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Selective inhibition of gene expression by RNA interference (RNAi) and wider oligonucleotide methods hold the potential to address significant unmet medical needs with importance across major disease areas such as cancer, genetic disorders, cardiovascular disease and autoimmune disorders.  Recent advances in small interfering RNA delivery and target selection provide unprecedented opportunities for clinical translation and positioning in the market place.

This conference offers the opportunity to engage with strategies for advancing RNAi–based therapies for rare and major diseases through devlopment and clinical trials to market, case study updates on approaches to optimising RNAi formulation and delivery and hear lessons learned through successful submissions from regulators.
 

 

 

  • Take away best practice learning on efficient and targeted efforts to maximise return on investment
  • Hear key case studies from leading companies and gain the edge in confronting the challenges of RNAi R&D
  • Discover the latest clinical data in RNAi and ASO medicine including the latest single strand antisense approaches
  • Review new conjugate/aptamer/lipid/polymer/nanoparticle delivery systems to enhance the efficacy and bioavailability of compounds
  • Update on the state of CRISPR technology for therapy
  • Enhance knowledge of IP and regulatory affairs
   

SMi's 2015 RNA Therapeutics event is a must attend for the following:

Research Scientist

Director

Chief Scientific Ofiicer

Professor of Medicine

PhD student

Academic research staff

Formulation Scientist

Research assistant

Clincal scientist

Biomedical scientist

Biotechnology

Post Doctoral Fellow

Clinical Investigator

Pharmacologist

Medicinal Chemist

Formulation scientist

Arlenda; AstraZeneca; Centre for Process Analytics & Control Technology; De Montfort University; Delft University Of Technology; Dream Pharma; F Hoffman - La Roche A G; Fujifilm Diosynth Biotechnologies; GlaxoSmithKline; GSK; hpa; Industrial Lab Automation; Innopharma Labs; Loughborough University; Mylan Pharmaceuticals; Novartis; Novartis Pharma AG; UCB; University College London; University Of Porto; Vienna University Of Technology; Voisin Consulting Life Sciences;

Conference programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Claude Paul Malvy

Claude Paul Malvy, Professor, Universite Paris Sud

9:10 RNAi therapeutics: Opportunities, obstacles and solutions

Michael Khan

Michael Khan, Chief Medical Officer, Silence Therapeutics

• Challenges for developing effective RNAi - based drugs
• Research on better delivery systems
• Validation in NHPs
• Identifying the right targets and indications
• Update on clinical status of Atu027
 

9:40 RNAi therapeutics for eye conditions

Covadonga Paneda

Covadonga Paneda, R&D Manager, Sylentis

• Update on the clinical programme of Bamosiran, an siRNA for the treatment of glaucoma
• New designs and candidates: impact on biodistribution within the eye
• Advantages of RNAi therapeutics in ophthalmology
 

10:10 Morning Coffee

10:40 From Bench to Bedside – The evolution of mirror-image oligonucleotides as new therapeutic modalities

Axel Vater

Axel Vater, VP Drug Discovery and Preclinical Research, Noxxon Pharma AG

• The benefits of Spiegelmers
• Preclinical & clinical data
• Overview NOX-E36 (drug for diabetic nephropathy)
 

11:10 RNA activation in therapy

Nagy Habib

Nagy Habib, Chairman and Co-Founder, MiNA Therapeutics

11:40 CRISPR-Cas as a “can opener” for genome editing?! First insights into early translational work for human therapeutics

Rodger Novak

Rodger Novak, CEO, CRISPR Therapeutics

• Introduction to CRISPR-Cas – a scientific thriller
• What makes CRISPR-Cas special? – Differentiation regarding other gene editing technologies
• Perspective provided on potential for translation into human medicines – challenges and opportunities
• CRISPR-Cas as a role model for personalized medicine
 

12:10 Hepatic targeting and avoidance of oligonucleotide drugs: What is the real story?

Bård Smedsrod

Bård Smedsrod, Chief Scientific Officer, D'Liver

• Understanding hepatic uptake of large molecule drugs, including oligonucleotides
• Compare uptake activity in different types of liver cells
• Insights into uptake receptors and metabolism
• A need for new ways of explaining hepatotoxicity caused by uptake of large molecule drugs
 

12:40 Networking Lunch

13:40 The use of LNA/UNA-aptamers for anticancer and delivery purposes

Jesper Wengel

Jesper Wengel, Professor, BioNEC Center Director, University of Southern Denmark

• Locked nucleic acid (LNA) is a structurally rigid modification that increases the binding affinity of a modified-oligonucleotide
• Compare the synthetic routes to both of these modifications, contrast the structural features, examine the hybridization properties of LNA and UNA modified duplexes
• LNA and UNA are also emerging as versatile modifications for aptamers

