Manufacturing cell and gene therapies: challenges in clinical translation
Open accessAbstract
The safety and efficacy of both cell and gene therapies have been demonstrated in numerous preclinical and clinical trials. Chimeric antigen receptor T (CAR-T) cell therapy, which leverages the technologies of both cell and gene therapies, has also shown great promise for treating various cancers. Advancements in pertinent fields have also highlighted challenges faced while manufacturing cell and gene therapy products. Potential problems and obstacles must be addressed to ease the clinical translation of individual therapies. Literature reviews of representative cell-based, gene-based, and cell-based gene therapies with regard to their general manufacturing processes, the challenges faced during manufacturing, and QC specifications are limited. We review the general manufacturing processes of cell and gene therapies, including those involving mesenchymal stem cells, viral vectors, and CAR-T cells. The complexities associated with the manufacturing processes and subsequent QC/validation processes may present challenges that could impede the clinical progression of the products. This article addresses these potential challenges. Further, we discuss the use of the manufacturing model and its impact on cell and gene therapy.
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Transportation variables including extended transit time, temperature fluctuations, and improper thawing can compromise viability or biological activity of living cells and EV-based products.
“Variables associated with transportation, including extended transit time, temperature fluctuations, and improper thawing procedures, can compromise the viability or biological activity of living cells and EV-based products (Lee and Chang, 2024; Gupta and Shaz, 2025).”
From Isolator-based point-of-care manufacturing: a practical solution for GMP-compliant cell and extracellular vesicles therapy production (2025) · cites this paper as Lee and Chang, 2024
The MSC-based cell therapies section lists freezing, storage, and shipment as steps in distributing cells, but does not say that transit time, temperature fluctuations, or improper thawing compromise viability. The AAV and LV-based gene therapies section discusses environmental effects on lentiviral-vector functionality during production, not transportation of living cells or EV-based products. Searches for the specified transportation and thawing effects found no passage addressing the claim. (LLM, conf=0.72)
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- Authors
- Lee, N. K., & Chang, J. W.
- Affiliations
- Samsung Medical Center (KR)
- Corresponding author
- Chang, Jong Wook
- Venue
- Annals of Laboratory Medicine
- Publisher
- Annals of Laboratory Medicine
- Published
- 2024-02-16
- Volume
- 44
- Issue
- 4
- Pages
- 314-323
- ISSN
- 2234-3806 (ISSN-L)
- 2234-3814
- PMC
- PMC10961620
- OpenAlex
- W4391881331
- Cited by
- 78
- References
- 64
- Citations by year
- 2026: 49 · 2025: 25 · 2024: 4
- FWCI
- 4.18
- Type
- journal article
- Language
- en
- Open access
- diamond
- doi.org
- Licence
- Locations
- Annals of Laboratory Medicine (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Funders
- Korea Health Industry Development Institute (HR22C1363)
- Ministry of Health and Welfare (HR22C1363)
- Topics
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Keywords
- cell therapy manufacturing, gene therapy manufacturing, clinical translation, CAR T-cell therapy, mesenchymal stem cells, viral vectors, quality control specifications, manufacturing model, production problems
- MeSH terms
- Receptors, Chimeric Antigen, Humans, Neoplasms, T-Lymphocytes, Genetic Therapy, Immunotherapy, Adoptive
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