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Intellia Therapeutics Announces New, Robust Genome Editing Data for Sickle Cell Disease at the American Society of Hematology Meeting

INTELLIA THERAPEUTICS (NTLA)  More Company Research Source: GlobeNewswire
US:NASDAQ Investor Relations: ir.intelliatx.com/investor-overview
PDF Data on ex vivo CRISPR/Cas9 genome-edited human hematopoietic stem cells shows increased production of fetal hemoglobinApproximately 80-95 percent editing was achieved in CD34+ cellsIncreased fetal hemoglobin levels were sustained for at least 16 weeks following transplant in mice ATLANTA, Dec. 11, 2017 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using the CRISPR technology, and its collaborator, Novartis, presented initial data from their research collaboration on genome-edited human hematopoietic stem cells. These data showed successful ex vivo editing of the erythroid specific enhancer of BCL11A, a gene associated with prevention of sickle cell disease, and the ability of these cells to stably engraft in mice while maintaining their desired properties. These data were presented today at the American Society of Hematology Annual Meeting in the platform presenta [Read more]

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