In a recent report published in the New England Journal of Medicine, Gupta, Sharma, Frangoul, Kanter, Mapara, Dalal, Alavi, and colleagues described interim results from the phase 1–2 BEACON study evaluating ristoglogene autogetemcel (risto-cel) in patients with sickle cell disease. This base-editing approach targets the HBG1 and HBG2 promoters to increase fetal hemoglobin production while reducing sickle hemoglobin. Among 31 treated patients, investigators observed rapid engraftment, sustained fetal hemoglobin expression, and no severe vaso-occlusive crises beyond 60 days after the last red-cell transfusion. These findings support continued evaluation of this therapeutic strategy despite notable treatment-related risks. (https://www.nejm.org/doi/full/10.1056/NEJMoa2504835)