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

Molecular Therapy. Methods & Clinical Development
Larissa LaptevaR K Puri

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.

Citations

Mar 20, 2021·Molecular Therapy. Methods & Clinical Development·Andreas L GimpelRichard D Braatz
Mar 24, 2021·Biomedicine & Pharmacotherapy = Biomédecine & Pharmacothérapie·Wuyuan Zhou, Xiang Wang
Apr 24, 2021·Human Gene Therapy·Joy A Cavagnaro
May 4, 2021·Frontiers in Cardiovascular Medicine·Isabel N SchellingerPhilip S Tsao
May 18, 2021·Molecular Therapy. Methods & Clinical Development·Daniela DragoAdora Ndu
Aug 10, 2021·Gene·Wuyuan Zhou, Xiang Wang

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Methods Mentioned

BETA
genetic modification
transfection
genetic modifications

Software Mentioned

INTERACT
OTAT
INitial

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