Our short courses are designed to be instructional, interactive and provide in-depth information on a specific topic. They allow for one-on-one interaction and provide a great way to explain more technical aspects that would otherwise not be covered during our main presentations.
PRE-CONFERENCE SHORT COURSES* | TUESDAY, SEPTEMBER 25 | 2:00 - 5:00 PM
SC1: Introduction to GPCR-Based Drug Discovery
This course will provide an understanding for some of the pharmacological complexities of G protein-coupled receptors (GPCRs) as well as for the tools used to study them in a drug discovery setting. The course is well suited for biologists, pharmacologists and medicinal chemists who have recently started working with GPCRs or for those who need a refresher on the latest technological advances and newest paradigms.
Instructor: Annette Gilchrist, PhD, Professor, Pharmacology, Midwestern University
SC2: Drug Metabolism and Its Role in Discovery and Drug Development
This short course will focus on concepts important for those wanting to understand how drug metabolism is applied to drug discovery and development. Topics will include how drugs are metabolized, what enzymes are involved, how drug metabolism concepts are applied during lead optimization, how drug metabolites are identified in preclinical studies and human clinical trials, the role of bioactivation in drug toxicity and the growing application of in silico tools in drug metabolism. Those scientists involved in medicinal chemistry, pharmacology and drug metabolism will benefit from this overview.
Instructor: John C. L Erve, PhD, DABT, Consultant, Jerve Scientific Consulting, Inc.
SC3: How to Best Utilize 3D Cells, Spheroids, and PDX Models in Oncology
The course will provide an overview of 3D cell culture and spheroid models currently available and where and how these models are being used, specifically for oncology research. The instructors will share their experiences on how they tested and evaluated various cell culture reagents and growth matrices, what worked, what didn’t and what you need to consider when setting up low- and high-throughput screening experiments using 3D cell cultures in your lab. The challenges working with 3D cell cultures, from experimental design to data analysis will be discussed.
Instructors: Madhu Lal-Nag, PhD, Group Leader, Trans-NIH RNAi Facility, National Center for Advancing Translational Sciences, National Institutes of Health
Geoffrey Bartholomeusz, PhD, Associate Professor and Director, Target Identification and Validation Program, Department of Experimental Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center
SC4: Covalent Fragments: Applications in Target-Based and Phenotypic Screens
This course will cover the design principles of covalent fragment libraries, target-based and phenotypic screens using covalent fragments, strategies to grow fragments into drug leads, and case studies.
Instructor: Alexander Statsyuk, PhD, Assistant Professor, Department of Pharmacological and Pharmaceutical Sciences, University of Houston
PRE-CONFERENCE DINNER SHORT COURSES* | TUESDAY, SEPTEMBER 25 | 6:00 - 8:30 PM
SC7: Enabling Macrocyclic Compounds for Drug Discovery: Opportunities, Challenges and Strategies
Macrocyclic compounds occupy an important underexplored chemical space between small molecules and biologics. However, these structures possess critical characteristics typically attributed to only one of these two broad categories, which makes them particularly attractive for modulating traditionally difficult target classes such as protein-protein interactions. With improvements in the past decade in the technologies for accessing these molecules and libraries thereof, significant progress has been realized; the number of synthetic macrocycles entering into clinical trials has steadily increased. This course will discuss important past and recent advances in the field, including an improved understanding of the properties of macrocyclic molecules, particularly as related to their PK-ADME profiles. Specific case studies of these compounds in drug discovery and development will be presented. The course is suitable for all those who wish to learn more about the current state and future potential of this evolving area regardless of their level of knowledge.
Instructor: Mark L. Peterson, PhD, COO, Cyclenium Pharma, Inc.
SC8: Targeting of Ion Channels with Monoclonal Antibodies
Ion channels are important therapeutic targets and currently represent the second largest target class addressed by therapeutic drugs. Significant opportunities exist for targeting ion channels with antibodies, but to date it has been challenging to discover therapeutic antibodies against them. This course will examine emerging technologies and strategies for enabling the isolation of functional anti-ion channel antibodies and highlight progress via case studies. The topics to be covered include: 1) antibody discovery, including methods to generate monoclonal antibodies and antigen preparation strategies; 2) assays to enable isolation of binding antibodies, including use of recombinant stable cell lines; 3) in vitro assays to measure functional activity of the antibody, including use of electrophysiology platforms and ion flux methods; and 4) review of promising ligand-gated and voltage-gated ion channel targets and antibodies in development. Open discussion will be actively encouraged.
