Pluripotent stem-cell-derived therapies in clinical trial: A 2025 update
Agnete Kirkeby, Heather Main, Melissa Carpenter
Abstract
Since the first derivation of human pluripotent stem cells (hPSCs) 27 years ago, technologies to control their differentiation and manufacturing have advanced immensely, enabling increasing numbers of clinical trials with hPSC-derived products. Here, we review the landscape of interventional hPSC trials worldwide, highlighting available data on clinical safety and efficacy. As of December 2024, we identify 115 clinical trials with regulatory approval, testing 83 hPSC products. The majority of trials are targeting eye, central nervous system, and cancer. To date, more than 1,200 patients have been dosed with hPSC products, accumulating to >10 11 clinically administered cells, so far showing no generalizable safety concerns.