Potent And Highly Soluble Pegylated Compstatin Peptide

Tech ID: 27122 / UC Case 2017-046-0


Lack of regulation in complement response is implicated in the pathology of several disorders, such as age-related macular degeneration, paroxysmal nocturnal hemoglobinurea, rare kidney diseases, chronic obstructive pulmonary disease, lupus, rheumatoid arthritis, asthma, adult respiratory distress syndrome, hemolytic anemia, rejection of xenotransplantation, stroke, heart attack, and ischemia reperfusion injuries. Regulating complement activation is important to control inflammation, autoimmune diseases, and infections. The compstatin family of peptides have been shown to be potent inhibitors of complement system activation and are promising candidates to become therapeutics for the treatment of age-related macular degeneration, and other complement-mediated diseases.

Brief Description

UCR researchers have developed novel compstatin peptides with polar amino acid extensions at the N-terminus and PEGylated extensions at the C-terminus. The new peptides have the following advantages compared to previously known compstatin peptides: (i) highly improved aqueous solubility while maintaining high inhibitory potency, and (ii) higher inhibitory efficacy against complement system activation in a human retinal pigmented epithelial cell-based assay that mimics the pathobiology of age-related macular degeneration. The combined solubility and inhibitory potency and efficacy properties render the new peptides excellent candidates to become therapeutics for the treatment of age-related macular degeneration.



A potent and highly soluble compstatin peptide shown in surface representation with an 8 PEG block C-terminal extension displayed in stick form. The surface of the compstatin analog is colored according to amino acid properties: gray for hydrophobic, green for polar neutral, blue for polar positively charged, red for polar negatively charged, yellow for cysteines of the disulfide bridge, and brown for glycine. The molecular image is generated using three-dimensional coordinates from a molecular dynamics simulation trajectory.




  • Inhibit complement activation by targeting protein C3, the converging point of all three complement activation pathways
  • Possess highly improved aqueous solubility properties and overcome the aggregation limitation for clinical translation of previously known compstatin peptides
  • Possess significantly improved inhibitory efficacy and retain inhibitory potency, compared to previously known compstatin peptides


  • Therapeutics for age-related macular degeneration (both dry and wet forms)
  • Potential therapeutics for other complement system-mediated inflammatory and autoimmune diseases, such as paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, C3 glomerulopathy, chronic obstructive pulmonary disease, lupus, rheumatoid arthritis, and ischemia reperfusion injuries

Patent Status

Patent Pending

Related Materials

Patent Status

Country Type Number Dated Case
United States Of America Published Application 20180057538 03/01/2018 2017-046


Learn About UC TechAlerts - Save Searches and receive new technology matches

Other Information


Age-related macular degeneration, compstatin, complement system, complement system-mediated diseases, autoimmune diseases, inflammatory diseases, rare diseases

Categorized As