George Q Daley
N Engl J Med . 2024 May 9;390(18):1642-1645. doi: 10.1056/NEJMp2314279. Epub 2024 Apr 24.
The recent regulatory approval of exagamglogene autotemcel (exa-cel [Casgevy, Vertex Pharmaceuticals/CRISPR Therapeutics]) in both the United States and United Kingdom for the treatment of sickle cell disease (SCD) and transfusion-dependent β-thalassemia marks the dawn of the era of gene editing in medicine.