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CHI's Beyond Genome: Tools to Therapies

Beyond Genome: Tools to Therapies

You’ve Sequenced the Genome – Now Use IT!

Fairmont Hotel, San Francisco, CA



RNAi for Target Validation

How to Best Utilize RNAi Screening Technologies for Target Discovery

June 9-10, 2008


As new research unravel the complex cellular pathways of RNA interference (RNAi), new technology platforms are being developed to exploit those RNAi-related mechanisms for use in early drug discovery. Drug discovery units in most companies now routinely use RNAi screening for identifying and validating potential drug targets in various therapeutic areas. The screens themselves, using small interfering RNA (siRNA) or short hairpin (shRNA) libraries, have become more sensitive, high-throughput and large-scale. However, is RNAi screening being done in a manner that’s most efficient and effective in generating valid drug targets? Can there be further improvements in assay design and experimental protocols to minimize off-target effects and obtain reliable data? CHI’s sixth annual RNAi for Target Validation meeting will bring together experts in the field to delve into the details of RNAi screening technologies and its use for target validation.

Topics to be covered include:


  • High-throughput and high-content in vitro siRNA and shRNA screens
  • In vivo siRNA and shRNA Screening Techniques
  • Best Practices in efficient use and design of siRNA and shRNA libraries 
  • Latest in RNAi delivery techniques
  • Approaches to minimizing off-target effects
  • Update on emerging biology in RNAi-related cellular mechanisms



RNAi-Based Therapeutics

Strategies for Effective Ttransition from the Lab to the Clinic

June 10-11, 2008


RNA interference (RNAi) has been a continually evolving area of research. Discovered primarily as a technique for gene silencing, RNAi has now emerged as a screening tool for target identification and validation in early drug discovery. Since establishing its presence as a screening tool, RNAi is now looking to make an impact in the therapeutic arena. Several RNAi-based compounds are already in various stages of clinical testing. Although still in its infancy, RNAi therapeutics are making rapid progress in the clinic, learning from mistakes made in the past by other gene therapy approaches. CHI’s sixth annual RNAi-Based Therapeutics meeting will discuss ways to overcome challenges relating to drug delivery, specificity and safety for RNAi-based therapeutics.

Topics to be covered include:


  • Case studies: Getting from the bench to the clinic
  • Progress report on RNAi therapeutics in clinical testing
  • Approaches for targeted RNAi delivery
  • Overcoming concerns around drug safety and specificity
  • Challenges related to scaling up from the lab to the clinic
  • Update on emerging biology in RNAi-related cellular mechanisms
  • Overview of the licensing, legal and intellectual property landscape



Genotyping and Large Scale Association Studies

The Impact of Copy Number and Insertion/Deletions on Disease Progression

June 9-10, 2008


Genotyping studies can provide important insight into the genetic causes for disease

such as breast cancer, heart disease and diabetes. Large scale efforts are underway

to collect DNA for the purpose of understanding an individual ’s risk of developing a

disease, and provide the basis for selecting the most appropriate treatment. This

collaborative area holds many new technologies for identifying genotypes as well as

associating genes with disease. Plan to take part and learn what the buzz is about

in  genotyping area, with topics covering:


  • Technologies for genotyping
  • Analysis of genotyping data
  • Large-scale population studies
  • Identification of genetic markers
  • Clinical application of genotyping and correlation with treatment selection and response
  • Genome-wide association studies
  • SNP linkage analysis vs. microsatellite markers
  • Linking genetic profiles with clinical data



Progression of Personal Genomics

Kit Development for Consumer Genomics

June 10-11, 2008


Continuing advances in genetic research combined with the Internet’s role in empowering individual’s personal health education are poised to revolutionize the healthcare industry. Kits utilizing DNA testing are obvious examples of how genetic technology can be applied to improve patient care. Proponents argue that putting personalized medical information directly into the hands of individuals allow for informed choices about their health. Skeptics point out that although genetic variation is linked to complex disease little is known about the interplay of genetic and non-genetic factors such as diet, exercise, smoking and pollution that also affect a person's risk for disease.  CHI’s Inaugural Progression of Personal Genomics: Kit Development for Consumer Genomics addresses:

•            Consumer education and contribution

•           Sound science-based innovations

•           Ethical, privacy, and reimbursement issues

•            Regulatory requirements

•            Strategies for consumer health opportunities


Join key thought leaders to exchange ideas and address the unique opportunities that arise from the convergence of genomic sciences, healthcare, and consumers.


Targeting Gene Therapy

Determining Targets and Optimizing Delivery

Therapeutic Modalities

Translation from Basic Research to Clinical Results

June 9-11, 2008


  • Potential targets – innumerable
  • Promising treatments – attainable
  • Plenty of Challenges – surmountable


The concept of gene therapy has long appealed to biomedical researchers and clinicians because of the promise to treat certain diseases at their origin.  However, negative results and publicity have necessitated a low profile for gene therapy research.  Researchers are, in fact, quietly making progress and are confident that gene transfer will be elevated from its current experimental status to a therapeutic modality.  Scientifically, major challenges remain: targeting the right gene to the right location in the right cells and expressing it at the right time, all while minimizing any adverse reactions.  In addition, collaborations between academia and industry are of critical importance for the future of gene therapy. Substantial knowledge as well as regulatory and financial resources is needed to move gene therapy from small-scale endeavors to clinical use.  CHI’s Inaugural Targeting Gene Therapy & Gene Transfer: Translation from Basic Research to Clinical Results assembles prominent scientific researchers, clinicians, businessmen, and regulators who represent wide areas of expertise to provide the forum for discussion for expanding the potential and promise of gene therapies.


10th Annual

Applying Systems Biology

Interrogate  Predict  Understand

June 9-11, 2008


The “Applying Systems Biology” meeting brings together leaders from around the world to present case studies about how Systems Biology is being ‘applied’ to achieve real-world solutions for battling critical targets, and improving drug discovery and development.


This year the meeting is featuring a comprehensive focus on “Collaboration” including international projects for modeling the immune system.  Along with data and project results, experts will address logistical issues in an interactive panel discussion addressing collaborating across areas of expertise, lingo and national borders.


The second half – “Abstraction” -- will look at the tools and technology that support this new age of science.  During “Abstraction,” the meeting will explore the journey from wet lab to dry lab in light of technological breakthroughs, including an indepth session focused on Gene Therapy.


Topics Include:


  • Drug Discovery & Development
  • Personalized Medicine
  • Cancer
  • The Immune System
  • Modeling
  • Pathways/Networks/ Systems
  • Gene Therapy
  • The Logistics of International Collaborative Projects
  • Unraveling Complexity




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