Considerations for development of gene-edited PSC-based therapies
Cell & Gene Therapy Insights 2023; 9(9), 1341–1352
PSCs provide a replenishable starting material for the consistent, scalable production of cell therapies with a broad range of therapeutic applications. The recent emergence of robust gene editing technologies offers the potential to significantly extend the reach and utility of PSCs by increasing potency and consistency of drug products derived from them. Full realization of the potential of this new platform will require careful consideration and planning with respect to the intended target products, approaches to process design, and the development of fit-for-purpose analytical control strategies.