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.

1. REGISTER BY 28th FEBRUARY AND RECEIVE A £300 DISCOUNT
REGISTER BY 31st MARCH AND RECEIVE A £100 DISCOUNT
SMi present their 6th conference on…
RNAi, miRNA and siRNA
Monday 27th and Tuesday 28th June 2011
Copthorne Tara Hotel, London
KEY SPEAKERS INCLUDE:
Troels Koch
VP, Research Division
Santaris Pharma
Rachel Meyers
Vice President, Research and RLD
Alnylam
Mike Webb
Head, API & Chemistry Analysis, UK
GSK
Dmitry Samarsky
Vice President, Technology Development
RXi Pharmaceuticals
Atilla Seyhan
Head, RNAi and Compound Deliver & Screens
Pfizer
RNAi is still a new and exciting Laura Sepp-Lorenzino
area of pharmaceutical Senior Director and Department Lead – RNA therapeutics Delivery Biology
Merck & Co
development, but with far to go
before its vast therapeutic
potential is fully utilised. This BY ATTENDING YOU WILL BE ABLE TO:
• Hear the latest in RNAi therapeutic progression
conference will showcase the • Learn about the ways top companies are utilising RNAi potential
latest progress made ensuring • Study cutting edge approaches at overcoming siRNA delivery issues
delegates leave fully informed • Network with some of the most important industry experts working in RNAi
• Develop a sound strategy of development in response to industry cuts
of industry developments.
PLUS A HALF-DAY POST-CONFERENCE WORKSHOP
Wednesday 29th June 2011, Copthorne Tara, London
Manipulating exons to treat disease
Hosted by Giles Campion, Chief Medical Officer and Senior Vice-President R&D, Prosensa
8.30am-12.30pm
www.rnai-event.com
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2. RNAi, miRNA and siRNA
Day One 27th June 2011 www.rnai-
8.30 Registration & Coffee 12.20 Overcoming a key challenge of RNAi therapeutics: Effective systemic
delivery of siRNA in humans
9.00 First Chair's Opening Remarks • Flexible and Scalable Manufacturing System
Dr Dmitry Samarsky, Vice President- Technology Development, RXI • Highly Attractive Safety Profile
Pharmaceuticals • Flexibility Across Organ Systems
• Expanding the Platform
STRATEGIC OVERVIEW Christopher Anzalone, CEO, Calando Pharmaceuticals
9.10 Contemporary RNA inhibition - Short single stranded antisense 12.50 Networking Lunch
• Over the past 10 years, Antisense has undergone a transformation and is
today “re-designed” 2.00 Second Chair’s Opening Remarks :
• The transformation is due to innovative chemistry and oligonucleotide Atilla Seyhan Head, RNAi and Compound Delivery & Screens, Pfizer
design, providing a combination of high potency & stability in short
oligonucleotides – uniquely represented by Short LNA antisense 2.10 sd-rxRNA™ – New class of RNAi compounds: Chemistry, in vivo efficacy
• Short LNA’s are taken up by cells un-assisted and that translates to potent and preclinical development
silencing in vivo • Addressing the key challenge of RNAi therapeutic development: in vivo
• The presentation will illustrate the translational nature of LNA and show delivery
the very latest pre-/clinical data • sd-rxRNA combine beneficial properties of RNAi and conventional
Troels Koch, VP, Research Division, Santaris Pharma. antisense technologies, which enables robust cellular uptake and gene
silencing
9.40 RNAi: Developing a new therapeutic platform • in vivo efficacy achieved using local administration of sd-rxRNA
• Unique new modality of RNAi therapeutics compounds
• Challenges remaining in unlocking this unique potential • RXi’s dermal anti-scarring (anti-fibrotic) and ocular preclinical
• The need for clear demonstration of efficacy in well-controlled human development programs
clinical study Dr Dmitry Samarsky, Vice President- Technology Development, RXI
• Translating the therapeutic potential of RNAi into clinical reality Pharmaceuticals
Rachel Meyers, Vice President, Research and RLD, Alnylam
2.40 Delivery and therapeutic potential of naked (non-formulated) siRNAs (local
10.10 Opportunities and challenges in siRNA therapeutics development and systemic administrations)
• siRNA lead discovery and optimization • Not all indications require specific siRNA formulations for effective
• Lipid nanoparticle siRNA lead optimization delivery
• Biophysical and biochemical tools • Naked synthetic chemically stabilized siRNA can be efficiently delivered to
• Pharmacokinetics and pharmacodynamic modeling the eye, inner ear, CNS, lung and kidney
• Species differences in LNP performance • Delivery of naked siRNA to these organs and tissues results in target gene
• Mitigation of delivery vehicle toxicities knockdown and alleviation of disease symptoms
Laura Sepp-Lorenzino, Senior Director and Department Head - RNA Elena Feinstein, Chief Scientific Officer, Quark Pharma
Therapeutics Delivery Biology, Merck & Co
3.10 Afternoon Tea
10.40 Morning Coffee
3.40 Pulmonary delivery of siRNA - Prospects for therapeutic intervention in
PANEL DISCUSSION respiratory disease
• Targeting epithelial expression in the lung with naked siRNA
11.10 PANEL DISCUSSION: The effect of pharma cuts on the further • Tailoring of the dosing strategy and the effect on siRNA distribution
development and commercialisation of RNAi technologies • Immune considerations in the lung
• Pharma in RNAi and Oligonucleotides: Market overview • Challenges and future prospects
• The immediate and lasting effect of 2010 cuts Emma Hickman, Lab Head, RDA Research/COPD, Novartis
• Internal v.s external development for pharma
• Pharma expectations and Biotech’s capabilities 4.10 Endosomolytic PepFect peptides for nucleic acid delivery
• Stimulating parallel developments and investment • pH titratable PF6 peptide for ubiquitous siRNA transfections of primary
Dmitry Samarsky, Vice President- Technology Development, RXI cells in vitro and for systemic in vivo delivery.
