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Click here for information on our co-located conference:Pharmaceutical Nanotechnology: Applications and Commercialisation, taking place on the 29th-30th June 2011 


SMi Presents their 6th annual conference on...

RNAi, miRNA and siRNA

About the conference


SMi is proud to present its 6th conference on RNAi, miRNA and siRNA which shall tackle some of the most prominent issues that stand in the way of the successful harnessing of the vast potential that the process possesses. RNAi is still a new and exciting area of pharmaceutical development, however significant progress is required in certain areas, in achieving successful targeted delivery and tackling off targeting as two examples.

This conference will display some of the most promising results achieved; from structural determination through to specific therapeutic applications, clinical trial considerations and negotiating the regulatory minefield, attendees can be sure to expect an invaluable learning and networking experience.



View the Programme | Register Your Place |

The conference will address:

  • RNA-based therapeutics
  • siRNA and oligoneucleotide delivery
  • The effect of pharma cuts on RNAi
  • miRNA in cardiovascular and immunology
  • Bioinformatics and computational support
  • Clinical trials and regulatory considerations
  • Epigenetics 

Interested in speaking at the conference?  Contact the Conference Producer.

For sponsorship and exhibition opportunities, Contact our Sponsorship Department.

 RNAi 3





Chief Executives, Chief Scientific Officers, Vice Presidents, Heads, Directors, Principal Scientists and Managers in the following areas:
  • RNAi
  • miRNA/microRNA
  • siRNA
  • RNA Silencing
  • Genomics Technologies
  • Bioinformatics
  • Gene Modulation
  • Chemistry and Manufacture
  • Translational Research
  • Discovery Chemistry
  • Molecular Medicine
  • Molecular Discovery Research
  • Molecular Biology and Chemistry
  • Regulatory Affairs
  • Business Development
  • Genomics
  • RNA Screening


Conference programme

8:30 Registration & Coffee

9:00 First Chair's Opening Remarks

Dmitry Samarsky

Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals

9:10 Single stranded antisense - today’s situation and the future’s potential

Troels Koch

Troels Koch, VP, Research Division, Santaris Pharma A/S

  • Overview of the RNAi industry
  • Current applications of siRNA, miRNA and oligonucleotides
  • Progress to make
  • 9:45 RNAi: Developing a New Therapeutic Platform

    Rachel Meyers

    Rachel Meyers, Vice President, Research and RLD , Alnylam Pharmaceuticals

  • Unique new modality of RNAi therapeutics
  • Challenges remaining in unlocking this unique potential
  • The need for clear demonstration of efficacy in well-controlled human clinical study
  • Translating the therapeutic potential of RNAi into clinical reality
  • 10:20 Opportunities and Challenges in siRNA Therapeutics Development

    Laura Sepp-Lorenzino

    Laura Sepp-Lorenzino, Senior Director, Merck & Co

  • siRNA lead discovery and optimization
  • Lipid nanoparticle siRNA lead optimization
  • Biophysical and biochemical tools
  • Pharmacokinetics and pharmacodynamic modeling
  • Species differences in LNP performance
  • Mitigation of delivery vehicle toxicities
  • 10:55 Morning Coffee

    11:15 PANEL DISCUSSION - The effect of pharma cuts on the further development and commercialisation of RNAi technologies

  • Pharma in RNAi and Oligonucleotides: Market overview
  • The immediate and lasting effect of 2009 cuts and 2010 cost repositioning by pharma
  • Internal v.s external development for pharma
  • Pharma expectations and Biotech’s capabilities
  • Progress for and potentials of alternative/parallel technologies and financing opportunities
  • Dmitry Samarsky

    Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals

    Troels Koch

    Troels Koch, VP, Research Division, Santaris Pharma A/S

    Emma Hickman

    Emma Hickman, Research Investigator, Novartis

    Laura Sepp-Lorenzino

    Laura Sepp-Lorenzino, Senior Director, Merck & Co

    Gary Carter

    Gary Carter, Market Research Director, Aligent Technologies

    Rachel Meyers

    Rachel Meyers, Vice President, Research and RLD , Alnylam Pharmaceuticals

    12:00 Guanidino modified siRNAs and EPR for structure determination

    Joachim Engels

    Joachim Engels, Professor, University of Frankfurt

  • Cationic siRNAs for stability and uptake
  • siRNA design rules
  • Hepatitis B virus  inhibition
  • Spin labeled RNA for structure studies
  • RNA structure and RNase H accessibility
  • 12:35 Overcoming a key challenge of RNAi Therapeutics: Effective Systemic Delivery of siRNA in Humans

