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Target Validation
7 April - 8 April 2003
Target Validation
'Target Validation’ defines the process by which it is determined whether a newly discovered molecule within the body can be manipulated to treat a disease. If a target can be identified and validated quickly and efficiently, it can provide a Pharmaceutical company with a crucial head-start against its competitors.

Due to the complexity of the drug discovery process and the huge demands on company resources, only a small number of targets can be developed at any given time. Therefore, any technologies or discoveries that can expedite the early stages of target validation are critical to drug discovery and development. Indeed, fast and accurate validation technologies are a necessity if the target selection bottleneck is to be overcome.

Following a very successful event in April 2002, SAE Media Group are eager to assess the progress in this field and to provide a forum where new developments and discoveries can be presented and an up-to-date assessment can be made of target validation systems in place.

Benefits of Attending
· MEET KEY EXPERTS at the cutting edge of new approaches for validating potential targets
· OVERCOME THE TARGET VALIDATION BOTTLENECK by keeping up to date with the very latest ideas for increasing the efficiency of drug discovery
· EVALUATE THE LATEST TECHNOLOGIES available for prioritisation of identified targets
· CUT THE COST AND TIME by understanding the risks and making the right go/no go decisions
· ASSESS FUNCTIONAL GENOMIC APPROACHES and take a closer look at their relative advantages
· LEARN FROM SPECIFIC CASE STUDIES from key companies and hear their experiences of different target validation solutions

A unique opportunity to learn from leading industry experts including:
· Dr Lee Beeley, Group Director, Target Genomics, Pfizer
· Dr Nicholas Dean, Vice President, Functional Genomics, ISIS Pharmaceuticals
· Dr Mark Lindsay, Project Leader, AstraZeneca
· Dr Neelam Jaiswal, Senior Scientist, Procter & Gamble
· Dr Eugene Brown, Senior Director, Expression Profiling Sciences, Wyeth
· Dr Mark Moore, Chief Scientific Officer, DeltaGen
· Dr Carl Pabo, Senior Vice President & Chief Scientific Officer, Sangamo BioSciences
· Dr Kyle Chan, Senior Director, Informatics & Functional Genomics, Celgene
· Dr Brian Zambrowicz, Executive Vice President, Research, Lexicon Genetics

"Excellent, one of the best I have been to. Wonderful meeting overall."
Christopher Marlor, Exploratory Biology, Achillion Pharmaceuticals Attendee, SAE Media Group’s Target Validation Conference 2002

Conference agenda

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8:30

Registration and Coffee

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9:00

Experimental design and data QC

  • Introduction: the transciptome as a phenotype
  • Experimental designs for target validation
  • Assessing biological and technical variability; replication
  • The utility of pilot experiments
  • Sample and data quality control
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    10:30

    Morning Coffee

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    11:00

    Data analysis and interpretation

  • Filtering: statistical and non-statistical methods
  • Defining useful end points for target validation
  • Mining annotation space: an overview of tools and resources
  • Integration of expression data into the target validation decision process
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    12:15

    Discussion and Questions: review of the session

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    12:30

    Close of Briefing

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    8:30

    Registration and Coffee

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    9:00

    Chairman's Opening Remarks

    Dr John Overington

    Dr John Overington, Vice President, Drug Discovery, Inpharmatica

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    9:10

    OPENING ADDRESS: ADVANCES IN TARGET VALIDATION

    Dr Mark Lindsay

    Dr Mark Lindsay, Project Leader, AstraZeneca

  • The increasing need to stay ahead in identifying and validating viable drug targets
  • Making the most of a wealth of information: overcoming the target validation bottleneck
  • Identifying the key developments in the industry
  • New tools available for faster, more efficient target validation
  • Practical considerations: identifying the obstacles
  • Where to next?
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    9:40

    AN INTEGRATIVE APPROACH TO TARGET PRIORITIZATION AND DRUG DISCOVERY

    Dr Kyle Chan

    Dr Kyle Chan, Senior Director, Informatics & Functional Genomics, Celgene

  • Strategies for identification of targets
  • Approaches for prioritization of identified targets
  • Screening approaches
  • Integration of functional genomics/proteomics approaches to SAR and optimisation
  • Discuss case study
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    10:20

    IN VITRO TARGET VALIDATION SYSTEMS

    Dr Neelam Jaiswal

    Dr Neelam Jaiswal, Senior Scientist, Procter & Gamble

  • The importance of target validation in the early stages of discovery
  • Prerequisites for in vitro target validation
  • Technological advances in in vitro validation
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    11:00

