Exagamglogene autotemcel is an autologous CRISPR-Cas9 modified hematopoietic stem cell therapy for the treatment of patients with sickle cell disease and recurrent vaso-occlusive crises. Sickle cell disease (SCD) is a genetic disorder characterized by the production of abnormal sickle-shaped red blood cells (called hemoglobin S, HbS) that initiate a pathophysiology resulting in severe pain, progressive multi-organ damage, and premature...
- Mechanism
- Curator reviewed · 1 reference
Exagamglogene autotemcel is an autologous gene therapy in which patient CD34+ hematopoietic stem cells are edited using CRISPR/Cas9 technology. A precise DNA double-strand break is made at a critical transcription factor binding site (GATA1) in the erythroid-specific enhancer region of the BCL11A gene, which disrupts GATA1 binding and reduces BCL11A protein expression. Following infusion and engraftment, the reduction in BCL11A expression results in an increase in γ-globin expression and downstream fetal hemoglobin production. Fetal hemoglobin expression reduces intracellular sickle hemoglobin concentration, preventing red blood cells from sickling and addressing the underlying cause of the disease.
- Primary indication
- Exagamglogene autotemcel (Casgevy) is a gene therapy indicated for the treatment of sickle cell disease in patients ≥12 years of age with recurrent vaso-occlusive crises (VOCs).Curator reviewed · 2 structured indications
- First approval
- Canada, 2024 · United States, 2023 · European Union, 2024
- Code names
- CTX-001
- Brand names
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What you can answer from here — as of July 29, 2026
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