Editas Medicine, Inc.(Nasdaq: EDIT), a leading genome editing company, today announced the journal Nature Medicine published the comprehensive, pre-clinical data demonstrating the pharmacology and specificity of EDIT-101, an experimental, CRISPR genome editing medicine being investigated for the treatment of Leber congenital amaurosis 10 (LCA10), a genetically-driven form of blindness. EDIT-101 is set to be the first in vivo, or editing inside the body, CRISPR-based medicine administered to people anywhere in the world.
“We are thrilled Nature Medicine published our paper sharing the comprehensive set of pre-clinical data for EDIT-101 and our approach to creating CRISPR-based genome editing medicines,” said Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. “The research presented in this important manuscript provided the foundation for our recently accepted Investigational New Drug (IND) application for EDIT-101 to treat LCA10, bringing us closer to the clinic and our goal of realizing the full potential of genome editing. We look forward to initiating the Phase 1/2 study to further evaluate the safety, tolerability, and efficacy of EDIT-101 for people living with LCA10, a disease currently with no treatment options.”
Published results detail the development of EDIT-101, an experimental genome editing medicine designed to remove the abnormal splice donor created by the IVS26 mutation in the CEP290 gene found in LCA10 patients and restore normal CEP290 expression. The paper summarizes in vitro experiments in human cells and retinal explants demonstrating the molecular mechanism of action and nuclease specificity.
Subretinal delivery of EDIT-101 in humanized CEP290 mice showed rapid and sustained CEP290 gene editing. A comparable surrogate non-human primate (NHP) vector also achieved productive editing of the NHP CEP290 gene at levels that met the target therapeutic threshold and demonstrated the ability of CRISPR/Cas9 to edit somatic primate cells in vivo. The results presented support further development of EDIT-101 for the treatment of patients with LCA10, as well as the application of genome editing approaches to treat a wide variety of inherited retinal diseases.
In the Phase 1/2 clinical trial, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) plan to initiate patient screening mid-year and begin patient dosing in the second half of 2019, enrolling 10-20 patients in the U.S. and Europe.
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