US FDA approves Vertex's gene therapy for sickle cell disease in children as young as two
- Posted on July 1, 2026
- By Bing News
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US FDA approves Vertex's gene therapy for sickle cell disease in children as young as two
July 1 (Reuters) - The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals' gene therapy in children as young as two with inherited blood disorders, including sickle cell disease, the first such treatment cleared for this age group. Casgevy, a one-time treatment made from a patient's own blood stem cells, was previously approved for patients aged 12 and older with
