Advances in biopharmaceutical products for hemophilia.

IF 4.6 2区综合性期刊 Q1 MULTIDISCIPLINARY SCIENCES iScience Pub Date : 2024-12-03 eCollection Date: 2024-12-20 DOI:10.1016/j.isci.2024.111436

Junzheng Wu, Xiaoling Liu, Huichuan Yang, Yanlin He, Ding Yu

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引用次数: 0

Abstract

Hemophilia is caused by the deficiency of clotting factors due to a single genetic abnormality. Replacement therapies have evolved from plasma-derived to recombinant coagulation factor concentrates but continue to have certain limitations. Monoclonal antibodies are clinical prophylactic treatment options unaffected by inhibitors and have better compliance than coagulation factor concentrates for patients with hemophilia. Gene therapy is a breakthrough in hemophilia treatment, as it drives the hepatic expression of factor VIII or factor IX and requires only a single administration to enable long-term replacement treatment in adult patients. Furthermore, biopharmaceutical products that target new pathways unaffected by inhibitors, including tissue factor pathway inhibitors, activated protein C, and antithrombin, as well as pharmaceutical technology advances to reduce dosing frequency, have demonstrated promising clinical results. This review provides a comprehensive overview of these biopharmaceutical products and explores the future of hemophilia treatment.

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来源期刊

iScience Multidisciplinary-Multidisciplinary

CiteScore

7.20

自引率

1.70%

发文量

1972

审稿时长

6 weeks

期刊介绍： Science has many big remaining questions. To address them, we will need to work collaboratively and across disciplines. The goal of iScience is to help fuel that type of interdisciplinary thinking. iScience is a new open-access journal from Cell Press that provides a platform for original research in the life, physical, and earth sciences. The primary criterion for publication in iScience is a significant contribution to a relevant field combined with robust results and underlying methodology. The advances appearing in iScience include both fundamental and applied investigations across this interdisciplinary range of topic areas. To support transparency in scientific investigation, we are happy to consider replication studies and papers that describe negative results. We know you want your work to be published quickly and to be widely visible within your community and beyond. With the strong international reputation of Cell Press behind it, publication in iScience will help your work garner the attention and recognition it merits. Like all Cell Press journals, iScience prioritizes rapid publication. Our editorial team pays special attention to high-quality author service and to efficient, clear-cut decisions based on the information available within the manuscript. iScience taps into the expertise across Cell Press journals and selected partners to inform our editorial decisions and help publish your science in a timely and seamless way.