discovery, development, diagnostics & beyond february 22-24, 2010 • sheraton miyako hotel •...
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www.IBCLifeSciences.com/AsiaTIDES
Delivering the Latest Research and Best Practices
from Around the World
IBC’s 2nd Annual
Bronze SponsorsSilver Sponsor Premier Publication
February 22-24, 2010 • Sheraton Miyako Hotel • Tokyo, Japan
Keynote PresentationsSynthesis and Biological Activity of the Nucleic Acids and Their Analogs
Marvin H. Caruthers, Ph.D. Distinguished Professor Chemistry and Biochemistry University of Colorado, USA
Therapeutic Applications of Dicer Substrate siRNAsJohn J. Rossi, Ph.D. Chairman, Lidow Family Endowed Research Professor, Molecular Biology Beckman Research Institute of City of Hope, USA Presentation supported by Dicerna Pharmaceuticals
Defying Difficult Diseases: Biomolecule-based Medicinal Science
Yoshiaki Kiso, Ph.D. Professor & Chairman Department of Medicinal Chemistry Kyoto Pharmaceutical University, Japan
RNAi and Peptide Intellectual Property Landscapes: Early and Evolving
Kathleen M. Williams, Ph.D., J.D. Partner, Intellectual Property Law Edwards Angell Palmer & Dodge LLP, USA
Fill your pipeline & expand your portfolio with RNAi, aptamer, microRNA, oligonucleotide and peptide based therapeutics
Advance your company’s research:
RNAおよびペプチド関連の最新技術• Find out what other companies are doing,
hear case studies and success stories presented by 37 North American and 7 European speakers.
Hear from these cutting-edge companies:• Alnylam • Amylin • Tekmira • RXi Pharmaceuticals • Aileron • Regado • Ipsen • Roche • Merck • Quark • Santaris • Regulus • Dicerna • Noxxon • Anges • Affymax • Allon • TriLink • Eurogentec
Grow your company’s development program and increase your knowledge:
RNAおよびペプチド医薬の市場性と企業パートナリング• Participate in sessions on worldwide regulatory
expectations, market landscape and partnering ideas.
Platinum Sponsor
Delivering the Latest Research and Best Practices
from Around the World
Discovery, Development, Diagnostics & BeyondFebruary 22-24, 2010 • Sheraton Miyako Hotel • Tokyo, Japan
Registered attendees of AsiaTIDES may join any of the main conference sessions of Drug & Biologics Japan (Monday afternoon through Wednesday) at no extra charge! IBC’s Drug & Biologics Japan includes sessions on open innovation, next generation biologics, personalized medicine and biomarkers, stem cells, and targeted cancer therapies.www.IBCLifeSciences.com/Japan
Increase your Knowledge Beyond Basic Academic Research – Fill your Pipeline & Expand your Portfolio with RNAi, Aptamer, microRNA, Oligonucleotide and Peptide Based Therapeutics
2 ToRegister,Tel:(+65)6514-3180•Fax:(+65)6733-5087•E-mail:[email protected]
AsiaTIDES is the only conference in Japan to focus on research, technology and product development, manufacturing and partnering in the field of oligonucleotide- and peptide-based therapeutics and diagnostics. You will hear updates from the most advanced research companies on new approaches to delivery and formulation as well as learn what potential drug candidates are currently in development, and what the most successful commercialization strategies are from both the industry and academic perspectives.
Hear from Drug Development Companies from Around the World:37 North American and 7 European speakers will offer case studies and success stories to help you benchmark your research and development in RNAi and aptamer, microRNA, oligonucleotide and peptide based therapeutics. There will also be 19 NEW companies and institutions presenting who were not represented at AsiaTIDES 2009, so you can gain new perspectives.
Plus: You will also be able to increase your skills by attending pre-conference tutorial courses on analytical methods and on DNA/RNA synthesis. Or, plan for the future by choosing to attend the plenary session of the concurrent Drug & Biologics Japan conference, on “Innovation in Japan – Is it Still Alive? Strategies for Open Innovation to Enhance Productivity and Creativity.”
By attending AsiaTIDES you will be able to learn how to advance your company’s research, build your network of partners and collaborators as well as grow your company’s development program – and your career.
