Download - RNAi, miRNA & siRNA
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SMi present their 6th conference on…
RNAi, miRNA and siRNAMonday 27th and Tuesday 28th June 2011
Copthorne Tara Hotel, London
KEY SPEAKERS INCLUDE:
RNAi is still a new and excitingarea of pharmaceuticaldevelopment, but with far to gobefore its vast therapeuticpotential is fully utilised. Thisconference will showcase thelatest progress made ensuringdelegates leave fully informedof industry developments.
Troels KochVP, Research DivisionSantaris Pharma
Rachel MeyersVice President, Research and RLD Alnylam
Mike WebbHead, API & Chemistry Analysis, UKGSK
Dmitry SamarskyVice President, Technology DevelopmentRXi Pharmaceuticals
Atilla SeyhanHead, RNAi and Compound Deliver & ScreensPfizer
Laura Sepp-LorenzinoSenior Director and Department Lead – RNA therapeutics Delivery BiologyMerck & Co
PLUS A HALF-DAY POST-CONFERENCE WORKSHOPWednesday 29th June 2011, Copthorne Tara, London
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
Manipulating exons to treat diseaseHosted by Giles Campion, Chief Medical Officer and Senior Vice-President R&D, Prosensa
8.30am-12.30pm
RNAi, miRNA and siRNADay One 27th June 2011
Register online at www.rnai-event.com • Alternatively fax yo
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Supported by
Who should attend:
• RNAi • miRNA/microRNA • siRNA • RNA Silencing • Genomics Technologies
Chief Executives, Chief Scientific Officers, Vice Presidents, Heads,Directors, Principal Scientists and Managers in the following areas:
8.30 Registration & Coffee
9.00 First Chair's Opening RemarksDr Dmitry Samarsky, Vice President- Technology Development, RXIPharmaceuticals
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 shortoligonucleotides – uniquely represented by Short LNA antisense
• Short LNA’s are taken up by cells un-assisted and that translates to potentsilencing in vivo
• The presentation will illustrate the translational nature of LNA and showthe very latest pre-/clinical data
Troels Koch, VP, Research Division, Santaris Pharma.
9.40 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 realityRachel Meyers, Vice President, Research and RLD, Alnylam
10.10 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 toxicitiesLaura Sepp-Lorenzino, Senior Director and Department Head - RNATherapeutics Delivery Biology, Merck & Co
10.40 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 Biotech’s capabilities• Stimulating parallel developments and investmentDmitry Samarsky, Vice President- Technology Development, RXIPharmaceuticalsEmma Hickman, Lab Head, RDA Research/COPD, NovartisGary Carter, Market Research Director, Agilent TechnologiesTroels Koch, VP, Research Division, Santaris PharmaLaura Sepp-Lorenzino, Senior Director and Department Head - RNATherapeutics 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
12.20 Overcoming a key challenge of RNAi therapeutics: Effective systemicdelivery of siRNA in humans• Flexible and Scalable Manufacturing System • Highly Attractive Safety Profile • Flexibility Across Organ Systems • Expanding the PlatformChristopher Anzalone, CEO, Calando Pharmaceuticals
12.50 Networking Lunch
2.00 Second Chair’s Opening Remarks : Atilla Seyhan Head, RNAi and Compound Delivery & Screens, Pfizer
2.10 sd-rxRNA™ – New class of RNAi compounds: Chemistry, in vivo efficacyand 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 genesilencing
• in vivo efficacy achieved using local administration of sd-rxRNAcompounds
• RXi’s dermal anti-scarring (anti-fibrotic) and ocular preclinicaldevelopment programs
Dr Dmitry Samarsky, Vice President- Technology Development, RXIPharmaceuticals
2.40 Delivery and therapeutic potential of naked (non-formulated) siRNAs (localand 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 symptomsElena Feinstein, Chief Scientific Officer, Quark Pharma
3.10 Afternoon Tea
3.40 Pulmonary delivery of siRNA - Prospects for therapeutic intervention inrespiratory 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 prospectsEmma Hickman, Lab Head, RDA Research/COPD, Novartis
4.10 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 Laboratorymedicine, Karolinska Institute
4.50 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 futureMike Webb, Head API Chemistry & Analysis UK, GSK
5.20 Second Chair’s Closing Remarks and Close of Day OneAtilla Seyhan, Head, RNAi and Compound Delivery & Screens, Pfizer
Europe
UnitedKingdom
USA
• Bioinformatics • Gene Modulation• Discovery Chemistry • Molecular Medicine• Molecular Discovery Research
• Molecular Biology andChemistry
• Regulatory Affairs • Business Development • Genomics
ur registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
RNAi, miRNA and siRNADay Two 28th June 2011-event.com
SPONSORSHIP AND EXHIBITION OPPORTUNITIESSMi offer sponsorship, exhibition, advertising and branding packages, uniquely tailored to complement your company’smarketing strategy. Prime networking opportunities exist to entertain, enhance and expand your client base within the contextof an independent discussion specific to your industry. Should you wish to join the increasing number of companies benefitingfrom sponsoring our conferences please call: Alia Malick on +44 (0) 20 7827 6168 or email: [email protected]
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 [email protected]
8.30 Re-registration & Coffee
9.00 First Chair's Opening RemarksThomas Thum, Director, Institute for Molecular and TranslationalTherapeutic 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 diseaseThomas Thum, Director, Institute for Molecular and TranslationalTherapeutic Strategies, Medical School Hannover
