“I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA commissioner Dr Scott Gottlieb after Luxturna’s approval.
As innovative gene and cell therapies continue to make the transition from the laboratory to the clinic, they are bringing with them the promise of truly personalised medicine. The last few years have seen the regulatory approval of the first gene therapies that take a patient’s own immune cells and genetically engineer them to target cancer cells more effectively.
These chimeric antigen receptor T-cell (CAR-T) therapies now represent a rapidly growing field, with Novartis’s Kymriah, the first CAR-T therapy approved by the US Food and Drug Administration (FDA) in August 2017 for the treatment of a rare blood cancer, seen as the tip of the iceberg for this treatment class’ potential. Approval of Kite Pharma’s Yescarta, a CAR-T treatment for certain forms of non-Hodgkin lymphoma, followed just a few months later.
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