What`s Inside... - IBC Life Sciences
Transcription
What`s Inside... - IBC Life Sciences
IBC’s 16th Annual May 12-15, 2014 • Rhode Island Convention Center • Providence, RI Event Guide Sponsor: What's Inside... Agenda Overview . . . . . . . . . . . . . . . . . . . . . . 4 Meeting Room Map . . . . . . . . . . . . . . . . . . . . 5 Event Sponsors . . . . . . . . . . . . . . . . . . . . . . . . . 6 Conference Agenda Monday Pre-Conference Workshops . . 8-9 Tuesday Sessions . . . . . . . . . . . . . . . . . . . . . . 10 Wednesday Sessions . . . . . . . . . . . . . . . 12-15 Thursday Sessions . . . . . . . . . . . . . . . . . . 16-19 Exhibition Hours and Information Exhibitor List and Hall Map . . . . . . . . . . . . 20 Poster Presentations . . . . . . . . . . . . . . . . . . . 24 Event Partners Gold Sponsors Silver Sponsors Bronze Sponsors Premier Publication Organized by AGENDA-AT-A-GLANCE Monday, May 12, 2014 • Pre-Conference Workshops 9:00 am 5:00 pm Workshop #1 (Room 555) Impurities in Oligonucleotides: Sources, Analysis and Control Workshop #2 (Room 551) Peptide Aggregation in Drug Substance and Drug Product Tuesday, May 13, 2014 • Main Conference Workshop #3 (Room 556) In vivo Delivery of Therapeutic Oligonucleotides: Challenges, Considerations and Solutions Workshop #4 (Room 552) Nucleic Acids Technologies for Molecular Diagnostics Exhibit Hall & Poster Viewing Hours: 3:15 pm – 6:45 pm 8:30 am 12:05 pm Keynote Presentations (Ballroom A) 1:25 pm 5:30 pm Plenary Presentations (Ballroom A): Updates on Progress of Leading Therapeutic Candidates Opening of Poster and Exhibit Hall (3:15 pm – Third Floor) Featured FDA Presentation 5:30 pm 6:45 pm Networking Wine and Cheese Reception in Poster and Exhibit Hall Sponsored by Lonza Wednesday, May 14, 2014 • Main Conference Oligonucleotide and Peptide Manufacturing, Technology and Product Development Manufacturing Advances, Process Using Conjugation Technology Improvements and Scale Up to Advance Therapeutic Sponsored by Bachem Oligonucleotides and Peptides (Room 552) (Ballroom B) Audience Interactive 8:15 am Strategy Discussion Forum: Spotlight Panel Discussion: 12:45 pm Assessing the Current Presentations Novel Strategies for the State of Conjugation, Scale-up of Oligonucleotide by Senn and Fusion, and Encapsulation and Peptides: Thinking Kinovate Technologies Outside the Box (Room 552) (Room 555) (Ballroom B) 12:45 pm 1:55 pm Audience Interactive Panel Discussion: 1:55 pm - Challenges with Novel Technologies 5:30 pm for the Analysis and Control of Oligonucleotides and Peptides (Ballroom B) 5:30 pm 6:30 pm Emerging Technologies GE Healthcare, BioSpring, Glytech and Corden Pharma (Room 552) Peptide Discovery and Development Medicinal Chemistry and Chemical Modifications to Improve Oligonucleotide Properties Peptide Discovery and Hit Finding Display Technologies and Discovery Platforms (Room 551) (Ballroom D) Computational Modeling and Design of Peptides Sponsored by CMD Biosciences Discovery and Preclinical Strategies Audience Interactive Panel Discussion: What Constitutes a Good Target for Oligonucleotide Modalities? Intracellular Delivery of Peptides Novel Chemistries for Peptidomimetic Libraries (Room 551) (Ballroom D) Networking Cocktail Reception in Poster and Exhibit Hall Thursday, May 15, 2014 • Main Conference Oligonucleotide and Peptide Manufacturing, Technology and Product Development Process Validation: Design, Qualify, Verify (Ballroom B) 8:25 am - Strategy Discussion Forum (11:15): 12:45 pm What’s Changed in our Approach to Process