The world's first CRISPR-based gene therapy has been approved for children as young as two years old, opening the possibility of a single, potentially curative treatment to thousands of American children with sickle cell disease before years of organ damage can narrow what medicine can do for them. On July 1, 2026, the FDA granted supplemental approval to Casgevy (exagamglogene autotemcel), extending its existing indication from patients aged 12 and older to any patient aged 2 and above with sickle cell disease with recurrent vaso-occlusive crises.
Approximately 5,500 additional American children are now eligible for this established one-time therapy, according to Vertex Pharmaceuticals, Casgevy's developer. Casgevy also covers transfusion-dependent beta-thalassemia in this new age indication.
