Pre Conference Short Courses - November 1, 2009

Sunday, November 1

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12:00 pm – 3:00 pm

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Strategies for Effective RNAi Screens (SC1)

This course is designed to provide in-depth information on how to go about setting up RNAi screening experiments and how to design assays for getting optimal results. The challenges working with different types of molecules i.e. siRNAs, shRNAs and the delivery systems to get them into the appropriate cells and tissues will be discussed. The instructors will also provide their input on best practices for the execution of experiments and interpretation of results when dealing with complex biology and informatics.

Topics to be covered:
Overview of assay designs for siRNA and shRNA screens
Testing and validating methods for RNA delivery
Use of appropriate reagents and cell types
Implementing proper quality control
Minimizing cytotoxicity and off-target effects
Data analysis and statistical considerations

Instructor(s):

Karol Kozak, Database Handling Unit, High Content Screening, LMC-RISC, ETH Zurich

Caroline Shamu, Ph.D., Director, ICCB-Longwood Screening Facility, Harvard Medical School

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Targeting GPCRs and Ion Channels with Antibodies (SC2)

This course is designed to be interactive and provide an in-depth update on the potentials and challenges of developing mAB candidates as therapeutics for conditions involving membrane proteins.

Topics to be covered:
Pros and cons of targeting multi-spanning membrane proteins with antibody approaches
Basic screening technologies for mAB against multi-spanners
GPCR challenges such as expression level, post-translational modifications and oligomerization
Ion channel specific methodologies
Optimizing leads
Case studies of clinical candidates

Instructor(s):

Tajib A. Mirzabekov, Ph.D., President & Chief Executive Officer, MSM Protein Technologies, Inc.

 

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3:30 pm – 6:30 pm

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Strategies for Optimizing RNAi Delivery (SC3)

This 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 RNAi delivery, both in vitro and in vivo, and offer guidance based on their knowledge and expertise in the field.

Topics to be covered:
Testing and validating methods for delivery
Overview of viral and non-viral vector systems
Chemical methods of improving delivery
New formulations to optimize delivery
Minimizing cytotoxicity and off-target effects
Examining tissue distribution and safety profiles

Instructor(s):

Roger Adami, Ph.D., Associate Director, Molecular Pharmaceutics, MDRNA, Inc.

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Combating Diabetes with Strategies for Enhanced Pancreatic Beta Cell Survival and Regeneration (SC4)

Topics to be covered:

Factors involved in islet neogenesis
Stimulating adult cell regeneration
Preventing b-cell death
Monitoring b-cells in animal models and evaluating their predictability to the clinic
Incretins and b-cell action

Instructor(s):

Jesper Gromada, Ph.D., Executive Director, Cardiovascular Metabolic Disease Area, Novartis Institutes for BioMedical Research, Inc.

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Structure-Based Design of Ion Channels (SC5)

Topics to be covered:

Overview of current technologies
Molecular modeling and simulation models
Human vs animal channels – how comparable are they?
Crystal structure of potassium channels, voltage gaited channels and sodium channels – different channels – different approaches?
High-throughput screening of ion channel inhibitors

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Cardiovascular Safety in Drug Development – From Preclinical to Phase I (SC6)

Topics to be covered:

Part One: Why is it important to deal with cardiovascular safety in an early stage?
Part Two: Preclinical Screening and Arrhythmia Assays
Part Three: How to use early data to impact Phase I program

Instructor(s):

Philip Sager, M.D., FACC, FAHA, FHRS, Chief Medical Officer, CardioDx, Inc.

Garry Gintant, Ph.D., Senior Group Leader, Integrative Pharmacology, Abbott Laboratories

*Separate Registration Required