Editas Medicine has reported positive outcomes for a gene editing treatment to treat severe sickle cell disease, a hereditary blood disorder with limited treatment options. Published today in the New England Journal of Medicine, results from the RUBY trial show a functional cure was achieved in 27 out of 28 patients, who had no more sickle cell crises in the two years after the therapy was administered.
The treatment in question, called renizgamglogene autogedtemcel (reni-cel), employs CRISPR/Cas12a gene editing to modify hematopoietic stem cells from the patient’s own bone marrow. The editing targets the HBG1 and HBG2 promoters to reactivate the production of fetal hemoglobin, whose production otherwise naturally halts after birth. This form of hemoglobin is known to improve symptoms and reduce the rate of painful crises caused by sickle cell disease, where misshapen red blood cells can interfere with normal blood flow.