Is It 'Gene Therapy'?

Journal of Law and the Biosciences, Vol. 5, pp. 786–793, 2018

8 Pages Posted: 5 Dec 2019

See all articles by Jacob S. Sherkow

Jacob S. Sherkow

University of Illinois College of Law; Center for Advanced Studies in Biomedical Innovation Law

Patricia J. Zettler

Ohio State University (OSU) - Michael E. Moritz College of Law

Henry T. Greely

Stanford Law School

Date Written: 2018

Abstract

What, exactly, is "gene therapy"? Crystallizing a modern definition of "gene therapy" has now become critically important on several fronts. Following the recent of passage the 21st Century Cures Act, FDA now relies on the term to give regulatory incentives to sponsors of certain ‘regenerative medicines’, including ‘gene therapies’. NIH Director Francis S. Collins and FDA Commissioner Scott Gottlieb recently announced that Recombinant DNA Advisory Committee ("RAC") approval would no longer be required for "gene therapy" because "there is no longer sufficient evidence to claim that the risks of gene therapy are entirely unique and unpredictable." And in July 2018 FDA issued six draft guidance documents on "gene therapy" but none with a substantially new definition. We review the contours ofthe term here, and propose that "gene therapy"— in the lay, scientific, and legal sense — should be defined as an intentional and expected permanent alteration of a specific DNA sequence of the cellular genome — that is, the sum of DNA that exists within a cell — for a clinical purpose. We also propose that gene therapy, as we define it, can be further categorized into at least three types: direct,compensatory, and augmenting. Defining gene therapy in this fashion would provide clarity to scientists and regulators alike.

Keywords: Gene Therapy, CRISPR, CAR-T, FDA, Law

JEL Classification: K11, K19, K39, O34, K00

Suggested Citation

Sherkow, Jacob S. and Zettler, Patricia J. and Greely, Henry (Hank) T., Is It 'Gene Therapy'? (2018). Journal of Law and the Biosciences, Vol. 5, pp. 786–793, 2018, Available at SSRN: https://ssrn.com/abstract=3490215

Jacob S. Sherkow (Contact Author)

University of Illinois College of Law ( email )

504 E. Pennsylvania Avenue
Champaign, IL 61820
United States

Center for Advanced Studies in Biomedical Innovation Law ( email )

Studiestraede 6
Studiestrade 6
Copenhagen, DK-1455
Denmark

HOME PAGE: http://jura.ku.dk/cebil/staff/

Patricia J. Zettler

Ohio State University (OSU) - Michael E. Moritz College of Law ( email )

55 West 12th Avenue
Columbus, OH 43210
United States

Henry (Hank) T. Greely

Stanford Law School ( email )

559 Nathan Abbott Way
Stanford, CA 94305-8610
United States
650-723-2517 (Phone)
650-725-0253 (Fax)

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