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research article

Clinically compliant cryopreservation of differentiated retinal pigment epithelial cells

Baque-Vidal, Laura
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Main, Heather
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Petrus-Reurer, Sandra
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April 1, 2024
Cytotherapy

Background aims: Age-related macular degeneration (AMD) is the most common cause of blindness in elderly patients within developed countries, affecting more than 190 million worldwide. In AMD, the retinal pigment epithelial (RPE) cell layer progressively degenerates, resulting in subsequent loss of photoreceptors and ultimately vision. There is currently no cure for AMD, but therapeutic strategies targeting the complement system are being developed to slow the progression of the disease. Methods: Replacement therapy with pluripotent stem cell-derived (hPSC) RPEs is an alternative treatment strategy. A cell therapy product must be produced in accordance with Good Manufacturing Practices at a sufficient scale to facilitate extensive pre-clinical and clinical testing. Cryopreservation of the final cell product is therefore highly bene ficial, as the manufacturing, pre-clinical and clinical testing can be separated in time and location. Results: We found that mature hPSC-RPE cells do not survive conventional cryopreservation techniques. However, replating the cells 2-5 days before cryopreservation facilitates freezing. The replated and cryopreserved hPSC-RPE cells maintained their identity, purity and functionality as characteristic RPEs, shown by cobblestone morphology, pigmentation, transcriptional pro file, RPE markers, transepithelial resistance and pigment epithelium-derived factor secretion. Finally, we showed that the optimal replating time window can be tracked noninvasively by following the change in cobblestone morphology. Conclusions: The possibility of cryopreserving the hPSC-RPE product has been instrumental in our efforts in manufacturing and performing pre-clinical testing with the aim for clinical translation. (c) 2024 International Society for Cell & Gene Therapy. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)

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Type
research article
DOI
10.1016/j.jcyt.2024.01.014
Web of Science ID

WOS:001225535000001

Author(s)
Baque-Vidal, Laura
Main, Heather
Petrus-Reurer, Sandra
Lederer, Alex Russell  
Beri, Nefeli-Eirini
Bar, Frederik
Metzger, Hugo
Zhao, Cheng
Efstathopoulos, Paschalis
Saietz, Sarah
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Date Issued

2024-04-01

Publisher

Elsevier Sci Ltd

Published in
Cytotherapy
Volume

26

Issue

4

Start page

340

End page

350

Subjects

Life Sciences & Biomedicine

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Key Words

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Age-Related Macular Degeneration

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Age-Related Clinical Translation

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Cryopreservation

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Manufacturing

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Retinal Pigment Epithelium

Editorial or Peer reviewed

REVIEWED

Written at

EPFL

EPFL units
UPLAMANNO  
FunderGrant Number

Swedish Research Council

Ragnar Soderberg Foundation

Ming Wai Lau Center for Reparative Medicine

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Available on Infoscience
June 5, 2024
Use this identifier to reference this record
https://infoscience.epfl.ch/handle/20.500.14299/208399
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