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

Kirkeby, Agnete LU ; Main, Heather and Carpenter, Melissa (2025) In Cell Stem Cell 32(1). p.10-37
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... (More)

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.

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Please use this url to cite or link to this publication:
author
; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
advanced therapy products, ATMP, clinical trial, pluripotent stem cells, transplantation
in
Cell Stem Cell
volume
32
issue
1
pages
28 pages
publisher
Cell Press
external identifiers
  • pmid:39753110
  • scopus:85213526696
ISSN
1934-5909
DOI
10.1016/j.stem.2024.12.005
language
English
LU publication?
yes
id
0cf92c0d-14f0-4260-a26b-4bdbc9e5e918
date added to LUP
2025-03-12 15:03:23
date last changed
2025-05-07 20:16:52
@article{0cf92c0d-14f0-4260-a26b-4bdbc9e5e918,
  abstract     = {{<p>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 &gt;10<sup>11</sup> clinically administered cells, so far showing no generalizable safety concerns.</p>}},
  author       = {{Kirkeby, Agnete and Main, Heather and Carpenter, Melissa}},
  issn         = {{1934-5909}},
  keywords     = {{advanced therapy products; ATMP; clinical trial; pluripotent stem cells; transplantation}},
  language     = {{eng}},
  number       = {{1}},
  pages        = {{10--37}},
  publisher    = {{Cell Press}},
  series       = {{Cell Stem Cell}},
  title        = {{Pluripotent stem-cell-derived therapies in clinical trial : A 2025 update}},
  url          = {{http://dx.doi.org/10.1016/j.stem.2024.12.005}},
  doi          = {{10.1016/j.stem.2024.12.005}},
  volume       = {{32}},
  year         = {{2025}},
}