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In pre-clinical experiments, human HSCs were edited ex vivo and infused into mice. Four months following transplant, the bone marrow was collected to evaluate engraftment and to assess HSC contribution to blood production. In this study, successful and durable repopulation of the blood system in vivo was observed. In addition, analysis of the relationship between editing frequency and the level of fetal hemoglobin production suggests this proprietary editing approach has the potential to be more potent than other approaches that have been previously reported. These data support the continued development of medicines for people with genetically-defined and genetically-treatable diseases of the blood.
“We are encouraged by these results demonstrating the potential for edited hematopoietic stem cells to provide a durable therapy for patients with β-hemoglobinopathies,” said
About Editas Medicine
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,” similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. 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 Annual Report on Form 10-K, which is on file with the
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