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In these experiments, NBSGW mice received an infusion of human CD34+ cells which had been edited either at the BCL11A erythroid enhancer (BCL11Ae) 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.
“We are encouraged by these preclinical results demonstrating cells edited at the HBG1/2 promoters repopulated all lineages of the blood system including, importantly, the red blood cell precursors. Editing at this site met our preclinical goals including robust, long-term induction of fetal hemoglobin and maintenance of normal hematopoietic stem/progenitor cell function,” said
As a leading genome editing company,
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the safety and efficacy of the Company’s editing approach to treating hemoglobinopathies. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the
Source: Editas Medicine, Inc.