The Shared Bulletin Board

Posted on: May 29, 2026 Author: C4U株式会社

C4U Completed the AMED Stage-Gate Evaluation for Stage 2

C4U Corporation ('C4U') is pleased to announce that we have successfully passed the interim (stage-gate) evaluation and have been approved to transition to Stage 2 under 'Strengthening Program for Pharmaceutical Startup Ecosystem' by the Japanese Agency for Medical Research and Development (AMED). C4U has been selected for this program in 2025.

For further details, please refer to the announcement from AMED (only in Japanese).
https://www.amed.go.jp/program/list/19/02/005_r8_chukan.html

With the support of this program, C4U will accelerate our research and development to demonstrate the safety and efficacy of a hematopoietic stem cell transplantation for primary immunodeficiency utilising CRISPR-Cas3 genome editing technology.

About C4U Corporation

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 Osaka University for use in eukaryotic cells thus simplifying sublicensing transactions which is in sharp contrast to the complex and heavily litigated CRISPR-Cas9 patent landscape. https://www.crispr4u.jp/en/

Contact

C4U Corporation
Yamadaoka 2-8, Suita, Osaka 565-0871, Japan
E-mail:info@crispr4u.com

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