Editas Medicine Reports Data Supporting Multiple Approaches to Create Treatments for Sickle Cell Disease and Beta-Thalassemia
Source: Nasdaq GlobeNewswire / 11 Dec 2017 18:00:20 America/New_York
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.
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