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Antisense & siRNA Technologies
16 February - 17 February 2004
Antisense & siRNA Technologies
Antisense and siRNA technologies are proving invaluable tools in Molecular Biology and their applications in drug discovery and development are at the cutting edge of Pharmaceutical R&D. Allowing for the down- or upregulation of gene expression, interfering with protein function and provoking immune responses, such technologies represent powerful tools. In recent years this has represented an area of major technological advance, potentially fuelling rapid expansion of product pipelines.

Following SAE Media Group’s very successful event in February 2003, this Conference will aim to track the new developments of the last year, looking at new clinical trial results and approaches. The event will also endeavour to discuss the medicinal chemistry behind effective therapeutic design, drug delivery, manufacture and purification. As the industry broadens its knowledge of this exciting area, this event will discuss the strategies and practices in place and ideas for the future, to analyse the prospects and commercial potentials for antisense technologies.

A unique opportunity to learn from leading industry experts including:
· Dr Sudhir Agrawal, President & Chief Scientific Officer, Hybridon
· Dr Eric Marcusson, Associate Director, Functional Genomics, ISIS Pharmaceuticals
· Prof Patrick Iversen, Senior Vice President, Research & Development, AVI BioPharma
· Dr Jim Wright, Chief Executive Officer, Lorus Therapeutics
· Dr Barry Polisky, Senior Vice President, Research & Development, Sirna Therapeutics
· Dr William Marshall, Executive Vice President, Research, Development & Production, Dharmacon

Benefits of Attending:
· CLINICAL TRIALS UPDATE: Learn about the latest advances in antisense and siRNA technologies from the market leaders
· ANTISENSE TECHNOLOGY: Discover the principles and developments behind antisense technology
· INTELLECTUAL PROPERTY RIGHTS: Examine the latest in patent law issues affecting antisense and siRNA inventions
· MANUFACTURE & PURIFICATION: Understand the current methods
· KEY INDUSTRY PERSONNEL: Meet the leaders in the field and learn from their experience

“The schedule looks excellent…it’s brought together the key leaders in the field”
Dr Charles Wilson, Chief Technology Officer, Archemix

Conference agenda

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8:30

Registration and Coffee

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8:30

Registration & Coffee

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9:00

General Issues in US Patent law

  • Patentability requirements
  • Patent term and adjustments
  • Inventorship and ownership
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    9:00

    RNA Interference: a gold standard for reverse genetic evaluation of gene function

  • Mechanistic insights into siRNA induced gene silencing
  • Impacting the drug discovery pipeline with siRNA
  • Design and validation of siRNA experimentation
  • Standardization and comparability of siRNA experimentation
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    9:30

    US patent law issues of particular significance to biotechnology inventions

  • Written description
  • Enablement
  • Restriction practise
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    9:30

    Design of active siRNA sequences

  • Why sequence design is necessary to optimize experimental outcomes
  • Factors important in active siRNA sequences
  • Experimental validation of rational design algorithms
  • Toward a genome-wide library of active gene silencing reagents
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    10:00

    Biotechnology patent prosecution

  • Claim strategies
  • Strategies for providing adequate claim support in the specification
  • Case studies
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    10:20

    Afternoon Tea

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    10:30

    Optimizing potency and specificity of siRNA

  • Hyper-functional siRNA
  • Global expression monitoring of gene knockdown
  • Eliminating sense strand targeting
  • Cell state profiling technologies
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    11:00

    Morning Coffee

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    11:15

    Biotechnology patent enforcement and related issues

  • Infringement
  • Claim interpretation
  • Research exemption
  • Research tool patents
  • FDA exclusivity
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    11:15

    In vivo applications of siRNA

  • Enhancing the stability of siRNA in biological samples
  • Correlations between activity, stability and longevity
  • Biodistribution and target knockdown
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    11:40

    Discussion and questions – review of the session

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    12:00

    Close of Executive Briefing

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    12:30

    Close of Executive Briefing

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    8:30

    Registration and Coffee

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    9:00

    Chairman's Opening Remarks

    Dr Charles Wilson

    Dr Charles Wilson, Chief Technology Officer, Archemix

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    9:10

    KEYNOTE ADDRESS

    Dr Sudhir Agrawal

    Dr Sudhir Agrawal, President & Chief Scientific Officer, Hybridon

  • Principle behind antisense technology
  • Lessons learned from first generation antisense
  • Mechanism of action of first generation antisense
  • Effects of immunostimulatory motifs
  • Design of second generation antisense agents and oral delivery
  • Studies of second generation antisense: from cell culture to clinic
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    9:40

