Biologics as Drug Therapy

This course is a brief introduction to and summary of the topic of biologics as drug therapy. It centers on the things that biologics can do (that small drug molecules also do) but more importantly what some biologics can do that small molecule drugs cannot do. In addition, safety issues with biologics can be different from those of small molecules and most certainly the pharmacokinetics of biologics is a unique challenge as compared to small molecules. The specific topics covered in this course include recombinant replacement protein biologics (assessment of biological activity, production, quality control, heterogeneity, removal of undruggable proteins through PROTACs), peptides (measurement of response, biased peptide signaling, methods to demonstrate target engagement), immunotherapy (vaccines, antibody approaches to modifying the immune system), antibodies (features, agonism, antagonism, antibody-drug complexes, antibody scavenging of endogenous species), and Nucleotide-based therapies (DNA- Gene therapy, CRISPR, RNA; exploiting the siRNA system). In addition, development issues with biologics will be addressed such as specific safety topics (immunogenicity) and importantly pharmacokinetics. Specifically, the delivery of biologics can be challenging and distribution and duration also a concern. Finally, mention of determining in vivo effectiveness will be discussed.