Grab a cup of coffee and 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.

Targeting Histone Methyltransferases and Demethylases
Targeting the Ubiquitin Proteasome System
Targeting the Microbiome
Lead Generation Strategies
NASH and Fibrosis
CRISPR for Disease Modeling and Target Discovery
NK Cell-Based Cancer Immunotherapy
Antibodies Against Membrane Protein Targets - Part 1
GPCR-Based Drug Discovery
Next-Generation Histone Deacetylase Inhibitors
Kinase Inhibitor Discovery
Autoimmune and Inflammation Drug Targets
Target Identification Strategies
Emerging Oligonucleotide Therapeutics
Targeting Ocular Disorders
Targeting Tumor Myeloid Cells
Antibodies Against Membrane Protein Targets - Part 2

Tuesday, September 26 | 5:05-6:05 pm

Targeting Histone Methyltransferases and Demethylases

Chemistry of Histone Demethylase Inhibitors

Moderator: Udo Oppermann, Ph.D., Professor, Molecular Biology; Director, Molecular Laboratory Sciences, Botnar Research Centre; Principal Investigator, Epigenetics and Metabolism, Structural Genomics Consortium, University of Oxford

  • Achieving potent and highly selective KDM inhibitors
  • Challenges in discovery of novel KDM chemical probes
  • New technologies supporting the discovery of KDM inhibitors

Considerations for Histone Methyltransferase Assay Development

Moderator: Karen Maegley, Ph.D., Associate Research Fellow, Biochemistry and Primary Screening, Pfizer Oncology

  • What are the options (direct enzymatic/binding assays vs. indirect assays)?
  • What are the limitations and opportunities of each approach?
  • How do different substrates or changes in nucleosome structure alter the enzyme active site?

Targeting the Ubiquitin Proteasome System

Small Molecule Immune-Oncology Drugs: Ready for First-Line Therapy?

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

  • Can biomarkers be developed to identify treatable patients and monitor therapy?
  • Can they be combined with other immune-oncology drugs and/or drugs that act directly on the tumor to achieve maximum efficacy?
  • How can the unleashed immune effect be tempered to minimize side effects?

Novel Targets for Cancer in the Proteostasis Space

Moderator: Benedikt Kessler, Ph.D., Professor of Biochemistry and Life Science Mass Spectrometry, Target Discovery Institute, Nuffield Department of Medicine, University of Oxford

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

Targeting the Microbiome

From Microbiome to Market: Exploring Successful Collaboration and Business Investment Models

Moderator: Keith F. Batchelder, M.D., CEO and Founder, Genomic Healthcare Strategies

Developing Microbiome Industry Standards

Moderator: Scott Jackson, Ph.D., Molecular Genetics and Microbial Genomics, National Institute of Standards and Technology

Ethical Issues in Microbiome Research and Medicine

Moderator: John M. Conley, J.D., Ph.D., William Rand Kenan, Jr. Professor of Law, University of North Carolina, Chapel Hill; Counsel, Robinson Bradshaw & Hinson

Lead Generation Strategies

DNA-Encoded Libraries

Moderator: Christopher Phelps, Ph.D., Manager, Drug Design & Selection Boston, RD Platform Technology & Science, GSK

  • 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 Drug Design Challenges

Moderator: Susanne Saalau, Ph.D., Director, Molecular Science, Astex Pharmaceuticals

  • FBDD in absence of x-al structure
  • Pros and cons of X-ray crystallography as primary screen
  • Orthogonal FBDD screening approaches: TSA, NMR, SPR, high content, phenotypic
  • Library design challenges

Integrating Phenotypic Screening into Lead Generation

Moderator: Paula M. Loria, Ph.D., Associate Research Fellow, Primary Pharmacology Group, Discovery Sciences, Pfizer

  • How used for HIT ID and validation - examples from the group
  • How used to support SAR
  • Setting up a screen from scratch

NASH and Fibrosis

Non-Invasive Assessment of Liver Fibrosis

Moderator: Bryan C. Fuchs, Ph.D., 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?

