First CRISPR gene editing therapy for inherited visual disorders

On the 30th November EDIT-101, developed by Editas Medicine, received the approval from the Food and Drug Administration (FDA) to be the first in vivo CRISPR medicine administered to people anywhere in the world. EDIT-101. EDIT-101 is an experimental CRISPR genome editing medicine for the treatment of Leber Congenital Amaurosis (LCA, an inherited early-onset visual disorder that leads to infantile and juvenile blindness). Editas Medicine, founded in 2013 by four well-known scientists who developed CRISPR tools for genome editing, is a leading genome editing company dedicated to treating patients with genetically defined, severe and untreatable diseases.

EDIT-101 is specifically addressed to patients who suffer from LCA due to mutations in the CEP290 gene. Around 2-3 children in 100.000 worldwide have LCA, and mutations in CEP290 account for 20-30% of these patients. EDIT-101 targets the most frequent mutation in LCA, a deep intronic mutation within CEP290 that generates nonfunctional mRNAs due to aberrant splicing events. The genome editing by EDIT-101 deletes the mutation and the surrounding region without introducing any other modification in the gene and reestablishing the correct splicing. The administration of EDIT-101 by subretinal injection close to photoreceptor cells. This CRISPR-mediated genome editing would only affect photoreceptors and other retinal cells of the patient without any consequence for other tissues and no transmission to progeny.

Editas Medicine and the globally based pharma company Allergan have entered and strategic alliance for performing the clinical assays and licensing the commercialization products. Allergan expects to enrol 10-20 patients for the type I/II clinical assays in order to assess safety, tolerability and efficacy of EDIT-101. (Read our blog entry 18/10/31)