ProQR are conducting a Phase 2/3 clinical trial (Illuminate) to measure the safety and effectiveness of RNA therapy, Sepofarsen which aims to treat LCA 10. Sepofarsen is designed to repair the CEP290 RNA defect and produce a normal functioning CEP290 protein, which will result in improved vision in participants. This study is ongoing in a number of clinical centres in North America and Europe, involving 30 patients over a 25 month period.
More information about the Illuminate trial can be found on the ProQR and Illuminate websites.