STAT+: European regulators endorse Casgevy, paving way for approval of CRISPR-based therapy

LONDON — The world’s first medicine based on the genome-editing technology CRISPR will almost certainly win approval in Europe in the coming months, following an endorsement Friday by a regulatory committee.

The medicine, a treatment for sickle cell disease and beta thalassemia called Casgevy, received a positive review from a European Medicines Agency’s committee. The European Commission will make the final decision, likely early next year, but almost always follows the EMA committee’s guidance. 

With the anticipated approval of the medicine, Europe will join regions including the U.S. and the U.K. that have recently given the green light to Casgevy, bringing the first CRISPR medicine to market just over a decade after the technology’s potential as a genome editor was described in academic research. The one-time therapy, also called exa-cel, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. 

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