Lessons learnt, and still to learn, in first in human stem cell trials.

Developing cellular therapies is not straightforward. This Perspective summarizes the experience of a group of academic stem cell investigators working in different clinical areas and aims to share insight into what we wished we knew before starting. These include (1) choosing the stem cell line and assessing the genome of both the starting and final product, (2) familiarity with GMP manufacturing, reagent validation, and supply chain management, (3) product delivery issues and the additional regulatory challenges, (4) the relationship between clinical trial design and preclinical studies, and (5) the market approval requirements, pathways, and partnerships needed.
Competing Interests: Conflict of interests C.J. is a co-founder of Aspera Biomedicines and Impact Biomedicines and receives royalties from Forty Seven Inc. C.M. is a senior vice president and equity holder in Sana Biotechnology. G.P. is a consultant for J-TEC, Gamagori, Japan, a member of the board of directors, and R&D director of Holostem Terapie Avanzate, Modena, Italy. J.S. is the founder and CEO of iPS Bio, Inc. The remaining authors report no conflicts.
(Copyright © 2022 The Authors. Published by Elsevier Inc. All rights reserved.)