Introduction to Biologics

This course is a brief introduction to the use of biologics as drug therapies. It centers on what biologics can do (that small-molecule drugs also do), but more importantly, what some biologics can do that small-molecule drugs cannot. In addition, safety issues with biologics can differ from those of small molecules, and the pharmacokinetics of biologics is a unique challenge 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, including specific safety topics (e.g., immunogenicity) and pharmacokinetics. Specifically, the delivery of biologics can be challenging and distribution and duration are also concerns. Finally, the determination of in vivo effectiveness will be discussed.

Who Should Attend?

• Biologists involved in drug discovery
• Medicinal chemists
• Students of pharmacology
• Academic pharmacologists

Course Benefits

• Gain new insights into state-of-the-art ideas on the utilization of biologics as therapy
• Learn new tools to quantitatively assess molecular effects of biologics
• Learn what biologics can do that small drug molecules cannot do
• Learn the unique challenges around biologics in terms of pharmacokinetics and safety

Course Topics

• General introduction to biologics
• Replacement proteins / production / quality control/ assessment of activity and purity
• Replacement peptides as drugs, biased signaling
• Methods to demonstrate target engagement
• Antibodies: general principles, Ab agonism, antagonism, scavenging ligands
• Immunotherapy (Abs, CAR T Cell therapy, immune checkpoint inhibitors)
• Vaccines
• Ab therapy for cancer
• Nucleotide-based therapies (DNA [Gene therapy], RNA)
• RNAi-based drug therapy
• Biologic Pharmacokinetics, delivery, distribution, duration, elimination
• Biologic safety; immunogenicity

Instructors

Terry Kenakin, PhD
University of North Carolina School of Medicine

Terry Kenakin received his BSc in chemistry and PhD in Pharmacology from the University of Alberta. After a post-doctoral fellowship at University College London, he joined Burroughs-Wellcome in Research Triangle Park, NC where he spent seven years in drug discovery. He then moved to GlaxoSmithKline for 25 years, followed by the University of North Carolina School of Medicine where he has been a Professor of Pharmacology since 2011. He has written 12 books on pharmacology, is the Editor-in-Chief of the Journal of Receptors and Signal Transduction, is on numerous editorial boards and has written many reviews and original papers on receptor pharmacology.