Document 6425299

Final Agenda
TARGETING GENE THERAPY
DETERMINING TARGETS
AND
OPTIMIZING DELIVERY
June 9-10, 2008
THERAPEUTIC MODALITIES
June 10-11, 2008
SESSIONS INCLUDE
KEYNOTE SPEAKERS
Andrea Califano, Ph.D., Professor, Director,
Columbia National Center for Biomedical
Computing, Associate Director, Herbert Irving
Comprehensive Cancer Center
Andrew Fire, Ph.D., Nobel Laureate and Professor,
Departments of Pathology and Genetics,
Stanford University School of Medicine
Linking Genes to Disease
Novel Delivery Systems
Mechanisms and Applications of Gene Repair
RNAi Delivery: A Therapeutic Challenge
Networks and Pathways
Hugh Young Rienhoff, Jr., M.D., Director,
MyDaughtersDNA.org
Eric J. Topol, M.D., Chief Academic Officer,
Scripps Health, Director of Scripps Translational
Science Institute
Case Studies
Register by March 14
and Save up to $350!
“You’ve Sequenced the Genome, Now Use IT”
Part of:
Applying Systems Biology
RNA Interference
Personal Medicine
June 8-11, 2008 • The Fairmont Hotel • San Francisco, CA
Corporate Support:
Targeting Gene Therapy
Lead Sponsoring Publications:
Cambridge Healthtech Institute
250 First Avenue, Suite 300
Needham, Massachusetts 02494
Telephone: 781-972-5400 or toll-free in the
U.S. 888-999-6288•Fax: 781-972-5425
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DETERMINING TARGETS
AND
OPTIMIZING DELIVERY
June 9-10, 2008
9:45
Sunday, June 8
SHORT COURSES*:
2:00 - 5:00pm
(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 Officer, 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
Why 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.
(SC4) Epigenetics—Unraveling the Secrets Beyond
Genes
TARGETING GENE THERAPY
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.
*Separate registration required.
Monday, June 9
7:30 am – 6:00 pm
Registration Open
7:30am
Morning Coffee
LINKING GENES TO DISEASE
8:45
Charperson’s Opening Remarks
Keynote Presentation
9:00
Cardiovascular Implications of Genome-Wide Association Studies
Eric J. Topol, M.D., Chief Academic Officer, Scripps Health, Director of Scripps Translational Science Institute, Professor of Translational Genomics, TSRI, Senior Consultant,
Division of Cardiovascular Diseases
State of the art genome wide association studies in cardiovascular complex traits has
yielded extraordinary new insights, particularly in myocardial infarction, coronary
artery disease, atrial fibrillation, and lipoprotein disorders. Prominently the 9p21 variants not
only are associated with atherosclerotic coronary disease, but also abdominal aortic aneurysm
and intracranial “berry” aneurysms, which represent a non-atherosclerotic, vessel wall medial
defect. We now have a substantial number of new genes, genomic loci, and pathways that will be
remarkable important substrate to proceed with functional genomics and preventive strategies in
the future.
Industrial-Scale Genotyping: Identifying, Validating, and
Translating Association Findings
Dietrich Stephan, Ph.D., Director & Senior Investigator, Neurogenomics, Translational Genomics Institute
Dissecting the genetic variants that subtley predispose to common and complex human disease requires careful study design considerations, a robust high-throughput environment, and evolving analysis paradigms. If
done correctly, the results can routinely be expected to result in therpeutic targets with an order of magnitude
more precision than historical strategies, as well as probabalistic risk assessment tools.
10:15
Networking Coffee Break
10:45
Genome Scan of 310 African-American Families for Genes Linked
to Higher Risk of Diabetes and Cardiovascular Disease
Michael Christman, Ph.D., President and Chief Executive Officer, Coriell Institute for Medical
Research
Obesity, hypertension, type 2 diabetes and their complications are more common among African Americans
than European Americans. Collectively, these diseases explain over 80% of the health disparity between
Americans of African descent and Americans of Western European descent. The Howard University Family
Study was developed as a population-based resource of multi-generational African American pedigrees to
study the genetic epidemiology of these diseases. We have used the Affymetrix SNP 6.0 arrays combined with
a family-based association analysis to identify common genetic factors influencing heritability of these common diseases in African Americans.
