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Pre-Conference Short Courses


Morning Short Courses

9:00am - 12:00 noon

(SC1) Imaging Technologies for Target Discovery
Over the last few years it has become evident, that imaging technologies are a very useful tool in drug discovery. This course is designed to provide information on optical and molecular imaging tools and the challenges, opportunities and limits will be discussed. An overview on optical and molecular imaging will be given.

Topics to be covered:

  • Overview and Introduction of current imaging technologies
  • Strength and limitations of using molecular imaging
  • Finding the optimal technology for specific applications
  • Imaging with fluorescent proteins
  • Live animal imaging

9:00 am  Richard M. Levenson, M.D., Vice President, CRI, Inc. 

9:55 Anatomical and functional  imaging using micro-CT and digital subtraction angiography in small animals
Cristian Badea, PhD, Assistant Professor of Radiology, Center for In Vivo Microscopy, Duke University Medical Center

10:45am-11:00am Coffee Break

11:00 am  imaging RNA in vivo
Natalia E. Broude, Ph.D., Research Associate Professor, Department of Biomedical Engineering, Boston University

12:00 pm End of Short Course

(SC2) Understanding the Structural Biology of Ion Channels to Guide Drug Discovery
During the past decade there have been two remarkable technological breakthroughs which have transformed ion channel-based drug discovery. The fi rst advance is using X-ray crystallography and solid-state NMR to solve the structures of ion channels, which enabled structure-guided drug design of ion-channel blockers. The other advance is the development of automated assays such as FLIPR and Ionworks which have high-throughput screening of ion channel blocking compounds a reality. This short course is an overview on the cutting-edge development of structure-based design of ion channel blockers. Several leading experts in the field will provide road maps to starters as well as contributing experiences of several complementary a proaches.
Topics to be covered:

  • Pinpointing ion channel domains critical for specificity and activity
  • Looking at 3-D structure for selectivity and specificty
  • Oligomerization of ion channels – implications for tissue specificity
  • Designing expression systems that produce truly representative channels for screening

Heike Wulff, Ph.D., Assistant Professor, University of California – Davis

(SC3) Advances in DNA Methylation Analysis
DNA methylation refers to the addition of methyl groups catalyzed by the enzyme, DNA methyltransferase to cytosines in CpG dinucleotides. This biochemical event has now come to be recognized as an important epigenetic phenomenon that affects gene expression. In recent years this field has been an area of intense activity and several new methodologies and applications for DNA methylation analysis have rapidly emerged. This course will cover the basics underlying the various techniques that have been developed and the context in which they have been successfully used. The advantages and limitations of the various methodologies will be discussed and appropriate case studies will be presented to demonstrate their use in biological, medical or epidemiological studies. The course will be offered in an informal, interactive setting to enable a free exchange of ideas and information.

8:30 am           Pre-Conference Short Course Registration

9:00     Daniel Weisenberger, Ph.D., Laboratory Manager, Epigenome Center, University of Southern California

Techniques for DNA Methylation

  • Overview of DNA methylation in mammals
  • Methods for analyzing DNA methylation such as, bisulfite-based detection methods including bisulfite genomic DNA sequencing, MSP, MethyLight (qMSP), Illumina GoldenGate, Illumina Infinium platforms, as   well as next generation sequencing approaches to better understand the DNA methylome.
  • Comparison of chemistries and platforms
  • Pros and cons of the various techniques

9:35     Christoph Bock, Ph.D., Junior Group Leader for Computational Epigenetics, Max Planck Institute for Informatics

Bioinformatic Methods for DNA Methylation Analysis and Epigenetic Biomarker Discovery

  • Challenges in epigenetic data analysis
  • Overview of powerful and user-friendly bioinformatics methods
  • Genome-wide annotation and prediction of DNA methylation
  • Discovery and validation of DNA methylation biomarkers for cancer diagnosis

10:10   Shannon Payne, Ph.D., Senior Scientist, Epigenomics Inc.

Clinical Applications of DNA Methylation

  • Genome-wide screening for DNA methylation changes
  • Overview of emerging applications of DNA methylation
  • Learnings from how and when to use the various methodologies
  • Factors affecting sensitivity and specificity

10:45 – 11:00 Networking Refreshment Break

11:00 – 11:45  Open discussion

11:45 – 12:00  Summary of key takeaways by the tutors

12:00               Course Adjourns

*separate registration required

Afternoon Short Courses

2:00pm - 5:00pm

(SC4) Tackling RNAi Delivery
The course is designed to provide both the beginner and the expert, with a comprehensive overview of current and emerging delivery systems and formulations facilitating RNAi delivery. Various methods for RNAi delivery such as viral vectors, liposomes, nanoparticles, chemical conjugates and other commonly used techniques will be compared and contrasted for different applications. The instructors will also discuss the challenges associated with the delivery of a wide array of RNA molecules such as siRNAs, shRNAs, aptamers and miRNAs and offer guidance based on their knowledge and expertise in the fi eld. The course is offered in an informal and interactive setting to enable free exchange of ideas and information.

