Target intelligence / Profile preview

Complement component (C)

Target
C
Molecular classification
Enzyme, Serine protease, Receptor, Serum protein, Other
01

Overview

The complement component system consists of a complex cascade of more than 30 plasma and cell-surface proteins that play a critical role in the innate immune system (StatPearls, PMID: 30020619). These proteins work through three main pathways—the classical, lectin, and alternative pathways—to promote opsonization, trigger inflammation via anaphylatoxins like C3a and C5a, and directly lyse pathogens through the formation of the membrane attack complex (MAC) (PubMed, PMID: 28438089). Dysregulation or overactivation of specific complement components is linked to various autoimmune and inflammatory diseases, including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (UniProt, P01031). Consequently, specific proteins within this system, most notably Complement component 5 (C5) and Complement component 3 (C3), have become high-value therapeutic targets for monoclonal antibodies and small molecules (NIH/NCBI, PMC7138379). Pharmacological intervention typically involves inhibiting protease activity or blocking receptor binding to prevent downstream tissue damage and chronic inflammation. However, because the system is vital for defense against certain bacteria, therapeutic inhibition carries a significant safety concern regarding life-threatening infections by encapsulated organisms such as Neisseria meningitidis (FDA Label, Soliris).

Other names
Complement system proteinsComplement cascadeComplement factorsComplement proteins
02

Mechanism of action

Inhibition of C5 cleavage to prevent MAC formation; C3 inhibition to block all activation pathways; Factor D or Factor B inhibition to block the alternative pathway; C5a receptor (C5aR1) antagonism to reduce inflammation.

03

Biological functions

Immune responseInflammationOpsonizationCell deathSignal transductionChemotaxis
04

Disease associations

InflammationInfectionParoxysmal nocturnal hemoglobinuria (PNH)Atypical hemolytic uremic syndrome (aHUS)Myasthenia gravisAge-related macular degeneration (AMD)Geographic atrophyNeuromyelitis optica spectrum disorder (NMOSD)
05

Safety considerations

Increased susceptibility to encapsulated bacterial infections (specifically Neisseria meningitidis)Requirement for meningococcal vaccination prior to treatmentRisk of breakthrough hemolysis in PNH patientsPotential for serious respiratory tract infections
06

Interacting drugs

Eculizumab

8 more in the full profile.

07

Biomarkers

CH50 (Total hemolytic complement activity)AH50 (Alternative pathway activity)Serum C3 and C4 levelsSoluble C5b-9 (Terminal complement complex)C5a levels

Beyond the preview

Go deeper on Complement component (C).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Complement component (C).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call