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. 2020 Nov 16;11(1):5821.
doi: 10.1038/s41467-020-19507-0.

Turning genes into medicines-what have we learned from gene therapy drug development in the past decade?

Katherine A High  1   2
Affiliations

Turning genes into medicines-what have we learned from gene therapy drug development in the past decade?

Katherine A High. Nat Commun. .

Abstract

Gene and cell therapy products approved over the past decade in Europe and North America have provided new therapeutic options for single gene disorders and for hematologic malignancies. Lessons learned, and limitations identified, are reviewed.

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Conflict of interest statement

The author declares the following competing interests: Formerly employed by and held equity in Spark Therapeutics (2014–2020); member of the Board of Directors, CRISPR Therapeutics (2019–present).

Figures

Fig. 1
Fig. 1. Investigational new drug applications (INDs) in gene therapy by year to US FDA.
CY calendar year. Reprinted with permission from Dr. Raj Puri.
See this image and copyright information in PMC

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