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 area of pharmaceutical development, but with far to go before its vast therapeutic potential is fully utilised. This conference will showcase the latest progress made ensuring delegates leave fully informed of industry developments.

Laura Sepp-Lorenzino Senior Director and Department Lead RNA therapeutics Delivery Biology Merck & Co

BY ATTENDING YOU WILL BE ABLE TO:

Hear the latest in RNAi therapeutic progression Learn about the ways top companies are utilising RNAi potential Study cutting edge approaches at overcoming siRNA delivery issues Network with some of the most important industry experts working in RNAi Develop a sound strategy of development in response to industry cuts

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
Register online and receive full information on all of SMis conferences Alternatively fax your registration to +44 (0) 870 9090 712 or call +44 (0) 870 9090 711

RNAi, miRNA and siRNA

Day One 27th June 2011
8.30 9.00 Registration & Coffee First Chair's Opening Remarks Dr Dmitry Samarsky, Vice President- Technology Development, RXI Pharmaceuticals STRATEGIC OVERVIEW 9.10 Contemporary RNA inhibition - Short single stranded antisense Over the past 10 years, Antisense has undergone a transformation and is today re-designed The transformation is due to innovative chemistry and oligonucleotide design, providing a combination of high potency & stability in short oligonucleotides uniquely represented by Short LNA antisense Short LNAs are taken up by cells un-assisted and that translates to potent silencing in vivo The presentation will illustrate the translational nature of LNA and show the very latest pre-/clinical data Troels Koch, VP, Research Division, Santaris Pharma. RNAi: Developing a new therapeutic platform 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 Rachel Meyers, Vice President, Research and RLD, Alnylam Opportunities and challenges in siRNA therapeutics development 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 Laura Sepp-Lorenzino, Senior Director and Department Head - RNA Therapeutics Delivery Biology, Merck & Co Morning Coffee PANEL DISCUSSION 11.10 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 2010 cuts Internal v.s external development for pharma Pharma expectations and Biotechs capabilities Stimulating parallel developments and investment Dmitry Samarsky, Vice President- Technology Development, RXI Pharmaceuticals Emma Hickman, Lab Head, RDA Research/COPD, Novartis Gary Carter, Market Research Director, Agilent Technologies Troels Koch, VP, Research Division, Santaris Pharma Laura Sepp-Lorenzino, Senior Director and Department Head - RNA Therapeutics Delivery Biology, Merck & Co siRNA, OLIGONUCLEOTIDES AND DELIVERY 11.50 Guanidino modified siRNAs and EPR for structure determination 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 Joachim Engels, Professor, University of Frankfurt 12.50 2.00 2.10 12.20

www.rnaiOvercoming a key challenge of RNAi therapeutics: Effective systemic delivery of siRNA in humans Flexible and Scalable Manufacturing System Highly Attractive Safety Profile Flexibility Across Organ Systems Expanding the Platform Christopher Anzalone, CEO, Calando Pharmaceuticals Networking Lunch Second Chairs Opening Remarks : Atilla Seyhan Head, RNAi and Compound Delivery & Screens, Pfizer sd-rxRNA New class of RNAi compounds: Chemistry, in vivo efficacy and preclinical development 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 RXis dermal anti-scarring (anti-fibrotic) and ocular preclinical development programs Dr Dmitry Samarsky, Vice President- Technology Development, RXI Pharmaceuticals Delivery and therapeutic potential of naked (non-formulated) siRNAs (local and systemic administrations) 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 symptoms Elena Feinstein, Chief Scientific Officer, Quark Pharma Afternoon Tea Pulmonary delivery of siRNA - Prospects for therapeutic intervention in respiratory disease 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 Emma Hickman, Lab Head, RDA Research/COPD, Novartis Endosomolytic PepFect peptides for nucleic acid delivery 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. Samir EL Andaloussi, Post Doc Researcher, Department of Laboratory medicine, Karolinska Institute Manufacture and control of oligonucleotides a new challenge for CMC 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 Mike Webb, Head API Chemistry & Analysis UK, GSK Second Chairs Closing Remarks and Close of Day One Atilla Seyhan, Head, RNAi and Compound Delivery & Screens, Pfizer

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Register online at www.rnai-event.com Alternatively fax yo

Who should attend:
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 Discovery Chemistry Molecular Medicine Molecular Discovery Research Molecular Biology and Chemistry Regulatory Affairs Business Development Genomics USA Europe

