PRE-CONFERENCE DINNER SHORT COURSES | September 25, 6:30-9:00 pm
SC1: Accelerated Patent Issuance and Funding Opportunities for Discovery Technologies Related to Cancer Cachexia, Microbiome, and Allergic and Inflammatory Diseases
This course will discuss how researchers and inventors can take advantage of the Cancer Moonshot Program and prioritized Track One examination at the U.S. Patent Office to vastly expedite the issuance of patents on their discoveries - thus speeding things up immensely with respect to obtaining funds for patented technologies. Patent prosecution tips with real-world working examples will be shared, including those covered under these three patents: 1) 9,585,920, 2) 9,408,880, and 3) 9,457,077.
Joe Kovarik, J.D., Patent Attorney with Bioscience Focus and Shareholder, Sheridan Ross, P.C.
SC2: 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.
Matthew Eddy, Ph.D., Postdoctoral Fellow, Ray Stevens Laboratory, The Bridge Institute, University of Southern California
Huixian Wu, Ph.D., Senior Scientist, Groton Center of Chemistry, Pfizer
SC3: Immunology Basics for Chemists
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 biopharmaceutical drug discovery and development will break it down for you by filling in 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.
Songqing Na, Ph.D., Senior Scientist, Biotechnology & Autoimmunity Res-AME, Eli Lilly and Company
Thomas Sundberg, Ph.D., Senior Research Scientist I, Center for Development of Therapeutics, Broad Institute of MIT and Harvard
SC4: Applications of Functional Screening Using CRISPR and RNAi
This course will offer details on how the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas technology works, how to set up CRISPR-based screens and how to complement it with existing RNAi-based screens using proper analysis and follow-up studies. The instructors will share their experiences on how to go about evaluating reagents/libraries, designing and setting up assays, and interpreting results when dealing with complex biology and informatics. The applications of such functional genomics screens for drug discovery and disease modeling will be discussed, along with design and workflows when working with different model systems. Ideas and best practices will be shared in an informal, interactive setting and attendees will walk away with practical advice and resources.
Jennifer Smith, Ph.D., Assistant Director, ICCB-Longwood Screening Facility, Harvard Medical School
John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT
Roderick Beijersbergen, Ph.D., Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center
Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University
SC5: 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.
Trevor Wilkinson, Ph.D., Associate Director, Antibody Discovery and Protein Engineering, MedImmune Ltd., United Kingdom
SC6: Covalent Fragments: Applications in Target-Based and Phenotypic Screens
The 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. Topics to be covered include design principles of covalent fragment libraries, target-based and phenotypic screens using covalent fragments and current technologies to conduct those screens, strategies and considerations to grow covalent fragments into drug leads, coupling covalent fragment growth with selectivity profiling in cells, using covalent fragments as toolkits to discover novel drug targets in phenotypic screens, and photocrosslinking methods to identify fragment drug targets in cells.
Alexander Statsyuk, Ph.D., Assistant Professor, Department of Pharmacological and Pharmaceutical Sciences, University of Houston
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. Beginning last decade, the advent of and improvements in the technologies for accessing these molecules and libraries thereof have led to significant progress being realized with this interesting chemical class and 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, in particular as they relate to their PK-ADME profiles, and specific case studies of these compounds in drug discovery and development. 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.
Eric Marsault, Ph.D., Professor, Department of Pharmacology-Physiology, Universite de Sherbrooke
Mark L. Peterson, Ph.D., COO, Cyclenium Pharma, Inc.
SC8: How to Best Utilize Organotypic 3D Cell Cultures Assays in Oncology
The course will provide an overview of the various 3D cell culture models available, their strengths and weaknesses, 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 and 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 of working with 3D cell cultures, from experimental design to data analysis will be discussed.
Sophie Lelievre, D.V.M., LLM, Ph.D., Professor, Department of Basic Medical Sciences; Associate Director, Collaborative Science, NCI-Designated Purdue Center for Cancer Research, Purdue University
Geoffrey Bartholomeusz, Ph.D., 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
Arvind Rao, Ph.D., Assistant Professor, Department of Bioinformatics and Computational Biology, The University of Texas MD Anderson Cancer Center
DINNER SHORT COURSES | September 27, 7:00-9:30 pm
SC9: Impact of Convergence of Immunotherapy and Epigenetics on Drug Discovery
In recent years, the understanding of both the immunotherapy and epigenetics of cancer has increased. This course will provide some details of how immunotherapy and epigenetic pathways interact and how they can be exploited to enhance the efficacy of current cancer treatments. The instructors will review recent scientific evidence and preclinical data that support the development of combination therapies and offer their perspectives on challenges that may have to be tackled along the way.
