- Gene therapy demonstrated clinically meaningful vision improvement in legally blind individuals.
- The therapy is capable of restoring vision even in advanced disease stages.
The Latest
A recent two-year phase 2b, randomized, double-masked, sham-controlled multicenter clinical trial by Boyer et al. and Nanoscope Therapeutics investigated mutation-agnostic gene therapy for the treatment of permanent and severe vision loss from of retinitis pigmentosa. The results build on an earlier trial that found 89% of patients injected with the gene therapy to experience an improvement of luminance levels across two visual tests compared to control group patients. This newest trial, named RESTORE, demonstrated significant improvement in best-corrected visual acuity after 52 weeks compared to the control arm. The results showed that gene therapy was well tolerated, with no treatment-related serious or severe adverse events reported.