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, USAJesse Dong, Ph.D., Senior Director, Medicinal Chemistry, IPSEN/Biomeasure, Incorporated, USAKazuko Hirabayashi, Ph.D., Principal Scientist, Pharmaceutical Information Department, Nippon Shinyaku Co., Ltd., Japan Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USAYoshiaki Kiso, Ph.D., Professor & Chairman, Department of Medicinal Chemistry, Director, Center for Frontier Research in Medicinal Science, Kyoto Pharmaceutical University, JapanDavid Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, USA Jesper Lau, Ph.D., Vice President, Diabetes Protein and Peptide Chemistry, Novo Nordisk, DenmarkShawn Lee, Ph.D., President and CEO, CPC Scientific, USA and ChinaRyuichi Morishita, M.D., Ph.D., Professor, Dept. of Clinical Gene Therapy, Graduate School of Medicine, Osaka University; Founder, Board Member, AnGes MG, Inc., Japan
Yoshikazu Nakamura, Ph.D., Professor, Department of Basic Medical Science, Institute of Medical Science, University of Tokyo; Founder, Ribomic, Japan
Pamela A. Pavco, Ph.D., Vice President, Pharmaceutical Development, RXi Pharmaceuticals, USA
Kal Reddy, Ph.D., Development Manager, Agilent Technologies, USA
Christopher A. Rhodes, Ph.D., Executive Director, Pharmaceutical Sciences, Amylin Pharmaceuticals, Inc., USA
Shigeki Sasaki, Ph.D., Professor, Division of Bioorganic and Synthetic Chemistry, Graduate School of Pharmaceutical Sciences, Kyushu University, Japan
Hideaki Sato, General Manager, Technical Support & Marketing Division, GeneDesign, Inc., Japan
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
AsiaTIDES®ScientificAdvisoryCommittee
Increase your Knowledge Beyond Basic Academic Research – Fill your Pipeline & Expand your Portfolio with RNAi, Aptamer, microRNA, Oligonucleotide and Peptide Based Therapeutics
Media PartnersSupporting Organizations
Co-Located with
Visitwww.IBCLifeSciences.com/AsiaTIDESforup-to-dateinformationonthisevent 3
8:00 Conference Registration Begins
Pre-ConferenceTutorialstoDevelopyourSkills Monday,February22,2010
MainConference•PlenarySession Monday,February22,2010 1:45 Chairperson’s Remarks
Yoshikazu Nakamura, Ph.D., Professor, Department of Basic Medical Science, Institute of Medical Science, University of Tokyo; Founder, Ribomic, Japan
KeynotePresentations 2:00 Therapeutic Applications of Dicer
Substrate siRNAs This presentation describes the use of Dicer substrate RNAs (dsiRNAs) for the treatment of HIV infection in a humanized mouse model for HIV infection. In addition we have developed dual targeting dsiRNAs for cancer treatment, in which each strand selectively targets a different message to trigger synthetic lethality in cancer treatment. Dicer substrate siRNAs present a unique platform for a variety of RNAi applications.John J. Rossi, Ph.D., Chairman, Lidow Family Endowed Research; Professor, Molecular Biology, Beckman Research Institute of City of Hope, USA Presentation supported by Dicerna Pharmaceuticals
2:45 Defying Difficult Diseases: Biomolecule-based Medicinal Science Based on the substrate transition state, we designed and synthesized novel classes of inhibitors of aspartic proteases such as renin, HIV protease, malarial plasmepsin II, ß-secretase (BACE1) containing the hydroxymethylcarbonyl (HMC) isostere. On the basis of our study with the “O-acyl isopeptide method”, we developed novel photo- and pH-triggered “click” peptides that readily convert to the native amyloid ß peptide 1-42 upon activation. Yoshiaki Kiso, Ph.D., Professor & Chairman, Department of Medicinal Chemistry, Kyoto Pharmaceutical University, Japan
3:30 Networking Refreshment Break with Poster and Exhibit Viewing
KeynotePresentations
4:00 RNAi and Peptide Intellectual Property Landscapes: Early and Evolving Short nucleic acids (RNAi and oligonucleotides) and peptides share the fundamental feature that they are a fragment of a longer molecule and possess regulatory function. Is there important IP to consider for such molecules? What has granted in the US, Europe, and Japan? Why do the country IP landscapes differ? Early RNAi patent filings include Fire, Tuschl I/II, Kreutzer, Glover, Rossi. How do the early patents affect the current market?Kathleen M. Williams, Ph.D., J.D., Partner, Intellectual Property, Edwards Angell Palmer & Dodge LLP, USA
4:45 Synthesis and Biological Activity of the Nucleic Acids and Their Analogs The lecture will focus on biochemical activities of boranephosphonate oligonucleotides, phosphonoacetate oligonucleotide, and new methods for synthesizing DNA and RNA.Marvin H. Caruthers, Ph.D., Distinguished Professor, Chemistry and Biochemistry, University of Colorado, USA
5:30 Networking Cocktail Reception with Poster and Exhibit ViewingReception Co-Sponsored by
and
Tutorial#1:AnalysisandBioanalysisofOligonucleotidesin
SupportofDrugDevelopmentTutorial Leader: G. Susan Srivatsa, Ph.D., President, ElixinPharma, USAThe tutorial will lead off with a review of US and European regulatory expectations for the analysis of oligonucleotides (antisense, siRNA, DNA decoys, aptamers, etc.). Assuring product quality through raw material testing, in process control and quality control testing of single strand and siRNA oligonucleotides in a manufacturing environment will be addressed. Special topics will include analysis of oligonucleotides in complex formulations and biological matrices, technology transfer/validation as well as a case study of a siRNA drug product analysis.
