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TIDES®
TIDES® EVENT INFOMATION
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IBC Life Science主辦
國際會議。展示會 TIDES®
Oligonucleotide and Peptide® Technology and Product Development
2008年5月18日(日)∼21日(三)
美國、拉斯維加斯、Red Rock Resort and Spa
| Pre-Conference Courses Sunday, May 18, 2008 | PRE-CONFERENCE COURSES | DAY ONE | DAY TWO | DAY THREE | | ||||
| 7:45 | Registration and Coffee | |||
| Full Day Course: 9:00 am - 4:00 pm | ||||
| 1 | Quality Assurance Considerations for Peptide and Oligonucleotide Manufacture | |||
| Instructor: K.A. Ajit-Simh, President, Shiba Associates | ||||
| In this short course, you will learn how to implement "graded" GMPs for the manufacture and control of peptides and oligonucleotides. The presentation will include current industry trends and practices as it relates to the checks and balances required for manufacturing under "appropriate" standards of quality. Examples from several manufacturing audits will be used to illustrate the compliance challenges faced by companies and how they were addressed effectively. This course is designed for those who are currently in R&D, who are synthesizing oligos and peptides for parenteral applications - particularly those who are contracting CMOs. It will benefit people who know GMPs but need more information on how to apply them in a phase-appropriate manner. Representatives of CMOs and sponsor companies alike will learn how to interpret and apply the regulations, guidelines and industry standards - and will gain a better understanding of each others' expectations. By attending this course you will:
Outline:
About the Instructor | ||||
| Morning Courses: 9:00 am - 1:00 pm | ||||
| 2 | Bioanalytical Techniques for Characterization of Peptides and Oligos | |||
| Course Leaders: Sandra Carriero, Ph.D., Senior Research Scientist/Manager, Immunochemistry, Laboratory Sciences, Charles River Laboratories, Preclinical Services Montreal Inc., Canada Hans Gaus, Ph.D., Director, Structural Biology, Isis Pharmaceuticals, Inc. | ||||
| Bioanalytical Methods for Oligonucleotide Therapeutics Selection of the Appropriate Bioanalytical Tool for Oligonucleotide Quantitation - Limitations and Considerations IND-Enabling Bioanalytical Strategies: Case Histories for Indications Involving Local and Systemic Administration of siRNAs Overcoming Bioanalytical Challenges of Peptide Therapeutics Panel Discussion with all Course #2 speakers. | ||||
| 4 | Introductory Course on the Formulation of Peptides and Oligonucleotides to Support Clinical Trials | |||
| Co-Instructors: Andrew Dibble, Ph.D., Associate Director, Pharmaceutical Development, Isis Pharmaceuticals, Inc. Qiang Ye, Ph.D., Associate Director, Product Development, Amylin Pharmaceuticals, Inc. | ||||
| This half-day course will teach you fundamentals of the drug development process to take a peptide or oligo NCE into the clinic. The focus will be on preformulation and formulation development activities relevant to parenteral products, but related CMC aspects and alternate dosage forms will be discussed briefly as well. Topics to be covered include:
About the Co-Instructors Andrew Dibble has over ten years industry experience in formulation development, working at Alza, Cardinal Health, and now Isis. Andrew has a Ph.D. in biochemistry from Cornell University, where he studied peptide-lipid interactions. His postdoctoral research at the University of Virginia explored the biophysical effects of peptide and protein binding to model membranes. While in industry, Andrew has worked on preformulation and formulation development of a variety of types of compounds, including anthracycline antibiotics, platinum coordination complexes, glutathione analogs, carbohydrates, pyrimidine nucleoside analogs, antisense oligonucleotides, and siRNAs. Qiang Ye has over thirteen years of pharmaceutical development experience in preformulation and formulation, working at DepoTech, Agouron, Pfizer, and now Amylin. Qiang has a Ph.D. in Biophysics (Interdisciplinary Program) from University of Virginia. He has published many research articles, review chapters, poster/podium presentations, and also filed eight US patents for novel pharmaceutical formulations (with emphasis on protein and peptide drugs). Over the years, Qiang has led preformulation and formulation development of >20 protein, peptide, small molecule, and oligonucleotide therapeutics and has gained extensive first-hand drug development experience at all stages, from Discovery to Preclinical to Phases 1, 2, and 3 to US NDA and EU MAA filing and approval. Additional speakers to be announced. | ||||
| Afternoon Courses: 2:00 pm - 6:00 pm | ||||
| 3 | Analytical Technologies for Peptides and Oligos | |||
| Chairperson: Daniel Capaldi, Ph.D., Executive Director, Development Chemistry, Isis Pharmaceuticals, Inc. | ||||
| The Course will illustrate how analytical chemistry contributes to the development of oligonucleotide and peptide therapeutics. The Course will comprise five separate presentations, each describing the application of novel analytical techniques to various aspects of oligonucleotide and peptide chemistry. Participants will learn about analytical methods that can be used to help understand and control the oligonucleotide starting material supply chain. Methods suitable for assessing the purity and stability of PEGylated peptides and single-stranded oligonucleotides will be presented. A novel ion-pair HPLC mass spectrometry method useful for characterizing RNA and DNA duplexes and a technique for assessing levels of aberrant 2´-5´ internucleotide linkages in RNA oligonucleotides will be described. The Course will conclude with a roundtable discussion. Using Analytical Controls to Design Quality into the Supply Chain of Therapeutic Oligonucleotides Characterization of PEGylated Peptides Methods for Assessing the Stability of Oligonucleotide Therapeutics Analytical Characterization of DNA & RNA Duplexes On-Line Preparation of Anion-exchange Separated Oligonucleotides for Mass Spectrometry Panel Discussion | ||||
| 5 | Manufacturing RNA Oligonucleotides: Can One Hydroxyl Really Make That Much of a Difference? | |||
| Course Leader: Fran Wincott, Ph.D., President, Wincott & Associates, LLC | ||||
| This course will address the processes by which RNA containing oligonucleotides are manufactured for preclinical studies and clinical trials. It is designed to illustrate the differences between manufacturing RNA and DNA and to give a broad overview of process needs and challenges facing manufacturers of RNA molecules through best practice strategies and case study examples. Participants will learn:
Representatives from major CMOs will provide their perspectives on RNA manufacturing, process development and scale-up - what they think you ought to know. You will also hear the sponsor's perspective on decisions regarding in-house manufacturing vs. external as well as when and how to execute technology transfer. The course will conclude with a roundtable discussion. Speakers: | ||||
| 6 | A Lifecycle View of Writing and Filing a CTD/eCTD Submission | |||
| Co-Instructors: James Blackwell, Ph.D., Senior Consultant, and Alex Kanarek, Ph.D., Quality Consultant, BioProcess Technology Consultants, Inc. | ||||
| In this short course, you will learn the key insights into writing and managing a CTD/eCTD submission filing from a lifecycle viewpoint -- from early development through commercialization. The focus will be on CMC ("Chemistry, Manufacturing, and Controls") sections for "tides" compounds, but insights will be gained that are beneficial for writing other sections. Writing and filing INDs, NDAs/BLAs are among the most important and challenging of drug development activities. Learn proven methods and gain a roadmap to resources available to assist with CTD submissions and speed time to market. By attending this course you will:
Agenda / Outline:
About the Instructors Alex Kanarek, Ph.D. has more than 30 years of experience in the biopharmaceutical industry with the Wellcome Foundation (now GlaxoSmithKline) and Connaught Laboratories (now Sanofi Pasteur) and has been an industry consultant since 1993. He has authored several GMP industry guides and he is now on the editorial advisory board of the BioProcess International journal. James Blackwell, Ph.D. has more than 16 years industry experience and has held senior technical and management positions with Repligen, Genzyme, and Abbott Laboratories and has been an industry consultant for several years. He is Past President of the Boston Chapter of ISPE and currently leads its Process and Product Development Community of Practice at the International level. He is on the advisory board of Pharmaceutical Processing. Their experience in development, manufacturing, and quality covers small and large molecules, and they have been involved with writing, filing and post-approval support for many filings. | ||||
| Day One Monday, May 19, 2008 | PRE-CONFERENCE COURSES | DAY ONE | DAY TWO | DAY THREE | | ||||
| Plenary Sessions | ||||
| 7:45 | Registration and Coffee | |||
| 8:45 | Chairperson's Introductory Remarks Fran Wincott, Ph.D., President, Wincott & Associates, LLC | |||
| Keynote Presentations | ||||
| 9:00 | FDA's Current Expectations on the Chemistry, Manufacturing and Controls of Peptide- and Oligonucleotide- Based Therapeutics Many peptide- and oligonucleotide-based products have been approved for clinical use as either therapeutic or as diagnostic agents. By their very nature, they have unique characteristics and provide exciting opportunities as well as many challenges. The presentation will discuss FDA's current expectations for the Chemistry, Manufacturing and Controls of peptide- and oligonucleotide-based therapeutic products, including characterization, control of the manufacturing process, specifications for drug substance, stability, reference standards, impurities, and specifications for the finished material. Blair A. Fraser, Ph.D., Director, Division of Pre-Marketing Assessment I, Office of New Drug Quality Assessment, Office of Pharmaceutical Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration | |||
| 9:45 | Merck's Perspective on siRNA as a Therapeutic Modality Big pharma companies have recently engaged in billion dollar alliances with companies with expertise in siRNA (small interfering RNA). Merck's recent purchase of Sirna Therapeutics is one example showing that big pharma has confidence that therapeutic siRNA can be harnessed to open a new frontier for drug development. Hear Merck's perspective on the utility of RNA therapeutics, including opportunities for vendors and external parties in the RNA synthesis, delivery, and pre-clinical space—and why the potential is so great. Alan Sachs, M.D., Ph.D., Vice President, RNA Therapeutics, Merck Research Laboratories | |||
| 10:30 | Networking Refreshment Break Sponsored by  | |||
| Featured Presentations | ||||
| 11:00 | Opportunities and Challenges of Developing Peptide Drugs in the Pharmaceutical Industry The peptide market is growing nearly twice as fast as that for all other types of pharmaceuticals due to the increased number of therapeutic targets and improved delivery methodologies. There are 67 therapeutic peptides on the market, 150 in clinical phases and 400 in preclinical stages. The market sizes for 2007 is estimated to be 3.01 billion US$. The important factors requiring consideration before starting peptide-based projects, including de-risking strategies, unmet medical needs, the competitive landscape and COG, are analyzed in this presentation. Waleed Danho, Ph.D., Distinguished Research Leader, Discovery Chemistry, Hoffmann-LaRoche Inc. | |||
| 11:30 | Toward a Non Coding RNA Revolution in the Cancer Society Alterations in miRNA genes play a critical role in the pathophysiology of many, perhaps all, human cancer: cancer initiation and progression can involve microRNAs. At the present time, the main mechanism of microRNoma alteration in cancer cells seems to be represented by aberrant gene expression, characterized by abnormal levels of expression for mature and/or precursor miRNA sequences in comparison with the corresponding normal tissues. Riccardo Spizzo, M.D., Senior Research Associate, The University of Texas M.D. Anderson Cancer Center | |||
| 12:00 | What Can Molecular Profiling Tell us About the Specificity of RNA Therapeutics? The specificity and efficacy of siRNAs, miRNAs, and anti-miRs will be critical factors for the realization of RNAi-based therapeutics. We have used gene expression profiling to characterize the specificity of these reagents in cultured cells and in animals. We discuss the implications of these findings for the design, chemical modification, and development of these small nucleic acids as therapeutics. Aimee Jackson, Ph.D., Senior Research Fellow, Molecular Profiling, Rosetta Inpharmatics, LLC., a wholly-owned subsidiary of Merck & Co., Inc. | |||
| 12:30 | Lunch on your own | |||
| Concurrent Break-Out Discussion Sessions | ||||
| 2:00 | Choose from 3 topics for in-depth discussion and your opportunity to ask questions to the experts: | |||
| Regulatory Expectations for CMC for Peptides and Oligos | ||||
| Moderator: G. Susan Srivatsa, Ph.D., President, ElixinPharma Panelists: Blair A. Fraser, Ph.D., Director, Division of Pre-Marketing Assessment I, Office of New Drug Quality Assessment, Office of Pharmaceutical Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration Dr. René Thürmer, Deputy Head Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany James V. McArdle, Ph.D., Vice President, Chemistry, Manufacturing and Controls, Archemix Corporation Seppe De Gelas, Head of Quality Assurance and Regulatory Affairs, Lonza Braine, Belgium Michael Verlander, D. Phil., Executive Vice President, PolyPeptide Laboratories, Inc. | ||||
| Patent Strategies in Light of the New Rules | ||||
| The patent office promulgated new rules that have significant impact on patent prosecution and litigation. This panel will discuss the implication of the rules that are stayed pending the outcome of the litigation with the PTO, in addition to several new cases regarding obviousness and details of written description. Attend to understand how to avoid new potential pitfalls and learn solutions for development of oligonucleotides, peptides and in therapeutics. Moderator: Richard Warburg, Ph.D., Partner, Foley & Lardner LLP Panelists: Janet M. McNicholas, Ph.D., Partner, Bell, Boyd and Lloyd, LLP Chris Steinhardt, Partner, Knobbe Martens Olson and Bear LLP Additional panelists to be announced | ||||
| miRNA: Opportunities and Challenges | ||||
| Discuss these questions, as well as your own:
Moderator: Aimee Jackson, Ph.D., Senior Research Fellow, Rosetta Inpharmatics, LLC Panelists: Troels Koch, Ph.D., Vice President, Research, Santaris Pharma A/S, Denmark William S. Marshall, PhD., President and Chief Executive Officer, Miragen Therapeutics Christine Esau, Ph.D., Project Leader, microRNA Biology, Regulus Therapeutics (invited) Alan D. Schreiber, M.D., Manager, ZaBeCor Pharmaceutical Company, LLC | ||||
| 3:30 | Networking Refreshment Break Sponsored by | |||
| 4:00 | Chairperson's Introduction of Highlight Keynote Speaker Masad J. Damha, Ph.D., F.C.I.C., James McGill Professor of Chemistry, McGill University, Canada | |||
| Highlight Keynote Presentation | ||||
| 4:10 | Companion Diagnostics: The Future Is Near The emergence of Companion (and Personal) Diagnostics has been slower than expected, yet the notion remains alive and healthy. Strong examples exist - Lipitor, anti-HIV drugs, and Herceptin are prescribed with companion diagnostics, more or less representing the breadth of the paradigm. The talk will focus on how proteomics broadly enable companion diagnostics by providing Biomarker Discovery for drug choice, dose, initial and sustained efficacy, and toxicity. Larry Gold, Ph.D., CEO and Chairman, SomaLogic, Inc. | |||
