Posttranslational modifications efficacy

Currently, 60 to 70 percent of all biotherapeutics based on recombinant proteins are produced in cultivated mammalian cells.8 Mammalian systems are often preferred over other hosts, such as bacteria, plants, and yeast, because of their capability for proper protein folding, assembly, and posttranslational modification. The quality and efficacy of a protein can thus be enhanced when expressed in mammalian cells. With the recent expansion in the biotherapeutics market, the demand for proteins derived from mammalian cells... 

The above example also gives an indication of the relative importance of carbohydrate analysis. Without question, protein glycosylation is the most complex of all posttranslational modifications made in eukaryotic cells, the importance of which cannot be underestimated. For many compounds, glycosylation can readily affect protein solubility (as influenced by folding), protease resistance, immunogenicity, and pharmacokinetic/pharmacodynamic profiles (i.e., clearance and efficacy) [36], Typical analytical methodologies used to assess carbohydrate content are also listed in Table 2. 

The potential impact of how posttranslational modifications, such as glycosyl-ation, can directly impact protein conformation and subsequently affect biological activity, including the overall safety and efficacy of the drug product. 

Expression of the nucleic acid product, including the normal steps in gene expression (transcription, franslation, and posttranslational modification). Expression is vital to the therapeutic efficacy of the gene in the sense that controlled expression of genes is important for proper biological function. Current delivery technologies result in continuous gene expression. 

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