Pharmaceuticals • A nuclear targeting peptide, PF14, for efficient delivery of splice switching
Emma Hickman, Lab Head, RDA Research/COPD, Novartis oligonucleotides in disease models caused by aberrant alternative splicing
Gary Carter, Market Research Director, Agilent Technologies • Solid formulations of PepFect peptides with nucleic acids.
Troels Koch, VP, Research Division, Santaris Pharma Samir EL Andaloussi, Post Doc Researcher, Department of Laboratory
Laura Sepp-Lorenzino, Senior Director and Department Head - RNA medicine, Karolinska Institute
Therapeutics Delivery Biology, Merck & Co
4.50 Manufacture and control of oligonucleotides – a new challenge for CMC
siRNA, OLIGONUCLEOTIDES AND DELIVERY • An overview of the current oligonucleotide manufacturing landscape
• Specific challenges for analytical control of oligonucleotides
11.50 Guanidino modified siRNAs and EPR for structure determination • The challenges for regulators and originators of oligonucleotide CMC
• Cationic siRNAs for stability and uptake filings
• siRNA design rules • Prospects for the future
• Hepatitis B virus inhibition Mike Webb, Head API Chemistry & Analysis UK, GSK
• Spin labeled RNA for structure studies
• RNA structure and RNase H accessibility 5.20 Second Chair’s Closing Remarks and Close of Day One
Joachim Engels, Professor, University of Frankfurt Atilla Seyhan, Head, RNAi and Compound Delivery & Screens, Pfizer
Register online at www.rnai-event.com • Alternatively fax yo
Who should attend: USA Europe
Chief Executives, Chief Scientific Officers, Vice Presidents, Heads,
Directors, Principal Scientists and Managers in the following areas:
• RNAi • Bioinformatics • Molecular Biology and
• miRNA/microRNA • Gene Modulation Chemistry
• siRNA • Discovery Chemistry • Regulatory Affairs
• RNA Silencing • Molecular Medicine • Business Development United
• Genomics Technologies • Molecular Discovery Research • Genomics Kingdom
Supported by

3. RNAi, miRNA and siRNA
-event.com Day Two 28th June 2011
8.30 Re-registration & Coffee 12.20 TGF-β gene silencing: experiences with competent
authorities/regulatory bodies and clinical trials
9.00 First Chair's Opening Remarks • Beginning clinical trials with gene silencing compounds.
• Interactions with EMA, FDA and PMDA for starting clinical trials.
Thomas Thum, Director, Institute for Molecular and Translational • TGF-β2 gene silencing in glioma, pancreatic carcinoma and malignant
Therapeutic Strategies, Medical School Hannover melanoma.
• Local versus intravenous application of gene silencing compounds
miRNA Karl-Hermann Schlingensiepen, Chef Executive Officer, Antisense Pharma
9.10 Cardiovascular miRNA therapeutics - Quo vadis? 12.50 Networking lunch
• miRNA target identification in cardiovascular disease EPIGENETICS
• Highthroughput identification of functional miRNAs in cardiovascular
disease 2.10 Epigenetic mechanisms of RNAi
• Therapeutic manipulation of miRNAs in CV disease • Introduction to the subject matter
Thomas Thum, Director, Institute for Molecular and Translational • Major issues in epigenetics and RNAi
Therapeutic Strategies, Medical School Hannover John Rossi, Dean, Graduate School of Biological Sciences, Beckman
Research Institute City of Hope
9.40 Strategies for inhibiting the miRNA pathway PANEL DISCUSSION
• miRNA pathway introduction
• Natural silencing of miRNAs - stress and oocytes 2.30 Epigenetics in RNAi
• Inhibition by eliminating protein components of the pathway • What can epigenetics bring to RNAi study and therapeutic
• Protein inhibitors development?
