US regulators approve two gene therapies for sickle cell disease

10 months ago 37

Blood disorder that can lead to premature death affects estimated 100,000 people in the United States, most of whom are Black

The US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene editing technology.

The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and Crispr Therapeutics.

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