ISSCR develops roadmap to accelerate pluripotent stem cell-derived therapies to patients

The International Society for Stem Cell Research (ISSCR) today announced the upcoming release of “Charting the Translational Pathway: ISSCR Best Practices for the Development of Pluripotent Stem Cell (PSC)-Derived Therapies,” a new paper highlighting some of the most critical aspects of the ISSCR’s breakthrough interactive resource designed to transform how PSC-derived therapies are developed, evaluated, and advanced toward clinical and commercial success. The paper was published today in Stem Cell Reports.

As more than 100 PSC-derived products move through clinical trials worldwide, developers face a highly technical, expensive, and fragmented translational landscape. The ISSCR Best Practices addresses this challenge head-on by consolidating critical scientific, manufacturing, and regulatory guidance into a single, authoritative, openly accessible resource for academia, biotechnology, pharmaceutical companies, and manufacturers.

“A defining strength of the ISSCR is the capacity to convene cross-sector, international expertise to address shared challenges and develop field-shaping guidance,” said Keith Alm, chief executive officer of the ISSCR. “The Best Practices for the Development of PSC-Derived Therapies exemplifies this commitment and reflects the extraordinary leadership of its authors and the dedication of hundreds of volunteers who contributed their time and expertise. This resource serves as a tool for navigating the uncharted, a benchmark for rigor, and a catalyst for safe, effective cell therapies for patients.”

Developed by an international steering committee of leading cell therapy experts with extensive regulatory and community experience, the Best Practices document charts the full translational pathway across seven essential areas. The paper shares insights that emphasize 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 regulatory considerations and drug product manufacturing.

“Focused research drives discovery, but without clear translational guidance, even the most promising therapies can stall before reaching patients,” said Jacqueline Barry, co-chair of the ISSCR Best Practices Steering Committee and one of the paper’s authors. “The Best Practices document transforms decades of collective, global experience into actionable steps. Our paper seeks to call out the key elements of the complex resource and share how we hope stem cell scientists will learn from the experience of our community.”

The Best Practices document emphasizes a risk-based, end-to-end development strategy – starting with informed choices about PSC starting material and scaling through manufacturing, quality control, and regulatory engagement. Presented in an intuitive, visual, and interactive format, it is designed to support informed decision-making at every stage of development and will be updated regularly to reflect the rapidly evolving scientific and regulatory environment.

“This is a much-needed translational playbook for the field as evidenced by the response – more than 1,400 downloads from around the world in just a couple of months,” said Kapil Bharti, one of the paper’s co-authors and co-chair of the Best Practices Steering Committee. “In the paper, we distill some of the principles and practices that were derived over nearly two years of work by a global task force that collectively has decades of experience in developing cell therapy products. By clarifying key decision points and regulatory expectations early, we can increase both the speed and rigor of development and, most importantly, the number and quality of stem cell-based therapies that ultimately reach patients worldwide.”

“Charting the Translational Pathway: ISSCR Best Practices for the Development of PSC-Derived Therapies” underscores the ISSCR’s ongoing commitment to advancing responsible, evidence-based stem cell science and ensuring that groundbreaking discoveries translate efficiently into real-world medical impact. The full Best Practices document is available free, and the ISSCR welcomes continued feedback from the global community as the resource evolves.

About Stem Cell Reports
Stem Cell Reports is the open access, peer-reviewed journal of the International Society for Stem Cell Research (ISSCR) for communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians. Stem Cell Reports is a Cell Press partner journal. Find the journal on X: @StemCellReports.

About ISSCR
Across more than 80 countries, the International Society for Stem Cell Research (@ISSCR) is the preeminent global, cross-disciplinary, science-based organization dedicated to advancing stem cell research and its translation to medicine.

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