Instructor: Trevor Wilkinson, PhD, Associate Director, Antibody Discovery and Protein Engineering, Medimmune Ltd., United Kingdom
SC9: CNS Translational Strategies
A greater understanding of CNS-related disease biology and the emergence of new, improved targets and technologies has bought renewed interest in neuroscience. This short course describes the critical stages needed to translate an exciting CNS target into an effective CNS therapy by addressing challenges in biomarkers design and validation, modelling, imaging and methods to bridge the preclinical/clinical translation gap.
Instructors: Viswanath Devanarayan, PhD, Executive Director and Global Head of Statistics & Data Sciences, Charles River Laboratories, Adjunct Professor, University of Illinois, Chicago
Changning Wang, PhD, Assistant Professor, Radiology, Massachusetts General Hospital, Harvard Medical School
SC10: Applications of Artificial Intelligence and Machine Learning in Drug Discovery and Development
This course aims to educate a diverse group of scientists-chemists, biologists, toxicologists, and those involved in translational and clinical research, about the growing use and applications of AI and ML. Talks start with explaining the basic terminology used and what it means, followed by discussions separating the hope from the hype. It goes into the caveats and limitations in AI and ML, while exploring ways in which it can be successfully applied in the drug discovery and development pipeline. There will be experts from various areas presenting case studies on how they have used AI/ML tools for lead optimization, target discovery, visualizing and classifying large datasets, patient stratification and more.
Instructors: Arvind Rao, PhD, Associate Professor, Department of Computational Medicine and Bioinformatics, University of Michigan
Deepak K. Rajpal, PhD, Senior Scientific Director, Computational Biology, GlaxoSmithKline R&D
Nicholas P. Tatonetti, PhD, Herbert Irving Assistant Professor of Biomedical Informatics and Director of Clinical Informatics, Herbert Irving Comprehensive Cancer Center, Columbia University
SC11: Mechanistic Understanding of Pharmacological Probes for the Ubiquitin-Proteasome System
This course is intended for the audience interested in drug discovery programs aimed to develop PROTACs (molecular degraders) and/or small molecule inhibitors targeting components of the ubiquitin-proteasome system (UPS). This course will cover basic mechanistic biochemistry of the ubiquitin-proteasome system, which includes E1, E2, E3, and deubiquitinating enzymes, and their macromolecular architecture. Subsequently, the course will transition into a general discussion of existing biochemical assays and technologies to discover PROTACs and small molecule inhibitors of E1, E2, E3, and deubiquitinating enzymes. Finally, existing PROTACs and pharmacological probes targeting E1, E2, E3, and deubiquitinating enzymes, their pharmacological properties, and basic control experiments that need to be done to inform a better design of these probes for preclinical and clinical studies will also be discussed.
Instructor: Alexander Statsyuk, PhD, Assistant Professor, Department of Pharmacological and Pharmaceutical Sciences, University of Houston
SC12: Clinically Relevant Animal Modeling for the Evaluation of Novel Antibacterial Approaches
The course will present a progression from small animal models (Galleria, mice) to more elaborate biofilm models (mice, rats, rabbits), and conclude with larger complicated models that include trauma reflective of where these infections cause hardware and implant problems (rats, pigs, goats). Each instructor will present typical evaluations of antibacterial products, both successes and failures. Each instructor will present examples of traditional small molecule antibiotic successes/controls and compare them to alternative antibacterial treatments such as vaccines, bacteriophage, etc. Important considerations for model development will be addressed such as: strain selection, biofilm, method of delivery, dosing, PK/PD considerations.
Instructors: Daniel V. Zurawski, PhD, Senior Scientist/Principal Investigator, Clinical RM, Inc., Walter Reed Army Institute of Research
Joseph C. Wenke, PhD, U.S. Army Institute of Surgical Research
Mark Shirtliff, PhD, Professor, University of Maryland
DINNER SHORT COURSES* | THURSDAY, SEPTEMBER 27 | 7:00 - 9:30 PM
SC13: GPCR Structure-Based Drug Discovery
Recent breakthroughs in obtaining high-resolution structures of G protein-coupled receptors (GPCRs) are rapidly impacting the pharmaceutical industry. This course will review how newly elucidated GPCR crystal structures have informed our current understanding of GPCR function. The instructors will explore how this new structural information is guiding rational drug design approaches for targeting GPCRs. This course will also review the role of conformational dynamics in GPCR function and structural biology techniques for studying the conformational dynamics of GPCRs, including the burgeoning field of applying nuclear magnetic resonance (NMR) to study GPCR structure and dynamics.