9:00 Analysis of Oligonucleotides: US and European Regulatory ExpectationsG. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
9:35 Analysis of Single Strand OligonucleotidesIpsita Roymoulik, Ph.D., Analytical Development Group Leader, Avecia Biotechnology, USA
10:10 Special Considerations for siRNAHüseyin Aygun, Ph.D., Chief Scientific Officer, BioSpring GmbH, Germany
10:45 Networking Refreshment Break with Poster and Exhibit Viewing 11:15 Analysis of Complex Stable Nucleic Acid
Lipid Particle (SNALP) Formulations CASE
STUDYNancy Fuselli, Director, Quality Control and Analytical Development, Tekmira Pharmaceuticals Corporation, Canada
11:50 Analytical Approaches to Determine Stability, PK Properties and Biodistribution of siRNAsIngo Roehl, Ph.D., Associate Director, Analytical Chemistry, Roche Kulmbach GmbH, Germany
12:25 Analytical Development for Early-Stage siRNA Products: A Case Study
CASESTUDY
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
1:00 Close of Tutorial
Tutorial#2:ScalingupOligonucleotideManufacturingProcess
Tutorial Leader: Lubomir Nechev, Ph.D., Senior Director, Process Chemistry, Alnylam Pharmaceuticals, Inc., USAThe tutorial will focus on the scale-up challenges of the critical steps in the oligonucleotide manufacturing process. The points of view of drug developers, oligonucleotide contract manufacturers, as well as synthesis hardware manufacturers will be presented. The effects of different modifications and scale on the manufacturing process as well as techniques for developing scaled-down process models will be discussed. 9:00 Introduction to the Tutorial
Lubomir Nechev, Ph.D., Senior Director, Process Chemistry, Alnylam Pharmaceuticals, Inc., USA
9:10 A History of DNA/RNA SynthesizersJeffrey Christopher Strauss, Vice President, BioAutomation Corp., USA
9:50 Challenges in the Preparation and Analysis of Conjugated Oligonucleotide Drug CandidatesKenneth W. Hill, Ph.D., Principal Scientist, Agilent Technologies, USA
10:30 Networking Refreshment Break with Poster and Exhibit Viewing 11:00 QbD Development Strategies for Oligonucleotide Therapeutics
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
11:40 Unique Challenges Associated with Large Scale Manufacturing of Different RNA Sequences Kathryn L. Ackley, Ph.D., Director of Operations, Girindus., USA
12:20 Effect of Scale on Oligonucleotide Manufacturing ProcessLubomir Nechev, Ph.D., Senior Director Process Chemistry, Alnylam Pharmaceuticals, Inc., Inc., USA
1:00 Close of Tutorial
AsiaTIDES®attendeesmayalsoattendtheplenarysessionoftheconcurrentconference,Drug&BiologicsJapan,asanoptional
sessionMondaymorning.Thesessionistitled:"InnovationinJapan–IsitStillAlive?StrategiesforOpen
InnovationtoEnhanceProductivityandCreativity."Please visit www.IBCLifeSciences/Japan for more information.
1:00 Networking Luncheon with Poster and Exhibit Viewing and Registration for Main Conference
4 ToRegister,Tel:(+65)6514-3180•Fax:(+65)6733-5087•E-mail:[email protected]
Oligonucleotide-Based Therapeutics Worldwide Advances in Peptides & Diagnostics
MainConference•ConcurrentTracks Tuesday,February23,2010
UpdatesonOligonucleotide-BasedTherapeuticsinPreclinicalDevelopment
8:30 Chairperson’s RemarksBob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, Inc., USA
8:45 Lead Generation for microRNA ModulatorsmicroRNAs regulate targets by binding to 3’UTRs with sequence complementarity to the microRNA seed region. The ability of microRNAs to target multiple transcripts enables fine-tuning of gene networks as a novel approach to therapy, but also presents unique challenges for lead candidate selection and development of microRNA therapeutics. We will discuss how we are addressing some of these unique challenges.Bal Bhat, Ph.D., Senior Director, Medicinal Chemistry, Regulus Therapeutics
9:15 Intratumoral Knockdown of the Oncogene KRAS with DsiRNADicer-substrate siRNAs (DsiRNAs) are capable of triggering potent and long lasting RNA interference effects. DsiRNAs targeting the oncogene KRAS have been used in vitro and in vivo to reduce KRAS expression to nearly undetectable levels in some normal tissues and in tumors. In vivo results focusing on KRAS-expressing orthotopic human xenograft tumor models, including both solid and hematological tumors, will be presented. Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, Inc.
9:45 microRNA as a Novel Modality for Cancer TherapyRNAi offers possible platform to identify potential drug candidates to any target even in undruggable or not amenable to conventional drugs. This paper introduces the examples of siRNAs and microRNAs delivery into tumors with metastasis in animal models, and discusses the potential there of these miRNAs as diagnostic and prognostic markers and as targets/molecules for molecular cancer therapy.Takahiro Ochiya, Ph.D., Head, Section for Studies on Metastasis, National Cancer Center Research Institute, Japan
10:15 Networking Refreshment Break with Poster and Exhibit Viewing
10:45 Preclinical and First Clinical Data from Mirror-Image RNA Oligonucleotides Functional mirror-image oligonucleotides, also called Spiegelmers® or L-aptamers, can be identified to bind and inhibit targets conceptually similar to antibodies. They have a direct mode of action and tremendous biostability. NOXXON's lead candidates are NOX-E36 and NOX-A12, targeting the chemokines MCP-1 and SDF-1, respectively. Data will be presented on NOX-E-36, which is in Phase I, and NOX-A12, which has completed regulatory tox and safety studies. Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany
11:15 Stereocontrolled Synthesis and Properties of Backbone-Modified Oligonucleotides
We have developed an oxazaphospholidine method for the stereocontrolled synthesis of phosophorothioate DNA and RNA. The method has been successfully applied to the synthesis of stereoregulated boranophosphate DNA. Quite recently, the method has also been used for the first synthesis of stereoregulated H-phosphonate DNA, which is useful as a versatile intermediate for the synthesis of P-chiral backbone-modified DNA analogs.Takeshi Wada, Ph.D., Department of Medical Genome Sciences, The University of Tokyo, Japan
