The first CRISPR gene-editing drug is coming—possibly as soon as next year

The first CRISPR gene-editing drug is coming—possibly as soon as next year


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The drug makers say they intend to submit exa-cel for regulatory approval in the U.S., U.K., and Europe by the end of this year, meaning the drug could receive marketing authorization sometime in 2023 as more and more biopharma companies pursue novel gene therapies.

Vertex and CRISPR Therapeutics’ therapy uses what’s called an “ex-vivo” application of CRISPR gene editing (one done outside the actual body): The patient’s stem cells are extracted, the cellular DNA is modified by exa-cel to spur the production of a type of hemoglobin that the body usually makes only in infancy, and the modified cells are put back into the patient in order to boost healthy hemoglobin and red blood cell production.

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