Background: RPE65-associated LCA (RPE65-LCA) is an inherited retinal degeneration caused by the mutations of RPE65 gene and gene therapy has been developed to be a promising treatment. This study aims to evaluate the association between changes in visual function and application of gene therapy in patients with RPE65-LCA. Methods: Several databases (PubMed, Cochrane Library, and Web of Science) were searched for results of studies describing efficacy of gene therapy in patients with RPE65-LCA. Six studies, which included one randomized and five prospective non-randomized clinical trials, 164 eyes met our search criteria and were assessed. Results: The BCVA significantly improved in treated eyes at 1 yr post treatment by − 0.10 logMAR (95% CI, − 0.17-0.04; p = 0•002), while there was no significant difference at 2-3 years post treatment (WMD: 0.01; 95% CI, − 0.00-0.02; p = 0•15). FST sensitivity to blue flashes also improved by 1.60 log (95% CI, 0.66-2.55; p = 0.0009), but no significant difference to red flashes (WMD: 0.86; 95% CI, − 0•29-2.01; p = 0.14) at 1 yr. There was no significant difference in central retinal thickness at 1 yr, but central retina in treated eyes appeared thinner at 2-3 years post treatment by 19.21 μm (95% CI, − 34.22-4.20; p = 0.01). Conclusions: Human gene therapy is a pioneering treatment option for RPE65-LCA. Although its efficacy appears to be limited to less than 2 yrs after treatment, it carries the potential for further improvement and prolongation of efficacy.

<b><i>Background:</i></b> Conbercept, as a novel vascular endothelial growth factor (VEGF) inhibitor, was approved for the treatment of neovascular age-related macular degeneration (nAMD) in China. <b><i>Objective:</i></b> This study aimed to compare the efficacy and safety between conbercept and ranibizumab in patients with nAMD. <b><i>Methods:</i></b> Several databases (PubMed, Web of Science, China National Knowledge Infrastructure, and WANFANG) were searched for the results of studies describing conbercept and ranibizumab for the treatment of nAMD. Sixteen randomized controlled trials including 1,224 eyes met our search criteria and were assessed. <b><i>Results:</i></b> Conbercept and ranibizumab had comparable effects on improving visual acuity at 3 months (standardized mean difference [SMD]: −0.19; 95% confidence interval [CI]: −0.46 to 0.08; <i>p</i> = 0.17) and 6–12 months (SMD: −0.01; 95% CI: −0.20 to 0.18; <i>p</i> = 0.90). At 3 months and 6–12 months, the differences in the change of central macular thickness in conbercept and ranibizumab groups were 1.06 μm (95% CI: −3.52 to 5.64; <i>p</i> = 0.65) and −0.12 μm (95% CI: −9.26 to 9.02; <i>p</i> = 0.98). In the short term, there was no significant difference between the 2 groups with respect to ocular adverse events (odds ratio [OR]: 0.86; 95% CI: 0.46–1.61; <i>p</i> = 0.63). No significant differences were observed in the recovery rate of choroidal neovascularization leakage between conbercept and ranibizumab at both 3 months (OR: 1.49; 95% CI: 0.83–2.68; <i>p</i> = 0.18) and 6–12 months (OR: 0.66; 95% CI: 0.18–2.43; <i>p</i> = 0.53). There were significant differences between conbercept and ranibizumab in terms of decreasing intraocular pressure (weighted mean difference [WMD]: −1.74; 95% CI: −2.28 to −1.20; <i>p</i> &#x3c; 0.00001), the plasma VEGF level (WMD: −21.49; 95% CI: −26.28 to −16.70; <i>p</i> &#x3c; 0.00001), and the C-reactive protein level (WMD: −1.16; 95% CI: −1.45 to −0.87; <i>p</i> &#x3c; 0.00001) in the short term. <b><i>Conclusion:</i></b> Conbercept was similar to ranibizumab in terms of efficacy and safety for the treatment of nAMD in China. Further studies with longer term observation are needed to support this conclusion.

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