About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published today in the New England Journal of Medicine.
Led by scientists from Mass Eye and Ear, Oregon Health Sciences University, and University of Pennsylvania, the BRILLIANCE trial evaluated the safety and effectiveness of EDIT-101, an experimental gene editing treatment developed by Editas Medicine that uses CRISPR technology. The experimental treatment was designed to edit a mutation in the CEP290 gene, which provides instructions to create a protein that is critical for sight.
People with this gene mutation have a rare condition that is commonly called Leber Congenital Amaurosis, or LCA, Type 10, for which there is currently no Food and Drug Administration-approved treatment. LCA’s various types occur in about 2 or 3 out of 100,000 newborns.
The new paper describes the study’s findings through February 2023 and details how the trial’s 14 participants — 12 adults and two children — responded to receiving EDIT-101 in one eye. The BRILLIANCE trial results showed 11 out of 14 treated participants experienced some improvements in vision and quality of life measures. Researchers also found that the CRISPR-based therapy was safe, with no dose-limiting toxicities reported.