    APTAMER THERAPEUTICS

    Dr Charles Wilson

    Dr Charles Wilson, Chief Technology Officer, Archemix

  • Aptamer overview
  • Aptamer activity in biochemical, cellular and animal disease models
  • Using chemistry to alter PK, distribution and activity
  • Technology advances in SELEX, post-SELEX optimization; alternative modes of action
  • Clinical development preview
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    10:20

    THERAPEUTIC OLIGONUCLEOTIDES - RNaseH MODULATION OF SPLICING AND SIRNA MECHANISMS

    Dr Eric Marcusson

    Dr Eric Marcusson, Associate Director, Functional Genomics, Isis Pharmaceuticals

  • Multiple mechanisms exist by which targeted oligonucleotides can affect gene expression
  • Oligonucleotides operating by any of these mechanisms have the potential to be used as therapeutics
  • The optimal chemistry for the oligonucleotide depends on the desired mechanism
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    11:00

    Morning Coffee

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    11:20

    TURNING TOOLS INTO DRUGS

    Dr Steffen Panzner

    Dr Steffen Panzner, Chief Executive Officer , Novosom

  • Delivery is the bottleneck of nucleic acids-based therapies
  • Liposomes are well approved in vitro but lack function in vivo
  • Charge reversible liposomes (Smarticles) were developed at novosom
  • Smarticles are functional in oligonucleotide transport or gene transfer in vivo
  • Targeting is achieved with different chemistries
  • Smarticles are safe and well tolerated upon repeated dosage
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    12:00

    APPLICATION OF XIAP ANTISENSE TO CANCER & OTHER PROLIFERATIVE DISORDERS

    Dr Jon Durkin

    Dr Jon Durkin, Vice President, Drug Discovery & Preclinical Development, Aegera Therapeutics

  • XIAP as validated cancer target
  • AEG35156/GEM640 as optimized second generation antisense drug
  • Results of preclinical studies
  • Clinical development program
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    12:10

    Discussion and questions – review of the session

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    12:40

    Networking Lunch

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    14:00

    SIRNA THERAPEUTICS: A CASE STUDY

    Dr Barry Polisky

    Dr Barry Polisky, Senior Vice President, Research & Development, Sirna Therapeutics

  • Developing a new class of nucleic acid based therapeutics based on RNA interference
  • Designing, stabilizing, manufacturing and delivering small interfering nucleic acids that facilitate the process of RNAi
  • Prospects for commercial success
  • Current research and/or clinical development
  • Case study: macular degeneration-targeting the Vascular Endothelial Growth Factor (VEGF)
  • Results from the current lead identification phase of development
    Updates from similar candidates in late stage clinical trials
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    14:40

    DRUG DEVELOPMENT FROM DISCOVERY TO CLINICS

    Dr Reimar Schlingensiepen

    Dr Reimar Schlingensiepen, Chief Operating Officer, Antisense Pharma

  • The RADAR® platform: rational and efficient antisense drug design
  • Antisense Pharma’s product pipeline
  • Case study AP 12009: antagonizing several cancer mechanisms for the treatment of malignant glioma
  • Future directions
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    15:20

    Afternoon Tea

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    15:40

    LNA AND IT’S USE IN ANTISENSE AND siRNA TECHNOLOGIES

    Dr Henrik Ørum

    Dr Henrik Ørum, Vice President, Business Development, Santaris Pharma

  • Basic chemistry of LNA
  • LNA antisense in vitro
  • LNA antisense in vivo
  • LNA SiRNA (siLNA)
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    16:20

    GENOME-WIDE SIRNA:

    Dr Eric Lader

    Dr Eric Lader, Associate Director, Business Development & RNAi Applications, Qiagen

  • Advanced siRNA design algorithms
  • Challenges in high throughput informatics for siRNA design
  • Design of the worlds highest throughput siRNA synthesis facility
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    17:00

