Turning cells into protein factories is at the very core of modern biopharma’s foundation. Therefore, it is no small thing that researchers have recently found a way to use bioinformatics and gene editing to remake cell line development. To the laymen, that means new protein drugs can be produced faster, safer, and cheaper.
That’s a very big deal because “recombinant protein therapeutics have been developed to treat a wide variety of clinical indications, including cancers, autoimmunity/inflammation, exposure to infectious agents, and genetic disorders,” according to a scientific paper published in the US National Library of Medicine at the National Institutes of Health (NIH).
Put another way, “therapeutic proteins can also be grouped based on their molecular types that include antibody-based drugs, Fc fusion proteins, anticoagulants, blood factors, bone morphogenetic proteins, engineered protein scaffolds, enzymes, growth factors, hormones, interferons, interleukins, and thrombolytics,” according to a NIH article .
Synthetically produced therapeutic proteins open a whole new world of possibilities in treating an almost endless list of diseases by copying how cells defend themselves naturally. This short, easy to follow video explains how cells fight off a virus, for example.
Producing therapeutic proteins is a very exacting exercise, however. If scientists are off the mark even by a little, the drug will not work as intended. It is nature’s recipe or bust.
“Naturally occurring proteins in our bodies are differently glycosylated depending on the cell producing them…so if you want to use some of these proteins as a therapeutic you want to be able to make the same version because otherwise the function, the specificity and the stability will differ and you may even get immunogenic [allergic] responses,” said Bjørn Voldborg, director of CHO cell line development at the Technical University of Denmark in an article in BioProcess International.
The cells used in this line of production are also natural. They are often referred to simply as CHO, which stands for Chinese Hamster Ovary cell.
“If you want to engineer a factory [cell line] you need to understand what machines you have in the factory. It’s the genome sequences of the CHO cells – and also the original hamster cells from which the CHO cells are derived – that give us the parts. Then we look at how the machinery is connected together,” Voldborg said at BPI Europe in July, adding bioinformatic analysis can be used to understand how protein secretion, metabolic and glycosylation pathways in CHO cells are linked, according to the BioProcess International article.
Even with all the opportunities to leverage in this line of cell development, Voldborg wants to use bioinformatics and gene editing to push ahead in drug development even further.
“We want to be able to make hard to produce proteins…we think we can make completely new protein-based drugs, unnatural proteins… proteins that cannot be made in CHO today,” Voldbery told BioProcess International.
“We are also looking at ways of moving from bench to pharmacy more quickly because we know what we are doing giving us the option of cheaper development and production.”