Scaling up pluripotent stem cell-based therapies - considerations, current challenges and emerging technologies: perspectives from the ISCT Emerging Regenerative Medicine Working Group

May 12, 2025Cytotherapy

Expanding stem cell therapies: key challenges, new technologies, and expert views

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Regenerative Health on OpenScience ↗PubMed ↗DOI ↗

Abstract

Manufacturing induced pluripotent stem cell (iPSC) therapies at the required scale for clinical trials remains a challenge.

Key considerations for stem cell therapy developers include specific manufacturing processes tailored to different development stages.
Decisions regarding the implementation of automation are critical in the manufacturing process.
The choice of technology affects the requirements for cell expansion and differentiation.
Scalable manufacturing technologies may enhance the application of quality-by-design approaches in product development.
An overview of existing technologies for expanding and differentiating iPSC-derived therapies highlights their compatibility with good manufacturing practices.

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Approval of induced pluripotent stem cells (iPSCs) for the manufacture of cell therapies to support clinical trials is now becoming realized after more than 20 years of research and development. However, manufacturing these therapies at the scale required for patient treatment, as well as for key clinical trial enabling activities such as preclinical and stability studies, remains a challenge. In 2022 the International Society for Cell and Gene Therapy (ISCT) established a Working Group on Emerging Regenerative Medicine Technologies, an area in which iPSC-derived technologies are expected to play a key role. In this article, the Working Group provides an overview of the considerations and challenges facing stem cell therapy developers, including development-stage specific manufacturing processes, the decision on when to implement automation, the choice of technology and different requirements of expansion and differentiation aspects of the process, and the integration of automation for both manufacturing and analytics in an end-to-end manufacturing process. The role of scalable manufacturing technologies in the application of quality-by-design approaches to product development, and the use of design-of-experiment approaches for increased product characterization, is discussed. Finally, we provide an in-depth review of the different technologies that have been used for expansion and differentiation of iPSC-derived therapies to date, including compatibility with good manufacturing practice requirements and process analytical technologies. We hope that this overview will summarize the existing knowledge in the field and reduce the challenges that therapy developers face in translating their research into clinical and commercial scale manufacturing.

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Scaling up pluripotent stem cell-based therapies - considerations, current challenges and emerging technologies: perspectives from the ISCT Emerging Regenerative Medicine Working Group

Abstract

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