Interactive Breakout Discussion Groups

Join a breakout discussion group. These are informal, moderated discussions with brainstorming and interactive problem solving, allowing participants from diverse backgrounds to exchange ideas and experiences and develop future collaborations around a focused topic.

This year’s breakouts with topics centered on each of the event’s 14 conference programs will take place on Tuesday evening, September 17 and – with breakfast – Thursday morning, September 19.

TUESDAY, SEPTEMBER 17 | 5:05 - 6:05 PM

Target Identification and Validation
Essex Ballroom

TABLE 1: New Trends in Functional Genomics-Based Screening and Technologies

Moderator: Roderick Beijersbergen, PhD, Group Leader, Division of Molecular Carcinogenesis and NKI Robotics and Screening Center, The Netherlands Cancer Institute

  • Screen validation and follow-up with single well CRISPR technology
  • Large-scale screening with cell sensing and response reporters
  • CRISPR screening in primary cells using co-culture systems

TABLE 2: Chemical Biology in Drug Discovery

Moderators: Jaimeen Majmudar, PhD, Principal Scientist, Chemical Biology, Pfizer, Inc.

Jeff Martin, PhD, Scientist II, Chemical Biology & Proteomics, Biogen, Inc.

  • Chemical biology approaches as an avenue for new targets
  • Chemo-proteomics approaches to understand protein degradation
  • Covalent fragment screening for the coupled discovery of targets and leads
  • How can chemical biology approaches complement CRISPR-based technologies?

TABLE 3: Use of Advanced Disease Models in Drug Discovery

Moderators: 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

Madhu Lal-Nag, PhD, Program Lead, Research Governance Council, Office of Translational Sciences, Center for Drug Evaluation & Research, U.S. Food and Drug Administration

  • Developing multi-cellular 3D cell culture models for high-throughput screening
  • Specific 3D models for target identification and validation screens
  • Challenges with high-throughput screens utilizing 3D spheroid models
  • Patient-derived xenograft ex vivo (PDXEx) models as a screening platform
  • Evaluation of microphysiological systems and models for efficacy, as well as safety studies

Lead Generation Strategies

TABLE 4: Integrating FBLD with Other Screening Methods

Moderator: Robert D. Mazzola, PhD, Director, Chemical Research, Merck Research Labs

  • Fragment screens vs. HTS vs. DNA-Encoded Libraries (DEL)? Or combos?
  • Prosecuting hits: Use separate teams of chemists? Collect data simultaneously?
  • Sharing examples/lessons learned

TABLE 5: New Biophysical Technologies

Moderator: Anup Upadhyay, PhD, Senior Scientist III, Drug Discovery Science & Technology, AbbVie  

  • Biophysical techniques for protein conformational plasticity and hydrodynamic properties, e.g., microscale thermophoresis (MST), second harmonic generation (SHG)
  • ‘Toolbox’ biophysical techniques: Which are most popular? Which to use for which applications?
  • Correlating results from various biophysical approaches: Identifying and validating true hits

TABLE 6: DNA-Encoded Libraries

Moderator: Svetlana Belyanskaya, PhD, Scientific Leader, Encoded Library Technologies, R&D Platform Technology & Science, GlaxoSmithKline Boston

NEW: Chris Phelps, PhD, Manager, R&D Platform Technology & Science, GlaxoSmithKline Boston

  • Different types/approaches (i.e., DNA recorded, DNA templated libraries)
  • Current constraints on DNA-Encoded Libraries (DNA compatible chemistry, library diversity, selection methods)
  • Applications/target classes

Emerging Ubiquitin and Autophagy Targets

TABLE 7: New Technologies and Assays to Study Protein Degraders

Moderators: Alexander Statsyuk, PhD, Assistant Professor, Department of Pharmacological and Pharmaceutical Sciences, University of Houston

Carles Galdeano, PhD, Serra Hunter Professor, Department of Pharmacy and Pharmaceutical Technology and Physical Chemistry, University of Barcelona

Yue Xiong, PhD, William R. Kenan Professor of the Biochemistry and Biophysics, University of North Carolina; Co-Founder, Cullgen

  • Key ubiquitination steps for inducing protein degradation
  • Biochemical, biophysical, and cellular-based approaches to monitor ternary complex formation
  • How to identify novel E3 ligases and E3 ligase ligands: Need and challenges
  • How do PROTACs and IMIDs affect the normal UPS function?