    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 Christian Schetter

    Dr Christian Schetter, Vice President, European Operations, Coley Pharmaceutical Group

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    9:10

    SPECIFICITY OF RNAi: TACKLING A NATURAL PROCESS TO OUR ADVANTAGE

    Dr Sumedha Jayasena

    Dr Sumedha Jayasena, Research Scientist, Amgen

  • Mechanistic overview of RNAi
  • Origin of off-target effects in RNAi
  • Tools for evaluating non-specific effects
  • Approaches for eliminating off-target effects
  • Rational design of siRNAs to minimize off-target effects
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    9:40

    CASE STUDY: ANTISENSE THERAPEUTICS LTD

    Dr Christopher Wraight

    Dr Christopher Wraight, Director, Research, Antisense Therapeutics

  • Antisense Therapeutics Ltd: background and business model
  • Strategic technology partnerships
  • A topical antisense therapy for psoriasis: ATL1101
  • Pipeline of second generation antisense drugs: preclinical validation strategy
  • ATL1102 for multiple sclerosis
  • Portfolio development for optimising risk vs return in the development of second generation antisense drugs
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    10:20

    SURVIVIN

    Dr Bharvin Patel

    Dr Bharvin Patel, Research Scientist, Cancer Research, Eli Lilly

  • Preclinical Biology
  • Target inhibition in Xenografts
  • Effects on biologic processes
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    11:00

    Morning Coffee

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    11:20

    CASE STUDY: AVI BIOPHARMA

    Prof Patrick Iversen

    Prof Patrick Iversen, Senior Vice President, Research & Development, AVI BioPharma

  • Third generation Neugene® anitisense technology
  • Six completed clinical trials, 200 human subjects and a variety of dosing regimes: the results
  • Potential ramifications: a platform from which to develop additional drug applications
  • Drugs currently in line for clinical trials, including antisense agents that target the West Nile Virus, prostate cancer and cholesterol lowering
  • New opportunities for strategic alliances
  • Bolus intravenous, subcutaneous, oral and transdermal delivery of Neugene antisense
    Moving forward – antisense for the future
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    12:00

    CASE STUDY: LORUS

    Dr Jim Wright

    Dr Jim Wright, Chief Executive Officer, Lorus Therapeutics

  • Drug targets and their role in cancer
  • Broad spectrum of anti-tumour activity
  • In vitro and in vivo evidence of antisense mechanism of action
  • Ongoing clinical studies: eg kidney cancer, breast cancer, AML and prostate cancer
  • Present and future antisense strategies in Lorus Therapeutics
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    12:40

    Networking Lunch

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    14:00

    INTELLECTUAL PROPERTY

    Dr David Harper

    Dr David Harper, Partner, McDonnell, Boehnen, Hulbert & Berghoff

  • US patent law issues impacting antisense and siRNA inventions
  • Developing a patent strategy
  • Defending your intellectual property rights
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    14:40

    THE MANUFACTURE OF OLIGONUCLEOTIDES

    Dr Mark Douglas

    Dr Mark Douglas, Strategy & IP Manager, Avecia

  • Current methods of manufacture
  • Cost effectiveness
  • Scaling up production
  • Managing the production of antisense agents
  • Regulatory challenges to the safe identification of oligonucleotides and their manufacture
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    15:20

    COMBINING BIOINFORMATICS, CHEMISTRY AND BIOLOGY TO PRODUCE HIGHLY POTENT siRNAS FOR TARGETED mRNA KNOCKDOWN

    Dr William Marshall

    Dr William Marshall, Executive Vice President, Research, Development & Production, Dharmacon

  • Design of highly functional siRNA is possible
  • Genome-wide siRNA targeting
  • Maximizing siRNA specificity
  • Considerations for in vivo application of siRNA
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    16:00

    Chairman's Closing Remarks and Close of Conference

    Workshops

    Practical Applications of siRNA Induced Gene Silencing

    Practical Applications of siRNA Induced Gene Silencing

    The Hatton, at etc. venues
    18 February 2004
    London, United Kingdom

    The Hatton, at etc. venues

    51/53 Hatton Garden
    London EC1N 8HN
    United Kingdom

    The Hatton, at etc. venues

    HOTEL BOOKING FORM

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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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