United States Food and Drug Administration Regulation of Human Cells, Tissues, and Gene Therapies

Sandhya Sanduja; Liz Lessey-Morillon; Rondine Allen; Xiaofei Wang; Gavin Imperato; Judith Arcidiacono

doi:10.1007/978-3-031-34567-8_5

United States Food and Drug Administration Regulation of Human Cells, Tissues, and Gene Therapies

Adv Exp Med Biol. 2023:1430:71-89. doi: 10.1007/978-3-031-34567-8_5.

Authors

Sandhya Sanduja¹, Liz Lessey-Morillon², Rondine Allen², Xiaofei Wang², Gavin Imperato², Judith Arcidiacono²

Affiliations

¹ Office of Therapeutic Products (OTP), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration (US FDA), Silver Spring, MD, USA. sandhya.sanduja@fda.hhs.gov.
² Office of Therapeutic Products (OTP), Center for Biologics Evaluation and Research (CBER), United States Food and Drug Administration (US FDA), Silver Spring, MD, USA.

PMID: 37526843
DOI: 10.1007/978-3-031-34567-8_5

Abstract

Research and development of gene therapies and cell- or tissue-based therapies has experienced exponential growth in recent decades and the potential for these products to treat diverse, often rare, clinical indications is promising. The Office of Therapeutic Products (OTP) in the Center for Biologics Evaluation and Research (CBER) at the United States Food and Drug Administration (US FDA) is responsible for the regulation of these products, among others, throughout the entire product lifecycle. This chapter provides an overview of the science- and data-driven approach to US FDA regulatory oversight of cell and gene therapy (CGT) products to ensure their safety and efficacy.

Keywords: Cell therapy; Clinical development; Clinical trial; Gene therapy; Investigational New Drug application; Marketing application; United States Food and Drug Administration.

Publication types

Review

MeSH terms

Drug Approval*
Genetic Therapy*
Humans
United States
United States Food and Drug Administration