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RNAi, miRNA and siRNADay One 27th June 2011                                                                               ...
RNAi, miRNA and siRNA-event.com                                                                                           ...
HALF-DAY POST-CONFERENCE WORKSHOP                                                                                 In assoc...
RNAi, miRNA AND siRNA                        Conference: Monday 27th and Tuesday 28th June 2011, Copthorne Tara Hotel, Lon...
RNAi, miRNA & siRNA
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RNAi, miRNA & siRNA

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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.

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RNAi, miRNA & siRNA

  1. 1. REGISTER BY 28th FEBRUARY AND RECEIVE A £300 DISCOUNTREGISTER 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-12.30pmwww.rnai-event.comRegister online and receive full information on all of SMi’s conferencesAlternatively fax your registration to +44 (0) 870 9090 712 or call +44 (0) 870 9090 711
  2. 2. RNAi, miRNA and siRNADay 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 humans9.00 First Chairs 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 Pharmaceuticals9.10 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 silencing9.40 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 (local10.10 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 Tea10.40 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 siRNA11.10 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 oligonucleotides11.50 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. 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 Chairs 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, Pfizerur 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 company’s 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
  4. 4. HALF-DAY POST-CONFERENCE WORKSHOP In association with Wednesday 29th June 2011 8.30am – 12.30pm Copthorne Tara Hotel, London Manipulating exons to treat diseaseOverview of workshop About the workshop leadersInvestigation 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’sRNAse 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 20neuromuscular orphan disease area, with the lead compound in phase IIItrials together with our corporate partners GSK. Principles of drug design years of experience in the pharmaceutical and biotechand development will be presented. The personalized approach to therapy in industry and is an expert in translational medicine. Hean orphan disease raises a number of developmental and regulatory issues has held posts of increasing seniority in both largewhich 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 Campion8.30 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 and9.00 Welcome & Introductions Giles Campion, Chief Medical Officer and Senior Vice-President Europe, including in the rare disease space. R&D, Prosensa9.10 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 early10.30 Morning Coffee drug development. Prior to joining Prosensa, Sjef was the project manager for a multidisciplinary drug11.00 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 NIMBAS12.00 Discussion session University.12.30 Close of WorkshopPHARMACEUTICAL FORWARD PLANNERFebruary 2011 June 201107/08 Parallel Trade 01/02 Pain Therapeutics ABOUT THE SMi PHARMACEUTICAL TEAM21/22 Advances and Progress in Drug Design 27/28 RNAi SMi have been involved in the pharmaceutical23/24 Stem Cells 29/30 Nanotechnology industry since 1993 and have developed a series of 29/30 Pharmaceutical Portfolio & ProductMarch 2011 Lifecycle Management informative and niche events, covering the latest07/08 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 professionals16/17 Superbugs & Superdrugs July 2011 and serving companies who have a focus on being at23/24 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 the30/31 Controlled Release 11/12 Social Media in the Pharmaceutical medium of both conferences and executive briefings. Industry Our pharmaceutical events are research-based andApril 2011 18/19 Clinical Trial Logistics Asia (Singapore) content driven with regular contact with major13/14 Asthma & COPD 20/21 Pre-Filled Syringes Asia (Singapore) industry personnel and cover a wide range of industryMay 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 brackets16/17 Clinical Trial Logistics
