Target intelligence / Profile preview

Complement factor (CF)

Target
CF
Molecular classification
Enzyme, Serine protease, Regulator, Plasma protein
01

Overview

Complement factors are a group of plasma proteins that serve as critical components and regulators of the complement system, an integral part of the innate immune response. This group includes essential enzymes such as Factor B and Factor D, which drive the alternative pathway amplification loop, as well as regulatory proteins like Factor H and Factor I that prevent excessive activation. Dysregulation of these factors is a primary driver in several complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and age-related macular degeneration (AMD). Therapeutic targeting of specific factors, particularly Factor B and Factor D, has led to the development of drugs like iptacopan and danicopan to control overactive complement cascades. However, the systemic inhibition of these factors poses a significant safety risk by increasing susceptibility to life-threatening infections from encapsulated bacteria, most notably Neisseria meningitidis. Monitoring of complement activity through biomarkers like C3 levels and AH50 is essential for managing treatment efficacy and safety. Overall, complement factors are vital therapeutic targets for modulating immune-mediated inflammation and tissue damage.

Other names
Complement system factorsComplement proteinsComplement components
02

Mechanism of action

Inhibition of the alternative pathway amplification loop and prevention of C3/C5 convertase formation.

03

Biological functions

Immune responseInflammationOpsonizationCell lysisPhagocytosis
04

Disease associations

Paroxysmal nocturnal hemoglobinuriaAtypical hemolytic uremic syndromeAge-related macular degenerationC3 glomerulopathySystemic lupus erythematosus
05

Safety considerations

Increased risk of encapsulated bacterial infections (e.g., Neisseria meningitidis)Risk of sepsisPotential for autoimmune flares
06

Interacting drugs

Iptacopan

3 more in the full profile.

07

Biomarkers

C3 levelsC4 levelsCH50AH50Bb fragmentC5asC5b-9

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