Validation? (Room 555) 12:45 pm 1:55 pm 4 Oligonucleotide Therapeutics Discovery Networking Luncheon in Poster and Exhibit Hall Sponsored by Nitto Denko Avecia Inc. Advances in Analytics and Control 1:55 pm – 5:00 pm Exhibit Hall & Poster Viewing Hours: 10:30 am – 6:30 pm Formulation and Delivery Technologies: Enabling Peptide and Oligo Product Opportunities (Room 552) Spotlight Presentation by Wyatt Technology (Room 552) Exhibit Hall & Poster Viewing Hours: 10:00 am – 2:00 pm Oligonucleotide Therapeutics Discovery Peptide Discovery and Development PK/PD and Toxicity of Oligonucleotides Peptides in Preclinical and Clinical Development (Room 551) Preclinical and Clinical Oligonucleotides (Ballroom D) Networking Luncheon in Poster and Exhibit Hall Sponsored by Nitto Denko Avecia Inc. Raw Materials and Supply Chain: Assessing and Mitigating Risk with Novel and Traditional Starting Materials (Ballroom B) Formulation and Delivery Technologies: Enabling Peptide and Oligo Product Opportunities (Room 552) Strategy Discussion Forum (3:30): Where Does Your Building Block Come From? Peptides Discussion (Room 554) Oligonucleotides Discussion (Room 555) Strategy Discussion Forum (3:30): Challenges in CMC Development of Novel Delivery Systems: Functional Excipients, Novel Formulations and Regulatory Expectations (Room 556) TIDES® Conference & Exhibition Preclinical and Clinical Oligonucleotides (Ballroom D) Endosomal Escape, Peptide Targeting and Intracellular Delivery (Room 551) MEETING ROOM MAP Fifth Level Nitto Denko Avecia Inc Corporate Suite Cepia Sanofi Corporate Suite 557 558 Peptide Strategy Forum (Thurs.) 554 Exhibit Hall/ Registration is located on Level 3 Escalators C for AIC Tedia (Tues) and Bachem (Wed.) Rotunda Workshop 3 Strategy Discussion Forum (Thurs.) 555 Workshop 1 Strategy Discussion Forums Ballroom B Ballroom A E Private Receptions 556 Ballroom D 553 553 552 550 551 Oligonucleotide and Peptide Manufacturing Track 1 Sessions Keynote Presentations Plenary Presentations Oligonucleotide Therapeutics Discovery Sessions Oligonucleotide and Peptide Manufacturing Track 2 Spotlight Presentations Workshop 4 Peptide Discovery and Development Workshop 2 Thermo Scientific Corporate Suite Agilent Corporate Suite www.IBCLifeSciences.com/TIDES5 EVENT SPONSORS Event Partners Gold Sponsors Silver Sponsors Bronze Sponsors Corporate Suite Sponsors Spotlight Presentation Sponsors Session Sponsors Networking Reception Sponsor Tote Bag Sponsor Luncheon Sponsor Lanyard Sponsor Event Guide Sponsor Literature Sponsors Get Immediate Access to the New Online Partnering Tool: TIDES Attendee Connect • View the attendee list TIDES Connect office hours: • Pre-schedule meetings Monday: 12:00pm – 1:30pm Wednesday: 12:30pm – 2:00pm • Share announcements Tuesday: 12:00pm – 1:30pm Thursday: 12:30pm – 2:00pm • Create a customized agenda 6 TIDES® Conference & Exhibition Located by the registration area Monday, May 12, 2014 • Pre-Conference Workshops 8:30 Registration and Coffee Workshop #1 Workshop #2 Impurities in Oligonucleotides: Sources, Analysis and Control Peptide Aggregation in Drug Substance and Drug Product (Room 555) (Room 551) Workshop Leaders: G. Susan Srivatsa, Ph.D., President, ElixinPharma Fran Wincott, Ph.D., President, Wincott & Associates, LLC 9:00Overview: Regulatory Expectations for the Control of Impurities in Oligonucleotides G. Susan Srivatsa, Ph.D., President, ElixinPharma Fran Wincott, Ph.D., Wincott and Associates 9:15Practical Considerations for the Implementation of Quality Criteria for Amidite Starting