Currently, Phase 2/3 trials sponsored by ProQR Therapeutics (NCT03913143, ILLUMINATE, and NCT04855045, BRIGHTEN) are testing the efficacy of an AONs-based therapy, sepofarsen, for the treatment of the most common mutation in the most frequently causative gene for LCA, CEP290 [153,154] (Table 1). This evidence concerns the gene CEP290 and Leber congenital amaurosis.