Gene therapy for beta thalassemia and sickle cell disease in Europe and the United States

SUMMARY

Following the lead of the United States (US) Food and Drug Administration (FDA), the European Medicines Agency (EMA) approved gene-editing therapy with exagamglogene autotemcel in early 2024 for the treatment of patients with transfusion-dependent β-thalassemia or severe sickle cell disease. A year earlier, the FDA granted marketing authorisation for gene therapy with lovotibeglogene autotemcel in patients with severe sickle cell disease. This article describes the results of the clinical trials on the basis of which these new treatments were authorised for marketing.

(BELG J HEMATOL 2024;15(8):313–4)