11:45 Technology Workshop Presentation Opportunity AvailableIBC’s Technology workshops offer supplier and service companies the opportunities to present product and service offers directly to the audience at the conference. For more information on presenting a technology workshop at this meeting, please contact:Asia: Catherine Wong, Phone: 65-6835-5128, email: [email protected]/Europe: Sherry Johnson, Phone: +1-508-614-1451, email: [email protected]
12:15 Networking Luncheon with Poster and Exhibit Viewing
UpdatesonPeptide-BasedTherapeuticsinPreclinicalandClinicalDevelopment
8:30 Chairperson’s RemarksChristopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USA
8:45 AL-309: A Preclinical Case Study for Peripheral Neuropathies
CASESTUDY
AL-309 is an all D-amino peptide derived from Activity-Dependent Neurotrophic Factor (ADNF). In preclinical models, AL-309 demonstrated protection of peripheral nerve damage after chemotherapy- or diabetes-induced neuropathy as well as an improvement in the pain response which develops in these models. The pharmacology, pharmacokinetics and preliminary toxicology suggest that AL-309 is an interesting drug candidate for the prevention of peripheral neuropathies.Bruce H. Morimoto, Ph.D., Vice President, Drug Development, Allon Therapeutics Inc., Canada
9:15 Medical Applications of Peptides with Angiogenesis or Antimicrobial PropertiesWe have been extensively investigating medical application of antimicrobial peptides with various pharmacological activities. A review will be provided of recent developments in this field and an overview of preclinical data regarding the projects: 1) medical application of antimicrobial peptide, and 2) utilization of antimicrobial peptide with angiogenesis property as wound healing agent.Shunsuke Sami, Ph.D., Executive Vice President, Drug Discovery Research, AnGes MG, Inc., JapanHideki Tomioka, Manager, Pharmaceutical Research Group, AnGes MG, Inc., Japan
9:45 Stapled Peptides: Leveraging the a-Helix to Create a New Class of Targeted TherapeuticsHydrocarbon “stapled” versions of the BH3-domain alpha-helices of BID and BIM, pro-apoptotic BCL-2 family members, possess potent and specific in vitro and in vivo anti-cancer activities for a broad diversity of solid and liquid tumors. AILERON is completing pre-clinical testing of BH3 Stapled Peptides for IND submission and is developing additional Stapled Peptide therapeutics for oncology, metabolic diseases, immunological disorders and viral infections.Huw W. Nash, Ph.D., Vice President, Corporate Development, Aileron, Inc., USA
10:15 Networking Refreshment Break with Poster and Exhibit Viewing 10:45 Development of Terlipressin as an Orphan
Drug in the US CASE
STUDYAn overview of the status of peptides as active pharmaceutical ingredients (APIs) will be presented. As a recent case study, challenges related to manufacturing and regulatory aspects of the development of Terlipressin, which is currently approved in the EU and South East Asia and pending approval as an orphan drug in the US, will be discussed. Robert Hagopian, Director, Business Development, PolyPeptide Group, USA
11:15 Update on Hematide™, a New Peptidic ESA being Evaluated for the Treatment of AnemiaWe have discovered a novel synthetic peptide-based compound (Hematide) that is a potent erythropoiesis-stimulating agent (ESA). A novel linker-spacer couples the peptide portion to PEG and prolongs its duration of action. In partnership with Takeda, Hematide is in global Phase 3 trials to establish the safety and efficacy after administration every four weeks in predialysis, dialysis, and PRCA patients. Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USA
11:45 RaPID (Random Peptide Integrated Discovery) System under Reprogrammed Genetic Code RaPID (Random Peptide Integrated Discovery) System is a new emerging technology that enables the expression of non-standard peptides using the translation machinery and rapid discovery of unique peptide drug leads. Particularly, when it is coupled with a cell-free display system, referred to as RaPID display, such peptides can be selected from a library with more than 100 billions of complexity. The lecture describes recent development of this technology.Hiroaki Suga, Ph.D., Professor, Chemical Biology and Biotechnology Lab, The University of Tokyo, Japan
12:15 Networking Luncheon with Poster and Exhibit Viewing
UpdatesonOligo-BasedTherapeuticsinClinicalDevelopment
1:30 Chairperson’s RemarksJames D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
1:45 Anti-Fibrosis Therapy by siRNAAbstract unavailable at press date. Please visit www.IBCLifeSciences.com/AsiaTIDES for program updates.Yasunobu Tanaka, Ph.D., Manager, Hokkaido Laboratory, Corporate Business Development, Nitto Denko Corporation, Japan
2:15 Phase I Evaluation of Stable Nucleic Acid Lipid Particles Containing Anti-ApoB siRNA Stable Nucleic Acid Lipid Particles (SNALP) that potently silence the conventionally non-druggable target, apolipoprotein B, have been shown to protect mice from developing atherosclerotic lesions in preclinical models of cardiovascular disease. Findings of clinically relevant RNAi-mediated gene silencing support the evaluation of ApoB-SNALP in hypercholesterolemic patients. Early results from a Phase I clinical trial will be presented.Ian MacLachlan, Ph.D., Executive Vice President & Chief Scientific Officer, Tekmira Pharmaceuticals Corp., Canada
2:45 RNA Silencing: Realization and Novel ConceptsExecuting the therapeutic potential of RNA silencing is of great interest. A single stranded approach using short, stable and highly potent oligonucleotides is the most direct strategy. Locked Nucleic Acid (LNA) represents such a direct strategy, and LNA can equally well be used for mRNA and microRNA silencing. The latest silencing concepts and mechanisms will be presented, together with the status of our LNA R&D programs.Troels Koch, Ph.D., Vice President, Research, Santaris Pharma, Denmark
3:15 Networking Refreshment Break and Last Chance for Poster and Exhibit Viewing
