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Editas Medicine announced results from experiments to demonstrate expanded CRISPR genome editing strategies in hematopoietic stem cells for the treatment of sickle cell disease and beta-thalassemia. In these experiments, the company demonstrated HBG1/2 promoter-edited CD34+ cells robustly engrafted in mice without lineage skewing of red blood cell precursors. The company reported these data in an oral presentation at the 60th Annual Meeting of the American Society of Hematology. In these experiments, NBSGW mice received an infusion of human CD34+ cells which had been edited either at the BCL11A erythroid enhancer site or at the HBG1/2 promoter site. Analysis of bone marrow collected eight to 16 weeks post-infusion demonstrated that robust fetal hemoglobin induction was achieved when targeting HBG1/2 promoters. Notably, editing HBG1/2 promoters upregulated fetal hemoglobin with superior repopulation of red blood cell precursors as compared to editing the BCL11Ae site. The red blood cell precursors from bone marrow edited at the BCL11Ae site had lower productive editing rates compared to other lineages and showed increased level of apoptosis, or programmed cell death, in erythroid culture compared to HBG1/2 promoter-edited cells. Increased production of fetal hemoglobin can be beneficial to patients with sickle cell disease or beta-thalassemia. Editing at the HBG1/2 site is a differentiated approach for development of a human therapeutic for the treatment of sickle cell disease and beta-thalassemia as compared to other medicines currently under development that edit at the BCL11Ae site.

Editas Medicine (EDIT) announced the FDA has accepted the company's investigational new drug, or IND, application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of Leber Congenital Amaurosis type 10, or LCA10. With the IND acceptance, Editas Medicine has earned a $25M milestone payment from Allergan (AGN) as part of the alliance between the companies to discover and develop experimental ocular medicines targeting serious, vision-threatening diseases. Editas Medicine and its partner, Allergan Pharmaceuticals International, a wholly-owned subsidiary of Allergan, expect to enroll 10 to 20 patients in a Phase 1/2 open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101.