Editas Medicine Presents Pre-Clinical Data for Treatment of Sickle Cell Disease and Beta-Thalassemia at the 24th Congress of the European Hematology Association
IND-enabling activities initiated for EDIT-301, a potentially best-in-class experimental medicine 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,
“We are encouraged by these pre-clinical results demonstrating cells edited at the beta-globin locus repopulated all lineages of the blood system including, importantly, the red blood cell precursors and the high percentage of fetal hemoglobin expression. Editing at this site continues to meet our preclinical goals for making a medicine including robust, long-term induction of fetal hemoglobin and maintenance of normal hematopoietic stem/progenitor cell function,” said
As a leading genome editing company,
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Source: Editas Medicine, Inc.