11:15
Copy-Number Variation in Control Population Cohorts
Richard F. Wintle, Ph.D., Assistant Director, Center for Applied Genomics, Hospital for Sick Children
Copy-number variation (CNV) is the most prevalent type of variation with respect to total nucleotide content
in the human genome. In order to understand the contribution of CNV to both normal human variation and
disease susceptibility, it is crucial to understand the range and characterisics of CNV variability in healthy population cohorts. In our group, a variety of approaches, both laboratory-based and analytical, are being applied to
variousdifferent control cohort populations. Here, we will describe recent work using high-density arrays from the
major vendors to ascertain genome-wide copy number. We also describe the development of a novel algorithm
for the determination of genomic copy number from these array platforms, and the application of the Database of
Genomic Variants containing normal variation data to disease studies.
11:45
Structural Genomic Variation in the Human Genome: The Impact
of Copy Number Variants (Cnvs) in Clinical Diagnoses
Charles Lee, Ph.D., FACMG, Director of Cytogenetics, Harvard Cancer Center, Assistant
Professor, Harvard Medical School, Associate Faculty Member, MIT Broad Institute, and
Department of Pathology, Brigham and Women’s Hospital
Genomic imbalances were traditionally thought to be rare and disease causing. However, over the past three
years we have come to appreciate that structural genomic variation (including copy number variants – CNVs)
are widespread and many can be very common among healthy individuals. This has complicated accurate
interpretation of data being generated from genome-wide comparative genomic hybridization (CGH) / genotyping platforms being used for clinical diagnoses. Strategies to determine if a particular CNV is pathogenic
or benign will be discussed, in the context of our recent studies that define the fine-scale genomic architecture
of hundreds of common CNVs.
12:15 pm Close of Morning Session
12:30
Luncheon Technology Workshops (Sponsorships Available)
or Lunch on Your Own
NOVEL DELIVERY SYSTEMS
2:00
Chairperson’s Remarks
Michael Barry, Ph.D., Professor, Internal Medicine, Mayo Clinic
2:05
Ex vivo Evaluation of Efficacy and Toxicity of Gene Therapy
Vectors Using Organ Cultures of Human Solid Tissues
Amos Panet, Ph.D., Chairman, Virology, Hebrew University-Hadassah Medical School
We have developed a generic technology to evaluate gene therapy vectors and transgenes using organ cultures
derived form normal and diseased human tissues such as skin, carcinomas, lung, colon etc. Using this approach we determine the tropism of Adeno, Lenti and herpes viral vectors to solid tissues of human origin.
This information was applied to evaluate oncolytic viruses and for the development of the Biological pump
technology to supply hormones systemically.
2:35
Targeting and Detargeting Gene Therapy Vectors
Michael Barry, Ph.D., Professor, Internal Medicine, Mayo Clinic
Viral gene therapy vectors and oncolytics hold promise to treat genetic diseases and cancer, but are plagued
by a lack of cell specificity in vivo. To circumvent this limitation, we have utilized peptide-presenting phage
libraries to select cell-binding peptides to supply these viruses with new ligands to target the cells of interest.
One fundamental problem with this approach is that translation of ligands from the structural context of a
phage library into the differing structural context of a virus can fail due to loss of ligand binding or disruption
of viral protein function. Approaches to improve phage selection and to avoid this context problem will be
discussed.
3:05
Facilitated delivery of siRNA to the CNS: A Therapeutic Approach
for Stroke
Carol M. Troy, M.D., Ph.D., Associate Professor, Pathology, Columbia University
Ischemia is a major cause of morbidity and mortality but at present no adequate therapies exist. Research on
the mechanisms of neuronal death in ischemia has yielded potential targets for therapeutic intervention but,
as with other diseases of the central nervous system, development of therapies has been hindered by lack of
accessibility to the brain. Another quandary has been specificity of action of the therapeutic. We have developed a novel non-transfection based delivery of siRNA and peptides in vitro, which employs a transduction
peptide, Penetratin1 (Pen1) to deliver the cargo to neurons with 100% efficiency. Pilot studies show that we
can use this approach to deliver siRNA in vivo to the CNS. Studies are in progress to test the efficacy of this
approach in a rodent model of stroke.
3:35
Technology Spotlight (Sponsorship Available)
3:50
Networking Refreshment Break
4:15
AAV Mediated Tissue Specific Gene Expression
Hua Su, Ph.D, Assistant Professor, Medicine, Anesthesia and Preoperative, University of
California, San Francisco
Untargeted gene expression can result in some unwanted side effects caused by cytotoxicity of viral vectors
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or excessive transgene expression. Each AAV serotypes has its own receptors and thus infect different tissue
with different efficiency. With selected AAV serotype combined with hypoxia response element and cardiac
specific promoter, we have achieved targeted gene expression in ischemic myocardium. We have expressed
genes in ischemic brain with AAV mediated gene transfer.
