Review of the clinical trials for bispecific antibody and gene therapy treatments for hemophilia that have approved
10.13699/j.cnki.1001-6821.2025.01.028
- VernacularTitle: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
- Author:
Zi-qiao CHEN
1
;
Xia CHEN
Author Information
1. 中国药科大学基础医学与临床药学学院,江苏南京 211198
- Publication Type:Journal Article
- Keywords:
bispecific antibody;
hemophilia;
gene therapy;
clinical development
- From:
The Chinese Journal of Clinical Pharmacology
2025;41(1):137-142
- CountryChina
- Language:Chinese
-
Abstract:
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.
