Durable Clinical Benefits in Severe Sickle Cell Disease with Exagamglogene Autotemcel

Abstract

BACKGROUND: Exagamglogene autotemcel (exa-cel) is a cell therapy that reactivates HbF via non-viral, ex vivo CRISPR/Cas9 gene-editing at the erythroid enhancer region of BCL11A in autologous CD34+ hematopoietic stem and progenitor cells (HSPCs). Exa-cel is approved for patients aged >=12 years with severe sickle cell disease (SCD). We report results from the ongoing Phase 3 CLIMB SCD-121 and CLIMB-131 trials.