Key Points
Dec 8 (Reuters) - The U.S. Food and Drug Administration (FDA) on Friday approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough CRISPR gene editing technology...
The agency approved Lyfgenia from bluebird bio (BLUE.O), and a separate treatment called Casgevy by partners Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN)...
In sickle cell disease, the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the body's tissues...
I prefer to call them a transformative therapy because patients will still have sickle cell disease on the other side of gene therapy," said Dr Sharl Azar, medical director of the Comprehensive Sickle Cell Disease Treatment Center at Massachusetts General Hospital...
Bluebird bio's sickle cell therapy is designed to work by inserting modified genes into the body through disabled viruses to help the patient's red blood cells produce normal hemoglobin...
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