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Complement factor D is a drug target for metabolic-associated fatty liver disease
1Department of Obstetrics and Gynecology, The First Affiliated Hospital of Gannan Medical University, Ganzhou, China.
Molecular Immunology
|November 27, 2025
Summary
Complement factor D (CFD) is a novel mediator in metabolic dysfunction-associated fatty liver disease (MAFLD). Targeting CFD with danicopan shows therapeutic potential for MAFLD by reducing liver fat and inflammation.
Area of Science:
- Hepatology
- Metabolic Diseases
- Complement System Biology
Background:
- Metabolic dysfunction-associated fatty liver disease (MAFLD) is a widespread condition with limited treatment options.
- Complement factor D (CFD), a key enzyme in complement activation, is implicated in metabolic disorders.
- The therapeutic potential of CFD inhibition, specifically with danicopan, for MAFLD is largely unexplored.
Purpose of the Study:
- To investigate the role of CFD in MAFLD pathogenesis.
- To evaluate the efficacy of danicopan, a CFD inhibitor, in treating MAFLD.
- To elucidate the molecular mechanisms underlying CFD's involvement and danicopan's therapeutic effects.
Main Methods:
- Utilized high-fat diet (HFD) induced mouse models of MAFLD.
- Employed CRISPR-Cas9 gene editing for CFD knockout in hepatocytes.
- Administered danicopan pharmacologically to obese mice and analyzed liver/serum samples from MAFLD patients.
Main Results:
- CFD was significantly upregulated in MAFLD mouse livers and human patient sera.
- Genetic ablation of CFD reduced lipid accumulation in hepatocytes.
- Danicopan treatment improved glucose tolerance, reduced liver enzymes (ALT), and alleviated hepatic steatosis in obese mice without causing weight loss.
Conclusions:
- CFD is identified as a novel mediator contributing to MAFLD.
- Danicopan demonstrates therapeutic efficacy and translational potential for MAFLD management.
- Targeting the CFD pathway represents a promising strategy for MAFLD treatment.
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