    Christopher  Anzalone

    Christopher Anzalone, CEO, Calando Pharmaceuticals

  • Flexible and Scalable Manufacturing System
  • Highly Attractive Safety Profile
  • Flexibility Across Organ Systems
  • Expanding the Platform
  • 13:10 Networking Lunch

    14:10 Second Chair's Opening Remarks

    Attila Seyhan

    Attila Seyhan, Head, RNAi and Compound Screens, Pfizer

    14:20 sd-rxRNA™ – New class of RNAi compounds: Chemistry, in vivo efficacy and preclinical development

    Dmitry Samarsky

    Dmitry Samarsky, Vice President of Technology Development, RXi Pharmaceuticals

  • Addressing the key challenge of RNAi therapeutic development: in vivo delivery
  • sd-rxRNA combine beneficial properties of RNAi and conventional antisense technologies, which enables robust cellular uptake and gene silencing
  • in vivo efficacy achieved using local administration of sd-rxRNA compounds
  • RXi’s dermal anti-scarring (anti-fibrotic) and ocular preclinical development programs
  • 14:55 Delivery and therapeutic potential of naked (non-formulated) siRNAs (local and systemic administrations)

    Elena Feinstein

    Elena Feinstein, Chief Scientific Officer, Quark Pharmaceuticals

  • Not all indications require specific siRNA formulations for effective delivery
  • Naked synthetic chemically stabilized siRNA can be efficiently delivered to the eye, inner ear, CNS, lung and kidney
  • Delivery of naked siRNA to these organs and tissues results in target gene knockdown and alleviation of disease
  • 15:30 Nanoparticle Dicer Substrate RNA (DsiRNA) Delivery System to Achieve Target Knockdown in Orthotopic and Subcutaneous Tumor Models

    Sujit  Basu

    Sujit Basu, Senior Director, Formulation, Dicerna Pharmaceuticals

    • Novel Dicer Substrate RNA-based therapeutics (DsiRNA) utilize an earlier entry step in the gene silencing process, namely the engagement of the enzyme Dicer
    • Compared to other RNAi approaches, this next generation RNAi strategy demonstrates greater potency and longer duration of action
    • DsiRNA payloads formulated in lipid nanoparticle delivery systems achieve >80% knockdown in mouse liver and >50% knockdown in Hep3B liver tumor models

    16:05 Afternoon Tea

    16:25 Pulmonary delivery of siRNA - Prospects for therapeutic intervention in respiratory disease

    Emma Hickman

    Emma Hickman, Research Investigator, Novartis

  • Targeting epithelial expression in the lung with naked siRNA
  • Tailoring of the dosing strategy and the effect on siRNA distribution
  • Immune considerations in the lung
  • Challenges and future prospects
  • 17:00 Endosomolytic PepFect peptides for nucleic acid delivery

    Samir  EL-Andaloussi

    Samir EL-Andaloussi, Post Doc Researcher, Karolinska Institute

  • pH titratable PF6 peptide for ubiquitous siRNA transfections of primary cells in vitro and for systemic in vivo delivery.
  • A nuclear targeting peptide, PF14, for efficient delivery of splice switching oligonucleotides in disease models caused by aberrant alternative splicing
  • Solid formulations of PepFect peptides with nucleic acids
  • 17:35 Manufacture and control of oligonucleotides – a new challenge for CMC

    Mike Webb

    Mike Webb, Head API Chemistry & Analysis UK, GSK

    An overview of the current oligonucleotide manufacturing landscape
    Specific challenges for analytical control of oligonucleotides
    The challenges for regulators and originators of oligonucleotide CMC filings
    Prospects for the future

    18:10 Second Chair’s Closing Remarks and Close of Day One

    Attila Seyhan

    Attila Seyhan, Head, RNAi and Compound Screens, Pfizer

    8:30 Registration & Coffee

    9:00 Chair's Opening Remarks

    Sterghios Moschos

    Sterghios Moschos, Principal Scientist, Pfizer

    9:10 Small RNAs as triggers for epigenetic control of gene expression

    John Rossi

    John Rossi, Dean, Graduate School of Biological Sciences , Beckman Research Institute City of Hope

  • Small RNAs and regulation of gene expression in yeast and plants
  • Transcriptional gene silencing triggered by small RNAs in mammals
  • Transcriptional gene activation triggered by small RNAs in mammals
  • Possible mechanisms and roles for argonaute proteins
  • 9:30 PANNEL DISCUSSION: Epigenetics in RNAi

