Two splice-switching oligonucleotide drugs targeting two retinal disorders, Leber’s congenital Amaurosis 10 (LCA10) and Usher syndrome type II, sepofarsen (or QR-110) targeting CEP290 [43] and QR-421a targeting USH2A [146], respectively (developed by ProQR Therapeutics) are moving to late-stage clinical trials, after good clinical results in phase I/II studies [147] (Table 3). The gene discussed is CEP290; the disease is retinal disorder.