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.  

WEDNESDAY, SEPTEMBER 26 | 5:05 - 6:05 PM

Small Molecules for Cancer Immunotherapy

Exploring Diverse Target Classes for Cancer Immunotherapy

Moderators: Paul Kassner, PhD, Vice President, Quantitative Biology, FLX Bio Inc.

Buvana Ravishankar, PhD, Scientist, Discovery Biology, FLX Bio Inc.

  • Enzyme targets vs. protein-protein interactions and approaches to target them
  • Immuno-metabolism targets
  • Kinase inhibitors for immunotherapy
  • TLRs
  • Chemokines

The Chemistry of Small Molecule Immunomodulators in the Clinic

Moderator: David Ferguson, PhD, Professor, Medicinal Chemistry, University of Minnesota

  • Single agents
  • Vaccine adjuvants
  • Combination therapies
  • Drug delivery and formulation 

NK Cell-Based Cancer Immunotherapy

Targeting Solid Tumors with NK Cells

Moderator: Dan Kaufman, MD, PhD, Professor, Director, Cell Therapy Program, University of California, San Diego

  • What antigen(s) are best to target?
  • CAR vs. antibody vs. NK cell engager strategies against solid tumors
  • How best to combine NK cells with other agents in clinical trials?

Addressing NK Cell Complexity in Clinical Trials

Moderator: Karl-Johan Malmberg, MD, PhD, Professor, Cancer Immunology, Institute for Cancer Research, Oslo University Hospital, Norway

  • Which subsets predict response?
  • Which phenotypes are generated by selection or expansion methods?
  • How to implement the emerging insights into the regulatory role of NK cells as a bridge to adaptive immunity?
  • How to address these questions systematically and consistently in clinical trials? 

Targeting the Ubiquitin-Proteasome System

Novel Targets for Cancer in the Proteostasis Space

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

  • Biological insights into the Ubiquitin-Proteasome System pathway
  • Target validation approaches for novel proteostasis targets
  • Emerging proteostasis targets

Screening Tools to Identify PROTACS, Kinases and Other Molecules

Moderator: Davide Gianni, PhD, Team Leader, Discovery Sciences, AstraZeneca

  • Biochemical, biophysical and cellular based approaches to monitor ternary complex formation
  • Approaches to identify novel E3 ligases and E3 ligase ligands

CNS and Neurodegenerative Targets

The Use of Precision Medicine in Drug Development for CNS Disorders

Moderator: Daniel Klamer, PhD, MBA, Vice President, Business Development & Scientific Strategy, Anavex Life Sciences Corp.

  • What questions should be asked before adding a new biomarker to your clinical trial?
  • What lessons can we learn from oncology?
  • Pros and cons using biomarkers as surrogate endpoints

Machine Learning Models in CNS Drug Discovery

Moderators: Istvan Enyedy, PhD, Principal Scientist, Biogen

Jane Yu, Technical Pre-Sales, Life Sciences, IBM Watson Health

  • Pros and cons of AI in drug discovery
  • Implementing AI in drug development – when and how
  • Methods and building models

Constrained Peptides and Macrocyclics

Conformational Searching and Macrocycles

Moderator: Maxwell D. Cummings, PhD, Senior Principal Scientist, Computational Chemistry, Discovery Sciences, Janssen R&D 

  • Is the binding mode known, suspected or unknown?
  • Selection of conformers and conformer sets: energy, RMSD, properties, etc.
  • Aspects of computational search methods relevant to macrocycles

Lead ID Using Macrocycle Libraries

Moderator: Adrian Whitty, PhD, Associate Professor of Chemistry, Boston University

  • What properties define a good macrocycle screening hit?
  • What represents good potency, and does this depend on library chemistry?
  • Specialized/biased versus general purpose libraries
  • Applications of machine learning

Drug Development Challenges of Stapled Peptides and Synthetic Macrocyclics

Moderator: Vincent Guerlavais, PhD, Director, Medicinal Chemistry, Aileron Therapeutics

  • Lead op challenges unique to stapled peptides or macrocyclics
  • Planning ahead: making it easier for formulation/scale-up
  • Converting a drug candidate into a macrocyclic 

Target Identification and Validation – Part 1

Exploiting CRISPR, RNAi and Single-Cell Analysis: What You Need to Know Before and After

Moderators: Sarah Boswell, PhD, Director of Sequencing Technologies, Laboratory of Systems Pharmacology and Director, Single-Cell Sequencing Core, Harvard Medical School

Roderick Beijersbergen, PhD, Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

  • Understanding inherent limitations and need for using complementary techniques
  • Examples of how multiple techniques have been put to good use for addressing biological questions
  • Evaluating and testing the reagents and tools
  • Insights on inherent challenges and ways to overcome it
  • Tackling data analysis

