US FDA approves two gene therapies for sickle cell disease

The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy, developed by partners Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN), and bluebird bio's (BLUE.O) Lyfgenia were approved for people aged 12 years and older.

Sickle cell disease (SCD) is a painful, inherited blood disorder in which the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the body's tissues.

The disease, which can be debilitating and lead to premature death, affects an estimated 100,000 people in the United States, most of whom are Black.