Landmark CRISPR Trial Shows Promise Against Fighting Blindness, but Is It a Cure?
Preliminary results from the open-label Phase1/2 BRILLIANCE clinical trial of its investigational CRISPR-based experimental medicine, EDIT-101, have rekindled hope for the patients suffering from blindness due to Leber congenital amaurosis 10 (LCA10).
The safety and efficacy assessments were presented at the XIXth International Symposium on Retinal Degeneration (RD2021) on September 29th.