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Review
. 2025 Jan 2;32(1):10-37.
doi: 10.1016/j.stem.2024.12.005.

Pluripotent stem-cell-derived therapies in clinical trial: A 2025 update

Agnete Kirkeby  1 , Heather Main  2 , Melissa Carpenter  3
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Free article
Review

Pluripotent stem-cell-derived therapies in clinical trial: A 2025 update

Agnete Kirkeby et al. Cell Stem Cell. .
Free article

Erratum in

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 revew the landscape of interventional hPSC trials worldwide, highlighting available data on clinical safety and efficacy. As of December 2024, we identify 116 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 >1011 clinically administered cells, so far showing no generalizable safety concerns.

Keywords: ATMP; advanced therapy products; clinical trial; pluripotent stem cells; transplantation.

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Conflict of interest statement

Declaration of interests A.K. is the owner of Kirkeby Cell Therapy APS, which holds royalty contracts and performs paid consultancy for Novo Nordisk A/S, Somite Therapeutics, and CCRM Nordic. A.K. is a co-inventor on several patents related to the generation of human neurons from stem cells and is engaged in the development of the STEM-PD product, which has been licensed to Novo Nordisk A/S for future development. H.M. is an owner of HOYA Consulting (ReGenMed Solutions AB), which provides consulting services on pluripotent stem cell start material including IP, quality, and commercial suitability. H.M. also holds a 50% role as project manager of ATMP Sweden. M.C. is president and owner of Carpenter Consulting Corporation, which provides consulting services for the development of stem cell therapeutics. M.C. is named inventor on a number of patents related to PSC manufacturing and product development.

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