Related Experiment Video
Updated: Jan 23, 2026

Method for Identifying Small Molecule Inhibitors of the Protein-protein Interaction Between HCN1 and TRIP8b
Published on: November 11, 2016
A small-molecule inhibitor of C5 complement protein.
Keith Jendza1, Mitsunori Kato1, Michael Salcius2
1Global Discovery Chemistry, Novartis Institutes of Biomedical Research, Cambridge, MA, USA.
Researchers developed the first small-molecule inhibitor targeting complement component 5 (C5). This discovery offers a new therapeutic strategy for diseases driven by uncontrolled complement pathway activation.
Area of Science:
- Immunology
- Drug Discovery
Background:
- The complement pathway is crucial for immune response.
- Uncontrolled complement activation contributes to various diseases.
- Human complement component 5 (C5) is a validated therapeutic target, with anti-C5 antibody therapies already approved.
Purpose of the Study:
- To identify and characterize the first small-molecule inhibitor of human complement component 5 (C5).
- To explore the mechanism of action of this novel C5 inhibitor.
Main Methods:
- Drug discovery and optimization processes.
- Biochemical assays to assess C5 inhibition.
- Mechanism of action studies.
Main Results:
- Successful identification and optimization of a novel small-molecule inhibitor of C5.
- Characterization of the inhibitor's mechanism of action.
Conclusions:
- A first-in-class small-molecule inhibitor of C5 has been developed.
- This inhibitor represents a potential new therapeutic approach for complement-mediated diseases.
More Related Videos
06:54Evaluation of the Interplay Between the Complement Protein C1q and Hyaluronic Acid in Promoting Cell Adhesion
Published on: June 15, 2019
10:33Development of Inhibitors of Protein-protein Interactions through REPLACE: Application to the Design and Development Non-ATP Competitive CDK Inhibitors
Published on: October 26, 2015
Related Concept Videos
Eukaryotic Transcription Inhibitors
Eukaryotic transcription inhibitors usually contain two distinct domains, a...
Complement System
Complementation Tests
Organisms heterozygous for different mutations are crossed pairwise in all combinations. If present on different genes, the mutations can complement each other by providing the missing...
Negative Regulator Molecules
Molecules and Compounds
Positive Regulator Molecules