Editas Medicine Inc (NASDAQ: EDIT) shares backed off Tuesday from Monday’s close, after the company issued data showing support for multiple approaches to create treatments for sickle cell disease, beta-thalassemia.
The company, based in the Harvard-headquartered town of Cambridge, Mass., 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.
Editas’ Chief Scientific Officer Charles Albright declared, "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.”
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.
Shares in Editas dropped 17 cents to $23.43, within a 52-week trading range of $13.12 to $31.81.