4:45
Panel Discussion with Afternoon Speakers
5:15
Welcoming Reception in the Exhibit Hall
6:30
Close of Day
Tuesday, June 10
7:00 am – 6:00 pm Registration Open
7:30 am Breakfast Workshop (Sponsorships Available)
MECHANISMS AND APPLICATIONS OF
GENE REPAIR
8:15
Chairperson’s Remarks
Eric Kmiec, Ph.D., Professor, Biology, University of Delaware/OrphageniX
8:20
A New Frontier in Gene Therapy
Eric Kmiec, Ph.D., Professor, Biology, University of Delaware/OrphageniX
Over the past seven years, we have pioneered the technique of gene repair wherein genetic mutations are
corrected directly within context of the chromosome. This repair is facilitated by oligonucleotides , non-viral
drug-like agents that does not induce an immune reaction or cause toxicity in humans. A number of genetic
diseases including sickle cell anemia, spinal muscular atrophy and muscular dystrophy have been successfully
treated in cell and animal models. Now, the focus is translating these results into a clinical trial.
8:50
Lasting Effects by Transient Gene Transfer: Epigenetics
Casey Case, Ph.D., Vice President Research, SanBio Inc.
9:20
Sequence-Specific Modification of Genomic DNA by
Oligonucleotides
Dieter Gruenert, Ph.D., Professor, Senior Scientist, Cell Biology, California Pacific Medical Center
Research Institute
We have developed a strategy small fragment homologous replacement (SFHR) for modifying specific targets
in the genomic DNA using small DNA fragments (SDF). Studies in hematopoietic stem cells, lymphoblasts,
epithelial cells, and embryonic stem cells have shown SFHR-mediated modification. This approach has
potential therapeutic applications as well as in the development of transgenic animals.
9:50
Networking Coffee Break, Poster and Exhibit Viewing
10:45
Nucleic Acid Delivery and Gene Repair in the Eye
John M. Nickerson, Ph.D., Professor, Department of Ophthalmology, Emory University
Short single stranded oligonucleotides (ODNs) can be delivered into photoreceptor cells of the neural retina
in vivo. We used a mouse strain bearing the retinal degeneration (rd1) lesion, a point mutation in a gene
encoding the beta-subunit of cGMP phosphodiesterase (beta-PDE). Delivery of therapeutic ODNs to rd1
mouse eyes resulted in genomic DNA conversion from mutant to wild type sequence at a low but observable
incidence. Correspondingly, observable beta-PDE immunoreactivity was detected. Rhodopsin immunopositive cells were detectable in the outer layers of the retina, suggesting that ODN-directed gene repair occurred
in about 0.2% of cells.
11:15
Selected Brief Poster Presentation
Keynote Presentation
11:45
Small RNAs as Markers, Effectors, and Targets of Genetic Change
Andrew Fire, Ph.D., Nobel Laureate and Professor, Departments of Pathology and Genetics,
Stanford University School of Medicine
The “Small-RNA-ome” of a cell or tissue sample gives us a remarkable window into ongoing processes of
gene regulation that have biological, procedural, and clinical consequences. This talk will provide several
examples in which we hope that Small-RNA-ome determination combined with other analysis will
contribute to understanding of (and control over) critical biological processes.
June 10-11, 2008
12:00 pm Registration Open
RNAi DELIVERY: A THERAPEUTIC CHALLENGE
2:00
Chairperson’s Remarks
Mark A. Kay M.D., Ph.D., Professor, Departments of Pediatrics and Genetics, Stanford University
2:05
Systemic RNAi Therapeutics for Treating Infection
Mark A. Kay M.D., Ph.D., Professor, Departments of Pediatrics and Genetics, Stanford University
Gene transfer vectors expressing shRNAs to target specific tissues have been utilized for treating different diseases. Recombinant AAV vectors expressing shRNAs have been shown to be effective in reducing hepatitis
B viral replication in a transgenic mouse model. Interestingly, over expression of shRNAs can be toxic and
even lethal because it interferes with normal microRNA processing. The rate-limiting steps in mammalian
tissues as well as effective strategies to maintain a high therapeutic index will be discussed.