Animal Models for Fibrosis

Moderator: Weilin Xie, Ph.D., Senior Principal Scientist, Biotherapeutics, Celgene

  • Who has used what?
  • Pros and cons of different models
  • What looks promising

NASH Drug Development Challenges

Moderator: Eric Lefebvre, M.D., Vice President, Head of Clinical Research and Development - NASH, Allergan

  • Translational tools
  • Role of biomarkers
  • FDA guidance
  • Defining target population

CRISPR for Disease Modeling and Target Discovery

CRISPR/Cas9 for Drug Discovery Applications

Moderators: James Inglese, Ph.D., Head, Assay Development & Screening Technologies, National Center for Advancing Translational Sciences, NIH
Patrick Collins, Ph.D., Senior Scientist, Genome Analysis Unit, Amgen

  • Impact of CRISPR/Cas9 for drug discovery in pharma and academia
  • Applications for functional screens, creating cell lines and disease models
  • Technical limitations
  • CRISPR delivery

Applications for Pooled CRISPR/Cas9 Screening: What Works and What Doesn't

Moderators: Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University
Roderick Beijersbergen, Ph.D., Group Leader, Netherlands Cancer Institute and Head, NKI Robotics and Screening Center

  • Best practices on setting up high-throughput CRISPR screens
  • Data analysis and validation; use of complementary methods
  • Insights on inherent challenges and ways to overcome it

Setting Up Pooled and Arrayed CRISPR Screens: What You Need to Know Before and After

Moderators: John Doench, Ph.D., Associate Director, Genetic Perturbation Platform, Broad Institute of Harvard and MIT
Jennifer Smith, Ph.D., Deputy Director, ICCB-Longwood Screening Facility, Harvard Medical School

  • Challenges and solutions for CRISPR gRNA design
  • Methods for detecting engineered changes
  • Evaluating and testing the reagents and libraries
  • Data analysis

NK Cell-Based Cancer Immunotherapy

Off-the-Shelf Cancer Immunotherapy

Moderator: Bahram (Bob) Valamehr, Ph.D., MBA, Vice President, Cancer Immunotherapy, Fate Therapeutics, Inc.

  • Genetic engineering of pluripotent cells
  • Derivation and expansion of single pluripotent cell clones
  • Generation of off-the-shelf NK and T cells

Addressing NK Cell Complexity in Clinical Trials

Moderator: Dean Anthony Lee, M.D., Ph.D., Professor, Pediatrics; Director, Cellular Therapy and Cancer Immunotherapy Program, Nationwide Children's Hospital; James Comprehensive Cancer Center/Solove Research Institute, The Ohio State University

  • Which subsets predict response?
  • Which phenotypes are generated by selection or expansion methods?
  • How to address these questions systematically and consistently in clinical trials

Antibodies Against Membrane Protein Targets - Part 1

Making Cell-Free Accessible - Expanding Protein Production Opportunities

Moderator: Erik Henrich, Researcher, Institute of Biophysical Chemistry, University of Frankfurt, Germany

  • Setting up cell-free expression systems
  • Hydrophobic expression environments
  • Tailored expression conditions
  • Toolbox of additive libraries: ligands, co-factors, inhibitors, etc.

In Vitro Binder Selections vs. Immunization Strategies for Membrane Protein Targets

Moderator: Pascal Egloff, Ph.D., Postdoctoral Researcher, Markus Seeger Group, Institute of Medical Microbiology, University of Zurich, Switzerland

  • Synthetic libraries and natural scaffolds
  • Target quality and selection biases
  • Importance of NGS for selections
  • State-specific binders

Selection of Target-Specific Antibodies

Moderator: Andrew Nixon, Ph.D., Vice President, Biotherapeutics Molecule Discovery, Boehringer Ingelheim

  • Antibody discovery using B cell selection
  • Integration of NGS into antibody discovery
  • Functional screening methodologies
  • Selecting for epitope diversity vs. affinity

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
  • Newer assay kits
  • Understanding signaling bias

Biophysical Approaches to GPCR Drug Discovery

Moderator: Phillip Schwartz, Ph.D., Senior Scientist, Biophysical Chemistry, Takeda

  • Physiological or mimetic, in what matrices should GPCRs be placed for biophysical characterization?
  • Promising new technologies to study GPCR-drug interactions
  • Fragment screening GPCRs: best methods and practices

Opioid Receptor Targeting

Moderator: Ying Xian Pan, M.D., Ph.D., Laboratory Head, Department of Neurology, Memorial Sloan Kettering Cancer Center

  • Which biased ligands show the most promise?
  • How to correlate in vitro biased signaling studies with in vivo results?
  • How can we integrate structural knowledge into new drug design?
  • What are the challenges in designing new biased ligands?

