Final Agenda - Day 1

Thursday, May 22, 2014

11:15 am KEYNOTE PRESENTATION: Structures, Chemical Probes, New Biology, New Targets for Drug Discovery: Is This the Right Sequence?

Chas BountraChas Bountra, Ph.D., Professor, Translational Medicine; Head, Structural Genomics Consortium, University of Oxford

In my presentation, I will discuss our partnership with nine large pharmaceutical companies to generate structure enabled, freely available, chemical probes; our collaborations with a network of academic labs to use these probes to dissect biological and disease networks; our plans to further improve target validation by using patient derived primary cells, and a new initiative to advance new clinical candidates into Phase IIa studies, pre-competitively.

12:00 pm Enjoy Lunch on Your Own

Phenotypic Screening, Target Identification and Mechanism of Action of Novel Bioactive Small Molecules 

12:55 Chairperson’s Opening Remarks

Jascha Blobel, Ph.D., Product Manager, Intelligent Pharma


1:00 Systematic Assembly of Chemical Probe Libraries to Explore and Validate Novel Biology

Iván Cornella-Taracido, Ph.D., Head, Chemical Biology; Associate Director, Discovery Sciences Chemistry Innovation Centre, AstraZeneca

Chemical probes, drug-like or not, have been used for years to identify new therapeutic targets as well as to perform validation studies directed to assess their efficacious engagement and pharmacological modulation. Herein I will elaborate on the physicochemical and biological features of a good tool compound, review historical work to assemble a comprehensive, properly annotated, collection of optimal chemical probes and discuss its use towards exploratory phenotype-driven biology (target discovery) and target validation.

1:30 Systematic Chemical Biology: Building the Novartis MOA Box

JeremyJenkinsJeremy L. Jenkins, Ph.D., Senior Investigator I, Developmental & Molecular Pathways, High-Throughput Biology, Novartis Institutes for BioMedical Research

Integrating chemical bioactivity knowledge from large-scale assay data is only the first step to carrying out chemical biology robustly. To use our integrated chemogenomics data effectively, we created an automated rule-based system to score tool compounds for targets based on common-sense assertions such as potency, selectivity, and fame. Consequently, the Mechanism-of-Action Box was created, a set of important tool compounds used widely for opportunistic discovery of targets that modulate phenotypic assays. Further, the chemogenomics data is used to create thousands of probabilistic models capable of predicting targets for new compounds.

Intelligent Pharma2:00 Pythia, The Way to Polypharmacology 

Ignasi_BeldaIgnasi Belda, Ph.D., CEO, Intelligent Pharma

Pharmacological applications of a molecule stand in close relationship with the molecule's principal target. To understand this relationship, Intelligent Pharma has developed a computational technology called Pythia, which uses ligand based approaches and experimental data to predict real and off-targets for a molecule.

2:15 Chemogenomic Profiling: A Systems-Level View of the Cellular Response to Small Molecules

GuriGiaeverGuri Nina Giaever, Ph.D., Associate Professor, Faculty of Pharmaceutical Sciences, The University of British Columbia

Genome-wide characterization of the cellular response to small molecules is fundamental to understanding the cell as a system and the mechanisms of drug action in a cellular context. We profiled 3,250 diverse small molecules genome-wide in a systematic and unbiased manner, identifying 317 compounds that specifically perturb 121 unique genes. Global analysis of the dataset revealed that the majority of the cellular response to small molecules can be described by a network of chemical moiety-associated biological signatures that provide a unique resource for the discovery of functional interactions between genes, chemicals and biological processes.

2:45 An Integrated Solution to Identify Your Small Molecule Targets and Accelerate Your Drug Discovery and Development Program

MarieEdithGourdelMarie-Edith Gourdel, Ph.D., Director, Chemistry, HYBRiGENiCS SERVICES

Identifying protein partners of a small bioactive molecule is of great interest in the drug discovery and development process. It is a support to (i) decipher the mechanism of action after i.e. phenotypic screening, (ii) study “off-target” effects, (iii) adjust therapeutic indications and (iv) consider drug repositioning. ULTImate YChemH™ is a chemical biology tool for direct target identification. This method is based on the well-established yeast two-hybrid technology for protein-protein interactions. It benefits from ULTImate Y2H™ platform whose relevance relies on highly complex protein fragment libraries using an optimized mating procedure and its sophisticated bioinformatics tools.

