Considerations for the development of iPSC-derived cell therapies: a review of key challenges by the JSRM-ISCT iPSC Committee

• Published in: Cytotherapy (Oxford, England) Vol. 26; no. 11; pp. 1382 - 1399
• Main Authors: Madrid, Marinna, Lakshmipathy, Uma, Zhang, Xiaokui, Bharti, Kapil, Wall, Dominic M., Sato, Yoji, Muschler, George, Ting, Anthony, Smith, Nathan, Deguchi, Shuhei, Kawamata, Shin, Moore, Jennifer C., Makovoz, Bar, Sullivan, Stephen, Falco, Veronica, Al-Riyami, Arwa Z.
• Format: Journal Article
• Language: English
• Published: England Elsevier Inc 01.11.2024
• Subjects: Cell- and Tissue-Based Therapy - methods; critical quality attributes (CQAs); good manufacturing practice (cGMP); Humans; Induced Pluripotent Stem Cells - cytology; iPSC-derived cell therapies; quality by design (QbD); Quality Control; quality control (QC); regenerative medicine; Regenerative Medicine - methods; the cost of goods (COGs); the cost of goods (COGs); regenerative medicine; quality by design (QbD); iPSC-derived cell therapies; critical quality attributes (CQAs); good manufacturing practice (cGMP); quality control (QC)
• ISSN: 1465-3249; 1477-2566; 1477-2566
• DOI: 10.1016/j.jcyt.2024.05.022

Since their first production in 2007, human induced pluripotent stem cells (iPSCs) have provided a novel platform for the development of various cell therapies targeting a spectrum of diseases, ranging from rare genetic eye disorders to cancer treatment. However, several challenges must be tackled for iPSC-based cell therapy to enter the market and achieve broader global adoption. This white paper, authored by the Japanese Society for Regenerative Medicine (JSRM) - International Society for Cell Therapy (ISCT) iPSC Committee delves into the hurdles encountered in the pursuit of safe and economically viable iPSC-based therapies, particularly from the standpoint of the cell therapy industry. It discusses differences in global guidelines and regulatory frameworks, outlines a series of quality control tests required to ensure the safety of the cell therapy, and provides details and important considerations around cost of goods (COGs), including the impact of automated advanced manufacturing.