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Tracking the FDA’s meeting on Vertex, CRISPR’s sickle cell therapy

October 31, 2023

A decade ago, scientists outlined the gene editing potential of CRISPR, turning the vestiges of a bacterial immune system into one of the biotechnology industry’s most powerful tools.

On Tuesday, a group of advisers to the Food and Drug Administration met to discuss the merits of what could be the first CRISPR medicine approved by the agency: a treatment for sickle cell disease from partners Vertex Pharmaceuticals and CRISPR Therapeutics.

Documents published last week show that FDA scientists are focused on the technical aspects of how CRISPR does its DNA-editing work. They seem relatively convinced the treatment, known as exa-cel, is effective.

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