Alan P. Kozikowski, Ph.D., Professor, College of Pharmacy, Department of Medicinal Chemistry and Pharmacognosy, University of Illinois at Chicago
Alejandro Villagra, Ph.D., Assistant Professor, Department of Biochemistry and Molecular Medicine, School of Medicine and Health Sciences, The George Washington University
Wayne W. Hancock, M.D., Ph.D., Professor of Pathology and Chief of Transplant Immunology, Children's Hospital of Philadelphia and University of Pennsylvania
Michael R. Green, Ph.D., Assistant Professor & Coordinator for Lymphoma Research, Eppley Institute for Research in Cancer and Allied Diseases and Member, Fred & Pamela Buffett Cancer Center, University of Nebraska Medical Center
SC10: Introduction to Allosteric Modulators and Biased Ligands of GPCRs
Aimed at scientists working on or moving into the field of G protein-coupled receptors (GPCRs), this course will provide information on the identification and validation of allosteric, pathway-biased drugs including emerging screening approaches and practical tips and tools for ligand identification and validation. Allosteric modulators and pathway-biased ligands represent novel therapeutic approaches for achieving more selective actions with regards to GPCRs. The protein structural basis underlying the drug activity of allosteric modulators and the emerging opportunities for computer-aided discovery of allosteric and biased ligands will also be covered.
Annette Gilchrist, Ph.D., Professor, Pharmacology, Midwestern University
Sid Topiol, Ph.D., CSO, 3D-2drug, LLC; Professor and Director, Structural and Computational Drug Discovery, Stevens Institute of Technology
SC11: Antibody-Mediated Membrane Protein Crystallography
Crystallography of membrane proteins remains a very challenging but rewarding endeavor. Approximately half of the currently prescribed drugs target membrane proteins, and therefore, generating structural information is of immense importance. One of the approaches that has been utilized successfully for many different membrane proteins is the use of antibody fragments as crystallization chaperones. This short course is designed to provide a background on antibody fragment-mediated membrane protein crystallization and discuss specific success stories that are published in the recent years. This course will also provide overall step-by-step guidelines for the beginners in this area, and how to design a systematic strategy with important troubleshooting steps.
Arun Shukla, Ph.D., Assistant Professor, Indian Institute of Technology, India
SC12: 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 screening programs. This presentation will cover a range of relevant topics with a goal of providing practical information to help prosecute such programs more effectively.
Fabien Vincent, Ph.D., Associate Research Fellow, Hit Discovery and Lead Profiling Group, Pfizer
SC13: 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.
Brian Gerstenberger, Ph.D., Principal Scientist, Medicinal Chemistry, Pfizer
Mark Schnute, Ph.D., Associate Research Fellow, Biotherapeutics Chemistry & Immunoscience Research, Pfizer Global R&D
SC14: Commercialization of Ophthalmic Drugs and Devices
The course is designed to cover the basic tenets needed to successfully commercialize drugs and devices in the ophthalmic space. Topics covered include: 1) Company/Business strategy and funding, 2) Preparing for commercial launch, 3) Ophthalmic drug and device pricing, and 4) KOL interaction. There will also be a question and answer session with experienced leaders in commercialization.
Emmett Cunningham, Partner, Clarus Ventures
Glenn Noronha, Ph.D., CSO, Clearside Biomedical, Inc.
Rick Beckman, M.D., CMO, Clearside Biomedical, Inc.
SC15: CRISPR-Based Gene Editing for Targeted Therapies
While the challenges and risks associated with oligonucleotide therapies still remain, there is a new and better understanding of how genes can be effectively manipulated and delivered. With the rise of gene editing tools and enhanced knowledge of targeted delivery, these therapeutic modalities are once again being embraced with renewed hope and enthusiasm. This course helps you understand how gene editing - particularly the one enabled by the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9 system - works, and how it can be used to help develop targeted therapies with good efficacy and delivery.
Clifford Steer, M.D., Professor of Medicine and Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School
Ciro Bonetti, Ph.D., Scientist, Regeneron Pharmaceuticals
Hao Yin, Ph.D., Research Scientist, The David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology
Eric Kmiec, Ph.D., Director, Gene Editing Program and Senior Research Scientist, Center for Translational Cancer Research, Helen F. Graham Cancer Center & Research Institute, Christiana Care Health System
* Separate registration required for Short Courses, Symposia, Training Seminars