• Inhibition by blocking miRNAs with modified oligonucleotides • Unexplored areas of fundamental biochemistry
• Small-compound inhibitors • Techniques and essential controls
Sterghios Moschos, Principal Scientist, Pfizer
Petr Svoboda, Department of Epigenetic Regulations, Institute of Petr Svoboda, Department of Epigenetic Regulations, Institute of
Molecular Genetics Molecular Genetics.
John Rossi, Dean, Graduate School of Biological Sciences, Beckman
10.10 Role of miRNAs and long intergenic non-coding RNAs (lincRNAs) in Research Institute City of Hope
respiratory and inflammatory disease
• miRNAs and lincRNAs in the regulation of the immune response 3.10 Afternoon Tea
• miRNAs in severe asthma and chronic obstructive pulmonary disease
3.40 GeneICE II - The Next Generation
• lincRNAs in non-small cell lung cancer • Targeted epigenetic gene silencing
Mark Lindsay, Reader in Biopharmaceutics, Imperial College London • Kinetics of induced epigenetic reprogramming
• Global expression of therapeutic and off target effects
10.40 Morning Coffee • Formulating GeneICE II for in-vivo efficacy
Mark Eccleston, Director of Business Development, ValiR
11.10 The miR-155/PU.1 axis regulates B cell function
• PU.1 expression is direclty regulated by miR-155 in activated B cells SCREENING AND COMPUTATIONAL SUPPORT
• mir-155 through regulation of PU.1 is requiered for class switching 4.10 Computational support of potential RNAi therapeutics
recombination • Minimise siRNA off-target signalling
• mir-155 through regulation of PU.1 is requiered for affinity maturation • Network analysis of paired mRNA-miRNA datasets
of B cells • Functional annotation of human miRNA
Elena Vigorito, Group Leader, Lymphocyte Signalling & Development, Paul Wilson, Bioinformatics Discovery and Analysis, GlaxoSmithKline
Babraham Institute
4.40 RNA interference as a research and therapeutic tool…Has its time
come?
IP AND REULATORY ISSUES A genome-wide RNAi screen identifies novel targets for neratinib
sensitivity leading to neratinib and paclitaxel combination drug
11.40 Second Chair’s Opening Remarks: treatments
Sterghios Moschos, Principal Scientist, Pfizer • RNAi, principles, technological aspects, and applications
• RNAi libraries for targeting HCV and SFV viruses
11.50 IP and oligonucleotides • Multiplexed RNAi (optional)
• Patent species: microRNA, anti-miR, delivery, use, process • Genome-wide RNAi screening for the Identification drug sensitizer
• Construing patent claims genes and novel drugs for breast cancer
• Breakthroughs versus increments Atilla Seyhan, Head, RNAi and Compound Delivery & Screens, Pfizer
• Long term issues with combination products
• IP strategy - to win, you have to be in the game 5.10 Second Chair’s Closing Remarks and Close of Day Two
Lorna Brazell, Partner, Bird & Bird Sterghios Moschos, Principal Scientist, Pfizer
ur registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
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marketing strategy. Prime networking opportunities exist to entertain, enhance and expand your client base within the context
of an independent discussion specific to your industry. Should you wish to join the increasing number of companies benefiting
from sponsoring our conferences please call: Alia Malick on +44 (0) 20 7827 6168 or email: amalick@smi-online.co.uk
Want to know how you can get involved?
Interested in promoting your pharmaceutical services to this market?
Contact Kiran Sharma, SMi Marketing on +44 (0) 207 827 6050, or email ksharma@smi-online.co.uk

5. HALF-DAY POST-CONFERENCE WORKSHOP In association with
Wednesday 29th June 2011
8.30am – 12.30pm
Copthorne Tara Hotel, London
Manipulating exons to treat disease
Overview of workshop About the workshop leaders
Investigation of the therapeutic potential of antisense oligonucleotides has in Giles Campion, Chief Medical Officer and Senior Vice-
the main been confined to knocking down protein expression via RNAi or President, R&D is responsible for the company’s
RNAse H. In this workshop we discuss the role of AONs to treat disease by discovery and development strategy and activities.
splice modification. Prosensa has a portfolio of such products in the Board certified in rheumatology, he has more than 20
neuromuscular orphan disease area, with the lead compound in phase III
trials together with our corporate partners GSK. Principles of drug design years of experience in the pharmaceutical and biotech
and development will be presented. The personalized approach to therapy in industry and is an expert in translational medicine. He
an orphan disease raises a number of developmental and regulatory issues has held posts of increasing seniority in both large
which will be discussed. pharmaceutical and biotech companies working in
Europe and the US, covering many different
therapeutic areas. During his many years in the industry, Dr Campion
8.30 Registration & Coffee has been involved in the development of over 40 therapeutics and
diagnostic agents and has led major filings and approvals in the US and
9.00 Welcome & Introductions
Giles Campion, Chief Medical Officer and Senior Vice-President Europe, including in the rare disease space.