| 5:00 | Poster and Exhibit Hall Opens with Reception Network with over 700 participants, learn about new products and services from over 80 suppliers and partners, and view posters with cutting-edge new science. | |||
| Day Two Tuesday, May 20, 2008 | PRE-CONFERENCE COURSES | DAY ONE | DAY TWO | DAY THREE | | ||||
| TIDES® Main Conference | ||||
| Process and Analytical Development: Phase 2 and Beyond | ||||
| 7:30 | Networking Coffee | |||
| 7:55 | Co-Chairperson's Remarks Christopher P. Holmes, Ph.D., Senior Director, Chemistry, Affymax, Inc. Paul McCormac, Ph.D., Director, Process and Analytical Development, Avecia OligoMedicines | |||
| Featured Presentation | ||||
| 8:00 | Clinical Phase-Based Approach to the Method Validation Continuum - Risk and Risk Mitigation Analytical development follows a continuum from pre-clinical examination to commercialization, punctuated by clinical studies. At points, appropriate to the clinical phase, method validation moves to a higher state of completion. Since patient safety is the overriding concern during clinical development, risk evaluation and management must take place vis-à-vis safety standards, such as toxicology, and the necessary validation must, as well, be consistent. Roger P. Micheli, Ph.D., Manager, Analytical Research and Regulatory Affairs, Roche Colorado Corporation | |||
| 8:30 |
The oligonucleotide therapeutic product life cycle requires API through Drug Product quality being assessed at multiple laboratories and on multiple instrument platforms. Some of the key challenges due to the resulting analytical variability encountered during method qualifications and subsequent validations will be discussed. Traditional versus statistical approaches to identify, address and predict these site-to-site adaptations will be elucidated for a set of case studies. Ipsita Roymoulik, Ph.D., Senior Analytical Chemist, Quality Control, Avecia Biotechnology | |||
| 9:00 |
Implementing a smooth transfer of manufacturing technology is critical to future GMP campaigns. While risk assessment plays a guiding role in the transfer process, it is not the only consideration in this essential partnership. The technology transfer process will be examined from the customer's perspective, and the resulting successful campaign will be described. Paul J. Hatala, Ph.D., Principal Investigator/CMC, Archemix Corp. | |||
| 9:30 |
A common strategy for ensuring adequate manufacturing capacity and security of supply is to source the same API from several manufacturers. While mitigating supply risk, this strategy significantly complicates management of the CMO relationship, particularly during process lock-down. This presentation will highlight the regulatory, intellectual property and technical challenges associated with using multiple manufacturers and how issues can be resolved. Michael S. Holfinger, Ph.D., Director, API Manufacturing and Process Development, Affymax, Inc. | |||
| 10:00 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 10:45 |
Many factors should be considered in process scale-up, such as the selection, sourcing, and justification of starting materials, minimization of processing bottlenecks, and development of sound analytical methodology. The synthetic process to prepare ABT-510, a nonapeptide angiogenesis inhibitor, evolved significantly as the program advanced into the clinic, with the primary goal always in sight. Highlights from this development effort will be discussed. John C. Tolle, Senior Group Leader, Global Pharmaceutical Products Division Process R&D, Abbott Laboratories | |||
| 11:15 | Process Change in Peptide Synthesis: Impact on Comparability and Global Regulatory Submissions Process changes in peptide synthesis will be discussed and the impact of making changes to an established process will be examined. Depending on the change the key quality attributes required for objective evaluation to show pre- and post-change product comparability should be carefully examined. The regulatory impact of changes for global submissions will be discussed. Gopal Krishna, Ph.D., Senior Director Technical Operations, The Medicines Company | |||
| 11:45 | Technology Transfer for Peptide Large Scale Manufacturing: Technical, Quality and Regulatory Challenges Large scale manufacturing of peptide API's at multi 10 or 100 Kg requires particular expertise during the transition between process development and manufacturing. Moreover pharmaceutical sponsors seek for security of supply through multiple production sites or between different CMO's. Our experiences of internal transfer and site transfer for API undergoing validation or registered will be presented. Regulatory requirements will be emphasized. Marie-Hélène Brichard, Ph.D., Head of Process Development, Lonza Braine, Belgium Oleg Werbitzky, Ph.D., Head, R&D Peptides and Oligonucleotides, Lonza Valais Works, Switzerland | |||
| 12:15 | Lunch and Roundtable Discussions in Poster and Exhibit Hall | |||
| 1:45 | Afternoon Chairperson's Remarks James Powell, General Manager, Agilent Technologies Jane Salik, President, PolyPeptide Laboratories, Inc. | |||
| 2:00 |
An increasing number of therapeutic candidates at various stages of development as well as of their licensing history are constantly contributing to the challenges faced by CMOs. The advantages and disadvantages of various approaches to the scale up of oligonucleotide manufacturing and process development will be investigated based on selected case studies. Tadeusz K. Wyrzykiewicz, Ph.D., Oligonucleotides Supervisor, Girindus America Inc. | |||
| 2:30 | Applying Process Analytical Technology to Increase Process Efficiency in Peptide Purification Operations Application of PAT in biotech processes has been seen by many as particularly challenging due to the complexities of the typical biotech product. A process scale chromatography step in a peptide manufacturing process will be presented to demonstrate the application of on-line HPLC as a PAT to enable increased product yield, decreased process variability, and decreased process cycle time. Rick E. Cooley, Manager, Process Analytics Center of Excellence, Dionex Corporation; Eli Lilly and Company (retired) | |||
| 3:00 |
The FDA is supporting Process Analytical Technology (PAT) innovation leading to a risk-based approach in drug development, API manufacturing and process control. The complexity and potential variability of oligonucleotide synthesis and downstream processing make this area of drug development ripe for applications of PAT. Opportunities for PAT in oligonucleotide API development and manufacturing will be presented with an example application. Paul Metz, Senior Director, Manufacturing Operations, Agilent Technologies Inc. | |||
| 3:30 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 4:00 | Process Lock-Down: Lock Late and Manage Change It is accepted that manufacturing processes will change as a compound progresses through development. Changes may be required to lower costs, facilitate scale-up, improve purity, or in response to changes in the supply chain. If the preceding is expected, how and when can we lock our processes? The presentation will describe potential process changes and Isis' strategies for managing them. Anthony Scozzari, Vice President, Development Chemistry, Isis Pharmaceuticals, Inc. | |||
| 4:30 |
Lyophilization is often a challenging and costly manufacturing step for oligonucleotides. This talk will discuss possible manufacturing and storage alternatives to the lyophilization of oligonucleotide drug substance. An overview of the design of experiments, potential benefits, and stability results for an oligonucleotide in development will be presented. Adam Schwab, Senior Scientist, Global Manufacturing Services, Pfizer Inc | |||
| Audience Interactive Panel Discussion | ||||
