Design and initial evaluation of a certified committee model for review of in vivo gene therapy and related technologies, together with ex vivo gene therapy, under the act on the safety of regenerative medicine

Introduction: The 2024 amendment to the Act on the Safety of Regenerative Medicine expanded the Act's regulatory scope to include in vivo gene therapy and related technologies. This created a need for certified committees for regenerative medicine to address associated scientific and regulatory issues. This study aimed to design and provide an initial evaluation of a practical and implementable committee model for reviewing newly covered technologies and ex vivo gene therapy under the amended Act. Methods: We conducted an institutional implementation study to develop a dual-committee model consisting of an independent second committee for gene therapy review, supported by selected shared foundational functions from an existing cell-based therapy committee. The success of the initial implementation was prospectively evaluated at the first review meeting using five predefined items: committee establishment, organizational structure, membership composition, workflow establishment, and operational feasibility. Results: A newly established specific certified committee for regenerative medicine for gene therapy review was certified by the Ministry of Health, Labour and Welfare on November 28, 2025. The predefined organizational model was implemented as an independent second committee with a shared institutional foundation. The required membership categories were fulfilled, including those specific to gene therapy review. A standardized review workflow incorporating prior administrative checks and a preliminary document review was established and successfully applied at the first review meeting, without major procedural disruption. Conclusions: A dual-committee model combining an independent second committee with a shared institutional foundation provided a practical and operationally feasible approach for implementing gene therapy review capacity within an existing regenerative medicine review infrastructure.