TABLE 8: Novel Applications and Approaches for PROTAC-Based Protein Degradation

Moderator: Tauseef R. Butt, PhD, President and CEO, Progenra, Inc.

  • Current limitations of PROTAC approaches – application of cereblon, cIAP, VHL, HDM2 ligases as degrader molecules ligase
  • Big hurdles in therapeutic potential of PROTACs – toxicity for chronic diseases, oral bioavailability, IP issues 
  • Expanding therapeutic potential of PROTACs – developing novel ligases for membrane, cytosol, or nuclear targets
  • How to overcome the above hurdles and not to develop “Me Too” PROTACs 

TABLE 9: Challenges with Developed Targeted Protein Degrader Molecules

Moderators: Susanta Samajdar, PhD, Senior Vice President and Head of Discovery, Aurigene Discovery Technologies Limited

Upendra Dahal, PhD, Senior Scientist, Pharmacokinetics and Drug Metabolism, Amgen, Inc.

  • Differential activity in cells and preclinical models
  • PK/PD relationships
  • Bell-shaped dose response
  • Achieving oral bioavailability
  • BBB permeability
  • CMC challenges

Targeting NASH

TABLE 10: Animal Models for NASH

NEW: Moderator: Rebecca Taub, MD, CMO & President, R&D, Madrigal Pharmaceuticals

  • Re-evaluating NASH animal models in the context of the new FDA guidelines for NASH cirrhosis
  • Which noncirrhotic NASH animal models have the most representative histology?
  • Are there NASH cirrhosis animal models that develop complications?
  • Which biomarkers will be reflective of improvements in histology or predictive of complications?

TABLE 11: Efficacy of MOA in the Treatment of NASH 

Moderator: Karin Conde-Knape, PhD, Corporate Vice President, Cardiovascular and Liver Disease Research, Novo Nordisk

  • What is decrease in liver fat telling us? 
  • What will be the needed efficacy to make a difference to patients?
  • Are we any closer to having surrogate markers of efficacy?

TABLE 12: Cirrhosis and NASH

Moderator: Peter Traber, MD, Partner, Alacrita Consulting; Adjunct Professor of Medicine, University of Pennsylvania School of Medicine

  • Targets in common between chirrhosis and NASH
  • Challenges in treating cirrhotic NASH
  • Regulatory guidance for cirrhosis NASH trials

Immuno-Oncology: Emerging Targets and Therapeutics

TABLE 13: STING: An IntereSTING IO Target

Moderators: Gottfried Schroeder, PhD, Senior Scientist, Department of Pharmacology, Merck Research Labs Boston

Charles A. Lesburg, PhD, Senior Principal Scientist, Computational and Structural Chemistry, Merck & Co., Inc.

  • Distinct structural features of STING to design modulators against
  • Biologics vs. small molecule approaches for modulating STING
  • MOA and physiological considerations, possible side effects, etc.

TABLE 14: Targeting the IDO/TDO-Kynurenine-Arylhydrocarbon Receptor Pathway and Beyond

Moderator: Thomas Hoffman, PhD, CFO, Phenex Pharmaceuticals

  • Pros and cons of IDO / TDO single / dual inhibitors vs. AHR antagonists?
  • Which tumor autonomous effects and which effects on the immune system can you expect?
  • For which cancers might such IDO / TDO / AHR inhibitors be applicable?

