Editas Medicine, Inc. announced positive Initial EDIT-301 Safety and Efficacy Data from the First Four Patients Treated in the RUBY Trial and the First Patient Treated in the EdiTHAL Trial. These promising data support the belief that EDIT-301 can be a clinically differentiated, one-time, durable medicine that can provide life changing clinical benefits to patients with sickle cell disease and beta thalassemia long term, specifically driving early and robust correction of anemia and sustained increases in fetal hemoglobin. EDIT-301 uses AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease, to edit the promoter regions of gamma globin gene 1 and 2 to increase the expression of HbF to mimic the naturally occurring mechanism of hereditary persistence of fetal hemoglobin to treat SCD.

The RUBY trial marks the first time AsCas12a was used to successfully edit human cells in a clinical trial. EHA Oral Presentation Details: Title: EDIT-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/II Clinical Trial (RUBY) of Patients with Severe Sickle Cell Disease Using Highly Specific and Efficient AsCas12a Enzyme. Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children's, Cleveland, OH, United States.