Also approved was another gene therapy made by Bluebird Bio that works differently.Ĭasgevy was approved in the United Kingdom last month after seeing a successful global trial in which 28 out of 29 patients reported being free of severe pain. The Food and Drug Administration on Friday approved a gene-editing therapy that's seen success in treating the most severe form of the disease.Ĭasgevy, manufactured by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first approved therapy based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.
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