Materials Todd B. Kreutzian, M.S., Director, Analytical Development, Agilent Technologies, Inc. NASD 10:00Manufacturing Strategies Regarding Impurities in Phosphoramidites Dr. Andreas Wolter, Site Manager, Sigma-Aldrich Biochemie GmbH, Germany 9:00 Overview: Peptide Stability Brian Lobo, Ph.D., Senior Scientist, MedImmune, Inc. 9:20 Case Study Formulation Challenges for a Therapeutic Peptide: Peptide Gelation Derrick S. Katayama, Ph.D., Senior Manager, Analytical Sciences, Boehringer Ingelheim 10:00 Preventing Aggregation of Therapeutic Peptides Jens Thostrup Bukrinski, Ph.D., Senior Formulation Scientist, Biopharma R&D, Novozymes Biopharma A/S, Denmark 10:40 Networking Refreshment Break 11:00 Peptide Synthesis and Downstream Processing Strategies to 10:30 Networking Refreshment Break 11:00Unpublished Data Detecting, Characterizing and Minimizing Impurities of Phosphorothioate Oligonucleotides Kym M. Bradley, Associate Director, Analytical Development and Quality Control, Isis Pharmaceuticals, Inc. 11:45Residual Solvents and Elemental Impurities: What to Test for, What to Characterize and How to Set Limits to Meet Regulatory Safety Expectations Emma Wright, D.Phil., Senior Director, Process Development, Nitto Denko Avecia Inc. 12:30 Lunch on your own Prevent Aggregation in Beta-Amyloid. Chris Bai, Ph.D., President and CEO, AmbioPharm, Inc. Emory Gunter, Associate Director of Process Development & Production, AmbioPharm, Inc. 11:45 Manufacturing Control of Peptide Aggregation :Pre- Manufacturing Assessment and Processing Control Strategies Gary F. Musso, Ph.D., President, Musso and Associates LLC 12:30 Lunch on your own 2:00 Suppression of Aggregation in Peptide Formulations by Non- Aqueous Solvents 2:00CMC Considerations for the Setting of Reporting, Identification and Qualification Thresholds Doug Brooks, Ph.D., Vice President, Manufacturing Development, Ophthotech Corporation 2:30Safety Considerations for the Justification of Qualification Thresholds for Synthetic Oligonucleotides Michael V. Templin, Ph.D., Technical Director, SNBL USA Ltd. 3:15 Networking Refreshment Break 3:45Impurities in Oligonucleotides: A Reviewer’s Perspective René Thürmer, Ph.D., Deputy Head Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany 4:30 Audience Interactive Panel Discussion 5:00 Close of Workshop Workshop Leader: Brian Lobo, Ph.D., Senior Scientist, MedImmune Neelima Phadnis, Ph.D., CMC Director, Xeris Pharmaceuticals, Inc. 2:45 Unpublished Data Analytical Challenges in Detection and Characterization of Peptide Aggregation and Instability Riccardo Torosantucci, Ph.D., Project Manager, Coriolis Pharma Research GmbH, Germany 3:30 Networking Refreshment Break 3:45 Peptide Aggregation and Instability in Combination Products and Impact on Subcutaneous Delivery Brian Lobo, Ph.D., Senior Scientist, MedImmune, Inc. 4:30 Audience Interactive Panel Discussion Moderator: Brian Lobo, Ph.D., Senior Scientist, MedImmune, Inc. 5:00 Close of Workshop TIDES Passport Contest Tuesday through Thursday – Win Prizes Don’t miss out on this fun and interactive experience as you tour through the Exhibit Hall. Stop by participating exhibitors and get your passport initialed and you could walk away with several prizes along with innovative ideas. 1. Visit each booth in the exhibit hall listed in your passport. Each booth will be labeled as a “Passport Stop”. 2. H ave each exhibiting company initial their destination on your passport. 