TABLE 15: Targeting the Adenosine Pathway

Moderator: Alwin Schuller, PhD, Senior Principal Scientist/Team Lead, Oncology, IMED, Biotech Unit, AstraZeneca

  • Targeting A2AR vs. CD73 vs. Dual Inhibitors
  • Other ways to modulate adenosine levels
  • Promising ‘synergistic’ combinations

Antibodies Against Membrane Protein Targets – Part 1
Antibody Forum – Part 1

TABLE 16: R&D for Emerging Modalities

Moderator: Zhiqiang An, PhD, Professor, Chemistry; Director, Texas Therapeutics Institute, University of Texas Health Science Center at Houston

  • Consideration of alternative modalities for intended target
  • Technologies for selections in final format
  • Emerging technologies for antibody discovery; NGS, single cell analysis, CRISPR, AI
  • Integrating traditional display platforms with new technologies
  • Multispecific platforms

TABLE 17: Fit-for-Purpose Membrane Protein Generation in Biologics Development

Moderator: Leyu Wang, PhD, Senior Scientist and Project Leader, Protein Sciences, AbbVie

  • Best practices to decide approaches for membrane protein production
  • Strategy for expression analysis for a novel multipanner
  • Challenges to close gap between membrane protein supply and demand
  • How to tap the external research organization or company

TABLE 18: Structural Biology Challenges in R&D for Membrane Protein Targets

Moderator: Xinchao Yu, PhD, Senior Scientist, Cryo-EM, Amgen

  • Producing sufficient quantity of minimally engineered human membrane proteins
  • Crystallography vs. cryo-EM, pros and cons
  • Detergents, nanodiscs or SMALPs
  • High-throughput structural biology for membrane proteins

THURSDAY, SEPTEMBER 19 | 7:30 - 8:30 AM

RNA as a Drug Target

TABLE 19: What Happens When You Get Off-Target Effects?

Moderator: Arthur A. Levin, PhD, Executive Vice President, R&D, Avidity Biosciences

  • How are off-target events for RNA-targeting drugs being avoided and assessed?
  • What approaches are being taken to broaden delivery, the major hurdle for RNA-targeting therapeutics?
  • After delivery what is the next challenge for RNA-targeting drugs?

TABLE 20: Predictiveness of Animal Models

Moderator: Marla Weetall, PhD, Vice President, Pharmacology, PTC Therapeutics

  • Discussion will revolve around using animal models to predict safety and efficacy as many splicing sites are not conserved across species

Kinase Inhibitor Discovery

TABLE 21: What Are the Next Actionable Precision Oncology Approaches?

Moderator: Vijaya Tirunagaru, PhD, VP, Head of Biology and Non-Clinical Development, Research, Rain Therapeutics

  • Technologies to identify novel oncogenic drivers
  • Mapping of driver mutations to specific patient populations
  • Synthetic lethality approaches

TABLE 22: Artificial Intelligence in Kinase Drug Discovery and Development

Moderator: Istvan J. Enyedy, PhD, Principal Scientist, Biogen

  • Use of AI in kinase inhibitor drug design and optimization
  • Pros and cons of AI in drug discovery
  • What is a novel kinase inhibitor and how can we expand the chemical space of kinase inhibitors?

PROTACs and Targeted Protein Degradation

TABLE 23: Designing and Optimizing Chemistry and Drug-Like Properties of Protein Degraders

Moderators: Ye Che, PhD, Head of Computational Design, Discovery Sciences, Pfizer, Inc.

Michael Plewe, PhD, Vice President, Medicinal Chemistry, Cullgen, Inc.

