Translating stem cell research into development of cellular drugs—a perspective from manufacture of stem cell products and CMC considerations

The development of human pluripotent stem cell (PSC)‐derived medicinal products has been gathering steam in recent years, but the translation of research protocols into GMP production remains a daunting task. The challenges not only reside with the nature of cellular therapeutics but are also rooted in the general inexperience in industry‐scale production of stem cell products. Manufacturers of PSC‐derived products should be aware of the technical nuances and take a holistic approach toward early planning and engagement with their academic partners. While not all issues will be readily resolved soon, the collective knowledge and consensus by the manufacturers and key stakeholders will help to guide rapid progression of the field. This paper highlights and discusses the range and timing of issues that must be considered in the development of cell‐based medicines. Considerable work can be done to assure the robustness of a manufacturing system during early research and development which can then be refined more smoothly during product development. Development of characterisation and safety testing proceeds from a stage and potency assay development may continue into clinical phases.