3:15 Refreshment Break in the Exhibit Hall with Poster Viewing

Case Studies in Epigenetics, Protein-Protein Interactions and Novel biology 

3:45 CREB Binding Protein Inhibition and Target Engagement

JonesLynLyn Jones, Ph.D., Head, Rare Diseases Chemistry; Head, Chemical Biology, Pfizer

This presentation will discuss the structure-based drug design and novel computational techniques applied to the creation of selective inhibitors of the CREBBP bromodomain. Synthetic library enablement and fragment replacement unearthed new bromodomain inhibitors, and a new technology was developed to assess bromodomain target engagement. These methods will facilitate target validation and drug discovery efforts in the epigenetic arena.

4:15 Targeting Protein-Protein Interactions as an Anticancer Strategy

HaianFuHaian Fu, Ph.D., Professor, Pharmacology, Hematology & Medical Oncology; Director, Emory Chemical Biology Discovery Center; Director, Discovery & Developmental Therapeutics Program of Winship Cancer Institute, Emory University

4:45 Chemical Probing Protein Interactions of the Urokinase Receptor in Cancer

SamyMerouehSamy Meroueh, Ph.D., Associate Professor, Biochemistry and Molecular Biology, Indiana University School of Medicine

The urokinase receptor (uPAR) is a GPI-anchored cell surface receptor at the center of an intricate network of protein-protein interactions. uPAR promotes ECM degradation and cell migration through its two immediate binding partners, the serine protease urokinase (uPA) and the extracellular matrix (ECM) component vitronectin (VTN). We present our efforts at the design of small-molecule probes to disentangle protein interactions of uPAR, and the subsequent biochemical, cellular, and computational studies to further define the contribution of these interactions in tumor invasion and metastasis.

5:15 KEYNOTE PRESENTATION: Selecting and Modulating Therapeutic Targets Using Human Biology and Chemical Biology

Stuart SchreiberStuart L. Schreiber, Ph.D., Director, Center for the Science of Therapeutics & Founding Member, Broad Institute of Harvard and MIT; Howard Hughes Medical Institute Investigator; Morris Loeb Professor, Chemistry and Chemical Biology, Harvard University

Human genetics can reveal ‘experiments of Nature’, even providing information related to a ‘dose/response’ through the analysis of allelic series of genetic variants that suggest the impact of caring about the activity of a target in terms of both efficacy and toxicity. Chemical biology can provide small-molecule modulators of the implicated targets, providing an independent means of target validation. This lecture will provide illustrations of an integrated approach to target validation using human genetics and chemical biology.

6:00 Close of Day and Workshop Registration

6:30 Dinner Workshop: Emerging Methods for Measuring Target Engagement*

The increase in usage of small molecule probes for the validation of novel therapeutic targets requires effective means of assaying target engagement in living systems. Importantly, measuring the extent to which a target is engaged (or not) provides critical information regarding probe selectivity and attributed efficacy modulating a biological process, while informing of any off-target interactions and potential toxicity. The workshop is designed to discuss emerging techniques and technologies for measuring target engagement.



-Erik Hett, Ph.D., Senior Scientist, Chemical Biology, Medicinal Chemistry, Pfizer


-Thomas Lundbäck, Ph.D., Assay Development & Screening, Chemical Biology Consortium Sweden; Senior Scientist, Division of Translational Medicine & Chemical Biology, Department of Medical Biochemistry & Biophysics, Karolinska Institutet


-Michael Dabrowski, Ph.D., CEO & Co-Founder, Pelago Bioscience AB


-Andrea M. Zuhl, Ph.D., Fellow, Neuroscience, Pfizer; Former Research Associate, Cravatt Lab, The Scripps Research Institute

*Separate Registration Required

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