| 5:00 | Pay Now or Pay Later - Process Development Strategies Co-Moderators: James Powell, General Manager, Agilent Technologies Jane Salik, President, PolyPeptide Laboratories, Inc. Panelists: Rick E. Cooley, Manager, Process Analytics Center of Excellence, Dionex Corporation; Eli Lilly and Company (retired) Tim Lannan, Senior Manager, Global Manufacturing Services, Pfizer Inc Paul McCormac, Ph.D., Director, Process and Analytical Development, Avecia OligoMedicines Roger P. Micheli, Ph.D., Manager, Analytical Research and Regulatory Affairs, Roche Colorado Corporation Anthony Scozzari, Vice President, Development Chemistry, Isis Pharmaceuticals, Inc. | |||
| 5:30 | Networking Reception in Poster and Exhibit Hall Dedicated Poster Viewing: Poster presenters are requested to be present at their posters. | |||
| Development Strategies of Leading Oligos in Clinical Development | ||||
| 7:30 | Networking Coffee | |||
| 8:25 | Chairperson's Remarks Bob D. Brown, Ph.D., Vice President, Research and Technology, Genta Incorporated | |||
| Featured Presentation | ||||
| 8:00 | Clinical Phase-Based Approach to the Method Validation Continuum - Risk and Risk Mitigation Analytical development follows a continuum from pre-clinical examination to commercialization, punctuated by clinical studies. At points, appropriate to the clinical phase, method validation moves to a higher state of completion. Since patient safety is the overriding concern during clinical development, risk evaluation and management must take place vis-à-vis safety standards, such as toxicology, and the necessary validation must, as well, be consistent. Roger P. Micheli, Ph.D., Manager, Analytical Research and Regulatory Affairs, Roche Colorado Corporation | |||
| 8:30 | Mipomersen: A First-in-Class Antisense Inhibitor of Apolipoprotein B Elevated plasma concentrations of apoB-containing atherogenic lipoproteins, e.g. LDL, are associated with increased risk for cardiovascular disease. Mipomersen is a 2'MOE modified antisense oligonucleotide designed to inhibit synthesis of human apoB-100 by the liver. Mipomersen has demonstrated equivalent pharmacological responses and favorable safety profiles in all hypercholesterol phenotypes tested in Phase 2. These results will be highlighted in this presentation. Brenda F. Baker, Ph.D., Director, Clinical Development, Isis Pharmaceuticals, Inc. | |||
| 9:00 |
Randomized trials including over 2,000 patients have yielded significant insights into the clinical utility of oblimersen (Genasense®) and more generally, about the systemic administration of oligonucleotides. Populations now can be identified by biomarker or clinical criteria as likely to benefit disproportionately from oblimersen. We have developed new schedules and routes of administration that enhance oligonucleotide uptake, while improving convenience and scheduling with other drugs. Preclinical and clinical results leading to the design of new trials and initial data will be presented. Bob D. Brown, Ph.D., Vice President, Research and Technology, Genta Incorporated | |||
| 9:30 | Development of an siRNA Targeting p53 to Protect Cells from Acute Injury AKIi-5 is a siRNA that temporarily inhibits expression of p53 and is the first siRNA to be administered systemically to patients. In acute settings such as renal failure following cardiac surgery, the temporary inhibition of p53 delays induction of cell death, thereby allowing natural repair mechanisms to restore cellular integrity. This talk will review the clinical programs for this siRNA that have been identified based on distribution following either local or systemic administration. Shai Erlich, Ph.D., Chief Development Officer, Quark Pharmaceuticals, Inc. | |||
| 10:00 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 10:45 | Progress on Clinical and Preclinical Studies of Defibrotide Defibrotide (DF) is a polydisperse mixture of 90% single-stranded polydeoxyribonucleotides. Actually, defibrotide is in Phase 3 clinical development in the US for the Treatment of severe hepatic Veno-Occlusive Disease (sVOD). Other trials on Prevention of VOD and Treatment of MM are starting and ongoing. Massimo Iacobelli, M.D., Senior Vice President and Scientific Director, Gentium S.p.A., Italy | |||
| 11:15 | Manipulating the Immune Response with Cell Penetrating Peptide Conjugated Phosphorodiamidate Morpholino Oligomers Antisense delivery to T cells and dendritic cells has been limited. Arginine-rich peptides (ARP) facilitate delivery of phosphorodiamidate Morpholino oligomers (PMO) into leukocytes. ARP-PMO targeted to genes expressed in T-cells and dendritic cells have been effective in diminishing allergic contact dermatitis, prevent development of type I diabetes in the NOD mouse and promote survival in Ebola virus lethal challenge models. Patrick Iversen, Ph.D., Senior Vice President, Research and Development, AVI BioPharma, Inc. | |||
| 11:45 | Development of the REG1 Anticoagulation System REG1 is an anticoagulation system consisting of the aptamer-based anticoagulant, RB006, and its specific antidote, RB007. The REG1 system has been evaluated in 3 Phase 1 studies and is currently being evaluated in Phase 2 proof-of-concept studies. This presentation will include the following:
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| 12:15 | Lunch and Roundtable Discussions in Poster and Exhibit Hall | |||
| Optimization Strategies of Leading Oligos in Preclinical Development | ||||
| 1:45 | Chairperson's Remarks James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc. | |||
| 2:00 | MicroRNA Manipulation as a Therapeutic Strategy for Cardiovascular Disease Heart failure remains a major unmet medical need, with more than 20 million patients worldwide and a 50% five year mortality rate. Recent studies have demonstrated the fundamental importance of microRNA perturbations in several forms of cardiovascular disease. By correcting microRNA levels we hope to develop breakthrough therapies that target the fundamental pathways responsible for this progressive, fatal condition. William S. Marshall, Ph.D., President and Chief Executive Officer, Miragen Therapeutics | |||
| 2:30 | Therapeutic Targeting of MicroRNAs with Antisense Oligonucleotides Regulus Therapeutics is focused on discovery of oligonucleotide therapies that modulate microRNA, shown to have important roles in many areas of biology, including cancer, metabolism, immunity, and others. Antisense targeting of miR-122 for HCV and metabolic disease is the most advanced preclinical program within Regulus, and progress in its development will be presented. Christine Esau, Ph.D., Project Leader, microRNA Biology, Regulus Therapeutics | |||
| 3:00 | Development of RNAi Therapeutics The design and effective delivery of synthetic RNAi compounds are critical factors for the successful use of RNAi in the clinic. Reduction of SOD1 using local (intrathecal) administration represents a potential therapeutic strategy for treating ALS, and nanoparticle formulations enable the distribution via systemic administration. Target reduction in mouse liver and other tissues at doses ≤1 mg/kg will be presented. Pamela A. Pavco, Ph.D., Vice President of Pharmaceutical Development, RXi Pharmaceuticals Corp. | |||
| 3:30 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 4:00 | Preclinical Development of Excellair™ as an siRNA Therapeutic for Asthma and Inflammation ZaBeCor is developing Excellair™, a siRNA directed at Syk kinase - an integral signaling molecule in multiple inflammatory pathways. Syk, a 72 kDa cytosolic protein tyrosine kinase expressed in many cell types, is critical to the signaling pathways of many receptors involved in inflammation in the lung and elsewhere. We will describe the development of Excellair™ leading to the initiation of clinical studies. Alan D. Schreiber, M.D., Manager, ZaBeCor Pharmaceutical Company, LLC | |||
| 4:30 | Single Stranded RNA Inhibition Improved oligonucleotide analogues - like LNA - have opened new perspectives for inhibition of RNA expression. A Single stranded approach with LNA offers a potent strategy to interfere with the expression of mRNA and microRNA. The presentation will cover:
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| 5:00 | Mechanisms of Immune Stimulation by RNA Innate immune defenses that can be activated by certain sequences or structures in RNA include Toll-Like Receptors (TLRs), RIG-I-Like Receptors (RLRs) and NOD-Like Receptors (NLRs). The immune stimulatory effects may be employed as vaccine adjuvants, or as therapeutics for cancer or infectious disease, but cause unwanted toxicities in other applications, such as RNAi. Arthur M. Krieg, M.D., Chief Scientific Officer and Executive Vice President of R&D, Coley Pharmaceutical Group, Inc. | |||
| 5:30 | Networking Reception in Poster and Exhibit Hall Dedicated Poster Viewing: Poster presenters are requested to be present at their posters. | |||
| Development Strategies of Leading Peptides in Clinical Development | ||||
| 7:30 | Networking Coffee | |||
| Featured Presentation | ||||
| 8:00 | Clinical Phase-Based Approach to the Method Validation Continuum - Risk and Risk Mitigation Analytical development follows a continuum from pre-clinical examination to commercialization, punctuated by clinical studies. At points, appropriate to the clinical phase, method validation moves to a higher state of completion. Since patient safety is the overriding concern during clinical development, risk evaluation and management must take place vis-à-vis safety standards, such as toxicology, and the necessary validation must, as well, be consistent. Roger P. Micheli, Ph.D., Manager, Analytical Research and Regulatory Affairs, Roche Colorado Corporation | |||
| 8:25 | Chairperson's Remarks Mark Fineman, Executive Director, Clinical Pharmacology, Amylin Pharmaceuticals, Inc. | |||
| 8:30 | Exenatide Treatment of Type 2 Diabetes: Development Path and Outcomes from Immediate and Long Acting Formulations Exenatide, an incretin mimetic, shares several of the glucoregulatory effects of GLP-1. In placebo-controlled clinical trials, exenatide twice-daily (BID) resulted in significant reductions in A1C and body weight in patients with type 2 diabetes. A once-weekly (OW) exenatide formulation is under development. In a 15-week study, exenatide OW had multiple metabolic effects, including significant reductions of A1C and body weight. Mark Fineman, Executive Director, Clinical Pharmacology, Amylin Pharmaceuticals, Inc. | |||
| 9:00 |
Nastech has developed an intranasal insulin formulation that exhibits a rapid onset and short duration of action that closely mimics the first-phase insulin response that is deficient in Type 2 diabetics. Intranasal insulin provides a convenient dosage form that exhibits excellent bioavailability in human clinical trials, and PK characteristics that may reduce the risk of treatment-induced hypoglycemia. Richard Mitchell, Ph.D., Scientific Director, Marketing and Business Development, Nastech Pharmaceutical Company, Inc. | |||
| 9:30 | Preclinical and Clinical Development of Orally Delivered Peptides Physiological barriers to the oral delivery of peptides have been overcome with an enteric-coated solid dosage formulation. Intestinal absorption is facilitated by the use of a protease inhibitor and an absorption enhancer. Preclinical and clinical studies have demonstrated bioavailability of intact, biologically active peptides. Results with several peptides such as calcitonin, GLP-1, desmopressin and leuprolide will be presented. Nozer Mehta, Ph.D., Vice President, Biological R&D, Unigene Laboratories, Inc. | |||
| 10:00 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 10:45 | Late-Stage Clinical Development of Synthetic Peptide MBP8298 for the Treatment of Multiple Sclerosis MBP8298, a synthetic peptide identical to a sequence from human myelin basic protein, is designed to specifically suppress autoimmune attack in MS patients with certain HLA types. Two pivotal Phase 3 trials in secondary progressive MS and a Phase 2 trial in relapsing remitting MS are currently underway. Drug discovery and development will be summarized, and status of the clinical trials will be presented. Mark J. Krantz, Ph.D., Vice President, Scientific Affairs, BioMS Medical Corp., Canada | |||
| 11:15 |
Romidepsin, a histone deactylase inhibitor (HDACi), is in late stage clinical development for cutaneous and peripheral T-cell lymphomas (CTCL and PTCL) and other hematologic and solid tumors indications. Gloucester's operational and regulatory strategies and experiences will be presented as a case study of product development by a start-up biotech company, with a focus on manufacturing, process transfer, and analytical comparability. Nicholas Vrolijk, Ph.D., Vice President, Manufacturing Operations, Gloucester Pharmaceuticals, Inc. | |||
| 11:45 | Presentation Title to be Announced Claudio D. Schteingart, Ph.D., Senior Director of Chemistry, Ferring Research Institute Inc. | |||
| 12:15 | Lunch and Roundtable Discussions in Poster and Exhibit Hall | |||
| Optimization Strategies of Leading Peptides in Preclinical Development | ||||
| 1:45 | Chairperson's Remarks Aleksander Swietlow, Ph.D., Principal Scientist, Pharmaceutics, Amgen Inc. | |||
| 2:00 |
Oxyntomodulin reduces food intake and ameliorates glucose intolerance in rodents. A clinical study has shown that preprandial oxyntomodulin for four weeks causes significant weight loss. TKS1225, a novel oxyntomodulin analogue, is being developed for the treatment of obesity and cardiometabolic disease. TKS1225 is potent and longer-acting and is a superior candidate to the native oxyntomodulin for development. John Burt, Ph.D., Chief Executive Officer, Thiakis Limited, United Kingdom | |||
| 2:30 | Discovery and Development of Peptides that Target FcRn for the Treatment of Autoimmune Disease The neonatal Fc receptor, FcRn, is responsible for the long half-lives of IgG molecules in vivo. A family of peptides that inhibits the FcRn:IgG protein-protein interaction was discovered using phage display screening. Data will be presented on the chemical optimization of this peptide family and their effects on IgG catabolism in mice and non-human primates. Adam R. Mezo, Ph.D., Associate Director, Chemistry, Syntonix Pharmaceuticals, Inc. | |||
| 3:00 | Intracellular Peptide Therapeutics: An Evolving Platform KAI is building a portfolio of peptides directed against intracellular protein targets, with our third such compound scheduled to enter the clinic in Q2 08. We will describe our targeted approach to enhance carrier peptide delivery technology to enable broader application of our platform, with examples from our pre-clinical and clinical studies. Derek Maclean, Ph.D., Director, Chemistry, KAI Pharmaceuticals, Inc. | |||
| 3:30 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 4:00 | Development of ABS201 as a New Antipsychotic Lead Argolyn Bioscience Inc. has developed a proprietary peptide modification technology to address the key problems of peptide drug development: stability, receptor selectivity and barrier crossing. Application of the technology enables efficient generation of leads from candidate peptides; this will be illustrated in the context of the development of ABS201 and ABS212, new leads for schizophrenia and pain respectively. Thomas A. Dix, Ph.D., Co-Founder and CSO, Argolyn Bioscience Inc. | |||
| 4:30 | Phase 1 IND for PL-3994, a New Peptide for Treatment of Congestive Heart Failure: CMC Strategy and Related Regulatory Experience PL-3994, Palatin's novel analog of atrial natriuretic peptide for acute decompensated congestive heart failure, is currently in Phase 1. This talk highlights the CMC strategy employed to support the IND filing, with emphasis on analytical characterization of drug substance and drug product. Key aspects of the interaction with the FDA on CMC issues during the IND filing are also discussed. Firoz D. Antia, Ph.D., Executive Director Chemical & Analytical Development, Palatin Technologies, Inc. | |||
