Results from the phase 1/2 BRILLIANCE trial, which is using CRISPR-based gene editing therapy to treat individuals who have Leber Congenital Amaurosis (LCA), showed that 11 out of 14 treated participants experienced some improvements in vision and quality-of-life measures. All trial participants, including 12 adults (ages 17 to 63) and 2 children (ages 10 and 14), were born with LCA, a form of inherited blindness. The participants received a single injection of the CRISPR/Cas9 genome editing medicine EDIT-101 in 1 eye. This trial, which included the first patient to ever receive a CRISPR-based investigational medicine, focused primarily on safety with a secondary analysis for efficacy. 

“This research demonstrates that CRISPR gene therapy for inherited vision loss is worth continued pursuit in research and clinical trials,” principal investigator Eric Pierce, MD, PhD, said in a news release. Dr. Pierce is director of the Ocular Genomics Institute and Berman-Gund Laboratory for the Study of Retinal Degenerations at Mass Eye and Ear and Harvard Medical School.