  • What can epigenetics bring to RNAi study and therapeutic development?
  • Unexplored areas of fundamental biochemistry
  • Techniques and essential controls
  • Sterghios Moschos

    Sterghios Moschos, Principal Scientist, Pfizer

    John Rossi

    John Rossi, Dean, Graduate School of Biological Sciences , Beckman Research Institute City of Hope

    Petr Svoboda

    Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics

    10:10 Strategies for inhibiting the miRNA pathway

    Petr Svoboda

    Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics

  • miRNA pathway introduction
  • Natural silencing of miRNAs - stress and oocytes
  • Inhibition by eliminating protein components of the pathway
  • Protein inhibitors
  • Inhibition by blocking miRNAs with modified oligonucleotides
  • Small-compound inhibitors
  • 10:45 Morning Coffee

    11:05 A performing approach to identify biological active miRNAs

    Gregoire  Prevost

    Gregoire Prevost, Chief Development Officer, InteRNA Technologies

  • Lentiviral-based miRNA expression library in 2D matrix
  • Determination of miRNA function using high content screens
  • miRNA expression data base in human tissues
  • 11:25 Role of miRNAs in angiogenesis - from mechansims to therapeutic apporaches

    Jan Fiedler

    Jan Fiedler , Institute of Molecular and Translational Therapeutic Strategies , Hannover Medical School

  • miRNA target identification in cardiovascular disease
  • Highthroughput identification of functional miRNAs in cardiovascular disease
  • Therapeutic manipulation of miRNAs in CV disease
  • 12:00 Role of miRNAs and long intergenic non-coding RNAs (lincRNAs) in respiratory and inflammatory disease

    Eleni Tsitsiou

    Eleni Tsitsiou, Research Associate, University of Manchester

    • miRNAs and the regulation of the innate immune response
    • miRNAs and asthma
    • Long non-coding RNAs and non-small cell lung cancer.


    12:35 Networking Lunch

    13:35 IP and Oligonucleotides

  • Patent species: microRNA, anti-miR, delivery, use, process
  • Construing patent claims
  • Breakthroughs versus increments
  • Long term issues with combination products
  • IP strategy - to win, you have to be in the game
  • Lorna Brazell

    Lorna Brazell, Partner, Bird & Bird

    14:10 Is silence still golden? Mapping the RNAi patent landscape

    Per Lundin

    Per Lundin, IP Attorney, Albihns Zacco AB

  • Despite recent turbulence within the RNAi field, the last couple of years have witnessed increasing patenting activity
  • A few corporate players dominate RNAi-related patenting but academia generates a large proportion of the IP within RNAi
  • Although crucial for many therapeutic applications, delivery only accounts for a small share of the IP
  • Key events have historically boosted RNAi patenting growth, making the outlook for the future interesting
  • 14:50 TGF-ß gene silencing: experiences with competent authorities/regulatory bodies and clinical trials

    Karl-Hermann Schlingensiepen

    Karl-Hermann Schlingensiepen, Chief Executive Officer, Antisense Pharma G Mb H

  • Beginning clinical trials with gene silencing compounds.
  • Interactions with EMA, FDA and PMDA for starting clinical trials.
  • TGF-β2 gene silencing in glioma, pancreatic carcinoma and malignant melanoma.
  • Local versus intravenous application of gene silencing compounds
  • 15:25 Afternoon Tea

    15:45 Computational support of potential RNAi therapeutics

    Paul Wilson

    Paul Wilson, Bioinformatics Discovery and Analysis, GlaxoSmithKline

  • Minimise siRNA off-target signalling
  • Network analysis of paired mRNA-miRNA datasets
  • Functional annotation of human miRNA
  • 16:20 RNA interference as a research and therapeutic tool…Has its time come?

    Attila Seyhan

    Attila Seyhan, Head, RNAi and Compound Screens, Pfizer

  • RNAi, principles, technological aspects, and applications
  • RNAi libraries for targeting HCV and SFV viruses
  • Multiplexed RNAi (optional)
  • Genome-wide RNAi screening for the Identification drug sensitizer genes and  novel drugs for breast cancer
  • 16:55 Chair’s Closing Remarks and Close of Day Two

    Sterghios Moschos

    Sterghios Moschos, Principal Scientist, Pfizer



    Manipulating exons to treat disease

    Manipulating exons to treat disease

    Copthorne Tara Hotel
    29th June 2011
    London, United Kingdom

    Copthorne Tara Hotel

    Scarsdale Place
    London W8 5SR
    United Kingdom

    Copthorne Tara Hotel

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.




    speaker image






    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.


    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.


    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.


    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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