| 5:00 | The Development of Hotspot Pharmacophores (HPs) for the Treatment of Cancer and Other Disease Indications Antyra has identified preclinical leads called HotSpot Pharmacophores which are derived from high diversity display libraries and can modulate critical regions of protein: protein interactions called “Hotspots”. Antyra has several lead candidates in addition to a sustainable pipeline for cancer and other disease indications. Antyra believes that HPs are competitive and viable alternatives to biopharmaceuticals such as monoclonal antibodies. Neil I. Goldstein, Ph.D., President, Antyra Inc. | |||
| 5:30 | Networking Reception in Poster and Exhibit Hall Dedicated Poster Viewing: Poster presenters are requested to be present at their posters. | |||
| Nucleic Acids Technologies for Diagnostics | ||||
| Regulatory Pathways and Quality Strategies | ||||
| 7:30 | Networking Coffee | |||
| 8:15 | Chairperson's Opening Remarks B. Melina Cimler, Ph.D., Vice President, Regulatory Affairs, Beckman-Coulter Inc. | |||
| Keynote Presentations | ||||
| 8:30 | Nucleic Acid Technologies for Diagnostics - FDA Perspectives on Why the Tortoise Won the Race FDA has been regulating diagnostics since implementation of the Medical Device Amendments of 1976. Regulation is science based and involves an overlapping series of well defined but flexible premarket and postmarket controls. By promoting sound science, FDA believes it adds value to marketed products and ensures that transition to new diagnostic frontiers occurs in a safe and effective manner that both protects and promotes public health. Steven I. Gutman, M.D., Director, Office of In Vitro Diagnostic Device Evaluation and Safety, CDRH, U.S. Food and Drug Administration | |||
| 9:00 | Increasing International Challenges in Regulatory and Compliance for Diagnostic Products The EU had been quite successful to harmonize the regulations within their member states into basically one common regulatory framework for In Vitro Diagnostic Medical Devices. Meanwhile the regulations have been multiplied and spread into other areas, like Latin America or Asia/ Pacific. Manufacturers are challenged by the diverse requirements and burdensome administration for worldwide product registrations. The presentation will highlight some of the major challenges and will give an outlook into the future. Petra Kaars-Wiele, Ph.D., Senior Director, International Regulatory Affairs and Affiliate Compliance, Diagnostic Division, Abbott Laboratories, Germany | |||
| 9:30 | A Regulatory Strategy for Emerging Molecular Diagnostics Recent advancements in genomic and molecular sciences hold great future promise for improving public health (e.g. personalized medicine). Novel technologies present great challenges for the current FDA premarket review paradigm where they compete with older, well-standardized tests for scarce review resources. The discussion will present principles and an approach for least burdensome regulation of new and established IVDs and laboratory developed tests. Michele M. Schoonmaker, Ph.D., Director, Government Affairs, Cepheid | |||
| 10:00 | Networking Poster and Exhibit Viewing Refreshment Break | |||
| 10:45 | Regulatory Development Issues: Companion Diagnostics and Therapeutics We are in an active era of targeted molecular therapy and understanding the role molecular assays will play in predicting sensitivity or resistance to these drugs. Selecting patients most likely to benefit from these agents has given rise to expanding interest in development of new companion diagnostics. Challenges and practical consideration in the co-development of drugs and diagnostics from a diagnostic regulatory perspective will be discussed. William J. Pignato, Senior Scientist, Regulatory Affairs, Genentech, Inc. | |||
| 11:15 | The Regulation of Molecular Diagnostics: An Overview of Global Trends Update your knowledge of evolving regulatory structures which govern molecular diagnostics in the United States, Canada, Europe and Australia. Compare national/regional approaches to issues such as risk classification, the regulation of laboratory developed tests and the role of third parties in device review. Special consideration will be given to the regulation of pharmacogenetic tests and the role of international bodies such as the Global Harmonisation Task Force. Stuart Hogarth, Visiting Research Fellow, Institute for Science and Society, Nottingham University, United Kingdom | |||
| Audience Interactive Panel Discussion | ||||
| 11:45 | Regulatory Pathways and Quality Strategies Moderator: B. Melina Cimler, Ph.D., Vice President, Regulatory Affairs, Beckman-Coulter Inc. Panelists: Steven I. Gutman, M.D., Director, Office of In Vitro Diagnostic Device Evaluation and Safety, CDRH, U.S. Food and Drug Administration Petra Kaars-Wiele, Ph.D., Senior Director, International Regulatory Affairs and Affiliate Compliance, Diagnostic Division, Abbott Laboratories, Germany William J. Pignato, Senior Scientist, Regulatory Affairs, Genentech, Inc. Michele M. Schoonmaker, Ph.D., Director, Government Affairs, Cepheid Stuart Hogarth, Visiting Research Fellow, Institute for Science and Society, Nottingham University, United Kingdom Peter Haima, Ph.D., Senior Project Manager In Vitro Diagnostics, Eurogentec S.A., Belgium | |||
| 12:15 | Lunch and Break-Out Discussion Roundtables | |||
| 1:45 | Chairperson's Remarks Timothy Stenzel, M.D., Ph.D., Vice President, Diagnostics R&D and Chief Medical Officer, Asuragen, Inc., A Spin-Off of Ambion | |||
| 2:00 | Quality Management in the Manufacturing of Primers and Probes for Molecular Diagnostics Assays Oligonucleotides are critical components of molecular diagnostic assays. Consistent functional performance is only assured with a GMP manufacturing process, requiring compliance with FDA 21CFR820 and ISO13485. IVD kits contain GMP oligos, whereas Lab Developed Tests often contain non-GMP oligos. Both approaches will be discussed in light of the Eurogentec GMP manufacturing process from a financial, quality, risk management and regulatory perspective. Peter Haima, Ph.D., Senior Project Manager In Vitro Diagnostics, Eurogentec S.A., Belgium | |||
| Analytical Methods and Validation | ||||
| 2:30 | The Development and Validation of Companion Diagnostics—Importance of the Clinical Laboratory The appropriate development of companion diagnostics (CDx) is critical to the future of the pharmaceutical industry. It is becoming increasingly clear that the clinical laboratory industry will be taking on greater responsibility for the success of CDx. The value of true collaborative efforts between the pharmaceutical industry, device manufacturers and clinical laboratories will be the focus of this presentation. Glenn A. Miller, Ph.D., Vice President, General Manager, Genzyme Analytical Services | |||
| 3:00 | Challenges in the Validation of Test Methods Use for Oligo Manufacturing A number of test methods are used during the manufacture of oligonucleotides including gel filtration chromatography, reverse phase chromatography, anion exchange chromatography, UV/VIS/FL spectroscopy and reporter over quencher fluorescence. These methods are applied to large number of oligonucleotides which make the method validation challenging. This challenge can be handled efficiently by grouping the oligonucleotides into families for the purpose of method validation. Michael D. Geier Ph.D., Director, Analytical Development and Test Method Validation, Roche Molecular Diagnostics | |||
| 3:30 | Networking Poster and Exhibit Viewing with Refreshments | |||
| 4:00 | IVD, ASR, RUO and Assay Validation: The Alphabet Soup of the Clinical Diagnostics Laboratory The presentation will discuss the regulations imposed upon the clinical diagnostic laboratory for the use of diagnostic assays with different regulatory designations. The ASR validation process will be specifically addressed. Diagnostic product manufactures with a keen knowledge of these regulations will better be able to position their products to be successful in a competitive market. Brian DuChateau, Ph.D., Vice President, Laboratory Operations, Converge Diagnostic Services | |||
