Age-related macular degeneration (AMD) is a multifactorial disease, which can culminate in irreversible vision loss and blindness in elderly. Nowadays, there is a big gap between dry AMD and wet AMD on treatment. Accounting for nearly 90% of AMD, dry AMD still lacks effective treatment. Numerous genetic and molecular researches have confirmed the significant role of the complement system in the pathogenesis of AMD, leading to a deeper exploration of complement inhibitors in the treatment of AMD. To date, at least 14 different complement inhibitors have been or are being explored in AMD in almost 40 clinical trials. While most complement inhibitors fail to treat AMD successfully, two of them are effective in inhibiting the rate of GA progression in phase II clinical trials, and both of them successfully entered phase III trials. Furthermore, recently emerging complement gene therapy and combination therapy also offer new opportunities to treat AMD in the future. In this review, we aim to introduce genetic and molecular associations between the complement system and AMD, provide the updated progress in complement inhibitors in AMD on clinical trials, and discuss the challenges and prospects of complement therapeutic strategies in AMD.

中文翻译：

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年龄相关性黄斑变性中的补体抑制剂：一种潜在的治疗选择

年龄相关性黄斑变性 (AMD) 是一种多因素疾病，可导致老年人不可逆的视力丧失和失明。如今，干性 AMD 和湿性 AMD 在治疗上存在很大差距。占 AMD 近 90% 的干性 AMD 仍缺乏有效的治疗方法。大量的基因和分子研究证实了补体系统在 AMD 发病机制中的重要作用，导致补体抑制剂在 AMD 治疗中的深入探索。迄今为止，在近 40 项临床试验中，至少有 14 种不同的补体抑制剂已经或正在 AMD 中进行探索。虽然大多数补体抑制剂未能成功治疗 AMD，但其中两种在 II 期临床试验中可有效抑制 GA 进展速度，并且均成功进入 III 期试验。此外，最近出现的补体基因治疗和联合治疗也为未来治疗 AMD 提供了新的机会。在这篇综述中，我们旨在介绍补体系统与 AMD 之间的遗传和分子关联，提供 AMD 补体抑制剂在临床试验中的最新进展，并讨论 AMD 补体治疗策略的挑战和前景。