9.40 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 inhibitorsPetr Svoboda, Department of Epigenetic Regulations, Institute ofMolecular Genetics
10.10 Role of miRNAs and long intergenic non-coding RNAs (lincRNAs) inrespiratory 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 cancerMark Lindsay, Reader in Biopharmaceutics, Imperial College London
10.40 Morning Coffee
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• 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 cellsElena Vigorito, Group Leader, Lymphocyte Signalling & Development,Babraham Institute
IP AND REULATORY ISSUES
11.40 Second Chair’s Opening Remarks:Sterghios Moschos, Principal Scientist, Pfizer
11.50 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 gameLorna Brazell, Partner, Bird & Bird
12.20 TGF-β gene silencing: experiences with competentauthorities/regulatory bodies and clinical trials• 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 compoundsKarl-Hermann Schlingensiepen, Chef Executive Officer, Antisense Pharma
12.50 Networking lunch
EPIGENETICS
2.10 Epigenetic mechanisms of RNAi• Introduction to the subject matter• Major issues in epigenetics and RNAiJohn Rossi, Dean, Graduate School of Biological Sciences, BeckmanResearch 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 controlsSterghios Moschos, Principal Scientist, PfizerPetr Svoboda, Department of Epigenetic Regulations, Institute ofMolecular Genetics. John Rossi, Dean, Graduate School of Biological Sciences, BeckmanResearch Institute City of Hope
3.10 Afternoon Tea
3.40 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 efficacyMark 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 miRNAPaul Wilson, Bioinformatics Discovery and Analysis, GlaxoSmithKline
4.40 RNA interference as a research and therapeutic tool…Has its timecome?A genome-wide RNAi screen identifies novel targets for neratinibsensitivity leading to neratinib and paclitaxel combination drugtreatments• 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 cancerAtilla Seyhan, Head, RNAi and Compound Delivery & Screens, Pfizer
5.10 Second Chair’s Closing Remarks and Close of Day TwoSterghios Moschos, Principal Scientist, Pfizer
HALF-DAY POST-CONFERENCE WORKSHOPWednesday 29th June 2011
8.30am – 12.30pmCopthorne Tara Hotel, London
Overview of workshopInvestigation of the therapeutic potential of antisense oligonucleotides has inthe main been confined to knocking down protein expression via RNAi orRNAse H. In this workshop we discuss the role of AONs to treat disease bysplice modification. Prosensa has a portfolio of such products in theneuromuscular orphan disease area, with the lead compound in phase IIItrials together with our corporate partners GSK. Principles of drug designand development will be presented. The personalized approach to therapy inan orphan disease raises a number of developmental and regulatory issueswhich will be discussed.
8.30 Registration & Coffee
9.00 Welcome & IntroductionsGiles Campion, Chief Medical Officer and Senior Vice-PresidentR&D, Prosensa
9.10 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 conceptJudith van Deutekom, Vice-President Drug Discovery, Prosensa
9.45 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
trialsSjef de Kimpe, Vice-President, Early Drug Development,Prosensa
10.30 Morning Coffee
11.00 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 approachGiles Campion, Chief Medical Officer and Senior Vice-PresidentR&D, Prosensa
12.00 Discussion session
12.30 Close of Workshop
About the workshop leadersGiles Campion, Chief Medical Officer and Senior Vice-President, R&D is responsible for the company’sdiscovery and development strategy and activities.Board certified in rheumatology, he has more than 20years of experience in the pharmaceutical and biotechindustry and is an expert in translational medicine. Hehas held posts of increasing seniority in both largepharmaceutical and biotech companies working inEurope and the US, covering many different
therapeutic areas. During his many years in the industry, Dr Campionhas been involved in the development of over 40 therapeutics anddiagnostic agents and has led major filings and approvals in the US andEurope, including in the rare disease space.
Judith van Deutekom, Vice-President, Drug Discoveryis a molecular biologist and has an extensive 17-yeartrack record in the genetic research of musculardystrophies. She has been dedicated to thedevelopment of a genetic therapy for DuchenneMuscular Dystrophy (DMD) since her postdoctoralstudies 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 forDMD by the Princess Beatrix fund in 2001 and by the LUMC (C.J. KokAward) in 2003.
Sjef de Kimpe, Vice-President, Early DrugDevelopment is responsible for pre-clinical and earlydrug development. Prior to joining Prosensa, Sjef wasthe project manager for a multidisciplinary drugdevelopment team bringing a lead compound fromacademic research towards phase IIa in an orphandrug indication. He was also a life science consultanton IP, licensing and technology assessment. Sjef hasover 18 years experience in pharmaceutical R&D. He
was assistant professor of pharmacology and worked as a seniorscientist with Nobel laureate Sir John Vane. Sjef studied pharmacy,holds a Ph.D in pharmacology and obtained an MBA at the NIMBASUniversity.
In association with
Manipulating exons to treat disease
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RNAi, miRNA AND siRNAConference: Monday 27th and Tuesday 28th June 2011, Copthorne Tara Hotel, London, UK Workshop: 29th June 2011, London
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