3:45 Development of Decoy Oligonucleotide DrugWe have developed decoy ODN against NFkB, an essential transcription factor for inflammation and adhesion. Currently, Phase III trial to treat atopic dermatitis will be scheduled. In addition, clinical trials of decoy eluting medical device will be initiated soon. In this session, together with the present clinical situation of NFkB decoy ODN, our recent modified decoy, to overcome the short half life, low efficiency of uptake, and degradation by endocytosis, will be introduced.. Ryuichi Morishita, Ph.D., Professor, Dept. of Clinical Gene Therapy, Graduate School of Medicine, Osaka University; Founder, Board Member, AnGes MG, Inc., Japan
4:15 Clinical Development of Synthetic siRNAs for Ocular and Renal DiseasesQuark Pharmaceuticals has a robust clinical development pipeline of synthetic siRNAs that includes two siRNAs administered via intravitreal injection for ocular indications and one siRNA administered systemically for two renal indications. This talk will describe the design and manufacture of these synthetic siRNAs, and provide an overview of the clinical experience from the Phase I safety studies and an update on the status of the Phase 2 studies. James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
4:45 Development of the REG1 Anticoagulation System for Acute Coronary SyndromesREG1 is an anticoagulation system consisting of the aptamer-based anticoagulant, RB006, and its specific control agent, RB007. The REG1 system has been evaluated in 3 phase 1 studies and a phase 2a feasibility study in PCI. This presentation will include:• Overview of the Regado drug-control agent technology• Overview of the REG1 System• Summary of the clinical findings to dateChristopher P. Rusconi, Ph.D., Senior Vice President, Discovery/Preclinical Development and Chief Scientific Officer, Regado Biosciences, Inc., USA
5:15 Close of Tuesday Sessions
Visitwww.IBCLifeSciences.com/AsiaTIDESforup-to-dateinformationonthisevent 5
UpdatesonPeptide-BasedTherapeuticsinPreclinicalandClinicalDevelopmentCont.
1:30 Chairperson’s RemarksJesse Z. Dong, Ph.D., Senior Director, Medicinal Chemistry, Ipsen, USA
1:35 GLP-1 Analogue Taspoglutide: From Discovery to Clinical DevelopmentTaspoglutide, a human GLP-1 analogue, is currently in phase 3 clinical development for the treatment of type 2 diabetes. The peptide is designed to be stable against enzymatic degradation and compatible with sustained-release formulations. It is administrated as a novel sustained-release formulation intended to be used as once weekly subcutaneous injection. The molecule design, chemistry, clinical safety, tolerance and efficacy data will be discussed.Raffaella Balena, M.D., Ph.D. Clinical Science Leader, F. Hoffmann-La Roche Ltd, SwitzerlandJesse Z. Dong, Ph.D., Senior Director, Medicinal Chemistry, Ipsen, USA
2:15 A Bivalent Influenza Peptide Conjugate Vaccine Based on Highly Conserved EpitopesDeveloping improved influenza virus vaccines that provide broader and more durable protection is an important, international public health objective. We have developed peptide-conjugate vaccines based on the M2 and HA proteins that are highly conserved and provide protection from disease against heterologous influenza infections in preclinical studies. These data and results from an initial clinical trial will be reviewed. John W. Shiver, Ph.D., Vice President, Worldwide Basic Research Franchise Head, Vaccines, Merck Research Laboratories, USA
2:45 Clinical Development for Peptide-Based Therapeutic Cancer Immunotherapy Targeting Oncoantigen and Tumor AngiogenesisOncoantigen and tumor angiogenesis are crucial targets for therapeutic cancer immunotherapy. Basic data showed potent antitumor effect using epitope peptides derived from target-molecules. We have performed several clinical trials, including the pivotal study against pancreatic cancer and Phase II clinical trial against cholangiocellular carcinoma. Data will be demonstrated in detail and future potential for therapeutic immunotherapy will be discussed.Takuya Tsunoda, M.D., Ph.D., Chief Operating Officer, OncoTherapy Science, Inc., Japan
3:15 Networking Refreshment Break and Last Chance for Poster and Exhibit Viewing
UpdatesonDiagnosticsinDevelopmentChairperson: Peter Haima, Ph.D., Director, IVD Services, EUROGENTEC-group, USA
3:45 New SNPs Typing Method Based on Abasic Site-Containing Probe DNA and Fluorescent LigandsA new SNPs typing method is proposed based on abasic site (AP site)-containing DNAs (AP-DNA) and fluorescent ligands. At an AP site in an AP-DNA/target DNA duplex, fluorescent ligands can recognize target nucleobases selectively. Using two ligands with different color emission, allele genotyping is demonstrated using PCR amplified products without any pre-treatment.Norio Teramae, Ph.D., Professor, Chemistry, Tohoku University, Japan
4:15 Development of a New GMP TAQ Polymerase for Improved PCR PerformancePCR is the method of choice for many diagnostic applications. A new hot-start method was developed that improves the efficiency, specificity, yield and sensitivity of the PCR reaction. HOT Diamond TAQ™ is manufactured according to a GMP-Pharma process; resulting in an exhaustively characterized, lot-to-lot reproducible enzyme, with extremely low residual DNA content.Peter Haima, Ph.D., Director, IVD Services, EUROGENTEC-group, USA
4:45 Modified dNTPs for improved PCR Performance: An Innovative Use of Nucleoside Protecting GroupsPCR is widely applied in high specificity detection assays. This presentation describes an approach in which the dNTPs, an essential PCR component, are modified with thermolabile dNTP protecting groups for application to Hot Start activation schemes in PCR. Studies describe the development of this technology, including the proof of principle experiments that identified the optimal temperature-sensitive 3'-nucleoside protecting group. Natasha Paul, Ph.D., Senior Staff Scientist, R&D, TriLink BioTechnologies, Inc., USA
5:15 Close of Tuesday Sessions
Oligonucleotide-Based Therapeutics Worldwide Advances in Peptides & Diagnostics
6:00 Networking Dinner in TokyoJoin fellow attendees and speakers for a memorable evening out in Tokyo. Space is limited and an additional fee applies. Please check box on registration page to join the dinner.
