Casgevy® (exagamglogene autotemcel)
for Sickle Cell DIsease
Casgevy (exagamglogene autotemcel) is a gene therapy given as a one-time intravenous infusion to treat adults and children aged 12 and older with sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT). It uses the patient’s own blood stem cells, edited with CRISPR/Cas9 to reduce BCL11A gene activity. The edited cells are reinfused after conditioning with busulfan, promoting the production of HbF and reducing VOCs in SCD and transfusion requirements in TDT.