3. Participants are to turn in their passport at the registration desk by 1pm on Thursday. Winners will be announced Thursday at 1:30pm. The drawing will be held in the exhibit hall. You must be present to win! 8 TIDES® Conference & Exhibition Monday, May 12, 2014 • Pre-Conference Workshops 8:30 Registration and Coffee Workshop #3 Workshop #4 in vivo Delivery of Therapeutic Oligonucleotides: Challenges, Considerations and Solutions Nucleic Acids Technologies for Molecular Diagnostics (Room 556) (Room 552) Workshop Leaders: Dick Keys, Ph.D., President, Advanced Biotectonics Consulting Marc M Lemaitre, Ph.D., President, ML_Consult Workshop Leader: Dmitry Samarsky, Ph.D., Executive Vice President, Technology and Business Development, RiboBio, China 9:00 Overview Dmitry Samarsky, Ph.D., Executive Vice President, Technology and Business Development, RiboBio, China 9:15 Delivery of Phosphorothioate Oligonucleotides to Cells C.A. Stein, M.D., Ph.D., Arthur and Rosalie Kaplan Professor and Chair, Department of Medical Oncology and Experimental Therapeutics, Deputy Director of Clinical Research, Comprehensive Cancer Center, City of Hope Medical Center 10:00 Advantages of Morpholino Oligonucleotides as Drug Candidates Ryszard Kole, Ph.D., Distinguished Scientist, Sarepta Therapeutics 10:45 Networking Refreshment Break 11:15 Delivery, Distribution and Cellular Uptake of LNA Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark 12:00 Unpublished Data Using Endosomal Escape Agents for siRNA Delivery in vivo David L. Lewis, Ph.D., Chief Scientific Officer, Arrowhead Research Corporation 12:45 Lunch on your own 2:00 The Use of Conjugates to Deliver Oligonucleotides in vivo Rachel Meyers, Ph.D., Vice President, Research and RLD (RNAi Lead Development), Alnylam Pharmaceuticals, Inc. 9:00 Overview: Novel Technologies and New Product Directions in Molecular Diagnostics Dick Keys, Ph.D., President, Advanced Biotectonics Consulting 9:15 Engineering Next-Generation Diagnostics with Synthetic Biology Timothy K. Lu, M.D., Ph.D., Assistant Professor, Deptartments of Electrical Engineering, Computer Science and Biological Engineering, Massachusetts Institute of Technology 9:50 Intellectual Property Update: How to Maintain a Genetic Diagnostic Claim Peter Carroll, Ph.D., Partner, Medlen & Carroll 10:25 Networking Refreshment Break 10:50 Informed Consent and Genetic Research Bertha deLanda, CIP - IRB/SCRO Panel Manager, Research Compliance Office, Stanford University 11:25 Risk Management of GMP Oligonucleotide Manufacturing Luc Marion, Vice President, Eurogentec North America 12:00 Lunch on your own 1:30 Advances in High-Throughput On-Chip Gene Synthesis Technology Jingdong Tian, Ph.D., Assistant Professor, Biomedical Engineering, Duke University 2:05 Unpublished Data Freeze Dried Hot Start PCR Mixes: The Evolution of Molecular Diagnostics 2:45 IMI COMPACT: A European Public-Private Partnership with the Aim to Develop Novel Delivery Systems for Biopharmaceuticals Enrico Mastrobattista, Ph.D., Associate Professor, Pharmaceutics, Utrecht University, The Netherlands 3:30 Networking Refreshment Break 4:00 Audience Interactive Panel Discussion Moderator: Dmitry Samarsky, Ph.D., Executive Vice President, Technology and Business Development, RiboBio, China 5:00 Close of Workshop Sabrina Shore, Senior Research Associate, TriLink BioTechnologies, Inc. 2:40 Unpublished Data Single Molecular Weight Discrete PEG Compounds (dPEG®s): Emerging Roles in Molecular Diagnostics Paul D. Davis, Ph.D., CEO, Founder, Quanta BioDesign, Ltd. 3:20 Networking Refreshment Break 3:50 Unpublished Data Case Study Aptamers in the Real World: