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Editas Medicine Demonstrates Dose-Dependent, In Vivo Editing with EDIT-101 in CEP290 Transgenic MiceCAMBRIDGE, Mass., Oct. 19, 2017 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced results from a pre-clinical study in transgenic mice demonstrating dose-dependent, in vivo editing using EDIT-101, Editas Medicine’s pre-clinical product candidate for the treatment of Leber Congenital Amaurosis type 10 (LCA10). The study was conducted in mice that have a human CEP290 common intron 26 knock-in (HuCEP290 IVS26 KI mice), an animal model for most common genetic change that causes LCA10. The results of this study further reinforce Editas Medicine’s belief in the transformative potential of EDIT-101 as a genome editing medicine to help patients with LCA10. LCA10 is an inherited retinal degenerative disease caused by mutations in the CEP290 gene that appears in childhood and leads to blindness. The Company reported these data today in a poster presentation at the 25th Anniversary Congress of the European Society of Gene and Cell Therapy (ESGCT) in Berlin. In this study, HuCEP290 IVS26 KI mice were treated with EDIT-101 by subretinal injection, resulting in efficient transduction and gene editing in the retinal photoreceptor cells of the mice, which is the cell type affected in LCA10 patients. The onset of CEP290 gene editing was rapid and was detectable as early as three days post-delivery with a further significant increase in editing observed by one week. The components were measured, and both Cas9 mRNA and guide RNA (gRNA) levels correlated with editing. At the administered dose of 1E+12 vg/mL vector concentration, editing levels exceeded levels predicted to be therapeutically relevant in 90 percent (27/30) of EDIT-101 treated eyes with a median of 31 percent of photoreceptors harboring productively edited CEP290 in the transduced neuroretina. The productive CEP290 gene editing rates were stable through six months of observation, and the expression of Cas9 mRNA and gRNA decreased over time. “To date, we have made significant progress in our LCA10 program. We have achieved predictive therapeutic levels of productive CEP290 gene editing in several pre-clinical settings, including in neural retinas in human cells, in transgenic mice, and in non-human primates. Collectively, these data demonstrate that we can deliver EDIT-101 to photoreceptors and can edit in the eye at levels well above the anticipated minimum therapeutic threshold, supporting the clinical development of EDIT-101 for the treatment of patients suffering from LCA10,” said Charles Albright, Ph.D., Chief Scientific Officer, Editas Mediine. Editas Medicine plans to submit an Investigational New Drug (IND) application for EDIT-101 in mid-2018. In March, Editas Medicine and Allergan Pharmaceuticals International Limited (“Allergan”) entered into a strategic research and development alliance under which Allergan received an exclusive option to license up to five of Editas Medicine’s genome-editing ocular programs, including Editas Medicine’s lead program for LCA10. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicine’s unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1. About Leber Congenital Amaurosis About Editas Medicine Forward-Looking Statements Contacts Investors: |