  • Design of protein degraders, linkers
  • Kinetics of binding and degradation
  • Ternary complex formation

TABLE 24: Novel Strategies for Protein Degradation

Moderator: Stewart Fisher, PhD, CSO, C4 Therapeutics 

  • Applying enzymology concepts to the optimization of targeted protein degraders
  • Developing biochemical and cell-based assays for studying protein degradation
  • Protein degradation beyond bi-functional degraders

TABLE 25: Overcoming Translation Challenges

Moderators: Peter Dragovich, PhD, Staff Scientist, Discovery Chemistry, Genentech

Shaomeng Wang, PhD, Warner-Lambert/Parke-Davis Professor of Medicine, Pharmacology and Medicinal Chemistry; Co-Director, Molecular Therapeutics Program and Director, Cancer Drug Discovery Program, University of Michigan

  • Pursuing previously undruggable protein targets
  • Exploring use of PROTACs and other protein degraders for oncology
  • Issues surrounding PK/PD, biotransformation, in vivo pharmacology, and delivery

Targeting Fibrosis

TABLE 26: Translational Tools for Studying Fibrosis

Moderator: Vanessa M. Morales-Tirado, PhD, Senior Scientist III, Translational Immunology, Immunology Discovery, AbbVie Bioresearch Center

  • Advanced physiological models in fibrosis (animal models for safety and efficacy)
  • Current and novel biomarkers in discovery
  • New in vitro/ex vivo assays (3D cellular and organotypic models)

TABLE 27: Promising New Fibrosis Targets

Moderator: Victor J. Thannickal, MD, Professor & Director, Division of Pulmonary, Allergy and Critical Care Medicine, University of Alabama at Birmingham

  • Cross-over Immuno-Oncology targets
  • Senolytics
  • Inflammation/Immune system targets

TABLE 28: Establishin Proof of Concept in Fibrosis

Moderator: Karl Kossen, PhD, Senior Vice President, Translational Science, Indalo Therapeutics

  • Biomarkers and imaging techniques
  • Enabling preclinical studies: opportunities and limitations
  • Notable clinical programs

GPCR-Based Drug Discovery

TABLE 29: Biased Signaling

Moderator: John Janetzko, PhD, Damon Runyon Postdoctoral Fellow, Kobilka Lab, Department of Molecular and Cellular Pharmacology, Stanford University

  • Structural determinants of ligand bias
  • The role of GRKs in biased signaling
  • Cryo-EM of GPCR complexes and its role in drug discovery
  • How can academic research help the development of new drugs?

TABLE 30: GPCR Targeted Lead-Development Challenges

Moderator: Phil Carpino, PhD, Scientific Director, Discovery Chemistry, Janssen Research & Development

  • Untangling biased signaling
  • Comparing various cell-based screens 
  • Medicinal chemistry challenges

TABLE 31: GPCR-Ligand Binding Kinetics

Moderator: Sam Hoare, PhD, Founder, Pharmechanics LLC

  • Compound residence time vs. compound in vivo efficacy
  • Off rate and duration of action
  • Kinetic artifacts in SAR assays

Antibodies Against Membrane Protein Targets – Part 2
Antibody Forum – Part 2

TABLE 32: Structure-Based Antibody Discovery and Design

Moderator: Christopher Corbeil, PhD, Research Officer, Human Health Therapeutics, National Research Council Canada

  • De novo design of antibodies: Useful and feasible?
  • Computational predictions in real-life projects: Better integration needed?
  • Using computational tools to access difficult-to-target proteins
  • Structure-based antigen design

TABLE 33: Opportunities in Antibody Discovery for Membrane Proteins

Moderator: Christyne Kane, PhD, Senior Scientist, Biologics Generation, AbbVie Bioresearch Center

  • In vivo vs. in vitro Approaches: The pros and cons
  • Antibody Databases: Requirements for the ideal database
  • IHC Tool Antibodies: Best practices for generation

TABLE 34: Characterization of Antibodies Against Membrane Proteins

Moderator: Joseph Rucker, PhD, Vice President, Research and Development, Integral Molecular, Inc.

  • Affinity and Kinetics: Useful approaches for characterizing antibody binding
  • Epitopes: Different techniques for epitope mapping; binning versus mapping; why do epitopes matter?
  • Cell Function: Integrating functional assays into antibody discovery and development