Editas Medicine, Inc. Announces Publication of Pre-Clinical Data Demonstrating the Pharmacology and Specificity of EDIT-101
January 21, 2019 at 09:30 pm IST
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Editas Medicine, Inc. 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. 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.
Editas Medicine, Inc. is a clinical-stage genome editing company. The Company is focused on developing potentially transformative genomic medicines to treat a broad range of serious diseases. It has developed a proprietary gene editing platform based on CRISPR technology. CRISPR uses a protein- ribonucleic acid (RNA) complex composed of an enzyme, including either CRISPR associated protein 9 (Cas9) or Cas12a (CRISPR from Prevotella and Francisella 1, also known as Cpf1), bound to a guide ribonucleic acid (RNA) molecule designed to recognize a particular deoxyribonucleic acid (DNA) sequence. It is engaged in the development of vivo administered gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body. Its lead program, reni-cel, is an experimental ex vivo gene-edited medicine to treat sickle cell disease (SCD), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (TDT).