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In experiments, CRISPR/Cas9 caused efficient and reproducible HDR in CD34+ cells with minimal impact on cell viability. In separate studies, CRISPR/Cpf1 efficiently edited at multiple sites, including targets associated with hereditary persistence of fetal hemoglobin (HPFH). These results confirm that Cpf1-directed editing expands the number of genomic sites accessible to develop genome editing medicines.
“We are encouraged by these results demonstrating efficient targeted integration at the beta-hemoglobin locus with CRISPR/Cas9 and results demonstrating efficient on target editing of adult human hematopoietic stem cells with CRISPR/Cpf1. These data together support multiple approaches to creating a superior therapy for the treatment of sickle cell disease and beta-thalassemia,” said
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 Company’s goal of developing therapies. 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.