Please note this is an extract of the original bulletin. MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia Page summary: Casgevy (exagamglogene autotemcel) is based on the innovative gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020 Change made: First published.
adminNovember 24, 2023