2:35
Delivering RNAi Therapeutics
Muthiah Manoharan Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals
3:05
Delivery of Therapeutic RNA Interference into the GI tract
Johannes Fruehauf, M.D., Ph.D., Vice President, Research, Cequent Pharmaceuticals Inc.
Cequent has developed a system that allows the delivery of therapeutic RNA interference into the gastrointestinal tract through oral application. This method, called Transkingdom RNA interference, (tkRNAi), uses
nonpathogenic bacteria that are modified to act as manufacturers and carrier vehicles of interfering RNA against
genes of interest. Activity has so far been shown across a wide range of targets. APCmin mice are a genetic model
of human colon cancer based on dysregulation of beta-catenin (CTNNB1) and the wnt pathway. They develop
multiple polyps in their gastrointestinal tract resulting in decreased life span due to chronic obstruction and
bleeding. Blockage of CTNNB1 in the gastrointestinal epithelium, e.g. through therapeutic RNA interference,
should result in therapeutic or preventive effects. Here we show, that chronic oral treatment of APCmin mice
(n=38) with tkRNAi bacteria resulted in a significant decrease of polyp formation through blockage of the
CTNNB1 pathway in the gut. Tumor sizes and numbers were reduced and animals displayed fewer polyps with
high grade dysplasia after oral treatment with tkRNAi bacteria conferring silencing against CTNNB1. These
findings open the possibility of developing RNAi-based drugs for organs and tissues outside of the areas targeted
by currently ongoing clinical trials, including the gastrointestinal tract, genitourinary tract, and the skin.
3:35
Technology Spotlight (Sponsorship Available)
3:50
Networking Refreshment Break, Poster and Exhibit Viewing
4:30
Construction of phi29 DNA-Packaging Motor for Applications in
Nanotechnology, Therapy, Diagnosis, and Drug Delivery
Peixuan Guo, Ph.D., Chair in Biomedical Engineering and Director of NIH Nanomedicine
Development Center, University of Cincinnati
Bacterial virus phi29 packaging RNA (pRNA) is an ATP-binding component of the DNA packaging motor.
pRNA contains aintermolecular interaction domain and a 5’/3’ helical domain. Its unique feature to form dimer,
trimer, hexamer and patterned superstructures via the interaction of two interlocking loops makes it a promising
tool in nanomedicine. Replacement or insertion of the 5’/3’helical domain with siRNA, ribozyme and receptorbinding aptamer or other therapeutic molecules does not interferer with the formation of the multimers, making
it a novel vehicle for targeted therapy, pathogen detection and drug delivery. The chimeric siRNA/pRNA
complex induced apoptosis in specific cancer cells, as tested in both cell culture and in animal trials. Such
protein-free nanoparticles as therapeutic reagents would allow repeated treatment for chronic diseases.
5:00
In vivo Imaging of siRNA Delivery and Silencing in Tumors
Anna Moore, Ph.D., Associate Professor, Department of Radiology and Director, Molecular
Imaging Laboratory, Massachusetts General Hospital
During the past years, RNAi has become an indispensable research instrument in virtually all fields of medical
and biological sciences. Its broad applicability (virtually any gene can be silenced), superior efficiency (1001000-fold compared to antisense oligonucleotides), and exquisite specificity (single nucleotide) could potentially
be used to develop a powerful novel treatment paradigm with global relevance to any disease amenable to
manipulation at the level of gene expression. The fast developing field of RNA interference requires monitoring of siRNA delivery to targeted organs and evaluating the efficiency of target gene silencing. Molecular
imaging techniques represent a powerful tool for real-time non-invasive monitoring of various events at a near
microscopic level and have superiour advantages over conventional in vitro and cell culture research techniques
in biology. Therefore, molecular imaging approach fits perfectly to fulfill the need to monitor siRNA delivery
and provides information in a fast, reproducible and non-invasive manner. This presentation will summarize the
existing information on various imaging modalities and their application for siRNA imaging.
5:30
Close of Day
SHORT COURSE*:
6:30 - 8:30pm
(SC5) Basics of RNAi Delivery
12:15 pm Close of Targeting Gene Therapy Conference
12:30
Luncheon Technology Workshops (Sponsorships Available)
Lunch on Your Own
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
PharmaceuticalCompany 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.