R&D, Prosensa
9.10 Overview/ Splicing modification and oligo design Judith van Deutekom, Vice-President, Drug Discovery
• Potential of AON to treat a variety of diseases through splicing is a molecular biologist and has an extensive 17-year
modification track record in the genetic research of muscular
• The principle of oligonucleotide design dystrophies. She has been dedicated to the
• Delivery, delivery, delivery development of a genetic therapy for Duchenne
• Animal models and preclinical proof of concept
Judith van Deutekom, Vice-President Drug Discovery, Prosensa Muscular Dystrophy (DMD) since her postdoctoral
studies at the University of Leiden (The Netherlands)
9.45 Preclinical safety and ADME of oligonucleotides and the University of Pittsburgh Medical Center
• First and later generations of oligonucleotides and their class (Pennsylvania, USA). She was awarded for her
profile research on antisense oligoribonucleotides as small molecule drugs for
• Specific aspects of safety and relevance to man DMD by the Princess Beatrix fund in 2001 and by the LUMC (C.J. Kok
• Distribution, Metabolism and pharmacokinetics Award) in 2003.
• Pharmacokinetic - pharmacodynamic modeling for clinical
trials
Sjef de Kimpe, Vice-President, Early Drug Development,
Prosensa Sjef de Kimpe, Vice-President, Early Drug
Development is responsible for pre-clinical and early
10.30 Morning Coffee drug development. Prior to joining Prosensa, Sjef was
the project manager for a multidisciplinary drug
11.00 A clinical case study — Duchenne muscular dystrophy development team bringing a lead compound from
• Duchenne Muscular Dystrophy – a devastating disease of academic research towards phase IIa in an orphan
dystrophin deficiency drug indication. He was also a life science consultant
• Clinical proof of concept
• Development in the orphan drug space on IP, licensing and technology assessment. Sjef has
• Regulatory consequences of a personalized approach over 18 years experience in pharmaceutical R&D. He
Giles Campion, Chief Medical Officer and Senior Vice-President was assistant professor of pharmacology and worked as a senior
R&D, Prosensa scientist with Nobel laureate Sir John Vane. Sjef studied pharmacy,
holds a Ph.D in pharmacology and obtained an MBA at the NIMBAS
12.00 Discussion session University.
12.30 Close of Workshop
PHARMACEUTICAL FORWARD PLANNER
February 2011 June 2011
07/08 Parallel Trade 01/02 Pain Therapeutics ABOUT THE SMi PHARMACEUTICAL TEAM
21/22 Advances and Progress in Drug Design 27/28 RNAi SMi have been involved in the pharmaceutical
23/24 Stem Cells 29/30 Nanotechnology
industry since 1993 and have developed a series of
29/30 Pharmaceutical Portfolio & Product
March 2011 Lifecycle Management informative and niche events, covering the latest
07/08 Imaging in Cancer Drug Development 29/30 KOL Europe (Munich, Germany) issues and developments surrounding the industry.
14/15 Pharmacovigilance Events bring together senior industry professionals
16/17 Superbugs & Superdrugs July 2011 and serving companies who have a focus on being at
23/24 Accelerating patient recruitment 06/07 ADMET the forefront of developments in this area. SMi aim to
& Retention in Clinical Trials 11/12 BioBanking generate informed and topical discussion through the
30/31 Controlled Release 11/12 Social Media in the Pharmaceutical medium of both conferences and executive briefings.
Industry Our pharmaceutical events are research-based and
April 2011 18/19 Clinical Trial Logistics Asia (Singapore)
content driven with regular contact with major
13/14 Asthma & COPD 20/21 Pre-Filled Syringes Asia (Singapore)
industry personnel and cover a wide range of industry
May 2011 sectors. For more information, please visit www.smi-
11/12 Generics, Supergenerics All conferences take place in central London, online.co.uk/pharma
and Patent Strategies UK – unless indicated otherwise in brackets
16/17 Clinical Trial Logistics

6. RNAi, miRNA AND siRNA
Conference: Monday 27th and Tuesday 28th June 2011, Copthorne Tara Hotel, London, UK Workshop: 29th June 2011, London
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