Instructors: Matthew Eddy, PhD, Postdoctoral Fellow, Ray Stevens Laboratory, The Bridge Institute, University of Southern California
Huixian Wu, PhD, Principal Scientist, Structural and Molecular Sciences, Discovery Sciences, Pfizer, Inc. Groton
SC14: Advancing Tools and Technologies for Fragment-Based Design
This course aims to introduce the fundamentals of Fragment-Based Lead Discovery (FBLD) to attendees. The first section will focus on the concepts of using fragments for hit generation. Special emphasis will be placed on practical pitfalls and the many ways to advance fragments to leads and drugs. The second part of the course will discuss the variety of fragment screening methods and when they are best applied. The composition of fragment libraries will also be discussed in detail. The attendees should come away from this course with a solid understanding of what FBLD is and how to apply it.
Instructors: Daniel A. Erlanson, PhD, Co-Founder, Carmot Therapeutics, Inc.
Mary Harner, PhD, Research Investigator II, Mechanistic Biochemistry, Bristol-Myers Squibb R&D
SC15: Introduction to Targeted Covalent Inhibitors
Covalent inhibitors of kinases have re-emerged as a drug design strategy due to more examples of their safety and efficacy in patients. Covalent inhibitors have the advantage of increased selectivity and longer action of duration but there are still important issues about their design and application that need to be better understood. This course will cover practical as well as theoretical issues that a medicinal chemist needs to keep in mind in developing covalent inhibitors.
Instructor: Mark Schnute, PhD, Associate Research Fellow, Medicine Design, Inflammation & Immunology Research, Pfizer, Inc.
SC16: Immunology Basics
Immunology is a difficult subject to master, even for immunologists. Newly discovered cell types and their associated function in human health and disease have been continuously revealed over the last decade. In this course immunologists (non-physicians) with extensive experience in the biopharmaceutical drug discovery and development will break it down for you by filling the gaps that most chemists have. It’s not a comprehensive course – but hopefully better – a useful course. The focus will range from basic background biology and terminology that immunologists take for granted and then jump to the biological underpinnings of the areas and targets a lot of medicinal chemists are developing compounds against.
Instructors: Songqing Na, PhD, Senior Scientist, Biotechnology & Autoimmunity Res-AME, Eli Lilly and Company
Thomas Sundberg, PhD, Senior Research Scientist I, Center for Development of Therapeutics, Broad Institute of MIT and Harvard
SC17: Technologies to Assess Permeability and Efflux in Gram-Negative Bacterial Pathogens
Our lack of understanding of the molecular basis for compound penetration into and efflux out of gram-negative bacteria has been identified as a key bottleneck for the rational discovery of novel antibacterial compounds. A main driver of this knowledge gap is the historical lack of assays, tools, and/or predictive models to provide medicinal chemists with structure-activity relationships that could guide optimization of whole cell penetration (and efflux avoidance). However, there have been some recent, promising advances in the field which set the stage for future innovative approaches.
Instructors: David Six, PhD, Investigator III, Infectious Diseases, Novartis Institutes for BioMedical Research
Ram Iyer, PhD, Principal Scientist (Bacteriology), Entasis Therapeutics, Inc.
SC18: Practical Phenotypic Screening
Phenotypic drug discovery is experiencing a Renaissance in the pharmaceutical industry, based on its successful track record in delivering first-in-class medicines. This approach offers the promise of delivering both novel targets and chemical matter modulating a disease phenotype of interest. Although phenotypic screening may appear at first sight to be similar to target-based screening, there are some significant differences between the two approaches. These need to be properly considered and addressed to ensure the greatest likelihood of success for phenotypic drug discovery programs. This presentation will cover a range of relevant topics with a goal of providing practical information to help prosecute such programs more effectively.
Instructor: Fabien Vincent, PhD, Associate Research Fellow, Hit Discovery and Lead Profiling Group, Pfizer, Inc.
* Separate registration required