Long-term follow-up demonstrates durable clinical benefits of exagamglogene autotemcel for sickle cell disease with recurrent vaso-occlusive crises: Final results of climb SCD-121

Abstract Background: Exagamglogene autotemcel (exa-cel) is a one-time, ex vivo CRISPR/Cas9 gene-edited, autologous cell therapy approved for patients ≥12 years old with severe sickle cell disease (SCD). We report results for durable, long-term efficacy and safety for the completed phase 3 CLIMB SCD-... View More