Editas presents pre-clinical data in Sickle Cell Disease, Beta-Thalassemia
Editas Medicine announced results from a follow-up study to assess two different CRISPR genome editing strategies, one targeting the BCL11A erythroid enhancer and one targeting the beta-globin locus, for the treatment of sickle cell disease and beta-thalassemia. In this study, NBSGW mice received an infusion of human CD34+ cells which had been edited either at the BCL11Ae or at the beta-globin locus. In vivo-derived erythroid cells from BCL11Ae-edited CD34+ hematopoietic stem/progenitor cells had reduced total indels and increased non-productive indels as compared to other tested lineages, a phenomenon not observed with beta-globin locus editing. Additionally, further optimization of nuclease and guide RNA combinations led to fetal hemoglobin expression of approximately 40 percent in the beta-globin locus-edited erythroid cells. Based on the data, Editas Medicine has initiated IND-enabling activities for EDIT-301, an experimental CRISPR medicine designed to durably treat sickle cell disease and beta-thalassemia by editing the beta-globin locus.