Development and Applications Gregory Penner, Ph.D., President and CEO, NeoVentures Biotechnology Inc., Canada 4:25 Audience Interactive Panel Discussion Moderator: Marc M Lemaitre, Ph.D., President, ML_Consult 5:00 Close of Workshop TIDES Passport Contest Tuesday through Thursday – Win Prizes Prize Pebble Smart Watch Gold Treasure Chest Valued at $150 BOSE SoundDock Portable Digital Music System/Speaker Donated by Asahi Kasei Booth 206 Bachem 403 Biopeptek, Inc. 107 $100 Gift Card Biotage 303 Apple TV BioTechLogic, Inc. 228 Swiss Army Knife Buchiglas USA 335 Pebble Smart Watch 16 GB iPad Mini CEM Corporation CS Bio Co. & ShengNuo Peptide 216 Prize Donated by Booth $100 Gift Card VinoIce Wine Cooler and a Lonza Wine Bottle Case Corden Pharma 302 $150 Gift Card Nitto Denko Avecia Inc. 215 Kindle NSF Pharmalytica 404 Chinese Art Craft RiboBio 234 TBA Cepia Sanofi 402 $100 AmEx Gift Card Tosoh Bioscience LLC 222 Lonza312 203/207 www.IBCLifeSciences.com/TIDES9 Tuesday, May 13, 2014 • Keynote Presentations (Ballroom A) Updates on Progress of Leading Therapeutic Candidates 7:30 Registration and Coffee 8:30 Chair Remarks Ved Srivastava, Ph.D., Head Peptide Chemistry, GlaxoSmithKline 8:45 Peptide R&D: Advances and Opportunities Nader Fotouhi, Ph.D., Chief Scientific Officer, Global Alliance for TB Drug Development (TB Alliance); Former Vice President, Head of Discovery Technologies, Hoffmann La Roche 1:25 Chairwoman’s Remarks Pamela A. Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals, Inc. 1:30 Velcalcetide (AMG 416): A Peptide Agonist of the Calcium Sensing Receptor for the Treatment of Secondary Hyperparathyroidism in Patients Receiving Hemodialysis 9:20 Case Study Pharmaceutical Protein and Peptide Engineering Lars F. Iversen, Ph.D., Corporate Vice President, Diabetes Protein Engineering, Novo Nordisk, Denmark Derek MacLean, Ph.D., Director, Process Technology, Amgen 1:55 Case Study Albiglutide: From Peptide to Patient Susan Johnson, M.D., Director of Clinical Development, GlaxoSmithKline 9:55 Networking Refreshment Break 10:25 Aptamer Nanotechnology for Therapeutic Nanoparticles 2:20 An Update on the Clinical Development of Fovista™, a Pegylated Anti-PDGF Aptamer for the Treatment of Wet AMD Omid Farokhzad, M.D., Associate Professor; Director, Laboratory of Nanomedicine and Biomaterials, Brigham and Women’s Hospital, Harvard Medical School 11:00 PeptiDream: From Bench to IPO Patrick C. Reid, Ph.D., Chief Scientific Officer, PeptiDream Inc. Jeffrey Nau, Vice President, Clinical and Medical Affairs, Ophthotech Corporation 2:45 Clinical Update on Alnylam ALN-TTR Programs for Transthyretin Amyloidosis Jared A. Gollob, M.D., Vice President, Clinical Research, Alnylam Pharmaceuticals, Inc. 11:35 Case Study Peptide-Based Vaccines in the New Era of Cancer Immunotherapy Eric von Hofe, Ph.D., President, Antigen Express, Inc. 3:10 Grand Opening of Poster and Exhibit Hall with Networking Refreshment Break 12:05 Lunch on your own 3:55 AZD9150, a Next Generation Antisense TWEET FROM TIDES Oligonucleotide Targeting STAT3 David C. Blakey, Ph.D., Chief Scientist, Oncology iMED, AstraZeneca, United Kingdom 4:20 Custirsen Development Update Anna Elgart, Ph.D., Clinical Pharmacology Leader, Phase 1 and Clinical Pharmacology, Innovative R&D, Teva Pharmaceutical Industries, Ltd., Israel Use hashtag #IBCTIDES to share what you are learning. Watch #IBCTIDES to find out what’s going on around the conference, hear peer comments on presentations, participate in real time discussions, discover contests and offers in the