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Vertex Pharmaceuticals
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On Friday, the Food and Drug Administration approved the first gene editing therapy ever to be used in humans, for sickle cell disease, a debilitating blood disorder caused by a single mutated gene.
The agency also approved a second treatment using conventional gene therapy for sickle cell that does not use gene editing.
For the 100,000 Americans with the disease, most of them Black, the approvals offer hope for finally living without an affliction that causes excruciating pain, organ damage and strokes.