A Milestone in Gene Editing: Prof. Locatelli Presents Durable 6-Year Data for Exa-cel in Thalassemia and Sickle Cell Disease
At the EBMT 2026 Annual Meeting, Professor Franco Locatelli from Bambino Gesù Children’s Hospital presented long-term follow-up data for exagamglogene autotemcel (exa-cel), a CRISPR/Cas9-based gene-editing therapy. The study included patients with transfusion-dependent thalassemia (TDT) and sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). With a median follow-up of over three years and some patients reaching over six years, the results provide robust evidence for the long-term efficacy and safety of gene editing in hereditary blood disorders.