exhibit hall and more. Join the Conversation by Becoming a Member of Our LinkedIn Groups: • Oligonucleotide and Peptide Development Professionals • New England Life Science Professionals Oligonucleotide Book Raffle Return Your Completed Conference Evaluation Form and Be Entered into a Drawing to Receive a Free Copy of the Following Book The Handbook of Analysis of Oligonucleotides and Related Products, Jose V. Bonilla, G. Susan Srivatsa (ISBN 9781439819937) is an essential resource on the practical application of modern and emerging analytical techniques for the analysis of this unique class of compounds in research, development, and manufacturing settings. For more information on this book title, pick up a flyer at the IBC marketing information section of the TIDES exhibit hall or visit http://www.crcpress.com/product/isbn/9781439819937 10 TIDES® Conference & Exhibition Featured FDA Presentation 4:45 Breakthrough Status: Are You Ready? Jeffrey Baker, Ph.D., Deputy Director, Office of Biotechnology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration 5:30 Boston Tea Party Celebrate Like It’s 1773! Networking, Wine and Cheese Reception in Poster and Exhibit Hall Relax with fellow attendees in the poster & exhibit hall over complimentary drinks & snacks, as we celebrate the 240th anniversary of the famous Boston Tea Party held in 1773. Sponsored by: Wednesday, May 14, 2014 • Main Conference Oligonucleotide and Peptide Manufacturing, Technology and Product Development Registration and Coffee 7:30 (Ballroom B) (Room 552) 8:15 Chairman’s Remarks Chairman’s Remarks Doug Brooks, Ph.D., Vice President, Manufacturing Development, Ophthotech Corporation Aaron M. Almeida, Ph.D., Senior Scientist, Chemistry, Arrowhead Research Corporation Manufacturing Advances, Process Improvements and Scale Up Session Sponsored by 8:30 Liquid API – Removing Lyophilization from the API Process Using Conjugation Technology to Advance Therapeutic Oligonucleotides and Peptides Analytical Roadmap for Lipid Nanoparticle Encapsulated siRNA’s Chandra Gamlath, Ph.D., Principal Scientist, Pfizer William Zedalis, Scientist, Analytical/CMC, Alnylam Pharmaceuticals, Inc. Oligonucleotide Drug Delivery and Cleavable Linker Technology 9:00 Unpublished Data Spray Drying: The Alternative Peptide Isolation Technology to Lyophilization Paul Tastenhoye, Dr.ir., Head of Program Management Peptides, Lonza, Belgium Next Generation Dynamic Polyconjugates for siRNA Delivery in vivo 9:30 Case Study Davunetide: Process Development and Scale-Up Aaron M. Almeida, Ph.D., Senior Scientist, Chemistry, Arrowhead Research Corporation Najib Maslouh, Ph.D., Vice President, Production, Bachem Americas, Inc. 10:00 Case Study Manufacture of GalNAc Conjugated Oligonucleotides: Process Development and Scale up Lubomir V. Nechev, Ph.D., Vice President, Process Sciences, Alnylam Pharmaceuticals, Inc. Kenneth W. Hill, Ph.D., Principle Investigator, Agilent Technologies, NASD Case Study Development of an Intein Based Conjugation Platform for the Synthesis of Fc-fusion Proteins Ryan Casey, Ph.D., Postdoctoral Fellow, Amgen Networking Refreshment Break in Poster and Exhibit Hall 10:30 Strategy Discussion Forum: Assessing the Current State of Conjugation, Fusion, and Encapsulation Technologies Spotlight Presentations (Ballroom B) (Room 552) (Room 555) 11:15 Unpublished Data Problems Associated with Acetyl Capping during Oligonucleotide Synthesis This discussion will focus on assessing established as Andrew A. Rodriguez, Ph.D., Senior Scientist, Process well as