(SC2) RNAi for Beginners
Presenters: Queta K.F. Smith, Ph.D., Associate Director, Technical Communications, Thermo Fisher Scientific, Inc.
Christophe J. Echeverri, Ph.D., Chief Executive Officer & Chief Scientific Offi cer, Cenix BioScience GmbH
Ian MacLachlan, Ph.D., Chief Scientific Officer, Protiva Biotherapeutics Inc.
Kevin V. Morris, Ph.D., Department of Molecular Medicine, The Scripps Research Institute
Overview:

• Overall Introduction to RNAi Technology

• From HT-RNAi Screens to in Vivo RNAi

• in Vivo Delivery

• Using Small RNAs to Direct Long-Term Stable Gene Silencing

Who Should Attend: Anyone who has started to use RNAi technology or has begun using the technology and wants to discuss issues and brainstorm with industry and academic leaders should attend this course for beginners.

(SC3) Integrated Genetic Association Analyses Using Bestof-Breed Tools and Data Resources: A Hands-on Workshop Introducing the Genetics Toolkit for the Galaxy Genomic Workbench
Presenters: Ross Lazarus, Ph.D., Anton Nekrutenko, Ph.D., and James Taylor, Ph.D., Channing Laboratory, Brigham & Women’s Hospital, Harvard Medical School
Overview:

• Translational genomic research road-blocks and solutions

• Introduction to the Galaxy framework

• Genotype data quality assessment and data fi ltering tools

• Statistical genetics analysis tools

• Genomic tools for prioritizing candidate markers

At the completion of the 3-hour workshop, participants will be familiar with the main features of the Galaxy genomic workbench. Participants will have performed a genotype quality control analysis and data cleaning, a genetic association case-control analysis, a genetic association continuous trait analysis, and a genetic association sample ancestry analysis. Participants will have used Galaxy tools for integrating data from the UCSC and other public data repositories with the results of statistical genetic analyses.

Who Should Attend?
Biologists and bioinformaticians involved in genetic disease association and pharmacogenetic research, including clinicians, genetic researchers, computer scientists and statisticians.

(SC4) Epigenetics—Unraveling the Secrets Beyond Genes
Epigenetics offers the possibility to alter our genetic destiny by controlling our essential molecules without changing the DNA sequence. Through Epigenetics’ control of genes, chromatic structure can be modified and genes can be silenced (turned off). The epigenome can be ‘unlocked’ to reveal the causes of disease and the complexity of humans’ development. This workshop will address how this new-found control can be used to detect disease, predict drug response, and create epigenetic therapies.

DNA Methylation Biomarkers for Cancer Detection and Drug Response Prediction
Christina Dahlstroem, Ph.D., Senior Vice President, Biomarker Solutions, Epigenomics, Inc.
The presentation will cover results from Epigenomics’ programs in early cancer detection in remote body fluids for colon, prostate and lung cancers. In addition, I will address Epigenomics’ differential methylation hybridization (DMH) microarray for discovery of predictive response and prognostic biomarkers in tissues. I will also discuss the use of DMH with cell lines that are sensitive and resistant to various drugs.

From Traditionnal to Novel IP Methods: Rapid LowCell# ChIPs and Methyl DNA IPs
Juana Magdalena, Ph.D., R&D Epigenetics Manager, Diagenode sa
We present here the very robust quality control (QC) that we established for our antibodies, which are mainly directed against targets relevant to the Epigenetics field such as modified histones, modifying enzymes and chromatin-interacting proteins. We first design immunogenic peptides in order to produce polyclonal antibodies directed against the target of interest. Both crude sera and purified antibodies are submitted to a similar step by step QC: I. Following immunizations, the rabbit crude sera is tested for immune response. Antibodies from crude sera will be affinity purified, tested in ELISA before and after purification. II. Whether or not the antibody is specific is determined during characterization (by Western Blot, Immunofluorescence and Dot Blot when applicable). III. Then specific antibodies are tested in ChIP. Our goal is also to characterize each antibody batch with an established QC and supply researchers with validated antibodies in a reproducible manner.

(SC5) Basics of RNAi Delivery
Instructors: Mark A. Kay M.D., Ph.D., Professor, Departments of Pediatrics and Genetics, Stanford University
John Rossi, Ph.D., Professor and Chair, Molecular Biology, Beckman Research Institute of the City of Hope
Muthiah Manoharan Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals
Roger Adami, Ph.D., Senior Research Scientist, Molecular Pharmaceutics, Nastech Pharmaceutical Company Inc.
The course is designed to provide both the beginner and the expert, an overview of the molecular mechanisms and recent technical advances for facilitating RNAi delivery. The instructors will discuss the challenges associated with the delivery of a wide array of RNA molecules such as siRNAs, shRNAs, aptamers and miRNAs and offer practical advice gained from their experience and expertise in the field. The course is offered in an informal and interactive setting to enable free exchange of ideas and information.

Topics to be covered:

• Overview of viral and non-viral vector systems

• Testing and validating methods for delivery

• The biochemistry of siRNA selection into RISC

• Design parameters for Dicer substrate siRNAs

• Aptamer siRNA conjugates for siRNA delivery

• Expression strategies for shRNAs/miRNAs

• Chemical methods of improving delivery

• Chemical conjugates and complexes

• Formulation approaches to deliver siRNAs

• Improving efficiency and reproducibility while minimizing cytotoxicity and off-target effects

(SC6) Tools to Therapies Forum

Join key thought leaders to exchange ideas and address the unique opportunities that arise from the convergence of genomic sciences, healthcare, and consumers in a unique interactive forum addressing:

• What does it take to transform the promise of an exciting tool into the reality of a therapy?

• Transforming genetic markers into meaningful genetic tests?

• How to finance genetic medicine?

• What are the new opportunities and strategies for direct consumer health?

Who should attend?
Decision makers and experts in licensing, alliance management, business development, strategic planning, technology evaluation, technology transfer, and portfolio management; capital funding firms including venture capitalists, public & private investors, angel investors, government funding agencies, corporate governance, incubators, and technology transfer centers; entrepreneurs and executives from start-up, early stage, established small to large biotechs, pharmaceutical companies and related companies; organizations interested in potential partners or to expand their technology portfolio; lawyers, exploring the legal issues, ethicists, looking at the "bigger" picture for society; and scientists who will be the developers as well as the consumers.

Speakers:
DNA Perspectives – An Independent Rating System for Genetics
Ryan Phelan, Ph.D., CEO & Founder, DNA Direct

IP and Creating Partnerships
Kristie Prinz, J.D., Founder, The Prinz Law Office

Funding
Gregory Bonfiglio, J.D., Managing Partner, Proteus Ventures, VC