United Kingdom

Supported by

-event.com
8.30 9.00 Re-registration & Coffee First Chair's Opening Remarks Thomas Thum, Director, Institute for Molecular and Translational Therapeutic Strategies, Medical School Hannover miRNA 9.10 Cardiovascular miRNA therapeutics - Quo vadis? miRNA target identification in cardiovascular disease Highthroughput identification of functional miRNAs in cardiovascular disease Therapeutic manipulation of miRNAs in CV disease Thomas Thum, Director, Institute for Molecular and Translational Therapeutic Strategies, Medical School Hannover Strategies for inhibiting the miRNA pathway 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 Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics Role of miRNAs and long intergenic non-coding RNAs (lincRNAs) in respiratory and inflammatory disease miRNAs and lincRNAs in the regulation of the immune response miRNAs in severe asthma and chronic obstructive pulmonary disease lincRNAs in non-small cell lung cancer Mark Lindsay, Reader in Biopharmaceutics, Imperial College London Morning Coffee The miR-155/PU.1 axis regulates B cell function PU.1 expression is direclty regulated by miR-155 in activated B cells mir-155 through regulation of PU.1 is requiered for class switching recombination mir-155 through regulation of PU.1 is requiered for affinity maturation of B cells Elena Vigorito, Group Leader, Lymphocyte Signalling & Development, Babraham Institute IP AND REULATORY ISSUES 11.40 Second Chairs Opening Remarks: Sterghios Moschos, Principal Scientist, Pfizer 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, Partner, Bird & Bird

RNAi, miRNA and siRNA

Day Two 28th June 2011
12.20 TGF- gene silencing: experiences with competent authorities/regulatory bodies and clinical trials Beginning clinical trials with gene silencing compounds. Interactions with EMA, FDA and PMDA for starting clinical trials. TGF-2 gene silencing in glioma, pancreatic carcinoma and malignant melanoma. Local versus intravenous application of gene silencing compounds Karl-Hermann Schlingensiepen, Chef Executive Officer, Antisense Pharma Networking lunch EPIGENETICS 2.10 Epigenetic mechanisms of RNAi Introduction to the subject matter Major issues in epigenetics and RNAi John Rossi, Dean, Graduate School of Biological Sciences, Beckman Research Institute City of Hope PANEL DISCUSSION 2.30 Epigenetics in RNAi What can epigenetics bring to RNAi study and therapeutic development? Unexplored areas of fundamental biochemistry Techniques and essential controls Sterghios Moschos, Principal Scientist, Pfizer Petr Svoboda, Department of Epigenetic Regulations, Institute of Molecular Genetics. John Rossi, Dean, Graduate School of Biological Sciences, Beckman Research Institute City of Hope Afternoon Tea GeneICE II - The Next Generation Targeted epigenetic gene silencing Kinetics of induced epigenetic reprogramming Global expression of therapeutic and off target effects Formulating GeneICE II for in-vivo efficacy Mark Eccleston, Director of Business Development, ValiR SCREENING AND COMPUTATIONAL SUPPORT 4.10 Computational support of potential RNAi therapeutics Minimise siRNA off-target signalling Network analysis of paired mRNA-miRNA datasets Functional annotation of human miRNA Paul Wilson, Bioinformatics Discovery and Analysis, GlaxoSmithKline RNA interference as a research and therapeutic toolHas its time come? A genome-wide RNAi screen identifies novel targets for neratinib sensitivity leading to neratinib and paclitaxel combination drug treatments 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 Atilla Seyhan, Head, RNAi and Compound Delivery & Screens, Pfizer Second Chairs Closing Remarks and Close of Day Two Sterghios Moschos, Principal Scientist, Pfizer

12.50

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ur registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
SPONSORSHIP AND EXHIBITION OPPORTUNITIES SMi offer sponsorship, exhibition, advertising and branding packages, uniquely tailored to complement your companys 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

