Driving the future of iPS-cell-based therapy in Japan: government strategies, regulatory review and clinical development

Japan has long been positioned as a global leader in regenerative medicine, and a critical moment occurred in 2025 when the world's first regulatory submission for an induced pluripotent stem (iPS)-cell-based therapy was filed. In this article, we detail the Japanese government's strategic investment in regenerative medicine since the early 2000s, and elucidate the advancement of regulatory guidelines tailored to the unique characteristics of regenerative medical products (RMPs) and designed to ensure safety while accelerating commercialization. We also provide an extensive summary of the latest clinical studies using iPS-cell-based therapies conducted by Japanese companies and academic institutions, as well as the current landscape of corporate involvement in this field. In addition, we address the challenges facing the development of RMPs by discussing the withdrawal cases that occurred in Japan last year.