CAMBRIDGE, Mass., May 11, 2017 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced results from its pre-clinical hematopoietic stem cell (HSC) program confirming the potential for CRISPR-based editing to treat sickle cell disease and beta-thalassemia. In an oral presentation today at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington, D.C., the Company presented data demonstrating a substantial increase in fetal hemoglobin protein using a novel and potent genome editing strategy.
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 Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. “We believe durable repopulation of the blood system with edited stem cells is a critical component for developing genomic medicines for patients with genetic blood diseases. With this in vivo proof-of-concept, we are continuing to advance our program to develop differentiated medicines with our novel, potent editing strategy.”
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.
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