Exploring Artificial Intelligence for Improving Drug Discovery and Healthcare

Moderators: Arvind Rao, PhD, Associate Professor, Department of Computational Medicine and Bioinformatics, University of Michigan

Nicholas P. Tatonetti, PhD, Herbert Irving Assistant Professor of Biomedical Informatics and Director of Clinical Informatics, Herbert Irving Comprehensive Cancer Center, Columbia University

  • In silico modeling of complex cellular phenotypes and disease models: paradigms to bridge the computational-experimental gap
  • Deconvolution of preclinical data and translating into clinical space: integrative modeling, generalizability, transportability and data harmonization
  • Why ML/AI in healthcare data will never work; bias, missingness, and confounding in secondary data use
  • Why ML/AI is our only hope to solve healthcare’s biggest problems; the power of scale, the complexity of medicine, and where the opportunities lie

Growing Use of 3D Spheroids and PDX Models in Drug Discovery

Moderators: 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

  • 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

Antibacterial Discovery and Development

Alternative Approaches to Treatment of Antibiotic-Resistant Pathogens

Moderator: Neeraj (Neil) Surana, MD, PhD, Assistant Professor of Pediatrics, Molecular Genetics and Microbiology, Duke University

  • Microbiota-based therapies
  • Phage-based therapies
  • Vaccines-based therapies

Considerations for the Discovery Scientist

Moderator: James Levin, PhD, Director of Preclinical Development, Cidara Therapeutics

  • What makes a good hit? What makes a good lead?
  • How do you build a screening paradigm?
  • How important is a target product profile? When should it be developed?
  • What clinical development aspects should be considered in the discovery process? When should they be incorporated?

NASH and Fibrosis

Non-Invasive Assessment of Liver Fibrosis

Moderator: Bryan C. Fuchs, PhD, Assistant Professor of Surgery, Harvard Medical School

  • Serum tests: Will omic analyses hold the key?
  • Which imaging technologies have the most promise?
  • Are tools that assess dynamic changes needed for early assessment of anti-fibrotics?

NASH Drug Development Challenges

Moderator: Rebecca Taub, MD, CMO & Executive Vice President, Research & Development, Madrigal Pharmaceuticals

  • Role of biomarkers
  • FDA guidance
  • Defining target population
  • Animal models

Cirrhosis and NASH

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

  • Challenges in treating cirrhosis and NASH
  • Common and unique targets between cirrhosis and NASH
  • Designing clinical trials for cirrhosis NASH 

Antibody Discovery Forum – Part 1

How Can NGS Support Library Production, Selection and Screening?

Moderator: Andrew Bradbury, PhD, MB BS (MD), CSO, Specifica, Inc.

  • Different NGS platforms
  • NGS traps
  • NGS in estimating library diversity
  • NGS in screening analysis

Tools for Enhancing Antibody Discovery

Moderator: Colby Souders, PhD, Chief Technology Officer, Abveris Antibody

  • Design and production of quality antigens and reagents required for discovery
  • Integration of next-generation sequencing in the discovery and engineering platform
  • Naïve discovery: library, hybridoma or B cell sorting?
  • Transitioning from low-throughput to high-throughput to full automation

Synthetic Single-Domain Antibody Libraries and the Future of Antibody Discovery

Moderator: Conor McMahon, PhD, Postdoctoral Fellow, Biological Chemistry and Molecular
Pharmacology, Harvard Medical School

  • Library design and discovery of binders
  • What makes a synthetic antibody library good?
  • Measuring library quality: Can we learn something from structural biologists?
  • Phage and yeast…is that the best we can do?
  • Pushing the limit: Interfacing with computer scientists to design ‘smart’ libraries
  • Will synthetic libraries ever be as good as animal derived? 

Antibodies Against Membrane Protein Targets – Part 1

How Will We Accomplish Future Progress in Generating Therapeutic Biologics to Challenging Membrane Targets?

Moderator: Catherine Hutchings, PhD, Independent Consultant, United Kingdom

  • GPCRs, ion channels, transporters, tetraspanins, etc.
  • Target selection and validation of emerging targets
  • Biology of disease, e.g., is there temporal and tissue ligand bias in disease?
  • Biology of function, e.g., active, inactive or allosterism, antibody-based formats
  • What has been an enablement (and what has been a distraction)?