promising new technologies for improving the Organic Chemistry, Isis Pharmaceuticals, Inc. Peptide-based Drug Discovery & pharmacokinetic and pharmacodynamic properties of Development, Common Misconceptions bioactive peptides and oligonucleotides. The panel will Laszlo Otvos, Ph.D., Professor, Temple University examine lessons learned from commercial successes and discuss the key objectives necessary for successful 11:45 Case Study Chemical Synthesis of “tide”-conjugate development. The panel will assess the Peptide Macrocycles advantages and limitations of the various approaches, Guenther Loidl, Ph.D., Vice President Research and Unpublished Data Case Study and answer the audience’s questions on this topic. Development, Bachem AG, Switzerland High Loading Polymeric Solid Support Discussion Moderator: for Efficient and Cost-Effective Aaron M. Almeida, Ph.D., Senior Scientist, Chemistry, Oligonucleotide Synthesis Arrowhead Research Corporation Mohammad Ahmadian, Ph.D., Associate Panelists: Director, Kinovate Life Sciences Ryan Casey, Ph.D., Postdoctoral Scholar, Amgen 12:15 Audience Interactive Panel Discussion Kenneth W. Hill, Ph.D., Principle Investigator, Agilent Technologies, NASD Novel Strategies for the Scale-up of Oligonucleotide and Peptides: Thinking Outside the Box William Zedalis, Scientist, Analytical/CMC, The panel members will provide additional opportunity to participate in discussions related to the Alnylam Pharmaceuticals, Inc. management of process scale-up through development and from a regulatory perspective. Moderator: Doug Brooks, Ph.D., Vice President, Manufacturing Development, Ophthotech Corporation Panelists: Mimoun Ayoub, Ph.D., Director, Global Peptides & Injectables Platform, Strategic Development, CordenPharma, Switzerland Chandra Gamlath, Ph.D., Principal Scientist, Pfizer Jon Holbech Rasmussen, Ph.D., Director, Global Development, PolyPeptide Group, Sweden Lubomir V. Nechev, Ph.D., Vice President, Process Chemistry, Alnylam Pharmaceuticals, Inc. Andrew Rodriguez, Senior Scientist, Isis Pharmaceuticals, Inc. Paul Tastenhoye, Dr.ir., Head of Program Management Peptides, Lonza, Belgium 12:45 Networking Luncheon in Poster and Exhibit Hall 12 TIDES® Conference & Exhibition Sponsored by: Wednesday, May 14, 2014 • Main Conference Oligonucleotide Therapeutics Discovery Peptide Discovery and Development Registration and Coffee 8:00 (Ballroom D) (Room 551) 8:15 Chairman’s Remarks Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark Medicinal Chemistry and Chemical Modifications to Improve Oligonucleotide Properties 8:30 Small Molecules That Enhance the Pharmacological Effects of Oligonucleotides Rudy Juliano, Ph.D., Professor, School of Pharmacy, University of North Carolina 9:00 New Structure Activity Determinants for LNA Oligonucleotides Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark Chairman’s Remarks Thomas Vorherr, Ph.D., Director, Peptide Discovery, Novartis Pharma AG, Switzerland Featured Presentation (8:20) A Renaissance of Peptide, Peptidomimetic and Proteomimetic Drug Discovery Tomi Sawyer, Ph.D., Past-Founding Chief Scientific Officer, Aileron Therapeutics and Adjunct Professor, University of Massachusetts and Northeastern University Peptide Discovery and Hit Finding (8:50) Application of In Vitro V(D)J Recombination to Generate Peptide-Grafted Antibodies to G Protein-Coupled Receptors Michael Gallo, Ph.D., President, Discovery, Innovative Targeting Solutions, Inc. (9:15) Venomics in Medicinal Chemistry: Reverse-Discovery Peptide Drug Discovery and Lead