…But Is It Gene Therapy? Debating a (Controversial) Definition

By WIRB-Copernicus Group
Published: September 2017

In August, the FDA announced the approval of the first "gene therapy" to receive such a label, tisagenlecleucel (KymriahTM, Novartis Pharmaceuticals) for certain forms of acute lymphoblastic leukemia (ALL). This paper looks at the various definitions of gene therapy used by scientific and regulatory organizations, and why the use of this term generates both confusion and passionate opinion.


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