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Exagamglogene Autotemcel (Casgevy; Vertex/CRISPR) for Sickle Cell Disease

Exagamglogene autotemcel (Casgevy; Vertex Pharmaceuticals and CRISPR Therapeutics) is an autologous, genome-edited, hematopoietic stem cell-based gene therapy indicated for the treatment of sickle cell disease (SCD) in patients aged 12 years and older with recurrent vaso-occlusive crises (VOCs).

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