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TARGETING GENE THERAPY
Our cell therapy product is produced by transient transfection of Mesenchymal Stem Cells. The vector used
encodes the Notch-1 IntraCellular Domain (NICD) a powerful regulator of developmental cell fate. Transfection with this vector causes the cells to assume neuronal precursor-like properties and to lose the ability to differentiate down alternative paths. This beneficial effect persists long after the gene transfer vector is gone. We
are exploring epigenetic mechanisms, such as CpG DNA methylation, to explain this phenomenon. These
cells have shown beneficial results in models of stroke, Parkinson’s disease and spinal cord injury.
THERAPEUTIC MODALITIES
(SC5) Basics of RNAi Delivery (continued)
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
1:50
A Synthetic Gene Delivery System for IL-12: From Bench to Clinic
Khursheed Anwer, Ph.D., Vice President, Research & Development, Expression Genetics, Inc.
Registration Open
Facilitated Break-Out Discussion Groups and Morning Coffee
NETWORKS AND PATHWAYS
8:15
Chairperson’s Remarks
Keynote Presentation
TARGETING GENE THERAPY
8:20
Luncheon Technology Workshops (Sponsorships Available)
Lunch on Your Own
1:45
Chairperson’s Remarks
Geoff Symonds, Ph.D., Senior Research Director, Global Product Leader, HIV Gene Therapy,
Johnson & Johnson Research
Wednesday, June 11
7:00am
12:30
CASE STUDIES
*Separate registration required.
7:00 am - 4:00 pm
12:15 pm Close of Morning Session
From Molecular Interaction Networks to the Master Regulators
of Neoplastic Phenotypes: Cancer Systems Biology Comes of
Age
Andrea Califano, Ph.D., Professor, Director, Columbia National Center for Biomedical
Computing, Associate Director, Herbert Irving Comprehensive Cancer Center
The identification of genes acting synergistically as master regulators of physiologic and pathologic cellular phenotypes is still an open problem in systems biology and
there are no biochemically validated examples for human cells. Here we apply a systems biology approach
to identify the repertoire of transcription factors (TFs) that constitute the master regulation module
responsible for synergistic activation of a tumor-specific signature.
8:50
Statistical and Computational Pharmacogenetics: Detecting
Genes for Drug Response
Rongling Wu, Prof., University of Florida Research Foundation Professor, Statistics, University of
Florida
I will present a conceptual framework for computing genes and genome for drug response by integrating mathematical and chemical aspects of drug reactions in the body. With this framework, specific DNA sequence
variants can be identified on the basis of the test of a few parameters that define the shape and pattern of
drug responses, which thus enhances the precision of parameter estimation as well as biological and clinical
relevance in pharmacogenetic and pharmacogenomic research.
This presentation describes the discovery and development of a synthetic lipopolymer for gene delivery of
IL-12 for cancer. Synthesis, formulation, scale-up, stability, animal safety/toxicity, biodistribution and results
from two clinical trials in ovarian cancer patients will be discussed. Application for additional cancer indications will also be described briefly.
2:20
Gene Therapy Development using HIV as a Specific Example
Geoff Symonds, Ph.D., Senior Research Director, Global Product Leader, HIV Gene Therapy,
Johnson & Johnson Research
Gene Therapy represents a different treatment paradigm and the presentation will address the development
process within the setting of big Pharma using the specific example of Gene Therapy for HIV. Similarities
and differences to small molecule and biologics development will be discussed, as well as the means by which
Gene Therapy can be ‘incubated’ to a point that it can stand alone.
2:50
Evidence of Neuroregeneration using Vascular Endothelial Growth
Factor Zinc Finger Protein Activator (SB-509) in Patients with
Diabetic Neuropathy: A Chronic Degenerative Polyneuropathy
Ely Benaim, M.D., Vice President, Clinical Affairs, Sangamo BioSciences, Inc.
Twenty four patients were treated with either VEGF Zinc finger protein plasmid DNA(SB-509 n=12) or
placebo (n=12) at a single treatment and were followed for clinical neurologic improvement for 6 months.
There was a statistically significant 25% improvement in Quantitative Sensory Testing in the lower extremities. Motor and Sensory Nerve Conduction Velocities showed a trend for improvement in a clinically relevant
magnitude. This study provided the basis for two Phase 2 trials in mild to moderate and moderate to severe
Diabetic Neuropathy.
