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, opening up two “transformative therapy” avenues for some patients.
The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and Crispr Therapeutics. Both therapies are made from the patients’ own blood stem cells and were approved for people aged 12 and older.
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