    Morning Coffee

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    11:20

    DRUG TARGET DISCOVERY

    Dr Chris Phelps

    Dr Chris Phelps, Principal Scientist, Inpharmatica

  • Target selection from genome scale data
  • Generic target mining approaches
  • Structural annotation of potential drug targets
  • Robust and scaleable strategies to lead discovery
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    12:00

    SCREENING THE DRUGGABLE GENOME

    Dr Kate Holt

    Dr Kate Holt, Senior Director, Molecular Genetics & Alliance Management, Lexicon Genetics

  • Pharma needs new drugs to new targets
  • A systematic approach is required to find these new targets
  • Knock-outs reliably model drug efficacy for the top targets of the pharmaceutical industry
  • Lexicon is knocking-out all druggable genes to identify the best new targets for drug discovery
  • Examples of high quality targets will be given
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    12:40

    Networking Lunch

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    13:40

    THE CHALLENGES OF ANIMAL MODELS FOR TARGET VALIDATION

    Dr Michael Schoor

    Dr Michael Schoor, Associate Director, Artemis Pharmaceuticals

  • The model system must modulate the expression of the target gene at the desired time and place
  • The modulation of target gene expression should be preferentially on a disease background
  • Developing faster technologies to quicken the process
  • The complexity of controlling gene expression
  • Sustaining the efficacy of the model system for long enough time periods
  • Establishing homology, not only of the genes in question but also of the resulting proteins

    How efficiently do current model systems meet these challenges?

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    14:20

    IN VIVO MAMMALIAN TECHNOLOGY

    Dr Mark Moore

    Dr Mark Moore, Chief Scientific Officer, Deltagen

  • Targeted mutagenesis approach to the knock-out of precise genes in mouse models
  • Accelerating the development of commercially-promising small molecules and secreted protein therapies
  • Phenotypic analysis programme to determine function and pharmaceutical relevance of genes and the proteins they encode
  • Detailed physiological, pathological and behavioural studies
  • Validation of the technology
  • Homology between the mouse and the human genome: mice, the model of choice?
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    15:00

    DEVELOPMENTS IN HIGH-THROUGHPUT GENETIC ANIMAL VALIDATION TECHNOLOGIES

    Dr Martin Augustin

    Dr Martin Augustin, Project Manager, Ingenium Pharmaceuticals

  • Novel approaches to deal with numerous potential targets at once
  • The development of new, superior target validation technologies
  • Providing rapid, more relevant and synergistic information about in vivo protein function
  • The challenge of developing robust and efficient tools
  • Integrating information
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    15:40

    Afternoon Tea

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    16:00

    SEAMLESS INTEGRATION OF TARGET VALIDATION, ASSAY DEVELOPMENT AND HTS

    Dr Johan Geysen

    Dr Johan Geysen, Vice President & Director, European Scientific Operations, Union Biometrica

  • Automated genetic/ enhancer/ suppressor screens
  • Bulk sorting of animals and tissues for biochemical/ microarray experiments
  • Dispensing of sorted organisms in multi-well plates for drug screening
  • Automated re-sampling of ‘in-well’ animal populations

    Visualize and quantify sub-visual signal (e.g. low expression levels)

  • Biological imaging using weak gene promoters

    Save on assay development time and assay cost

  • Integration of flow sorting and automated biological imaging
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    16:40

    INTRABODIES: A TOOL FOR TARGET VALIDATION

    Dr Kirsten Mundt

    Dr Kirsten Mundt, Business Development, ESBATech

  • Identification of highly stable, well-expressed scFv frameworks
  • Selection of specific antibodies against target of interest
  • A novel screening method to identify highly stable and soluble antibodies
  • Selection of specific antibodies against the target of interest
  • Validation of functionality in yeast and mammalian cell systems
  • Optimisation of existing scFvs for target validation
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    17:20

    Chairman’s Closing Remarks and Close of Day One

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    8:30

    Re-registration and Coffee

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    9:00

    Chairman's Opening Remarks

    Dr Kate Holt

    Dr Kate Holt, Senior Director, Molecular Genetics & Alliance Management, Lexicon Genetics