HALF-DAY POST-CONFERENCE WORKSHOP

Wednesday 29th June 2011
8.30am 12.30pm Copthorne Tara Hotel, London

In association with

Manipulating exons to treat disease

Overview of workshop Investigation of the therapeutic potential of antisense oligonucleotides has in the main been confined to knocking down protein expression via RNAi or RNAse H. In this workshop we discuss the role of AONs to treat disease by splice modification. Prosensa has a portfolio of such products in the neuromuscular orphan disease area, with the lead compound in phase III trials together with our corporate partners GSK. Principles of drug design and development will be presented. The personalized approach to therapy in an orphan disease raises a number of developmental and regulatory issues which will be discussed. 8.30 9.00 Registration & Coffee Welcome & Introductions Giles Campion, Chief Medical Officer and Senior Vice-President R&D, Prosensa Overview/ Splicing modification and oligo design Potential of AON to treat a variety of diseases through splicing modification The principle of oligonucleotide design Delivery, delivery, delivery Animal models and preclinical proof of concept Judith van Deutekom, Vice-President Drug Discovery, Prosensa Preclinical safety and ADME of oligonucleotides First and later generations of oligonucleotides and their class profile Specific aspects of safety and relevance to man Distribution, Metabolism and pharmacokinetics Pharmacokinetic - pharmacodynamic modeling for clinical trials Sjef de Kimpe, Vice-President, Early Drug Development, Prosensa Morning Coffee A clinical case study Duchenne muscular dystrophy Duchenne Muscular Dystrophy a devastating disease of dystrophin deficiency Clinical proof of concept Development in the orphan drug space Regulatory consequences of a personalized approach Giles Campion, Chief Medical Officer and Senior Vice-President R&D, Prosensa Discussion session Close of Workshop About the workshop leaders Giles Campion, Chief Medical Officer and Senior VicePresident, R&D is responsible for the companys discovery and development strategy and activities. Board certified in rheumatology, he has more than 20 years of experience in the pharmaceutical and biotech industry and is an expert in translational medicine. He has held posts of increasing seniority in both large pharmaceutical and biotech companies working in Europe and the US, covering many different therapeutic areas. During his many years in the industry, Dr Campion has been involved in the development of over 40 therapeutics and diagnostic agents and has led major filings and approvals in the US and Europe, including in the rare disease space. Judith van Deutekom, Vice-President, Drug Discovery is a molecular biologist and has an extensive 17-year track record in the genetic research of muscular dystrophies. She has been dedicated to the development of a genetic therapy for Duchenne Muscular Dystrophy (DMD) since her postdoctoral studies at the University of Leiden (The Netherlands) and the University of Pittsburgh Medical Center (Pennsylvania, USA). She was awarded for her research on antisense oligoribonucleotides as small molecule drugs for DMD by the Princess Beatrix fund in 2001 and by the LUMC (C.J. Kok Award) in 2003. Sjef de Kimpe, Vice-President, Early Drug Development is responsible for pre-clinical and early drug development. Prior to joining Prosensa, Sjef was the project manager for a multidisciplinary drug development team bringing a lead compound from academic research towards phase IIa in an orphan drug indication. He was also a life science consultant on IP, licensing and technology assessment. Sjef has over 18 years experience in pharmaceutical R&D. He was assistant professor of pharmacology and worked as a senior scientist with Nobel laureate Sir John Vane. Sjef studied pharmacy, holds a Ph.D in pharmacology and obtained an MBA at the NIMBAS University.

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PHARMACEUTICAL FORWARD PLANNER

February 2011
07/08 21/22 23/24 07/08 14/15 16/17 23/24 30/31 13/14 11/12 16/17 Parallel Trade Advances and Progress in Drug Design Stem Cells Imaging in Cancer Drug Development Pharmacovigilance Superbugs & Superdrugs Accelerating patient recruitment & Retention in Clinical Trials Controlled Release Asthma & COPD Generics, Supergenerics and Patent Strategies Clinical Trial Logistics

June 2011
01/02 27/28 29/30 29/30 29/30 06/07 11/12 11/12 18/19 20/21 Pain Therapeutics RNAi Nanotechnology Pharmaceutical Portfolio & Product Lifecycle Management KOL Europe (Munich, Germany) ADMET BioBanking Social Media in the Pharmaceutical Industry Clinical Trial Logistics Asia (Singapore) Pre-Filled Syringes Asia (Singapore)
ABOUT THE SMi PHARMACEUTICAL TEAM SMi have been involved in the pharmaceutical industry since 1993 and have developed a series of informative and niche events, covering the latest issues and developments surrounding the industry. Events bring together senior industry professionals and serving companies who have a focus on being at the forefront of developments in this area. SMi aim to generate informed and topical discussion through the medium of both conferences and executive briefings. Our pharmaceutical events are research-based and content driven with regular contact with major industry personnel and cover a wide range of industry sectors. For more information, please visit www.smionline.co.uk/pharma

March 2011

July 2011

April 2011 May 2011

All conferences take place in central London, UK unless indicated otherwise in brackets

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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