  5. 5. RNAi, miRNA AND siRNA Conference: Monday 27th and Tuesday 28th June 2011, Copthorne Tara Hotel, London, UK Workshop: 29th June 2011, London 4 WAYS TO REGISTER www.rnai-event.comFAX your booking form to +44 (0) 870 9090 712 POST your booking form to: Events Team, SMi Group Ltd, Great GuildfordPHONE on +44 (0) 870 9090 711 Business Square, 30 Great Guildford Street London, SE1 0HS, UK EARLY BIRD □ Book by 28th February 2011 to receive a £300 off the conference price DISCOUNT □ Book by 31st March 2011 to receive a £100 off the conference price CONFERENCE PRICES I would like to attend: (Please tick as appropriate) Fee Total □ Conference & Half-Day Workshop £1998.00 +VAT £2397.60 □ Conference only £1399.00 + VAT £1678.80Unique Reference Number □ Half-Day Workshop only £599.00 + VAT £718.80Our Reference LVY18 PROMOTIONAL LITERATURE DISTRIBUTIONDELEGATE DETAILS □ Distribution of your company’s promotional literature to all conference attendees £999.00 + VAT £1198.80Please complete fully and clearly in capital letters. Please photocopy for additional delegates.Title: Forename: GROUP DISCOUNTS AVAILABLESurname: The Conference fee includes refreshments, lunch, conference papers and CD ROM containing all of the presentations.Job Title:Department/Division:Company/Organisation: VENUE Copthorne Tara Hotel, Scarsdale Place, Kensington, London W8 5SREmail: □ Please contact me to book my hotelAddress: Alternatively call us on +44 (0) 870 9090 711, email: hotels@smi-online.co.uk or fax +44 (0) 870 9090 712Town/City: CD ROMS/DOCUMENTATIONPost/Zip Code: Country:Direct Tel: Direct Fax: I cannot attend but would like to purchase the following CD ROMs: (Shipped 10-14 days after the event) Price TotalMobile: □ Conference presentations on CD ROM (Slides only) £499.00 +VAT £598.80Switchboard: □ The conference presentations – paper copy £499.00 - £499.00Signature: Date:I agree to be bound by SMis Terms and Conditions of Booking. PAYMENTACCOUNTS DEPT Payment must be made to SMi Group Ltd, and received before the event, by one of theTitle: Forename: following methods quoting reference Y18 and the delegate’s name. Bookings made within 7 days of the event require payment on booking, methods of payment are below. PleaseSurname: indicate method of payment:Email: □ UK BACS Sort Code 300009, Account 00936418Address (if different from above): □ Wire Transfer Lloyds TSB Bank plc, 39 Threadneedle Street, London, EC2R 8AU Swift (BIC): LOYDGB21013, Account 00936418 IBAN GB48 LOYD 3000 0900 9364 18Town/City: □ Cheque We can only accept Sterling cheques drawn on a UK bank. □ Credit Card □ Visa □ MasterCard □ American ExpressPost/Zip Code: Country: All credit card payments will be subject to standard credit card charges.Direct Tel: Direct Fax: Card No: □□□□ □□□□ □□□□ □□□□Terms and Conditions of Booking Valid From □□/□□ Expiry Date □□/□□Payment: If payment is not made at the time of booking, then an invoice will be issued and must be CVV Number □□□□ 3 digit security on reverse of card, 4 digits for AMEX cardpaid immediately and prior to the start of the event. If payment has not been received then credit carddetails will be requested and payment taken before entry to the event. Bookings within 7 days ofevent require payment on booking. CD Roms will not be dispatched until payment has been received. Cardholder’s Name:Substitutions/Name Changes: If you are unable to attend you may nominate, in writing, anotherdelegate to take your place at any time prior to the start of the event. Two or more delegates maynot ‘share’ a place at an event. Please make separate bookings for each delegate. Signature: Date:Cancellation: If you wish to cancel your attendance at an event and you are unable to send a I agree to be bound by SMis Terms and Conditions of Booking.substitute, then we will refund/credit 50% of the due fee less a £50 administration charge,providing that cancellation is made in writing and received at least 28 days prior to the start of the Card Billing Address (If different from above):event. Regretfully cancellation after this time cannot be accepted. We will however provide theConference documentation on CD ROM to any delegate who has paid but is unable to attend for anyreason. Due to the interactive nature of the Briefings we are not normally able to providedocumentation in these circumstances. We cannot accept cancellations of orders placed forDocumentation or CD ROM as these are reproduced specifically to order. If we have to cancel theevent for any reason, then we will make a full refund immediately, but disclaim any further liability.Alterations: It may become necessary for us to make alterations to the content, speakers, timing,venue or date of the event compared to the advertised programme.Data Protection: The SMi Group gathers personal data in accordance with the UK Data ProtectionAct 1998 and we may use this to contact you by telephone, fax, post or email to tell you about otherproducts and services. Unless you tick here □ we may also share your data with third parties VAToffering complementary products or services. If you have any queries or want to update any of thedata that we hold then please contact our Database Manager databasemanager@smi-online.co.uk VAT at 20% is charged on the attendance fees for all delegates. VAT is also charged on CDor visit our website www.smi-online.co.uk/updates quoting the URN as detailed above youraddress on the attached letter. ROMs and Literature Distribution for all UK customers and for those EU customers not supplying a registration number for their own country here: _______________________ If you have any further queries please call the Events Team on tel +44 (0) 870 9090 711 or you can email them at events@smi-online.co.uk