Editas test CRISPR to treat beta-thalassemia & sickle cell disease

Editas Medicine Inc., a U.S.-based pharmaceutical company recently announced results from trial of two different CRISPR-Cas9 genome editing strategies, one targeting the beta-globin locus and the other targeting the erythroid enhancer, for treatment of beta-thalassemia & sickle cell disease. The data was presented by the company at the 24th Congress of the European Hematology Association in Amsterdam, report sources.

For the record, Editas Medicine Inc., is a leading genome editing pharmaceutical company, that focus on potential of CRISPR-Cas9 & Cas12a genome editing strategies in treatment of patients with serious diseases. The company aims to develop, manufacture & commercialize durable, transformative precision genomic medicines for wide-ranging class of diseases.

Charles Albright, Chief Scientific Officer, Editas Medicine, was reported to say that the pre-clinical results validating cells edited at the beta-globin locus repopulated all lineages of the blood system including RBC precursors & the high percentage of fetal hemoglobin expression, showed potential to treat beta-thalassemia & sick cell disease. Editing at the site continues to meet the company’s pre-clinical goals for making the medicine for durable induction of fetal hemoglobin & maintaining normal hematopoietic stem cell function, Albright added.

In this study, NBSGW mice (supports engraftment of human hematopoietic stem cells without irradiation) were reportedly given an infusion of human CD34+ cells which were edited either at beta-globin locus or the BCL11Ae. In vivo-derived erythroid cells from BCL11Ae and edited CD34+ hematopoietic stem cells had increased non-productive indels and reduced total indels, as compared to other lineages that have been tested (a phenomenon not observed with beta-globin locus editing). Also, further optimization of guide RNA & nuclease combinations led to fetal hemoglobin expression of approximately 40% in the beta-globin, locus-edited erythroid cells.

Based on the data, the pharmaceutical company has initiated IND-enabling activities for an experimental CRISPR medicine, EDIT-301. The drug is designed to durably treat beta-thalassemia & sickle cell disease by editing the beta-globin locus, cite sources.

Source credits: http://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-presents-pre-clinical-data-treatment-sickle-cell