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.
Continue reading...