3:20
Networking Refreshment Break, Last Chance for Poster and
Exhibit Viewing
CLOSING PLENARY SESSION
4:00
Poster Awards in the Exhibit Hall
Plenary Keynote Presentation
4:15
My Daughter’s DNA: Networking the Dots for a Diagnosis
Hugh Young Rienhoff, Jr., M.D., Director,
MyDaughtersDNA.org
Photo Credit: Cody Pickens
9:20
Clotting, Cascades, and Computers - Systems Biology in
Personalized Medicine
Michael Roehrl, M.D., Ph.D., Pathology and Laboratory Medicine, Massachusetts General
Hospital
The human blood clotting system is a complex and highly regulated network of biomolecular interactions. We demonstrate in this talk how data from careful biochemical measurements can be integrated into
quantitative and predictive computational models of blood coagulation. Millions of patients receive the
oral anticoagulant Coumadin to prevent fatal thromboembolic events. Yet personalized Coumadin dosing is
both cumbersome and expensive (requiring frequent blood draws and lab testing) and potentially dangerous.
Coumadin is among the top 10 drugs with the largest number of serious adverse event reports submitted to
the FDA. We show how a novel Systems Biological approach can be used in the clinical setting to personalize
Coumadin dosing and to achieve safe therapeutic goals.
4:45
Closing Panel Discussion:
Collaboration Across Areas of Expertise
Neoplastic transformation and progression is driven by deregulated cellular pathways that control cell fate,
growth, differentiation and survival. Although significant progress has been made to identify and characterize
oncogenes, tumor suppressors and the molecular pathways that they regulate, it remains largely unclear what
pathways play a critical role in the development of different tumor types. Post-genomic era technology in
gene expression profiling has provided a powerful tool to study gene regulations in cancers at the molecular
level. In this study, we developed and applied a novel approach to derive gene signatures for cancer prognosis
in the context of known biological pathways.
Increasingly, advances in the post-genomic era draw upon multiple areas of expertise. Melting
silos of jargon, perspectives, and modus operandi is essential in order to achieve significant
progress in the quest to conquer disease and fully understand biological forms. Melting egos may
also play a part in working together toward a common goal. As collaboration grows ever more
ubiquitous in the life sciences, its challenges are encountered more frequently. This panel discussion will focus on how to overcome some of the inherent problems that arise in collaborations,
including academic/industry projects and international teams. Basic logistical issues will also be
addressed.
•
How to function across barriers of time, space, and language
•
How to set up efficient teams – structure of collaboration
•
Advantages/disadvantages of collaboration
•
Differences between academic and industry perspectives
•
Building respect into multi-cultural teams
•
Communicating in-between multiple areas of expertise
•
Outlook of collaboration in the life sciences
11:15
5:30
9:50
Networking Coffee Break, Poster and Exhibit Viewing
10:45
Identify Pathway Specific Gene Signatures for Cancer Prognosis
using Gene Expression Profiling Data
Dan Li, Ph.D., Principle Research Scientist, Informatics, Integrative Biology, Eli Lilly and Company
Genome-wide Transcriptional Fingerprinting of Hepatotoxicity
Regulatory Networks Using Multiplex Parallel High Throughput
ChIP-on-Chip Assays
Jeff Falk, Ph.D., Director, Technology Applications, Molecular Biology, Aviva Systems Biology
A genomewide transcription factor mapping consortium is currently being assembled to facilitate the
dissection of key disease and toxicology-related regulatory networks. The consortium will facilitate global
identification of key toxicity and disease-related networks and biomarkers by providing reference fingerprints
of transcription factor-mediated pathway modulations in key tissues that can then be compared with similar
profiles derived from disease-related or therapeutic compound treated samples. We will describe the initial
phase of experiments utilizing our next generation ChIP-on-chip technology for mapping of transcriptional
networks that pinpoint critical pathways and biomarkers associated with hepatotoxicity.
11:45
Systems Biology of Melanoma
William Kaufmann, Ph.D., Professor, Pathology and Laboratory Medicine, University of North
Carolina, Chapel Hill
I propose to describe a model of human carcinogenesis that is based upon the interaction of an external stress
(sunlight) with mutations in the MAPK signaling pathway in melanocytes to cause deletions in the CDKN2A tumor suppressor locus that encodes p16INK4A and ARF. Systems biology approaches to the model
include generation of genetic and physical interaction networks to model the DNA damage response, global
analysis of gene expression to identify melanoma subtypes, and mathematical models of nucleotide excision
repair and G2 DNA damage checkpoint responses.
