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author：Wikipedia 2025-09-03 15:20:27 - browse：565

Christine Kao/STAT

The Food and Drug Administration’s approval on Friday of two gene therapies for sickle cell disease sets up what could be an intense competition between Bluebird Bio, maker of Lyfgenia, and the partnership of Vertex Pharmaceuticals and CRISPR Therapeutics, whose drug is Casgevy.

For the first time, physicians and patients will have a choice of potentially curative treatments for an inherited disease that affects nearly 100,000 Americans.

The Vertex/CRISPR team would seem to have the edge. As the first CRISPR-based therapy, Casgevy has the advantage of being powered by the latest and buzziest biological technology, though some physicians and patients may be more comfortable with the lentiviral vector technology behind Lyfgenia, which dates back to the 1990s — even with a “black box” warning of a risk of blood cancer added to its label.

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