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Review
. 2022 Aug;36(4):687-699.
doi: 10.1016/j.hoc.2022.03.004. Epub 2022 Jun 27.

Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle

Najat Bouchkouj  1 , Megha Kaushal  2 , Poornima Sharma  2 , Kristin Baird  2
Affiliations
Review

Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle

Najat Bouchkouj et al. Hematol Oncol Clin North Am. 2022 Aug.

Abstract

The recent progress in gene and gene-modified cellular therapies has shown great promise for numerous pediatric diseases. Nevertheless, the development of these products is complicated, and the regulatory pathway is rigorous. There are, however, several opportunities for programmatic support within the Food and Drug Administration. This article highlights the life cycle of product development through approval and many of the available resources and programs to support product development.

Keywords: Cell therapy; Food and drug administration (FDA); Gene therapy; Pediatric; Regulations.

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Conflict of interest statement

Disclosure The authors have nothing to disclose.