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Close of Conference
Contact: Jon Stroup, Manager, Business Development
[email protected] • 781-972-5483
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Phone: 415-772-5000
Fax: 415-772-5013
PRESENT A POSTER AND SAVE $50
Discounted Room Rate: $239 s/d
Reduced Room Rate Cutoff Date: May 13, 2008
To reserve your hotel room, please call the hotel directly to make
your room reservation. Identify yourself as a Cambridge Healthtech
Institute conference attendee to receive the reduced room rate.
Reservations made after the cut-off date or after the group room block
has been filled (whichever comes first) will be accepted on a spaceand-rate-availability basis. Rooms are limited, so please book early.
TRAVEL INFORMATION
FLIGHT DISCOUNTS:
To receive a 5% discount on American Airlines, American Eagle and
American Connections call and make your flight reservations at
1-800-433-1790 or go online at aa.com. Please refer to the authorization
number AN# A2418SS via phone or enter it in the promotion discount
box online.
CAR RENTAL INFORMATION:
Special discount rentals have been established with AVIS for this
conference. Call AVIS directly at 800-331-1600 and reference the Avis
Worldwide Discount (AWD) Number J868190.
5
BeyondGenome.com
Reasons You Should Present Your Research Poster
at Beyond Genome:
•
•
•
•
Your poster will be exposed to over 600 delegates
Receive $50 off your registration fee
Your poster abstract will be published on our conference CD
Your research will be seen by leaders from top pharmaceutical,
biotech, academic and government institutes
•
Your poster abstract will be published on our conference CD
•
Poster competition with cash prizes
Please submit your abstract and register for the meeting.
To secure a poster board and inclusion in the conference CD, your abstract
must be submitted, accepted and registration paid in full by May 5, 2008.
Maximize your experience onsite at Beyond Genome!
The Intro-Net offers you the opportunity to set up meetings with
selected attendees before, during and after this conference,
allowing you to connect to the key people you want to meet.
This online system was designed with your privacy in mind and is
only available to registered session attendees of
this event.
Registered conference attendees will receive
more information on accessing the Intro-Net in
the weeks leading up to the event!
REGISTRATION INFORMATION:
REGISTER 3 — 4th IS FREE
❒ Mr. ❒ Ms. ❒ Mrs. ❒ Dr. ❒ Prof.
Individuals must register for the same conference or conference
combination and submit completed registration forms together for
discount to apply. Please reproduce this registration form as needed.
86508 F
Name
Job Title
Div./Dept.
Company
Address
City/State/Postal Code
June 9-11, 2008 • The F a i r m o n t H o t e l
San Francisco, CA
Country
Telephone
Would you like to receive event updates via fax?
❒ Yes
❒ No
Fax
Email*
*Email is not a mandatory field. However, by excluding your email you will not receive notification about online access to pre-conference presenter materials, conference
updates, networking opportunities and requested eNewsletters.
PRICING INFORMATION:
SHORT COURSE S :
Commercial
Academic, Government, Hospital-Affiliated
One Short Course
Two Short Courses
❒ $595
❒ $895
❒ $295
❒ $495
Choose the short course(s) you will most likely attend. Please select ONLY ONE for each conference day.
June 8 (2:00 - 5:00pm)
❒ (SC2) Intro to RNAi
❒ (SC3) Genetic Association Analyses
❒ (SC4) Epigenetics
PREMIUM-Attend the ENTIRE
June 10 (6:30 - 8:30 pm)
❒ (SC5) RNAi Delivery
❒ (SC6) Tools to Therapies
Commercial
Academic, Government, Hospital-Affiliated
❒ $1645
❒ $795
PRESENT A POSTER AND SAVE $50
Cambridge Healthtech Institute encourages attendees to gain further exposure by presenting
their work in the poster sessions. To secure a
poster board and inclusion in the conference CD,
your abstract must be submitted, accepted and
registration paid in full by May 5, 2008. Register
online to use the Poster Abstract Submission form
or, if you register by phone,fax, or mail, you will
receive Poster Abstract Submission guidelines via
email. I am interested in presenting a poster at:
❒ Beyond Genome
and will submit a completed one-page abstract by
May 5, 2008 (Please Note: Registration must be paid in
full to present poster.)
