从阵发性睡眠性血红蛋白尿药物靶点出发的补体C5抑制剂在疾病治疗中的应用

罕见病的治疗是全球性的医疗难题，其患病人数也在逐年上升。阵发性睡眠性血红蛋白尿（PNH）作为罕见病的一种，其靶向治疗药物的靶点均为补体成分5（C5），显现出该靶点具有极大的临床研究意义。C5作为补体系统的重要组分之一，在先天免疫中发挥作用，参与机体免疫保护并且调节适应性免疫反应。目前多种疾病的发生发展均被证实与C5的异常激活相关，因此研究其参与疾病进展的作用机制及进行药物开发已成为新药研发的热点。如今C5抑制剂已有药物上市，同时现阶段靶向C5的药物中也有单克隆抗体、多肽、核酸类药物等进入临床试验阶段。因此，通过对目前C5激活机制以及C5抑制剂类药物的研发进展进行综述，以期为相关药物的研发及其深层次应用提供参考。

Application of Complement C5 Inhibitor in Disease Treatment Based on Paroxysmal Nocturnal Hemoglobinuria Drug Target

The treatment of rare diseases is a global medical problem, and the number of patients with rare diseases has also been increasing year by year. Paroxysmal nocturnal hemoglobinuria (PNH), a rare disease, is targeted at complement component 5 (C5) in all targeted therapeutic drugs, which shows great clinical significance of the target. As one of the important components of complement system, C5 plays a role in innate immunity, participates in immune protection and regulates adaptive immune response. At present, the occurrence and development of many diseases have been confirmed to be related to abnormal activation of C5. Therefore, it has become a hot spot for new drug research and development to study the mechanism of its participation in disease progression and drug development. Nowadays, C5 inhibitors have been marketed. At the same time, C5-targeted drugs like monoclonal antibodies, polypeptides and nucleic acid drugs have entered the clinical trial stage. Therefore, based on the current research on C5 activation mechanism, the research and development progress of C5 inhibitors is reviewed, which provides reference for the research and development and deep application of related drugs.