A new “functional cure” for sickle cell disease may come in the form of an experimental one-time gene editing cell therapy, renizgamglogene autogedtemcel (reni-cel), according to findings published in the New England Journal of Medicine.
Reni-cel is designed to modify a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease, increasing fetal hemoglobin levels to prevent red blood cells from forming into sickle-shaped cells, and improving overall hemoglobin levels to reduce complications from the disease.