Revolutionizing genetic disease treatment: The case of Exagamglogene autotemcel

Recent approval of exagamglogene autotemcel (exa-cel) on November 16, 2023 in the United Kingdom (UK) developed by Vertex pharmaceuticals and CRISPR Therapeutics, represents the first cell-based therapy for treatment of sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β-thalassemia (TDT) in patients aged 12 and above. Following suit, the United State Food and Drug Administration (FDA) authorized ex-cel for the same indications on December 8, 2023. Exa-cel represents the first commercially approved CRISPR-based gene therapy, offering hope to approximately 25,000 eligible patients in the U.S. and Europe. The significance of exa-cel extends beyond revolutionizing therapeutic intervention in genetic medicine; it also raises profound ethical considerations and societal implications related to genome manipulation.