14:10 Transfection: Viral and synthetic techniques converge

Steffen Panzner

Steffen Panzner, Managing Director, Lipocalyx

  • What can be learned from viral delivery?
  • Convergence between a viral and synthetic system
  • Development of the Viromer technology
  • Cell uptake and delivery data
  • 14:40 Delivering macromolecules to the right address - many hands make light work

    Steven Hood

    Steven Hood, Director, Scinovo, GSK

    • Synthesis of drug/polyterpene bioconjugates
    • Design of the nanoassemblies and their functionalization with recognition ligands
    • Cell and tissue imaging
    • Pharmacological evaluation in vitro (cell culture) and in vivo
     

    15:10 Afternoon Tea

    15:40 Protecting biotech inventions, how much for how little?

    James Legg

    James Legg, Patent Attorney , Boult Wade Tennant

    • Why use a patent to protect a biotech invention?
    • An underlying principle - The extent of patent protection should be justified by the technical contribution to the art.
    • Application of this principle during prosecution of patent applications and post-grant
    • Implications for when to file and what to include in your patent application
     

    16:10 Quick Tips

    John Johnston

    John Johnston, Clinical Assessor, Biologicals & Biotechnology Unit, MHRA

    • The hypothesis and the objective of a clinical study
    • Clinical end points / surrogate markers
    • The Innovation Office at the MHRA
    • Points will be illustrated by examples in the public domain
     

    16:40 Biochemically modified messenger RNA encoding nucleases for in vivo gene correction of severe inherited lung diseases

    Michael Kormann

    Michael Kormann, Group Leader, Pediatric Infectiology and Immunology, Eberhard-Karls-University Tuebingen

    • Modified messenger RNA
    • In vivo treatment
    • Life-saving efficacy
    • Limiting off-target effects

    17:10 Round Table Discussion –Developing RNA therapeutics for rare diseases

    Michael Kormann

    Michael Kormann, Group Leader, Pediatric Infectiology and Immunology, Eberhard-Karls-University Tuebingen

    • Identifying and facing early stage and clinical challenges
    • How to best understand and seizing opportunities in the market place

    17:50 Chairman’s Closing Remarks and Close of Day One

    Claude Paul Malvy

    Claude Paul Malvy, Professor, Universite Paris Sud

    8:30 Registration & Coffee

    9:00 Chairman's Opening Remarks

    Michael Khan

    Michael Khan, Chief Medical Officer, Silence Therapeutics

    9:10 RNA Therapeutics: The 3rd major engine of pharmaceutical value creation?

    Dirk Haussecker

    Dirk Haussecker, Consultant, RNA Therapeutics

    • Review of RNA Therapeutics technology
    • Current state of RNA Therapeutics clinical pipeline
    • Assessment of RNA Therapeutics pre-clinical pipeline
    • RNA Therapeutics business and financial market considerations
     

    9:40 RNA Therapeutics: Unlocking the potential with locked nucleic acids

    Bo Rode Hansen

    Bo Rode Hansen, VP, Drug Discovery & Alliance, Roche

    • What is new after several decades of learnings from developing RNA therapeutics?
    • Why has the success rate of RNA therapeutics drug development been low with very few marketed drugs as result?
    • What is the most recent thinking on how to increase the productivity of RNA therapeutics development?
    • Why has Roche recognized Locked Nucleic Acids as the preferred chemistry and modality for RNA therapeutics development?
     

    10:10 Networking towards clinical application of antisense mediated exon skipping

    Annemieke Aartsma-Rus

    Annemieke Aartsma-Rus, Associate Professor , Leiden University Medical Center

    • Overview of how exon skipping can be exploited to treat different diseases with unmet medical need
    • Discussion of path from proof of concept to clinical trials using Duchenne muscular dystrophy as a showcase
    • Discussion of joint efforts to overcome translational challenges for clinical implementation

    10:40 Morning Coffee

    11:10 Tackling Intractable targets in oncology – progress towards viable therapeutic oligonucleotides platforms

    David Blakey

    David Blakey, Chief Scientist, AstraZeneca

    • Robust activity in tumours has been limited with initial  ASO chemistries
    • Next generation ASOs demonstrate  robust target knock down in multiple preclinical cancer models including primary tumour explants  and human tumour xenografts
    • AZD9150, a constrained ethyl ASO vs STAT3 has completed initial trials in cancer patients and shown encouraging signs of clinical activity
    • Preclinical and clinical data will be shared that suggest next generation ASO technology is poised to become a key therapeutic modality to modulate intractable oncology targets

     

