Scientists developing new medicines have a simple goal – to find new treatments that can change the course of human disease. The end result may be a medicine that helps manage symptoms, stops a disease from progressing or that even cures a disease entirely. Ideally, these new medicines will treat disease with minimal impact to healthy tissue in the body, targeting treatment to where it is needed.
Scientists who work in protein engineering pursue more targeted medicines by designing proteins – either antibodies or enzymes – with new features and functions programmed to somehow disrupt the normal function of a disease target, in the hopes of having a therapeutic effect.
But what does it mean to “design” a molecule and how do they do it? We talked with some of AbbVie’s protein engineers, who explained the process in five simple steps.
The protein engineering team works with disease experts to understand the disease biology and possible targets, and come up with protein designs that may impact how the disease behaves in the body.
Armed with their understanding of the target biology and protein design, the protein engineers begin to identify and engineer molecules that are able to engage the disease target by blocking it, activating it or breaking it down. The hope is that these molecules will ultimately change the way the disease behaves.
Depending on the features of the disease target, the protein engineers choose the right approach to engage the target. They could choose to engineer an antibody drug conjugate, which recognizes the tissue where the target is located and delivers a drug directly to the diseased tissue, a bispecific, which engages two or more targets at the same time, or a number of other approaches.
The scientists then conduct extensive testing to learn about the molecules they have designed and generated. The goal is to identify the one that has all of the important features needed to have the desired disease-modifying effects in people. They also conduct necessary safety testing before giving the experimental medicines to humans.
Once the molecule successfully passes all the testing, it begins clinical studies where they can see its safety and tolerability in humans, and whether it has the desired therapeutic impact on human disease.