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author：hotspot 2025-10-03 00:22:38 - browse：142

illustration of one blue dna strand — Adobe

The Food and Drug Administration is convening a meeting of outside experts on Tuesday to review exa-cel, a CRISPR-based treatment for sickle cell disease made by Vertex Pharmaceuticals and CRISPR Therapeutics.

Tuesday’s meeting is set up a bit differently than most FDA advisory panels. The agency has not raised any concerns about exa-cel’s efficacy or safety, and there will not be a typical vote at the end of the day on whether the data from exa-cel’s pivotal clinical trial support an approval as a treatment for sickle cell disease.

Instead, the FDA is seeking input about the methods used by Vertex and CRISPR Therapeutics to evaluate the risk of inadvertently making unwanted changes to patients’ DNA — so-called off-target editing — when exa-cel is administered.

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