Becker's Healthcare October 30, 2023
An advisory committee is meeting Oct. 31 to discuss an experimental drug for sickle cell disease, which could be the first approved therapy made with CRISPR technology — but the candidate faces skepticism.
CRISPR is a gene-editing tool that stands for clustered regularly interspaced short palindromic repeats, and cancer drug researchers have been investigating its treatment potential for years.
The candidate is exagamglogene autotemcel, or exa-cel, which is a drug product developed by Boston-based Vertex Pharmaceuticals and Switzerland-based CRISPR Therapeutics. The drugmakers applied for the drug to be approved for sickle cell disease patients 12 and older.
Sickle cell disease affects about 100,000 people in the U.S., and the painful blood disorder is most prevalent among people with...
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