| 4:30 | A New Concept for Standardization of NAT Assays across Different Platforms, Laboratories and Technologies A fundamental goal of laboratory medicine is that results for patients' samples will be comparable independent of the medical laboratory that produced the results." (W. Greg Miller et al. Clinical Chemistry 2006;52:553-554). A strategy for achieving this goal for NAT assays using metrologically traceable and commutable control materials will be presented. E. Ralf Schönbrunner, Ph.D., Vice President, Research and Development, AcroMetrix | |||
| Featured Future Directions Presentation | ||||
| 5:00 |
Preventive therapy with statins results in a substantial reduction of primary and secondary coronary events. Despite the overall cholesterol lowering benefits of statins, a fraction of patients on statin therapy appear to receive little or no survival benefit from the treatment. The results for a common biomarker for both risk of coronary heart disease and statin response will be presented. Andrew Grupe, Ph.D., Senior Director, Pharmacogenomics and Director, CNS Research, Celera | |||
| 5:30 | Networking Reception in Poster and Exhibit Hall Dedicated Poster Viewing: Poster presenters are requested to be present at their posters. | |||
| Day Three Wednesday, May 21, 2008 | PRE-CONFERENCE COURSES | DAY ONE | DAY TWO | DAY THREE | | ||||
| TIDES® Main Conference | ||||
| Delivery of Oligo and Peptide-Based Therapeutics: Characterization, Manufacturing, Regulatory, IP and Economic Considerations | ||||
| 7:45 | Networking Coffee | |||
| 8:15 | Morning Co-Chairperson's Opening Remarks Mark E. Davis, Ph.D., Warren and Katharine Schlinger Professor of Chemical Engineering, California Institute of Technology Christopher A. Rhodes, Ph.D., Executive Director, Pharmaceutical Sciences, Amylin Pharmaceuticals, Inc. | |||
| Plenary Presentations | ||||
| 8:30 | Systemic Delivery of Small Molecules and Nucleic Acids via Targeted Lipidic Nanoparticles: Challenges and Opportunities Systemic delivery of many therapeutic molecules requires the ability to target specific cell types and to deliver the therapeutic across the cell membrane and into the cytosol. We have developed antibody fragment targeted lipidic nanoparticles capable of delivering small molecules and nucleic acids intracellularly. Factors governing successful targeting will be presented as well as current barriers that require further study. James D. Marks, M.D., Ph.D., Department of Anesthesia and Pharmaceutical Chemistry, Member, Comprehensive Cancer Center, University of California, San Francisco | |||
| 9:00 |
In 1990, Inhale Therapeutic Systems (now Nektar Therapeutics) began the commercial development of the first inhaled insulin. The company partnered with Pfizer in l995 and after a highly challenging development program, succeeded in gaining regulatory approval for inhaled insulin, named Exubera, in January 2006 in both Europe and the United States. The approval was front page news worldwide. Most patients clearly preferred Exubera over injections. However, sales were weak and Pfizer returned the product to Nektar in October of 2007. Is inhaled insulin dead? John S. Patton, Ph.D., CSO and Founder, Nektar | |||
| 9:30 | Pulmonary Delivery of GLP-1 Using Technosphere Platform Technology Abstract to come. Dr. Peter Richardson, MRCP, Corporate Vice President and Chief Scientific Officer, MannKind Corporation | |||
| 10:00 | Networking Poster and Exhibit Viewing with Refreshments Sponsored by | |||
| 10:45 | Integrating Development of Peptide Therapeutics and Delivery Systems Peptides comprise an increasing number of therapeutic candidates at discovery and development stages, highlighting the opportunity for delivery to play an enabling role. Peptide delivery options are illustrated through various commercial examples. Key issues include appreciating the unique physical and chemical properties of peptides; systematically evaluating the technology options; and managing risk, according to the technology integration point. Paul A. Burke, Ph.D., Principal, Drug Delivery Consulting, LLC; Consultant, former Executive Director, Pharmaceutics, Amgen Inc. | |||
| 11:15 | A Platform Approach to Albumin Fusion Proteins - Can it be Applied to Formulation? At CoGenesys, a development platform has been established to rapidly advance HSA fusion proteins from development to the clinic. While many aspects of this approach are successful due to the biochemical dominance of the large albumin moiety over the entire fusion protein, particular care must be taken in formulation, where the fusion partner may define primary degradation pathways. Jason Bock, Ph.D., Senior Manager, Pharmaceutical Development, CoGenesys | |||
| 11:45 | Towards Clinical Investigation of CALAA-01, a Systemically-Administered, siRNA-Containing Nanoparticle Formulation Calando is advancing CALAA-01, a polymer-based, transferrin-ligated, siRNA-containing nanoparticle formulation targeting ribonucleotide reductase subunit M2 (RRM2), for clinical investigation in patients with solid tumors. Pre-clinical safety and efficacy, and also characterization, formulation and CMC issues of CALAA-01 will be discussed, as well as clinical protocol and status. Jeremy Heidel, Ph.D., Chief Scientific Officer, Calando Pharmaceuticals | |||
| 12:15 | Lunch in Poster and Exhibit Hall | |||
| 1:45 | Afternoon Co-Chairperson's Remarks John P. Mayer, Ph.D., Senior Research Advisor, Eli Lilly and Co. Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals | |||
| 2:00 | Using Selected Nucleic Acids for Delivery The Ellington and Sullenger labs have utilized a previously selected anti-PSMA aptamer to deliver siRNA and other cargoes to cells. Inhibition of gene expression works in both cell and animal models, and can lead to tumor regression. We will discuss additional routes and applications for RNA-mediated delivery of RNAs. Andrew D. Ellington, Ph.D., Wilson and Kathryn Fraser Research Professor in Biochemistry, The University of Texas at Austin; Co-Founder, b3 bio, Inc. | |||
| 2:30 | Small Peptides as Drug Delivery Agents Alba's technology provides the ability to physiologically, transiently and reversibly modulate tight junctions and alter paracellular permeability. These small peptides facilitate the absorption of co-administered biologically active macromolecules across paracellular barriers. These peptides are chemically synthesized and do not require chemical conjugation, providing advantages in formulation and manufacturing. Mark J. Ginski Ph.D., Director Product and Analytical Development, Alba Therapeutics Corporation | |||
| 3:00 | Mechanisms and Optimization of in vivo Delivery of Lipophilic siRNAs and Lipidoid Complexed siRNAs We synthesized a variety of lipophilic siRNAs and used them to elucidate the requirements for siRNA delivery in vivo (Nature Biotechnology 25, 1149 - 1157 (2007). Efficient and selective uptake of these siRNA conjugates depends on interactions with lipoprotein particles, lipoprotein receptors and transmembrane proteins. Our results demonstrate that conjugation to lipophilic molecules enables effective siRNA uptake through a common mechanism that can be exploited to optimize therapeutic siRNA delivery. Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals | |||
| 3:30 | Networking Refreshment Break Sponsored by | |||
| 4:00 | PolyTran™ Technology for Systemic Delivery of siRNA Therapeutics in Oncology To enable systemic administration of siRNA, we designed a biodegradable, cationic polymer-based delivery system called PolyTran. RNAi nanoplexes formed by condensation of PolyTran with siRNA against validated cancer genes were characterized, then studied in vitro for gene knockdown and in vivo for tumor growth inhibition in multiple tumor xenograft mouse models. Modifications to achieve PEGylation and ligand targeting are underway. Steven M. Chamow, Ph.D., Senior Vice President, CMC, Intradigm Corporation | |||
