Aggregate Forms of Recombinant Human Erythropoietin With Different Charge Profile Substantially Impact Biological Activities

J Pharm Sci. 2020 Jan;109(1):277-283. doi: 10.1016/j.xphs.2019.05.036. Epub 2019 Jun 16.

Abstract

Recombinant human erythropoietin (rHuEPO) as a glycoprotein growth factor has been considered a biological drug for treatment of anemic patients with chronic renal failure or who receive cancer chemotherapy. Biological activity and circulation time are 2 parameters that are important to achieve EPO's efficacy. Previous efforts for increasing EPO's efficacy have focused on glycosylation modification via adding more sialic acid antenna and generates more negative charged protein. Evidences cleared that EPO's activity increased by numbers of N-glycan moieties with presence of sialic acids at their terminus. Correlation between bioactivity and glycosylation with terminal sialylation is theoretically achieved using the calculation of the amount of charge profile of the EPO variants called "I-number." Here, we studied and compared the relationship between bioactivities of different EPOs that contained various I-numbers and the effect of their secondary and tertiary protein structures on measured in vivo efficacy. Eight recombinant EPOs batches were produced under the same condition. I-numbers found out by EPO's charge profiles determination using capillary electrophoresis and activities were studied upon erythroid precursor cell stimulation in mice. Analyzing the bioactivity, I-number, and structural studies revealed that in spite of I-number, conformational changes in protein structure and presence of aggregated species impact bioactivity substantially.

Keywords: I-number; aggregation; biological activity; erythropoietin; glycosylation.

MeSH terms

  • Animals
  • Dynamic Light Scattering
  • Electrophoresis, Capillary
  • Erythropoietin / chemistry*
  • Erythropoietin / pharmacology
  • Glycosylation
  • Injections, Subcutaneous
  • Mice
  • N-Acetylneuraminic Acid / analysis
  • Particle Size
  • Protein Aggregates*
  • Protein Structure, Secondary
  • Protein Structure, Tertiary
  • Recombinant Proteins / chemistry*
  • Recombinant Proteins / pharmacology
  • Reticulocytes / drug effects

Substances

  • EPO protein, human
  • Protein Aggregates
  • Recombinant Proteins
  • Erythropoietin
  • N-Acetylneuraminic Acid