    11:40 Panel Debate - Collaborative working from academia to big pharma

    Michael Khan

    Michael Khan, Chief Medical Officer, Silence Therapeutics

  • Preparing to colaborate with big pharma - do's and dont's
  • Exploring collaborative models
  • The role of platform providers

     

  • 12:20 Networking Lunch

    13:30 In vivo RNAi and functional genomics

    Victor Kotelianski

    Victor Kotelianski, Director of the Centre for Research, Education and Innovation, Skolkovo Institute of Science and Technology

    • The use of siRNAs in vivo to study gene function in the liver of mouse models
    • Demonstration of progress in siRNA delivery to mouse hepatocyte
    • RNA interference to examine the activities of specific genes in animal models
    • Analysis of three different cases of multiple mRNAs knockdown in studies of endocytosis, hypoxia and HYPPO pathway
     

    14:00 From RNA splicing to Eteplirsen trials: a drug for Duchenne muscular dystrophy (DMD)

    Ryszard Kole

    Ryszard Kole, Distinguished Scientist, Sarepta Therapeutics

    • Invention of RNA splicing modulation
    • From thalassemia to DMD
    • Insight in to Eteplirsen induced exon skipping of 51 in dystrophin pre-mRNA in DMD patients
    • The results of the continuing trial after 120 weeks of treatment will be presented

    14:30 Afternoon Tea

    15:00 Fusion-oncogene junctions in cancer and interfering RNA: A review

    Claude Paul Malvy

    Claude Paul Malvy, Professor, Universite Paris Sud

    • Double targeting for Ewing sarcoma
    • A  new siRNA delivery agent for Ewing cells
    • Literature review of siRNA and other cancers with fusion oncogene

    15:30 Taking siRNA beyond the liver: Multifunctional nano-complexes for targeted siRNA delivery to brain, lung and tumours

    Stephen Hart

    Stephen Hart, Professor in Molecular Genetics , UCL

    • Targeted lipid-peptide nanocomplex formulations: modulation of surface properties for improved targeting specificity and toxicity profiles
    • Self-assembly protocols by microfluidics for improved siRNA nanoparticle homogeneity, stability and storage
    • Silencing of therapeutic targets in tumour, lung and brain
    • Therapeutic efficacy of siRNA nano-complexes by systemic delivery to tumours
     

    16:00 Chairman’s Closing Remarks and Close of Day Two

    Michael Khan

    Michael Khan, Chief Medical Officer, Silence Therapeutics

    +

    FEATURED SPEAKERS

    Annemieke Aartsma-Rus

    Annemieke Aartsma-Rus

    Associate Professor , Leiden University Medical Center
    Bo Rode Hansen

    Bo Rode Hansen

    VP, Drug Discovery & Alliance, Roche
    David Blakey

    David Blakey

    Chief Scientist, AstraZeneca
    Nagy Habib

    Nagy Habib

    Chairman and Co-Founder, MiNA Therapeutics
    Rodger Novak

    Rodger Novak

    CEO, CRISPR Therapeutics

    Annemieke Aartsma-Rus

    Associate Professor , Leiden University Medical Center
    Annemieke Aartsma-Rus

    Axel Vater

    VP Drug Discovery and Preclinical Research, Noxxon Pharma AG
    Axel Vater

    Bård Smedsrod

    Chief Scientific Officer, D'Liver
    Bård Smedsrod

    Bo Rode Hansen

    VP, Drug Discovery & Alliance, Roche
    Bo Rode Hansen

    Claude Paul Malvy

    Professor, Universite Paris Sud
    Claude Paul Malvy

    Covadonga Paneda

    R&D Manager, Sylentis
    Covadonga Paneda

    David Blakey

    Chief Scientist, AstraZeneca
    David Blakey

    Dirk Haussecker

    Consultant, RNA Therapeutics
    Dirk Haussecker

    James Legg

    Patent Attorney , Boult Wade Tennant
    James Legg

    Jesper Wengel

    Professor, BioNEC Center Director, University of Southern Denmark
    Jesper Wengel

    Joerg Kaufmann

    Chief Scientific Officer, Silence Therapeutics
    Joerg Kaufmann

    John Johnston

    Clinical Assessor, Biologicals & Biotechnology Unit, MHRA
    John Johnston

    Michael Khan

    Chief Medical Officer, Silence Therapeutics
    Michael Khan

    Michael Kormann

    Group Leader, Pediatric Infectiology and Immunology, Eberhard-Karls-University Tuebingen
    Michael Kormann

