Enhancement of therapeutic protein in vivo activities through glycoengineering

Nat Biotechnol. 2003 Apr;21(4):414-21. doi: 10.1038/nbt799. Epub 2003 Mar 3.

Abstract

Delivery of protein therapeutics often requires frequent injections because of low activity or rapid clearance, thereby placing a burden on patients and caregivers. Using glycoengineering, we have increased and prolonged the activity of proteins, thus allowing reduced frequency of administration. Glycosylation analogs with new N-linked glycosylation consensus sequences introduced into the protein were screened for the presence of additional N-linked carbohydrates and retention of in vitro activity. Suitable consensus sequences were combined in one molecule, resulting in glycosylation analogs of rHuEPO, leptin, and Mpl ligand. All three molecules had substantially increased in vivo activity and prolonged duration of action. Because these proteins were of three different classes (rHuEPO is an N-linked glycoprotein, Mpl ligand an O-linked glycoprotein, and leptin contains no carbohydrate), glycoengineering may be generally applicable as a strategy for increasing the in vivo activity and duration of action of proteins. This strategy has been validated clinically for glycoengineered rHuEPO (darbopoetin alfa).

Publication types

  • Evaluation Study

MeSH terms

  • Anemia / drug therapy
  • Animals
  • CHO Cells / metabolism
  • COS Cells / metabolism
  • Cricetinae
  • Drug Delivery Systems / methods*
  • Erythropoietin / genetics
  • Erythropoietin / metabolism
  • Erythropoietin / therapeutic use
  • Excipients / chemistry
  • Female
  • Genetic Enhancement / methods
  • Glycoproteins / biosynthesis*
  • Glycoproteins / genetics*
  • Glycoproteins / metabolism
  • Humans
  • Leptin / biosynthesis
  • Leptin / deficiency
  • Leptin / genetics
  • Leptin / therapeutic use
  • Mice
  • Mice, Inbred BALB C
  • Protein Engineering / methods*
  • Proteins / administration & dosage
  • Proteins / therapeutic use*
  • Recombinant Proteins / biosynthesis
  • Recombinant Proteins / genetics
  • Recombinant Proteins / therapeutic use
  • Thrombopoietin / biosynthesis
  • Thrombopoietin / therapeutic use

Substances

  • Excipients
  • Glycoproteins
  • Leptin
  • Proteins
  • Recombinant Proteins
  • Erythropoietin
  • Thrombopoietin