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    9:10

    GENOMICS AND TARGET VALIDATION IN THE CONTEXT OF THE PRODUCTIVITY GAP

    Dr Lee Beeley

    Dr Lee Beeley, Group Director, Target Genomics, PharmaMatters

  • A reality check after nearly a decade of genomics-based drug discovery
  • Are there learnings from our history that help us achieve consistent productivity?
  • The two faces of genomics: ‘understanding the substrate’ and ‘data to knowledge’
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    9:40

    FROM HUMAN TISSUE TO VALIDATED TARGET

    Dr Robert Coleman

    Dr Robert Coleman, Chief Scientific Officer, Pharmagene Laboratories

  • Identifying drug targets from human gene expression information
  • Establishing the functional relevance of gene expression data
  • Gaining a balanced view of the importance of disease association in target identification
  • Validation of targets through use of protein expression and functional pharmacology
  • Spotting the positive but not ignoring the negative
  • Identifying exploitable alternatives: switching the indication
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    10:20

    RETINOID SIGNALLING DURING NEUTROPHIL DIFFERENTIATION

    Dr Tarif Awad

    Dr Tarif Awad, Senior Scientist, Genomics Collaborations, Affymetrix

  • An overview of expression profiling with GeneChip arrays
  • Retinoic acid (RA) signalling in neutrophil development and leukaemia
  • The use of a dominant negative approach to disrupt RA signalling
  • Using pathway information to guide data analysis and interpretation
  • IRF-1 and Myo-related cascades
  • Cell cycle inhibition and activation of apoptosis
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    11:00

    Morning Coffee

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    11:20

    CASE STUDY: GENEICETM

    Dr Jake Micallef

    Dr Jake Micallef, Director, Technology & Operations, Gene Expression Technologies

  • Chromatin configuration and gene expression
  • Silencing specific genes by triggering the cell’s natural mechanism for gene silencing
  • In vitro and in vivo delivery
  • Sustainability of silencing
  • Specificity of silencing
  • How has the technology been put into practice?
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    12:00

    CELL-BASED SCREENS AND TRANSCRIPTIONAL PROFILING IN DRUG DISCOVERY

    Dr Eugene Brown

    Dr Eugene Brown, Senior Director, Expression Profiling Sciences, Wyeth

  • Identifying targets and mechanism of action for compounds selected from a high-throughput, cell-based screen
  • Performing the screen: HCT116 p21+/+ and HCT116 p21 -/- isogenic colon cancer cell lines
  • Recapitulating cancer cells that are defective in the p53-p21 growth arrest pathway
  • Transcriptional profiles from the treated and untreated isogenic cells
  • Suggested mechanisms of action for lead compounds
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    12:40

    Networking Lunch

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    14:00

    LETTING FUNCTION GUIDE DISCOVERY

    Dr Mark Powell

    Dr Mark Powell, Scientist, Rigel Pharmaceuticals

  • A different starting point: identifying the protein target first
  • Identifying proteins that interact in cellular pathways with the original protein
  • Developing sets of cellular assays that model key events in the disease process
  • Using retroviruses to deliver a library of peptide modulators
  • Confirming function of the protein
  • Advantages of looking at protein-protein interactions for target validation
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    14:40

    THE USE OF ADENOVIRAL GENE EXPRESSION TECHNOLOGY TO VALIDATE THE USE OF SPECIFIC GENES AS NOVEL DRUG TARGETS

    Dr Gert-Jan Arts

    Dr Gert-Jan Arts, Senior Scientist, Galapagos Genomics

  • Adenoviral vectors: a powerful gene delivery tool
  • Efficient transduction of primary human cells in multi-well format
  • Knock-down vectors expressing small panhandle RNAs
  • Arrayed knock-in and knock-down libraries enabling high-throughput screening using disease-relevant cellular assays
  • Examples: an update of validation technologies in use
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    15:20

    ZINC FINGER PROTEIN TRANSCRIPTION FACTOR (ZFP TF) TECHNOLOGY FOR GENE REGULATION

    Dr Philip Gregory

    Dr Philip Gregory, Senior Scientist & Group Leader, Sangamo BioSciences

  • Modular approach to the design of engineered ZFP TFs
  • Applying the knowledge of chromosomal architecture
  • Selective recognition and regulation of target genes and their protein products
  • Applying the technology to validate targets in animal models of human disease
  • Addressing the technical challenges associated with validation studies in animals

    Delivery of ZPF TFs into cells for in vitro and in vivo approaches

  • Recent examples of applications of ZFP TFs in target validation
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    16:00

    Chairman's Closing Remarks followed by Afternoon Tea
    Close of Conference

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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