Detergent-Free Production of Membrane Proteins

Moderator: Tim Dafforn, PhD, Professor, Biotechnology, University of Birmingham, United Kingdom

  • Challenges of using detergent
  • Variety of approaches to detergent free extraction
  • Comparisons of each method
  • Challenges of using detergent-free systems
  • Use of detergent-free systems in drug discovery pipelines

The Role of MS in Pharma: From Small to Large Molecules and Beyond

Moderator: Iain D. G. Campuzano, Principal Scientist, Discovery Attribute Sciences, Amgen

  • Native-MS in pharma: Is it established or still niche?
  • Required improvements for native-MS to become mainstream in pharma
  • LC-MS and native-MS in membrane protein analysis: What role does it have?
  • Specific MS improvements which would highly benefit pharmaceutical research

Structure and Function of Membrane Proteins for Ab Production and Drug Development

Moderator: Bonnie Ann Wallace, PhD, Professor, Institute of Structural and Molecular Biology, Birkbeck College, United Kingdom

  • Expression of membrane proteins (eukaryotic and prokaryotic)
  • Structure and modelling of membrane proteins
  • Antibody production against membrane proteins
  • Drug binding (in vitro and in silico) to channels and receptors  

FRIDAY, SEPTEMBER 28 | 7:30 - 8:30 AM

Autoimmune and Inflammation Drug Targets

The Microbiome and Autoimmunity

Moderator: Ulrich Thienel, MD, PhD, CMO, Finch Therapeutics, Inc.

  • Promising drug targets shared across platforms (microbiome/small molecules/biologics) in treating autoimmune disease  
  • Treating autoimmune disease with full microbiome spectrum vs. single strain
  • Drug development challenges for microbiome-based treatments

IBD Drug Development

Moderator: David Y. Liu, PhD, CSO, Protagonist Therapeutics

  • Why so much activity recently?
  • What are the promising targets?
  • What will the key unmet needs be in 5 years? 
  • What are the challenges ahead?

Developing Kinase Inhibitors for Chronic Indications

Moderator: Philip A. Harris, PhD, Senior Director, Pattern Recognition Receptor DPU, GlaxoSmithKline

  • Covalent vs. noncovalent
  • Utility of kinase selectivity profiling data
  • Integrating PK/PD to predict safety margins 

Targeting the Microbiome

Standards for the Microbiome Therapeutics Industry: Navigating the Regulatory Landscape

Moderator: Scott A. Jackson, PhD, Leader, Complex Microbial Systems Group, Biosystems and Biomaterials Division, National Institute of Standards and Technology

  • Validated analytical methods for demonstrating purity, identity, potency, stability of live biotherapeutic products
  • The need for reference materials
  • Leveraging current standards:  e.g. USP

Targeting Tumor Myeloid Cells

Therapeutic Targeting of Myeloid Derived Suppressor Cells

Moderator: David A. Eavarone, PhD, Senior Scientist, Siamab Therapeutics

  • In vitro assays
  • Specific targeting of MDSC
  • Effective payloads  

Kinase Inhibitor Discovery

Challenges in Kinase Inhibitor Discovery

Moderator: Istvan Enyedy, PhD, Computational Chemistry, Biogen

  • Selectivity for safety
  • Blood-brain barrier permeability

Kinase Targets for Cancer Immunotherapy

Moderator: Guido Zaman, PhD, Managing Director & Head of Biology, Netherlands Translational Research Center B.V.

  • What are the new and promising kinase targets for cancer immunotherapy?
  • Lessons learned from combination studies with immune checkpoint modulators
  • Technologies and approaches for target identification and validation 

GPCR-Based Drug Discovery

New Ways to Screen GPCRs

Moderator: Samantha J. Allen, Ph.D., Senior Scientist, Lead Discovery - Screening, Janssen Research & Development

  • Physiologically relevant cells and assays
  • High-content screening
  • Understanding signaling bias

New technologies for GPCR Structure-Function Insights and Drug Discovery Impact

Moderator: Matthew Eddy, PhD, Assistant Professor, Chemistry, University of Florida

  • XFEL and other crystallography advances
  • NMR applications for drug screening
  • cryoEM: current applications and future directions 

SMALP/Nanodisc Technology for Membrane Proteins

Moderator: Mahmoud l. Nasr, RPh, PhD, Instructor, Biological Chemistry and Molecular Pharmacology Department. Harvard Medical School

  • Similarities and differences of lipid interactions with SMA (styrene-maleimide polymers) v. MSP (membrane scaffold proteins) nanodiscs
  • Advantages and limitations of SMALP particles and MSP nanodiscs in solution
  • Covalently Circularized Nanodiscs 
  • DNA-Corralled Nanodiscs
  • Possible applications of small and large nanodiscs in biotechnology and medicine
  • Future directions and problems 

Lead Generation Strategies

Degradation-Induced Therapeutics

Moderator: Joe Patel, PhD, Director, Structural Biology, C4 Therapeutics

  • Ubiquitin-mediated protein degradation strategies 
  • Which technique to try first?
  • Hurdles to their therapeutic potential
  • Stories to share?

