用于血友病治疗的双特异性抗体与基因治疗产品的临床研发现状  

Review of the clinical trials for bispecific antibody and gene therapy treatments for hemophilia that have approved

作　　者：陈子乔 陈霞 CHEN Zi-qiao;CHEN Xia(School of Basic Medicine and Clinical Pharmacy,China Pharmaceutical University,Nanjing 211198,Jiangsu Province,China;Hangzhou Tigermed Consulting Co.,Ltd,Hangzhou 310051,Zhejiang Province,China)

机构地区：[1]中国药科大学基础医学与临床药学学院,江苏南京211198 [2]杭州泰格医药科技股份有限公司,浙江杭州310051

出　　处：《中国临床药理学杂志》2025年第1期137-142,共6页The Chinese Journal of Clinical Pharmacology

摘　　要：血友病是一种罕见的单基因遗传性凝血因子缺乏性疾病,患者自幼儿期发病,重症患者常有自发性出血或关节肌肉出血史,需要长期频繁接受因子输注治疗。本文回顾了双特异性抗体药艾美赛珠单抗与2种基因疗法ROCTAVIAN和HEMGENIX针对不同血友病亚型的临床研发过程,以此总结罕见病药物研发的策略和基因治疗产品临床研发的关键要素,为今后同类药物在类似适应证中的研发提供借鉴。Hemophilia is a rare monogenic inherited coagulation factor deficiency disease,which begins in early childhood,and severe patients often have a history of spontaneous bleeding or joint muscle bleeding,requiring long-term frequent transfusion of clotting factor.This article reviews the clinical development process of bispecific antibody drug EMICIZUMAB and two gene therapies ROCTAVIAN and HEMGENIX for the corresponding hemophilia subtypes,so as to summarize the strategies for rare disease drug development and the key elements of gene therapy drug development,and to provide reference for the development of similar drugs in similar indications in the future.

关 键 词：双特异性抗体 血友病 基因治疗 临床研发 

分 类 号：R973[医药卫生—药品]