A roadmap for developing pluripotent stem cell-derived cellular therapies
The International Society for Stem Cell Research (ISSCR) has released "Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies," a comprehensive, interactive new resource designed to accelerate the translation of human pluripotent stem cell (PSC)-derived therapies into clinical trials and commercial use.
Developed by leading international experts in cell therapy, this groundbreaking document outlines the key principles and decision points required to develop safe, effective, and regulatory compliant PSC-derived products.
Organized into seven sections, the guidance offers jurisdictionally neutral information on topics ranging from PSC line selection and raw material use to regulatory considerations, preclinical studies, and clinical trials.
"The creation of this resource reflects the power of collaborative leadership to deliver something truly impactful for the stem cell community," said ISSCR President Hideyuki Okano.
"I am deeply grateful to the ISSCR Best Practices Task Force for their vision and dedication in producing this blueprint for advancing new cellular therapies. While the clinical applications of pluripotent stem cells will continue to evolve, this initiative ensures that the best practices available today are shared broadly with our community now and in the future through regular updates."
"Focused research is the foundation of innovation, but without clear translational guidance, promising therapies can stall before reaching patients for reasons that could have been anticipated and prevented," said Jacqueline Barry, co-chair of the Best Practices Steering Committee and Chief Clinical Officer at Cell and Gene Therapy Catapult, UK.
"This document distills the collective experience of global experts to provide actionable best practices that can reduce development time, cut costs, and bring more therapies to clinical trial and market approval."
The ISSCR's Best Practices document emphasizes the importance of choosing appropriate PSC starting material, meeting regulatory requirements across jurisdictions, and ensuring quality at every stage—from cell banking and ancillary materials to drug product manufacturing.
Presented in an easy-to-follow visual format, the resource also includes links to critical, jurisdictionally relevant regulatory information and will be updated annually to reflect the rapidly evolving landscape.
Nine jurisdictions, including Australia, Brazil, Canada, China, the European Union, Japan, South Korea, the United Kingdom, and the United States are part of the regulatory drug development timelines and operational and strategic considerations in the resource, giving the scientific community consolidated access to the vital steps to take on the path to cell therapy development.
"This resource is a much-needed tool for academic researchers, biotech innovators, regulators, and industry stakeholders preparing to transition from proof-of-concept studies to first-in-human trials," said Kapil Bharti, co-chair of the Best Practices Steering Committee and scientific director at the National Institutes of Health's National Eye Institute, U.S.
"Through the Best Practices and their anticipated effect on the speed and rigor of the translational process, we hope to increase the number and quality of stem cell-based therapies reaching patients around the world."
More information:
Download the ISSCR Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies.
Provided by International Society for Stem Cell Research