Thursday, September 28 | 8:00-9:00 am

Next-Generation Histone Deacetylase Inhibitors

Non-Oncology Indications for HDAC Inhibitors

Moderator: Timothy A. McKinsey, Ph.D., Associate Professor and Associate Division Head for Translational Research, Director, Consortium for Fibrosis Research and Translation, Department of Medicine, Division of Cardiology, University of Colorado Denver

  • Are isoform-selective HDAC inhibitors really necessary?
  • Can we improve the therapeutic index of HDAC inhibitors with episodic dosing and/or combination therapy?
  • Why do HDAC inhibitors work in so many preclinical models?

HDAC Inhibitors in Immuno-Oncology

Moderator to be Announced

Kinase Inhibitor Discovery

New Kinase Targets in Cancer Immunotherapy

Moderator: Guido J.R. Zaman, Ph.D., Managing Director & Head of Biology, Netherlands Translational Research Center B.V. (NTRC)

  • Biological insights into the immune pathways
  • Target validation approaches for novel kinase immunotherapy targets
  • Predicting rational combinations

Strategies for Achieving Kinase Inhibitor Activity and Selectivity

Moderator: Istvan Enyedy, Ph.D., Senior Scientist, Drug Discovery, Biogen

  • Ligand- and structure-based methods
  • Limitations of current methods
  • Emerging tools and strategies

Autoimmune and Inflammation Drug Targets

Developing Kinase Inhibitors for Chronic Indications

Moderator: John Robinson, Ph.D., Director, Medicinal Chemistry, Array Biopharma

  • Utility of kinase selectivity profiling data
  • Safety assessment as an experiment rather than a progression gateway
  • Integrating PK/PD to predict safety margins

Drug Development for Inflammatory Bowel Disease

Moderator: Kamal Puri, Ph.D., Director, Inflammation & Immunology, Celgene Corporation

  • Why so much activity recently?
  • What will the key unmet needs be in 5 years?
  • Biomarkers to predict response and complications; e.g. structuring or fistualizing disease
  • Personalized approach to treatment: how do we use markers to get the right drugs to the right patients?

Current Issues in Patentability of Biotech/Pharma Innovations

Moderator: Sanjeev Mahanta, Ph.D., J.D., Associate Attorney, Intellectual Property and Technology Practice Group, Posternak, Blanskstein and Lund LLP

  • The first-to-invent patent regime expands the scope of prior art, making it critical that patent applications be filed as early as possible
  • The new regime makes it easier to invalidate patents, making it very important that patents sought are of high quality
  • Challenge of 'personalized medicine' patents 

Targeting IL17 Pathway

Moderator: Joseph Wahle, Ph.D., Senior Scientist, Immunology and Respiratory Disease Research, Boehringer Ingelheim

  • Other promising targets in IL17 pathway besides ROR?
  • ROR challenges
  • Downstream development issues/safety signals
  • Animal models

Target Identification Strategies

Exploiting the Complementarity of CRISPR and RNAi

Moderator: Stephanie Mohr, Ph.D., Lecturer, Genetics & Director of the Drosophila RNAi Screening Center, Harvard Medical School

  • Use of CRISPR v/s RNAi
  • Understanding inherent limitations and need for using complementary techniques for validation
  • Examples of how both techniques have been put to good use

Exploring Computational Techniques for Improving Drug Discovery

Moderator: Jeffrey Barrett, Ph.D., Director, Open Targets and Group Leader, The Wellcome Trust Sanger Institute, Genome Research Limited

  • In silico modeling of complex cellular phenotypes and disease models
  • Deconvolution of preclinical data and translating into clinical space
  • Understanding limitations and applications of computational approaches

Advances in Chemical Biology and Phenotypic Screening for Target Discovery

Moderator: Jeff Piotrowski, Ph.D., Principal Scientist, Target Identification, Yumanity Therapeutics

