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Updated: Jul 3, 2025
Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production
Published on: August 18, 2023
Manufacturing Cell and Gene Therapies: Challenges in Clinical Translation
Na Kyung Lee1,2, Jong Wook Chang1,2,3
1Department of Health Sciences and Technology, Samsung Advanced Institute for Health Sciences and Technology (SAIHST), Sungkyunkwan University, Seoul, Korea.
Abstract:
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.
Insights
Manufacturing cell and gene therapies, including CAR-T cells, faces challenges. Addressing these manufacturing and QC hurdles is crucial for clinical translation of these promising cancer treatments.
Area of Science:
- Biotechnology
- Regenerative Medicine
- Oncology
Background:
- Cell and gene therapies show promise for treating diseases, particularly cancers.
- Chimeric antigen receptor T (CAR-T) cell therapy combines cell and gene technologies for cancer treatment.
- Despite advancements, manufacturing challenges hinder the clinical translation of these therapies.
Purpose of the Study:
- To review general manufacturing processes for cell and gene therapies.
- To identify and discuss challenges in manufacturing and quality control (QC).
- To explore the impact of manufacturing models on cell and gene therapy progression.
Main Methods:
- Literature review of cell-based, gene-based, and CAR-T cell therapies.
- Analysis of manufacturing processes, challenges, and QC specifications.
- Discussion of manufacturing models and their influence.
Main Results:
- Manufacturing processes for mesenchymal stem cells, viral vectors, and CAR-T cells were reviewed.
- Complexities in manufacturing and QC/validation pose significant challenges.
- The manufacturing model impacts the clinical progression of cell and gene therapies.
Conclusions:
- Manufacturing complexities and QC hurdles are key obstacles for cell and gene therapy clinical translation.
- Addressing these challenges is essential for advancing these innovative treatments.
- Understanding manufacturing models is vital for successful therapeutic development.
