Proteins are the fastest growing class of pharmaceuticals for the treatments of many diseases, from cancers to immune disorders. To exert their therapeutic effect, most protein drugs require glycosylation, with one or more carbohydrate molecules (glycans) attached to their surface to improve stability and solubility.1 Changes in glycan coverage can greatly affect the efficacy, half-life, and immunogenicity of proteins as therapeutics.2
In this study, we have focused on the glycoprotein, vascular endothelial growth factor (VEGF), a 192 amino acid residue protein.3 Recombinant VEGF is used clinically to stimulate blood vessel formation in ischemic, injured and bioengineered tissues. VEGF contains a single conserved N-glycan, while several variants differ in the O-glycosylation pattern. Recombinantly-produced VEGF is correctly folded and bioactive despite the heterogeneous glycosylation states,4 although glycan differences appear to impact protein solubility,5 proteolysis6 and receptor binding.7
The total chemical synthesis was undertaken by native chemical ligation8 in a convergent synthesis with polypeptide fragments equipped with thioesters and containing multiple different glycans at Asn-75. These were folded into the 3-D homodimer active protein and assessed for receptor binding, bioactivity, and immunogenicity and compared to native, partial and unglycosylated forms.