Also consider attending
April 25-28, 2010 • Hynes Convention Center • Boston, MA, USACo-located with TIDES:
Applications of Nucleic Acids Technologies for Molecular DiagnosticsApril 27-28, 2010 • Hynes Convention Center • Boston, MA, USA
www.IBCLifeSciences.com/TIDES
Oligonucleotide and Peptide® Technology and Product DevelopmentTIDES
8:30 Chairperson’s RemarksMuthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc., USA
8:45 Chemical Strategies for Achieving Systemic Delivery of siRNAsAn update will be presented on our progress toward systemic delivery of small interfering RNAs (siRNAs) using various chemical approaches.Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc., USA
9:15 RNAi Chemical and Structural Optimization for Therapeutic Development Nucleotide chemistry is being used to improve therapeutic oligonucleotide properties such as potency, stability and specificity. Moreover, we have developed a new class of modified RNAi compound, self-delivering rxRNA (sd-rxRNA™), that is characterized by spontaneous cellular and tissue uptake. This translates to dramatically enhanced in vivo cellular uptake locally following direct administration and to liver following intravenous administration. Kinetics and mechanism of cellular uptake as well as preclinical development of sd-rxRNA™ will be discussed.Pamela A. Pavco, Ph.D., Vice President, Pharmaceutical Development, RXi Pharmaceuticals, USA
9:45 Planning for Success: Scale Up and Manufacturing of Stable Nucleic Acid Lipid Particles for Phase I and BeyondStable Nucleic Acid Lipid Particle (SNALP) products containing siRNA payloads are currently being evaluated in several clinical and preclinical studies. This presentation will describe the scale up, manufacturing and characterization of these products to enable initial clinical trials and will also provide an overview of strategies to further develop the platform for late-stage clinical trials and, ultimately, commercialization.Peter Lutwyche, Ph.D., Vice President, Pharmaceutical Development, Tekmira Pharmaceuticals Corporation, Canada
10:15 Networking Refreshment Break
10:45 Mimicking Endogenous Peptide Secretion Using Dry Powder InhalationTechnosphere® dry powder formulations in combination with a novel inhalation device comprise a versatile peptide delivery platform that mimics the pharmacokinetics of intra-arterial injection and is characterized by very rapid systemic drug absorption. This physiologic delivery profile provides advantages over the same drugs administered by injection and has applicability over a broad range of therapeutics agents including insulin, GLP-1, PYY, OXM, and PTH. Andrea Leone-Bay, Ph.D., Vice President, Pharmaceutical R&D, MannKind Corporation, USA
Intellectual Property Panel Discussion 11:15 Market Landscape: Demystifying Intellectual Property
for Oligos and Peptides around the WorldPanelists will discuss the most pressing issues in intellectual property for oligos and peptides in an audience interactive discussion. Topics to be discussed:1. Challenges involved with filing for patents and avoiding intellectual
property disputes. 2. Specific examples/case studies on how patent disputes are being dealt
with in the genomics area. 3. Peptides vs. oligos, how does IP landscape differ? Is there overlap?Moderator: Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, USA Panelists:Steven Lee Highlander, Partner, Fulbright and Jaworski L.L.P., USA Antonio Maschio, Ph.D., Partner, Edwards Angell Palmer & Dodge UK LLP, United Kingdom Kathleen M. Williams, Ph.D., J.D., Partner, Intellectual Property, Edwards Angell Palmer & Dodge LLP, USA
6 ToRegister,Tel:(+65)6514-3180•Fax:(+65)6733-5087•E-mail:[email protected]
Delivery Challenges and Solutions for Oligos and Peptide Therapeutics
Manufacturing and Analytical Development for Peptides and Oligos
MainConference•ConcurrentTracks Wednesday,February24,2010
Session Sponsor:
8:30 Co-Chairpersons’ RemarksSteven J. Prestrelski, Ph.D., M.B.A., Vice President, Pharmaceutical R&D, Amylin Pharmaceuticals, Inc., USAEmma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
8:45 The Oligonucleotide Development Journey CASESTUDYA discussion of oligonucleotide development strategies and
requirements through clinical phases, including the application of targeted process development, control of process changes and development of design history, raw materials sourcing tactics and the development of process controls and specifications. The presentation will include key examples to illustrate the areas covered.Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
9:15 The Challenges of Supply Chain Management with an Emerging TechnologyThe speaker will explore the numerous issues related to managing an uninterrupted supply of clinical material for a development company expanding the scope of the fundamental technology. Some of the topics will be: changing raw material requirements; limited visibility for forecasted production; flexible inventory management; capacity planning for an evolving product portfolio.David Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, Inc., USA
9:45 Cost-Effective Synthesis of siRNA and Aptamers Using CEM as the 2'-hydroxyl Protecting Group:
CASESTUDY
