Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle

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.