Experiments support opportunities to develop best-in-class therapies for hemoglobinopathies
CAMBRIDGE, Mass., Dec. 11, 2017 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced results from experiments to demonstrate expanded CRISPR genome editing strategies in hematopoietic stem cells for the treatment of hematologic diseases such as sickle cell disease and beta-thalassemia. In these proof of concept experiments, the Company demonstrated CRISPR/Cas9 homology directed repair (HDR) and CRISPR/Cpf1-directed editing in human CD34+ cells. The Company reported these data today in a poster presentation at the 59th Annual Meeting of the American Society of Hematology (ASH) in Atlanta.
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 Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. “Combined with our previously reported data showing the upregulation of fetal hemoglobin, we are continuing to advance our program to develop best-in-class, durable therapies for patients with hemoglobinopathies.”
About Editas Medicine
Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The Company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients. To learn more about Editas Medicine, please visit www.editasmedicine.com.
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 Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Editas Medicine, Inc.