Genetically Modified Cell Therapy Reduces Severe Vaso-occlusive Crises in Patients With SCD

Treatment with ristoglogene autogetemcel (risto-cel) cell therapy led to rapid engraftment, improved fetal hemoglobin (HbF) expression, and reduced vaso-occlusive crises in patients with sickle cell disease (SCD), according to the phase 1/2 BEACON trial published in the New England Journal of Medicine.1

Risto-cel, an investigational autologous CD34+ cell therapy, was developed by using base editing to target the HBG1 and HBG2 promoters and inhibit binding of the BCL11A protein without modifying BCL11A expression. This mechanism of action boosts production of anti-sickling HbF while reducing the level of sickle hemoglobin (HbS).