| 4:30 | Delivery and Efficacy Challenges for Research and Development of siRNA Therapeutics Effective delivery is essential for RNAi. Dicer substrates are highly potent, and these longer sequences allow for conjugation of delivery and targeting entities, e.g., peptides, without loss of activity. Various formulations have been tested for their ability to provide efficacy while limiting toxicity. Activity has been demonstrated in vitro and with local and systemic administration of siRNA. Michael V. Templin, Ph.D., Director, Pharmacology and Toxicology, Nastech Pharmaceutical Company, Inc. | |||
| 5:00 | Close of TIDES® | |||
| Nucleic Acids Technologies for Diagnostics | ||||
| New Technologies: Planning for Tomorrow's Diagnostics | ||||
| 7:45 | Networking Coffee | |||
| 8:00 | Chairperson's Remarks Marc Lemaitre, Ph.D., Director, R&D, Glen Research Corp. | |||
| Keynote Presentation | ||||
| 8:15 | Unnatural Base Pair Systems for DNA/RNA-Based Biotechnology The creation of unnatural base pairs, compatible with the natural A-T(U) and G-C base pairs, could expand the genetic alphabet for the site-specific incorporation of extra, functional components into nucleic acids and proteins. We have developed a series of unnatural base pairs, which exhibit unique specificity in in vitro replication, transcription or translation. This unnatural base pair system would provide variety of biotechnology applications. Ichiro Hirao, Ph.D., Team Leader, Nucleic Acid Synthetic Biology Research Team, RIKEN Genomic Sciences Center (GSC); President, TagCyx Biotechnologies, Japan | |||
| 9:00 | Molecular Imaging with Oligonucleotides Molecular Imaging can assess gene expression non-invasively, repeatedly and quantitatively in living subjects. Pharmaco-Imaging of oligonucleotides allows quantifying in 3-D and in the whole body of animals and humans the bio-distribution time course of antisense, aptamers, interfering RNAs, ribozymes, etc. Use of oligonucleotides as contrast agents requires that a sufficient contrast is obtained and that the correlation between tracer and target concentrations is demonstrated. Bertrand Tavitian, M.D., Ph.D., Head of the Laboratoire d'Imagerie Moleculaire Experimentale, Institute for Biomedical Imaging, CEA, France | |||
| 9:30 | Designing Multiplex STR Genotyping Assays for Human Identification Using our non-nucleotide linkers and 5-dye fluorescent technology we have developed highly discriminating multiplex short tandem repeat (STR) assays for human identification. In this presentation, we will present some of the challenges we encountered and the solutions we implemented when developing these multiplexed STR assays to meet the needs of the forensic community. Lori K. Hennessy, Ph.D., Director, R&D, Human Identification Group, Applied Markets Division, Applied Biosystems | |||
| 10:00 | Poster and Exhibit Viewing Refreshment Break | |||
| 10:45 | Ferrocene DNA Oligonucleotides and Electrochemical Microarrays Phosphoramidites containing ferrocenes with discrete electrode potentials are used to synthesize short DNA oligonucleotides. These structures present analytical, synthetic and purification challenges for which we have developed processes used in downstream manufacturing. Using electrode surface capture and electrochemical detection of amplicon:ferrocene-oligonucleotide hybrids we have developed low to medium density multiplex arrays useful for mutation characterization of the Cystic Fibrosis CFTR gene and Warfarin metabolism. Dick Keys, Ph.D., Senior Director, Chemistry and Product Development, Osmetech Molecular Diagnostics | |||
| 11:15 | MicroRNA: Emerging Biomarkers and Advances in Molecular Diagnostics A deepening understanding of miRNA biology underscores the importance of expression alterations in these small RNAs that are now associated with many diseases, including cancer. As a result, miRNAs are increasingly promising biomarkers for both diagnostic and therapeutic applications. In this presentation, we will describe how technology innovations have illuminated clinical opportunities for miRNA. Gary J. Latham, Ph.D., Head of Technology Development, Asuragen, Inc. | |||
| 11:45 | High Throughput microRNA Expression Profiling Using a Bead Based Multiplex System Recent publications have shown that cancers have specific miRNA profiles that either classify the a cancer, or give prognostic information. A bead based multiplexed assay was developed to address both specificity needs and throughput needs for an assay to profile miRNA in tissues. The profiles of various cancers will be shown and discussed. Keld Sorensen, Ph.D., Director, R&D Bioscience Group, Luminex | |||
| 12:15 | Lunch in Poster and Exhibit Hall | |||
| 1:45 | IsoAmp® Molecular Analyzer, a Disposable Molecular Diagnostic Device for Point-of-Care Pathogen Detection BioHelix has developed the IsoAmp® Molecular Analyzer, a simplified diagnostic platform for nucleic acid testing without specialized instrumentation. The platform consists of manual sample prep, isothermal Helicase-Dependent Amplification; and detection with the BEST™ Cassette. IsoAmp® tests achieve single-copy sensitivity and use target probes and internal controls to ensure specificity. Assays under development include Clostridium difficile, Staphyloccus aureus, and Methicillin-resistant Staphylococcus aureus. Tamara Ranalli, Ph.D., Manager, Business Development and Quality Systems, BioHelix Corporation | |||
| Technology IDOL | ||||
| 2:15 | Diagnostic Applications of DNA Sequencing Based on the popular television program, this session is an opportunity for developers of DNA sequencing for diagnostic applications to present new data. Presentations will be strictly limited to ten minutes each. After the presentation, the panel will question the presenter for 5 minutes. Participants to be announced. For information on participation, please contact Sherry Johnson, Senior Business Development Manager, IBC Life Sciences 508-614-1451 or email sjohnson@ibcusa.com | |||
| Business Considerations for Successful Launch in Clinical Market | ||||
| Chairperson: Loren "Dick" Keys, Ph.D., Senior Director, Chemistry and Product Development, Osmetech Molecular Diagnostics, Inc. | ||||
| 3:00 | Nanotechology Medical Device Development and Product Launch Nanosphere's Verigene® System is a bench top molecular diagnostics workstation uniquely suited to meet present-day needs and drive the future potential of the biomarker-driven field of diagnostics. The development of the Verigene system was a multidisciplinary technology development and integration effort. The Verigene System® is the first nanotechnology based clinical diagnostic platform in the world with FDA Clearance. William Cork, Chief Technology Officer and V.P. Research & Development, Nanosphere, Inc. | |||
| 3:30 | Networking Refreshment Break | |||
| 4:00 | An Overview of The Current State of PCR Licensing As patents for simple PCR and some related technologies have expired, the licensing landscape appears to have become far more complex. Now, complicated diligence is necessary in determining what licenses may be needed for new product development in several fields. This presentation will give a brief overview of the current status of PCR licensing: the fields, territories, some technologies and some of the issues associated with these elements. Monte Wetzel Ph.D., Director of Licensing, Roche Molecular Systems Inc. | |||
| 4:30 | Qualifying Manufacturing Material Suppliers for Molecular Diagnostics and Therapeutics The responsibility of ensuring that raw materials used in the manufacture of molecular diagnostics and therapeutics is ultimately the responsibility of the finished product manufacturer. Manufacturers must define, communicate, and enforce the quality expectations that they have for their raw material suppliers. This presentation explores the challenges and methods of identifying, qualifying, and managing raw material suppliers in this growing industry. Jim Darnell, Manager, Life Sciences, Clarkson Consulting | |||
| 5:00 | Close of Nucleic Acids Technologies for Diagnostics Conference | |||
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