Editas Medicine announces publication of EDIT-101 data in Nature Medicine
Editas Medicine announced the journal Nature Medicine published the 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, 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," the company said in a statement. 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.