exagamglogene autotemcel

Exagamglogene autotemcel is an autologous gene therapy that uses CRISPR/Cas9 genome editing technology to modify the patient’s own CD34+ hematopoietic stem and progenitor cells. The therapy disrupts a regulatory region of the BCL11A gene in erythroid cells, leading to reduced BCL11A expression, increased γ-globin production, and elevated fetal hemoglobin (HbF) levels. This mechanism addresses the underlying cause of sickle cell disease by reducing sickling of red blood cells and also treats transfusion-dependent beta thalassemia by improving hemoglobin balance. Exagamglogene autotemcel is approved for patients aged 12 years and older with sickle cell disease who have recurrent vaso-occlusive crises or with transfusion-dependent beta thalassemia requiring regular blood transfusions[1][2][3][6][8]. It was developed by Vertex Pharmaceuticals in collaboration with CRISPR Therapeutics.

Brand names

Casgevy

Other names

exagamglogene autotemcelCTX001CTX-001CTX 001Casgevy