From Bulk-Scale CEM Amidites to OligoribonucleotidesWe have manufactured all four 2'-O-(2-cyanoethoxymethyl) (2'-O-CEM) amidites on a multi-kilogram scale and successfully used them for the synthesis of RNA oligonucleotides. Data will be presented demonstrating that CEM chemistry significantly improves the efficiency of manufacturing of RNA oligonucleotides with or without modified nucleotides by comparison with TBDMS chemistry. The syntheses of 21mer to 58mer RNA and modified aptamers from micrograms to grams for basic research and preclinical development have been accomplished.Kyeong Eun Jung, Ph.D., Director, Research Center, Samchully Pharm. Co. Ltd., Korea
10:15 Networking Refreshment Break 10:45 Exenatide Once Weekly: A New Therapy for Type 2 Diabetes
Exenatide Once Weekly (EQW) is a long-acting formulation of exenatide, the active ingredient in Byetta® (exenatide injection) based on the Medisorb technology from Alkermes, Inc. EQW has the potential to be a diabetes therapeutic with superior efficacy, tolerability and patient convenience. This presentation will provide an overview of aspects of drug development including formulation development, scale-up and commercial manufacturing and results of clinical studies.Steven J. Prestrelski, Ph.D., M.B.A., Vice President, Pharmaceutical R&D, Amylin Pharmaceuticals, Inc., USA
11:15 DSP Challenges in Peptides: Getting the Right Things DoneHPLC typically accounts for 20-50% of peptide production costs, forcing manufacturers to find trade-offs between yield and productivity, while keeping quality, robustness and flexibility. Several case studies will be discussed in which Lonza has tackled this challenge by introducing new process development and optimization tools, including impurity tracking, DoE techniques, and numerical modeling. Strategies for the integration of these tools into QbD methods will also be offered.Alessandro Butté, Ph.D., Group Leader Peptides R&D, Lonza AG, Switzerland
11:45 Applying Manufacturing Sciences to Increase Productivity and ProfitabilityProcess capability is adversely affected by system variation, which, in turn, renders the process to be irreproducible and products to be out of specifications, and eventually increases manufacturing costs. By using Six Sigma approach to systematically identify and to eliminate the root cause of variability in liquid chromatography processes, reduced manufacturing cost along with improved product quality can be realized.Michael Li, Ph.D., Manager, Process Sciences, Asahi Kasei TechniKrom, USA
12:15 Networking Luncheon
SponsorsandExhibitorsPlatinum Sponsor
Expertise.Innovation.Reliability. When your needs call for a contract services organization, Avecia OligoMedicines stands ready. Whether it’s in the development, manufacturing or analysis of therapeutic oligonucleotides, Avecia is
committed to exceeding your expectations. You can rely on our FDA-inspected facility to manufacture active pharmaceutical ingredients (API) that meet the international requirements of ICH Q7. We have more than 15-years experience synthesizing siRNA, aptamers, chimeras, DNA, RNA and molecular decoys. It’s no wonder we are the manufacturing partner for more than 1,000 different sequences at production scale. Whether you are in preclinical stages, or ready for a commercial launch, you can be assured of the best in both technology and service when you turn to the world-leading capabilities of Avecia.
Silver Sponsor Established in 1981, ChemGenes, the industry leader in Oligonucleotide Reagent manufacturing, has consistently provided the highest quality Phosphoramidites and Solid Supports in the market. Our Massachusetts
facility is setup for therapeutic grade phosphoramidites and DNA/RNA synthesis products for GMP grade oligonucleotide manufacturing. Currently, our bulk synthesis capacities are 150kg/base yearly for 2’-O-Methyl and DNA phosphoramidites and 60kg/base yearly for RNA phosphoramidites. Additionally, ChemGenes carries the widest variety of modified phosphoramidites and supports currently used in oligonucleotide synthesis including Microarray Technology, Oligonucleotide Therapeutics, Oligonucleotide Based Probes and other areas of Nucleic Acid research. ChemGenes remains devoted to providing you with invaluable customer service and comprehensive technical support.
Bronze SponsorsAgilent Technologies Nucleic Acid solutions Division (NASD), based in Boulder, Colorado is the leading innovator in providing flexible therapeutic oligo development services and manufacturing solutions. In addition to manufacturing
cGMP oligo materials Agilent offers an array of turn-key services, including development of analytical methods and processes, stability studies, and regulatory support. Focusing on long-term client partnerships, NASD has made significant investments in facilities and technical personnel to provide our customers with an unparalleled range of expertise and manufacturing capacity. Understanding that every client’s goals are unique, NASD’s team approaches every project to deliver the maximum benefit and value.
GeneDesign offers contract manufacturing of oligonucleotides including siRNA, Aptamer, Antisense (LNA/BNA), CpG oligo, decoy, IVD oligo from screening assay
use to over hundreds gram under the control of GMP guideline with sterilized condition. Through the process development, we can support your successful oligo therapeutics and diagnostics development.
