ARTICLE | Regulation
Two sickle cell gene therapies enter U.S. market, but can both succeed?
FDA approves pair of sickle cell therapies with curative potential, but safety and pricing concerns cloud bluebird’s commercial path
December 9, 2023 1:07 AM UTC
It was a landmark day for sickle cell disease patients. The decision by FDA to simultaneously approve Casgevy from partners CRISPR Therapeutics and Vertex and Lyfgenia from bluebird promises to transform the indication from a serious, painful, lifelong affliction to a potentially curable disease.
Doubts remain, however, about whether there’s opportunity in the market for both products...