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Research progress in small molecule inhibitors of complement factor B
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Shuai WEN1, Yao ZHAO1, Yan WANG1, Xing LI1, Yi MOU1, *, Zheng-yu JIANG2, *
Acta Pharmaceutica Sinica | 2025, 60(1) : 37 - 47
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Acta Pharmaceutica Sinica | 2025, 60(1): 37-47
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Research progress in small molecule inhibitors of complement factor B
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Shuai WEN1, Yao ZHAO1, Yan WANG1, Xing LI1, Yi MOU1, *, Zheng-yu JIANG2, *
Affiliations
  • 1. College of Pharmacy and Chemistry & Chemical Engineering, Taizhou University, Taizhou 225300, China
  • 2. Jiangsu Key Laboratory of Drug Design and Optimization, School of Pharmacy, China Pharmaceutical University, Nanjing 210009, China
Published: 2025-01-12 doi: 10.16438/j.0513-4870.2024-0600
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The alternative pathway (AP) of the complement system is a key contributor to the pathogenesis of several diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), C3 glomerular disease (C3G) and age-related macular degeneration (AMD). Complement factor B (CFB) is a trypsin-like serine protein that circulates in the human bloodstream in a latent form. As a key node of the alternative pathway, it is an important target for the treatment of diseases mediated by the complement system. With the successful launch of iptacopan, the CFB small molecule inhibitors has become a current research hotspot, a number of domestic and foreign pharmaceutical companies are actively developing CFB small molecule inhibitors. In this paper, the research progress of CFB small molecule inhibitors in recent years is systematically summarized, the representative compounds and their activities are introduced according to structural types and design ideas, so as to provide reference and ideas for the subsequent research on CFB small molecule inhibitors.

complement system  /  alternative pathway  /  complement factor B  /  complement factor B small molecule inhibitor
Shuai WEN, Yao ZHAO, Yan WANG, Xing LI, Yi MOU, Zheng-yu JIANG. Research progress in small molecule inhibitors of complement factor B[J]. Acta Pharmaceutica Sinica, 2025 , 60 (1) : 37 -47 . DOI: 10.16438/j.0513-4870.2024-0600
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Year 2025 volume 60 Issue 1
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Article Info
doi: 10.16438/j.0513-4870.2024-0600
  • Receive Date：2024-06-27
  • Online Date：2025-11-07
  • Published：2025-01-12
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History
  • Received：2024-06-27
  • Revised：2024-11-05
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Affiliations
    1. College of Pharmacy and Chemistry & Chemical Engineering, Taizhou University, Taizhou 225300, China
    2. Jiangsu Key Laboratory of Drug Design and Optimization, School of Pharmacy, China Pharmaceutical University, Nanjing 210009, China
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