DNA-Encoded Libraries

Co-moderators: Svetlana Belyanskaya, PhD, Scientific Leader, Encoded Library Technologies, R&D Platform Technology & Science, GSK Boston

Ghotas Evindar, PhD, Manager, ELT Chemistry, GSK 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

Fragment-Based Libraries

Moderator: Ashley Adams, PhD, Senior Scientist, Discovery Chemistry and Technology, AbbVie, Inc.

  • Revamping libraries – what have we learned?
  • When to use which type of library?
  • Pros and cons of various commercial libraries 

Target Identification and Validation – Part 2

Chemical Biology Approaches for Target ID and Target Discovery

Moderators: Doug Johnson, PhD, Director, Chemical Biology & Proteomics, Biogen

Jaimeen Majmudar, PhD, Senior Scientist, Chemical Biology, Pfizer, 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? 

Targeting Gram-Negative Pathogens

Building an Understanding of Porin-Permeation in Gram-Negative Pathogens

Moderator: Ram Iyer, PhD, Principal Scientist (Bacteriology), Entasis Therapeutics, Inc.

  • Addressing the knowledge gap for gram-negative pathogens
  • Specific approaches, ie, TOMAS
  • Identifying compounds that will disrupt the outer membrane

Filling in the Gaps in the MDR Gram-Negative Pipeline

Moderator: Todd A. Black, PhD, Executive Director, Infectious Diseases, Basic Research, Merck Research Laboratories

  • Narrow spectrum antibiotics
  • Non-traditional / non-antibiotic approaches
  • Challenges for clinical trial designs
  • Diagnostic challenges of identifying the right patients and strategies
  • Future steps 

Antibody Discovery Forum – Part 2

Considerations in Selecting Bispecific Antibody Formats for Immunotherapies

Moderator: Gundo Diedrich, PhD, Director, Antibody Engineering, MacroGenics

  • Target considerations in selection of bispecific pairs
  • Criteria to select a format: valency, half-life, effector function
  • Manufacturing challenges: byproducts, aggregation, titer
  • Considerations to modulate the affinity to both targets: cis vs. trans-binding, target expression
  • MOAs of bispecifics that cannot be addressed with combinations of monospecific antibodies

Considerations in Target Selection for Antibody-Drug Conjugates (ADCs)

Moderator: Jennifer J. Hill, PhD, Team Lead, MS & NMR Analytics, National Research Council Canada

  • Characteristics of real-world ADC targets and how these compare to the ideal ADC target
  • Prediction and screening methods to determine target suitability for ADC development
  • ADC target expression in healthy tissues: what is acceptable?
  • Matching targets with optimal drug-linkers

Going Beyond Dual Antibody Targets to Tackle the Complexity of Cancer and the Immune System

Moderator: Marc Mansour, PhD, Senior Consultant, Leidos Health

  • Could single targets (e.g., PD-1/ PDL-1) and dual targets (e.g., PD-1 and CTLA4) be the exception rather than the rule for effective immune modulation of cancer?
  • Combining antibody-based therapies with other modalities such as other biologics or small molecules/targeted therapies – can we do better than simply follow rational (but generally blind) development plan?
  • How do we assess success in our search of a clinical signal when testing complex combinations? 

Antibodies Against Membrane Protein Targets – Part 2

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

How Can we Optimize Therapeutic Antibody Discovery for Multi-Spanning Membrane Proteins (GPCRs, Ion Channels and Transporters)?

Moderator: Trevor Wilkinson, PhD, Associate Director, Antibody Discovery and Protein Engineering, MedImmune Ltd., United Kingdom

  • What are the most efficient methods to isolate antibodies that modulate function?
  • What are the most efficient screening methods?
  • What are the preferred formats for antigens?
  • Can we establish rules for how to address specific target classes?

Production Challenges for Membrane Proteins

Moderator: Nicola Burgess-Brown, PhD, Principal Investigator, Structural Genomics Consortium, University of Oxford, United Kingdom

  • Construct design: preferred tags, proteases, how many constructs to screen?
  • Challenges of expressing integral membrane proteins: which expression host?
  • Problems of scale of production for structural biology: resource and cost issues
  • Strategies to improve purification and stability of membrane proteins: detergents, lipids, affinity reagents, liposomes, nanodiscs

Droplet Microfluidics for Antibody Discovery

Moderator: Christoph Merten, PhD, Group Leader, Microfluidics, European Molecular Biology Laboratory (EMBL), Germany

  • What kind of screens can be done on primary plasma cells from mice and humans?
  • What are the specific advantages compared to other screening formats?
  • How to get access to the technology?

* 活动内容有可能不事先告知作更动及调整。

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