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What’s in a name?

How FDA’s RMAT eligibility will benefit gene, CAR T therapies

FDA’s description of Kymriah tisagenlecleucel from Novartis AG as the “first gene therapy” approved in the U.S. provoked a debate about taxonomy that distracted from an important regulatory decision.

What matters is that FDA has decided certain gene therapies can be eligible for the Regenerative Medicine Advanced Therapy (RMAT) designation. Defining CAR T as gene therapy means they could qualify for RMAT designation, potentially speeding their development.

RMAT, created by the 21st Century Cures Act, applies the principles pioneered in FDA’s breakthrough designation to regenerative

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