    Nagy Habib

    Chairman and Co-Founder, MiNA Therapeutics
    Nagy Habib

    Rodger Novak

    CEO, CRISPR Therapeutics
    Rodger Novak

    Ryszard Kole

    Distinguished Scientist, Sarepta Therapeutics
    Ryszard Kole

    Steffen Panzner

    Managing Director, Lipocalyx
    Steffen Panzner

    Stephen Hart

    Professor in Molecular Genetics , UCL
    Stephen Hart

    Steven Hood

    Director, Scinovo, GSK
    Steven Hood

    Sven Klußmann

    Co-Founder & Chief Technology Officer, Noxxon Pharma
    Sven Klußmann

    Victor Kotelianski

    Director of the Centre for Research, Education and Innovation, Skolkovo Institute of Science and Technology
    Victor Kotelianski

    Sponsors and Exhibitors

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    Workshops

    Interview Dirk Haussecker

    Download

    Interview with Michael Kormann

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    Sponsors and Exhibitors


    Lipocalyx

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    http://www.lipocalyx.de/

    Lipocalyx is active in nucleic acid delivery. Our Viromers are novel synthetic vectors emulating viral uptake and endosome escape and provide a safe and efficient way to deliver siRNA, mRNA or DNA. The company was founded in 2011 in Halle(Saale), a German biotech hub. Lipocalyx is offering off-the shelf research reagents and development services for the industry.


    Silence Therapeutics Plc

    Sponsors and Exhibitors
    www.silence-therapeutics.com

    Silence Therapeutics is a leading RNA therapeutics company. It has developed proprietary modifications to improve the robustness of RNA sequences together with advanced liposomal chemistries to enhance the delivery of its therapeutics. Its technology can selectively silence or replace the expression of any gene in the genome, modulating expression up as well as down in a variety of organs and cell types, in vivo. This allows the development of therapeutics for diseases with high unmet clinical need.

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    Contract-Biotechnology.com is a web-based platform for laboratory outsourcing solutions. We are an online R&D matching tool that connects Scientifics and organizations involved in discovery, development and delivery of new healthcare products with service and products providers worldwide. Life science information technology is undergoing a revolution. Part of that revolution involves new, unexplored business models, open innovation, open data and collaboration. If you have a project or idea that you would like to propose, develop or run, we can help. A New Collaboration Network Model for Discovery Research and Development • Effective Platform: the 95% • 5.000 registered users // > 15.000 Newsletter subscribers • Geographical background: 45,5% Europe / 37,5% North America / 16,5% Asia / 0,5% ROW • Professional background: Outsourcing Manager, Project Manager, CEO, Business Developer, CSO, Account Manager, Sales & Marketing People, Scientists, R&D. For further information, please visit our website www.contract-biotechnology.com


    British Pharmacological Society

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    http://www.bps.ac.uk

    The British Pharmacological Society (BPS) is the primary UK learned society concerned with research into drugs and the way they work. Our members work in academia, industry, regulatory agencies and the health services, and many are medically qualified. The Society covers the whole spectrum of pharmacology, including laboratory, clinical, and toxicological aspects. Clinical pharmacology is the medical speciality dedicated to promoting safe and effective use of medicines for patient benefit. Clinical pharmacologists work as consultants in the NHS and many hold prominent positions in UK Universities.


    Genetic Engineering News

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    http://www.genengnews.com

    Genetic Engineering & Biotechnology News (GEN) is the most authoritative and widely read biotechnology news publication in the world.Published 21 times a year,GEN's unique news and technology focus include the entire bioproduct life cycle from early-stage R&D to applied research and bioprocess through to commercialization. GEN is the only publication that provides the full range of the biotechnology market coverage in areas such as omics, drugs and biomarker discovery, bioprocessing, clinical research, molecular diagnostics and biobusiness. For a free subscription go to: http://www.genengnews.com/subscriptions.aspx

    Marriott Regents Park

    128 King Henry's Road
    London NW3 3ST
    United Kingdom

    Marriott Regents Park

    This 4 star north London hotel in zone 2 is the perfect destination for the astute business traveler as well as the leisure guest that knows how convenient north London hotels are, as a base from which to explore the city .Bond Street is just 3 stops from Swiss Cottage underground station on the Jubilee Line, so you can be shopping, exploring the sights and taking in one of London’s world-renowned West End shows in less than 15 minutes when you stay at this hotel near central London. At the same time, the hive of activity that is Camden Town, the chic shops, cafes and restaurants of Primrose Hill and ZSL’s London Zoo in Regents Park are all just a short walk from this hotel in north London.

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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, SMI 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@smi-online.co.uk

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    Contact SMi GROUP LTD

    UK Office
    Opening Hours: 9.00 - 17.30 (local time)
    SMi Group Ltd, 1 Westminster Bridge Road, London, SE1 7XW, United Kingdom
    Tel: +44 (0) 20 7827 6000 Fax: +44 (0) 20 7827 6001
    Website: http://www.smi-online.co.uk Email: events@smi-online.co.uk
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