Title
Beyond Genome Event (June 9-11)
Early Registration until March 14, 2008
Best Value
Advanced Registration until April 25, 2008
❒ $1795
❒ $875
Registration after April 25, 2008 and On-site
❒ $1995
❒ $945
Yes! I would like to receive a
FREE eNewsletter subscription to:
❒
The latest industry news, commentary
and highlights from Bio•IT World
Choose the conference COMBINATION you will most likely attend. Please select ONE from EACH option:
Option 1: (June 9-10)
Option 2: (June 10-11)
AND
❒ Systems Biology – Collaboration
❒ Systems Biology – Abstraction
❒ RNAi for Target Validation
❒ RNAi-Based Therapeutics
❒ Gene Therapy – Targets and Delivery
❒ Gene Therapy – Therapeutic Modalities
❒ Genotyping and Large Scale Association Studies
❒ Progression of Personal Genomics
STANDARD Attend one conference ONLY (1.5 Days)
Commercial
Academic, Government, Hospital-Affiliated
Early Registration until March 14, 2008
❒ $1195
❒ $575
Advanced Registration until April 25, 2008
❒ $1245
❒ $645
Registration after April 25, 2008 and On-site
❒ $1495
❒ $775
Please Choose the SINGLE conference you will most likely attend:
June 9-10
OR
June 10-11
❒ Systems Biology – Collaboration
❒ Systems Biology – Abstraction
❒ RNAi for Target Validation
❒ RNAi-Based Therapeutics
❒ Gene Therapy – Targets and Delivery
❒ Gene Therapy – Therapeutic Modalities
❒ Genotyping and Large Scale Association Studies
❒ Progression of Personal Genomics
Poster Discount
❒ $50
❒ ISCB Member Discount
❒ 10% OFF
❒ I cannot attend but would like to purchase the Beyond Genome CD for $750 (plus shipping). Massachusetts delivery will include 5% sales tax.
❒ Please send information on exhibiting and opportunities to present workshops.
PAYMENT INFORMATION:
❒ Enclosed is a check or money order payable to Cambridge Healthtech Institute, drawn on a U.S. bank, in U.S. currency.
❒ Invoice me, but reserve my space with credit card information listed below.
Invoices
unpaid
two weeks
prior
to conference will be billed to credit card at full registration rate. Invoices must be paid in full and checks
Please
refer to
the Registration
Code
below:
received by the deadline date to retain registration discount. If you plan to register on site, please check with CHI beforehand for space
availability.
❒ Please charge: ❒ AMEX (15 digits) ❒ Visa (13-16 digits) ❒ MasterCard (16 digits) ❒ Diners Club (14 digits)
Card #
Cardholder
Signature
Cardholder’s Address (if different from above)
City/State/Postal Code
Country
Exp. Date
❒
The premier e-news source on
technology for healthcare
❒
Tools, strategies and companies driving
integrative biology
CHI INSIGHT PHARMA REPORTS
A series of reports that evaluate the salient trends in pharmaceutical technology, business, and therapy markets. Keep
abreast of the latest advances in pharmaceutical R&D, their
potential applications and business impacts, and their current
and future position in the marketplace. For a list of reports,
visit InsightPharmaReports.com, or contact Rose LaRaia,
[email protected], 781-972-5444
ADDITIONAL REGISTRATION DETAILS
Each registration includes all conference sessions, posters and
exhibits, food functions, and a copy of the conference CD.
GROUP DISCOUNTS
Special rates are available for multiple attendees from the
same organization. Contact David Cunningham at 781-9725472 to discuss your options and take advantage of the savings.
HANDICAPPED EQUAL ACCESS
In accordance with the ADA, Cambridge Healthtech
Institute is pleased to arrange special accommodations for attendees with special needs. All requests
for such assistance must be submitted in writing to
CHI at least 30 days prior to the start of the meeting.
SUBSTITUTION/CANCELLATION POLICY
In the event that you need to cancel a registration, you may:
• Transfer your registration to a colleague within your
organization
• Credit your registration to another Cambridge Healthtech
Institute program
• Request a refund minus a $100 processing fee per conference
• Request a refund minus the cost ($750) of ordering a copy
of the CD
NOTE: Cancellations will only be accepted up to two
weeks prior to the conference.
Program and speakers are subject to change.
Video and or audio recording of any kind is
prohibited onsite at all CHI events.
FAX or MAIL your registration to:
Cambridge Healthtech Institute
250 First Avenue, Suite 300,
Needham, Massachusetts 02494
T: 781-972-5400 or toll-free in the U.S. 888-999-6288
F: 781-972-5425 • www.healthtech.com