The US Food and Drug Administration today approved a first-of-its kind medical treatment that uses Crispr gene editing.
Called Casgevy, the therapy is intended to treat patients with sickle cell disease, an inherited blood disorder that affects more than 100,000 people in the United States. The UK first approved the groundbreaking treatment on November 16. The therapy is made by Vertex Pharmaceuticals of Boston and Crispr Therapeutics of Switzerland.
The treatment aims to eliminate episodes of debilitating pain that are a hallmark of sickle cell disease. It involves editing patients’ cells outside the body to override the genetic defect that causes the disease. Adults and children 12 and older with frequent
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