Clinical Development of Gene Therapies: The First Three Decades and Counting

Larissa Lapteva; Tejashri Purohit-Sheth; Mercedes Serabian; Raj K Puri

doi:10.1016/j.omtm.2020.10.004

Clinical Development of Gene Therapies: The First Three Decades and Counting

Mol Ther Methods Clin Dev. 2020 Oct 10:19:387-397. doi: 10.1016/j.omtm.2020.10.004. eCollection 2020 Dec 11.

Authors

Larissa Lapteva¹, Tejashri Purohit-Sheth¹, Mercedes Serabian¹, Raj K Puri²

Affiliations

¹ Division of Clinical Evaluation and Pharmacology/Toxicology, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.
² Division of Cellular and Gene Therapies, Center for Biologics Evaluation and Research, Office of Tissues and Advanced Therapies, U.S. Food and Drug Administration, Silver Spring, MD, USA.

Abstract

In the past three decades the field of gene therapy has made remarkable progress, surging from mere laboratory experiments to Food and Drug Administration (FDA)-approved products that bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. Herein, we review the evolution of the gene therapy clinical research landscape and describe the gene therapy product development programs evaluated by the FDA in Investigational New Drug applications received in 1988-2019. We also discuss the clinical development programs of the first six oncolytic and gene therapy products approved in the United States.

Keywords: CBER; Center for Biologics Evaluation and Research; FDA; IND; Investigational New Drug application; OTAT; Office of Tissues and Advanced Therapies; gene therapy; natural history; product development; rare diseases.

Publication types

Review