1:30 pm           Pre-Conference Short Course Registration

 2:00     Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals

 Improved Delivery by Chemical Modifications

  • Overview of the siRNA delivery landscape and inherent challenges
  • Methods for improving delivery and potency
  • Chemical modifications of proven value
  • Chemically conjugated siRNAs
  • Liposomes
  • Lipoplexes and oligoplexes

 2:35     Ian MacLachlan, Ph.D., Chief Scientific Officer, Tekmira Pharmaceuticals Inc.

 Improved Delivery by Formulation Design

  • Modular delivery solutions
  • Delivery to the target cell, intracellular uptake and escape
  • Pre-clinical pharmacology
  • Immunological considerations

 3:10     Speaker to be announced

 Improved Delivery Using Viral Vectors

  • Factors limiting vector efficacy
  • Testing and predicting off-target effects
  • Vector dose and delivery route
  • Learnings from gene therapy

 3:45 – 4:00      Networking Refreshment Break

 4:00 – 4:45      Open discussion

 4:45 – 5:00      Summary of key takeaways by the tutors

 5:00                 Course Adjourns

(SC5) Ion Channel Assays for Safety Screening
Ion channels are involved in a complex and intricate signaling system that play an important role in affecting the cellular response to a drug and hence to the overall patient safety. For instance, the hERG potassium channel plays an important role in repolarization of cardiac myocytes and other sodium and calcium channels also control of ionic current fl ow in various cells. Drug-induced alterations in the translation and traffi cking of the ion channel proteins and drug-induced blockade of channels resulting in reductions in ionic current are all thought be contribute to drug-related adverse events. in vitro assays using isolated cells, cell lines, and expression systems cloned for specifi c ion channels are now routinely used to predict the drug response. Electrophysiology experiments, using conscious or anaesthetized animals, are also conducted to identify potential drug liabilities. Over the years there have been significant improvements in both the technology and in the scientifi c understanding of how ion channels can impact drug safety. This course provides a detailed overview of the types of ion channelbased screening assays and technologies that are in use and how they are being applied to effectively monitor and predict drug safety.

1:30 pm           Pre-Conference Short Course Registration

2:00     Harry Witchel, Ph.D., Senior Lecturer in Physiology, Brighton and Sussex Medical School

Cardiac Ion Channels and Their Role in Drug-Induced Toxicity

  • General overview: cardiac ion channels: relation to ventricular repolarisation, QT interval and conduction
  • The importance of hERG : relevance  to arrhythmias, TdP and the story of Terfenadine and Verapamil
  • Role of hERG’s structure in drug toxicity: channel promiscuity
  • Exceptions to the scheme: Relation between hERG and clinical outcomes: QT prolongation, ventricular arrhythmia and Torsades de Pointes
  • Early detection: methods, criteria, risks

2:35     Laszlo Urban, M.D., Ph.D., Executive Director, Global Head, Preclinical Safety Profiling, Novartis Institutes for Biomedical Research Inc.

Addressing cardiac safety in drug discovery (up to clinical candidate)

  • Strategy and logistics at lead prioritization and lead selection: predictive value, ROI
  • Addressing hERG channel promiscuity:  SAR, effects of physicochemical properties
  • hERG inhibition, trafficking, co-factors
  • Integrative risk assessment for cardiac safety

3:10     Huabin Sun, M.D., Senior Research Investigator, Cardiovascular Safety Pharmacology, Discovery Toxicology, Bristol-Myers Squibb

Ex vivo and in vivo assays for assessing off-target ion channel liabilities

  • Overview of pre-clinical safety testing focusing on ex-vivo and in-vivo models for off-target ion channel assessment and relevance to human diseases
  • Mainly focusing on sodium/calcium channel inhibition: clinical significance, ex-vivo and in-vivo testing strategies for screening
  • Cardiovascular safety pharmacology case studies

3:45 – 4:00      Networking Refreshment Break

4:00 – 4:45      Open discussion

4:45 – 5:00      Summary of key takeaways by the tutors

5:00                 Course Adjourns

(SC6) Screening For Potential Drug Targets - Design Strategies for Novel-Generation Kinase Inhibitors
The structural coverage of the human kinome has signifi cantly increased in the last few years. This course will provide useful insights and information on how fragment-based design offers an opportunity for assembling novel inhibitors, monitoring their binding kinetics and improving the residence time. Specific focus on screening technologies such as X-rays will be given.
Topics to be covered:

  • Fragment-Based Inhibitor Assembly
  • Monitoring Binding Kinetics
  • Aiming at long residence time on target
  • Privileged Fragments with cross-target promiscuity within the kinase family fragments with front-loaded bindng kinetics characteristics


2pm Introduction
Gerhard Müller, Ph.D., Chief Scientifi c Offi cer, Proteros Fragments, GmbH

2:10 Binding Kinetics in Drug Discovery
Gerhard Müller, Ph.D., Chief Scientifi c Offi cer, Proteros Fragments, GmbH

2:55 Design Principles of Novel Kinase Inhibitors
Peter Sennhenn, Ph.D., Director,  Med Chem, Proteros fragments, GmbH

3:40 – 4:00 Refreshment Break

4:00 Kinase Inhibitor – directed Chemistry and Libraries
Frank Leemhuis, Ph.D., Managing Director, MercaChem

4:45 Fragments, Kinetics and Kinase Inhibitors
Gerhard Müller, Ph.D., Chief Scientifi c Offi cer, Proteros Fragments GmbH

 5:00 End of Course

*separate registration required


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