR-Cas9: A type of widely used genome editing technology. Cas9 binds with a guide RNA, and selectively cleaves DNA complementary to a part of the guide RNA (20-base guide sequence). By changing the guide sequence, it can selectively cleave DNA with various base sequences. It was developed by multiple researchers in the US and Europe and has a complex background of numerous intertwined patents, with patent litigation currently ongoing in the US and other countries.