Exhibitors (As of September 18, 2009)
Visitwww.IBCLifeSciences.com/AsiaTIDESforup-to-dateinformationonthisevent 7
InvestmentandLicensingOpportunties 1:30 Innovation Showcase
Co-Chairs: Gary Carter, Business Development Manager, Nucleic Acids Solutions Division, Agilent Technologies Inc., USADavid Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, Inc., USAFour emerging drug development and technology companies will give 15-minute updates on their business plan, technology, and partnering objectives. For more information on how to present in the Innovation Showcase, please see guidelines on the website or contact Ellen C. King of IBC Life Sciences at [email protected]
2:30 Networking Refreshment Break
RegulatoryUpdates:StrategiesforNavigatingtheApprovalPathway
3:00 Chairperson’s RemarksG. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
3:15 Regulatory Considerations in the Development of RNAi TherapeuticsRegulatory issues in the development of small interfering RNA (siRNA) as therapeutics will be discussed from the perspective of nonclinical pharmacology and toxicology as well as chemistry, manufacturing and controls (CMC). Experience with FDA as well as with European and Canadian authorities in regulatory filings to enable Phase 1 and Phase 2 clinical trials will be shared. Saraswathy (Sara) V. Nochur, Ph.D., Vice President, Regulatory Affairs, Alnylam Pharmaceuticals, Inc., USA
3:45 Considerations for the CMC Development of Oligonucleotides in Complex Delivery SystemsRecent advances in oligonucleotide-based therapeutics include the development of complex formulations for targeted delivery to various sites. Utilisation of these novel systems for the delivery of complex oligonucleotides presents exceptional challenges for characterisation and control. This presentation will provide strategies for addressing the regulatory expectations for assuring quality and performance of these delivery systems in support of preclinical and clinical development.G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
4:15 Development of Peptide-Based Cancer Vaccines up to Phase II – Regulatory Challenges and OpportunitiesIMA901 for the treatment of renal cell carcinoma and IMA910 for the treatment of colorectal cancer are two product candidates currently developed in two ongoing phase II cancer vaccine trials in Europe. Both immunotherapy candidates consist of 10 and 13 HLA-restricted tumor-associated peptides (TUMAPs), respectively. We will report on regulatory challenges and opportunities in this "multi-peptide multi-national multi-center" clinical development setting.Peter Lewandrowski, Ph.D., Director and Head of CMC, immatics biotechnologies GmbH, Germany
4:45 Close of AsiaTIDES®
MainConference•PlenarySession Wednesday,February24,2010(continued)
ConferenceLanguageTheconferencewillbeconductedinEnglishwithouttranslation.
InterestedinSponsoringorExhibiting?• Gain international exposure through our specialized marketing campaign • Network with international players in the industry• Meet face-to-face with top-notch researchers and executives from
pharmaceutical and biotechnology companies• Showcase your latest technology to our targeted audience of
key decision-makers• Build invaluable relationships and form new partnerships that give you a
competitive advantage in the US, Europe and Asia-PacificAsia-PacificSalesContact: CatherineWong,Tel:+65-6835-5134
E-mail:[email protected].&EuropeanSalesContact: SherryJohnson,Tel:+1-508-614-1451
E-mail:[email protected]
AbouttheOrganizersThis event is brought to you by the organizers of TIDES Peptide and Oligonucleotide® Technology and Product Development and EuroTIDES conferences, as well as Drug Discovery and Development of Innovative Therapeutics World Congress in Boston and Japan, IBC Life Sciences, an informa business. Other informa businesses include: Datamonitor, MedTRACK, SCRIP and Pharmaprojects. www.IBCLifeSciences.com and www.informa.com
CallforPosters Limited space is available for poster presentations at this event. If you have new results/data on topics relevant to this conference, we encourage you to submit a poster abstract for consideration. To present a poster, complete the conference registration form and submit poster title and one page poster abstract online at www.IBCLifeSciences.com/AsiaTIDES by February 2, 2010. See registration form for poster fees.
MakingHotelReservationsSpecial room rates have been contracted with Sheraton Miyako Hotel Tokyo for IBC’s delegation. To take advantage of this special rate, please visit the conference website at www.IBClifesciences.com/AsiaTIDES and select the Hotel Info Link. More information on how to reserve your room is available there.
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On or before On or before On or before Standard RateRegistrationFees November 20, 2009 December 18, 2009 January 22, 2010 After January 22, 2010
Industry Main Conference plus Monday morning* o USD1699 o USD1899 o USD1999 o USD2099Main Conference Only (Mon afternoon - Wed) o USD1399 o USD1599 o USD1699 o USD1799
Academia/Government Discounts – 40% SavingsRate extended to full-time employees of government, universities and university-affiliated hospitals who have NO affiliation to a for profit entity. Visit the registration page at www.IBCLifeSciences.com/AsiaTIDES for academic packages and discounted pricing information.
Present a Poster o USD100 - Commercial o FREE - Academic *PleaseselecttheMondaymorningpre-conferenceoptionyouwishtoattend:
o Tutorial #1: Analysis and Bioanalysis of Oligonucleotides in Support of Drug Development o Tutorial #2: Scaling up Oligonucleotide Manufacturing Process o Plenary Session of Drug & Biologics Japan Conference
Pleasetellusyourprimaryareaofinterest:o Oligonucleotides o Peptides o Diagnostics
Attend Networking Dinner o USD 120
46103
46150B