Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases—by DahShu Innovative Design Scientific Working Group (IDSWG): Therapeutic Innovation & Regulatory Science

Chenkun Wang; Junrui Di; Mercedeh Ghadessi; Rui Tang; Caroline Mulatya; Daoyuan Shi; Tu Xu; Wenquan Wang; Chaoqun Mei; Susan Wang; Bryan McComb; Robert A. Beckman; Gianna McMillan

doi:10.1007/s43441-025-00853-4

Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases—by DahShu Innovative Design Scientific Working Group (IDSWG): Therapeutic Innovation & Regulatory Science

Chenkun Wang
, Junrui Di
, Mercedeh Ghadessi
, Rui Tang
, Caroline Mulatya
, Daoyuan Shi
, Tu Xu
, Wenquan Wang
, Chaoqun Mei
, Susan Wang
, Bryan McComb
, Robert A. Beckman
, Gianna McMillan

Loyola Marymount University

Research output: Contribution to journal › Review article › peer-review

Abstract

The rapid advancement of cell and gene therapies (CGT) in the past ten years has inspired biopharmaceutical companies, biotechnologies, and nonprofits to tackle diseases that have traditionally been challenging to treat. Rare diseases, where roughly 80% have a genetic basis, have enjoyed this scrutiny, but the complexity of CGT trial design and implementation have proven challenging. This manuscript offers general guidance for CGT clinical development, current regulatory requirements and guidelines governed by FDA and EMA, considerations around preclinical development, safety monitoring and the need for long-term monitoring and follow up.

Original language	English
Pages (from-to)	1336-1355
Journal	Ther Innov Regul Sci
Volume	59
Issue number	6
DOIs	https://doi.org/10.1007/s43441-025-00853-4
State	Published - 2025
Externally published	Yes

ASJC Scopus Subject Areas

Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
Public Health, Environmental and Occupational Health
Pharmacology (medical)

Keywords

Cell and gene therapy
Clinical development
Regulatory requirements and guidelines
Safety monitoring
Genetic Therapy/methods
United States
Cell- and Tissue-Based Therapy/methods
Humans
Clinical Trials as Topic
Rare Diseases/therapy
United States Food and Drug Administration

Access to Document

10.1007/s43441-025-00853-4

Cite this

Wang, C., Di, J., Ghadessi, M., Tang, R., Mulatya, C., Shi, D., Xu, T., Wang, W., Mei, C., Wang, S., McComb, B., Beckman, R. A., & McMillan, G. (2025). Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases—by DahShu Innovative Design Scientific Working Group (IDSWG): Therapeutic Innovation & Regulatory Science. Ther Innov Regul Sci, 59(6), 1336-1355. https://doi.org/10.1007/s43441-025-00853-4

Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases—by DahShu Innovative Design Scientific Working Group (IDSWG): Therapeutic Innovation & Regulatory Science. / Wang, Chenkun; Di, Junrui; Ghadessi, Mercedeh et al.
In: Ther Innov Regul Sci, Vol. 59, No. 6, 2025, p. 1336-1355.

Research output: Contribution to journal › Review article › peer-review

Wang, C, Di, J, Ghadessi, M, Tang, R, Mulatya, C, Shi, D, Xu, T, Wang, W, Mei, C, Wang, S, McComb, B, Beckman, RA & McMillan, G 2025, 'Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases—by DahShu Innovative Design Scientific Working Group (IDSWG): Therapeutic Innovation & Regulatory Science', Ther Innov Regul Sci, vol. 59, no. 6, pp. 1336-1355. https://doi.org/10.1007/s43441-025-00853-4

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abstract = "The rapid advancement of cell and gene therapies (CGT) in the past ten years has inspired biopharmaceutical companies, biotechnologies, and nonprofits to tackle diseases that have traditionally been challenging to treat. Rare diseases, where roughly 80\% have a genetic basis, have enjoyed this scrutiny, but the complexity of CGT trial design and implementation have proven challenging. This manuscript offers general guidance for CGT clinical development, current regulatory requirements and guidelines governed by FDA and EMA, considerations around preclinical development, safety monitoring and the need for long-term monitoring and follow up.",

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