  • What is covered and what is missing when accessing available target space?
  • Latest breakthroughs in on- and off-target identification
  • Avoiding potential pitfalls in target identification

Emerging Oligonucleotide Therapeutics

Considerations for Therapeutic Translation of CRISPR/Cas9

Moderators: Clifford Steer, M.D., Professor of Medicine and Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School
Eric B. Kmiec, Ph.D., Director, Gene Editing Institute and Senior Research Scientist, Center for Translational Cancer Research, Helen F. Graham Cancer Center & Research Institute, Christiana Care Health System

  • Potential human applications of genome editing to somatic cells
  • Scientific and technical considerations for design and application of genome editing strategies
  • Ethical and regulatory issues associated with somatic cell genome editing
  • Development of reproducible and robust strategies that can enable precise point mutation repair or gene insertion
  • Types of experimental variabilities with the use of different cell lines; established cell lines, iPSCs and primary cells in general

Designing and Delivering Better Oligonucleotide Drugs

Moderators: Dan Peer, Ph.D., Director, Laboratory of Precision NanoMedicine, Tel Aviv University 

Bruce Given, M.D., COO, Arrowhead Pharmaceuticals

  • Is delivery the only challenge that will change the fate of oligonucleotide therapies?
  • What are the challenges and opportunities in modulating the immune system with oligonucleotide drugs?
  • Can we design improved and local oligo therapeutics? In what indications? Examples from siRNA, modified mRNA and CRISPR /Cas will be considered. 

Targeting Ocular Disorders

Small Molecules or Big Molecules: Current Ophthalmic Drug Development Status?

Moderator: Bo Liang, Ph.D., President, R&D, IVIEW Therapeutics, Inc.

  • Current challenges for ocular disorders?
  • Biologics setting up a gold standard or small molecules to combat eye diseases?
  • Drugs and diagnostics?

Optimizing Recruitment for Ocular Rare Diseases

Moderator: Sharon Klier, Vice President, Ophthalmology, Medical, Quark Pharmaceuticals

  • Why? Incentives for pharmaceutical companies.
  • Where? Finding suitable clinical sites/geographical areas.
  • Who? Diagnosis challenges.
  • How? Protocol design.

Is Neuroprotection for Glaucoma Treatment a Dream?

Moderator: Naj Sharif, Ph.D., FARVO, FBPhS, Executive Director; Head, Global Alliances & External Research, Santen, Inc.

  • What is neuroprotection and how do we achieve it?
  • Why have previous clinical trials failed?
  • What does the future hold for Glaucoma treatment?

Targeting Tumor Myeloid Cells

Preclinical Efficacy Assessment of Drug Candidates Targeting Tumor Myeloid Cells

Moderator: Bernard Vanhove, Ph.D., COO, OSE Immunotherapeutics

  • Cell-based assays for myeloid function
  • Setting up "in sitro" assays from patient tissues
  • Surrogate animal models and drugs
  • Humanized mice models

Combination Cancer Immunotherapy and New Immunomodulatory Targets

Moderator: Kipp A. Weiskopf, M.D., Ph.D., Resident Physician, Medicine, Brigham and Women's Hospital

  • What are emerging strategies for immunotherapy combinations?
  • How can preclinical and clinical data determine rational combinations?
  • How can we use biomarkers to inform new combinations?

Antibodies Against Membrane Protein Targets - Part 2

Emerging Methods for Expression and Reconstitution of GPCRs

Moderator: Scott R. Prosser, Ph.D., Professor, Chemistry and Biochemistry, University of Toronto, Canada

  • E. coli - challenges with GPCRs
  • Yeast - opportunities and developments
  • Purification, extraction, reconstitution
  • Micelles versus HDLs versus liposomes
  • Biosynthetic labeling and the use of isotopes
  • Insect cells & HEK cells - efficiencies and opportunities

Characterization of Antibodies Against Membrane Proteins

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

  • Affinity and Kinetics: Useful approaches for characterizing antibody binding; interpreting complex binding.
  • Epitopes: Different techniques for epitope mapping; binning versus mapping; does epitope matter?
  • Cell Function: Integrating functional assays into antibody discovery and development; ion channel assays beyond electrophysiology.
  • Endocytosis: What are the best approaches for measuring antibody endocytosis? Recycling versus degradation.
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