Optimization Tools Inspired by Nature 9:30 RNAi Therapeutics Using GalNAc-siRNA Conjugate Delivery Platform Reto Stocklin, Ph.D., President and CEO, Atheris Laboratories, Switzerland (9:40) Venoms to Drugs Muthiah (Mano) Manoharan, Ph.D., Senior Vice President, Drug Discovery, Paul Alewood, Ph.D., Professor and Head, Division of Chemistry and Structural Biology, Institute for Molecular Bioscience, University of Queensland, Australia Alnylam Pharmaceuticals, Inc. 10:00 Carba-LNA Modified Oligos (siRNA & Antisense) for Cellular mRNA Targeting Jyoti Chattopaohyaya, Ph.D., Professor, Chemical Biology Program, Department of Cell & Molecular Biology, University of Uppsala, Sweden Display Technologies and Discovery Platforms (10:05) High Throughput Discovery and Optimization of Peptide Target Binders with Peptide Display and PEPperCHIP® Peptide Microarrays Volker Stadler, Ph.D., CEO, PEPperPRINT, Germany Networking Refreshment Break in Poster and Exhibit Hall 10:30 Medicinal Chemistry and Chemical Modifications to Improve Oligonucleotide Properties 11:15 DNA Strand Invading Locked Nucleic Acid Oligonucleotides Downregulate Huntingtin Gene Expression C. I. Edvard Smith, M.D., Ph.D., Professor, Laboratory Medicine Karolinska Institutet, Sweden Discovery of Membrane Permeable Peptides Able to Hit MicroRNA Targets Stephen Fiacco, Ph.D., CEO, EvoRx Technologies 11:45 Self-Delivering Bioreversible, PhosphoTriester RiboNucleic Neutral (siRNN) Prodrugs Steven Dowdy, Ph.D., Professor, Cellular and Molecular Medicine, UCSD School of Medicine 12:15 Role of Chemical Modification in Improving RNA Therapeutics Xianbin Yang, Ph.D., Director, R&D, AM Biotechnologies Direct Selection of Macrocyclic Peptides Targeting Intracellular Protein-Protein Interactions Kris Josephson, Ph.D., Director of Molecular Biology, Ra Pharmaceuticals, Inc. PeptiDream’s PD Platform: Innovative Peptide Discovery Platform Patrick C. Reid, Ph.D., Chief Scientific Officer, PeptiDream, Inc., Japan 12:45 Networking Luncheon in Poster and Exhibit Hall Sponsored by: www.IBCLifeSciences.com/TIDES13 Wednesday, May 14, 2014 • Main Conference Oligonucleotide and Peptide Manufacturing, Technology and Product Development (Ballroom B) (Room 552) 1:55 Chairman’s Remarks Chairman’s Remarks Matthias Kretschmer, Ph.D., Director, Analytical Sciences, Alnylam Pharmaceuticals, Inc. Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion Advances in Analytics and Control of Oligonucleotides Emerging Technologies 2:00 Establishing Specification Limits for Phosphorothioate Oligonucleotides Claus Rentel, Ph.D., Executive Director, Analytical Development Quality Control, Presentation TBA Isis Pharmaceuticals, Inc. 2:30 Analytical Roadmap for GalNAc Ligated siRNA’s Matthias Kretschmer, Director, Analytica/CMC, Alnylam Pharmaceuticals, Inc. First Manufacturing Results from the Next Generation OligoProcess Synthesizer Dr. Hüseyin Aygün, CSO, BioSpring GmbH 3:00 Analytical Challenges in the Quality Control of PEGylated Spiegelmers Johannes Hoos, Senior Director Chemical Analytics, NOXXON Pharma AG, Germany Improving Peptide Drug Properties by Glycosylation via Chemical Methods Michael F. Haller, Ph.D., Business Development, GlyTech, Inc Networking Refreshment Break in Poster and Exhibit Hall 3:30 Spotlight Presentation 4:15 Quantitation of Oligonucleotide Therapeutics and Biomarkers in Biological Matrices: Sensitivity vs. Specificity Laixin Wang, Ph.D., Associate Laboratory Director, Method Development, Tandem Labs Case Study Adventures in the Scale up of C-type Natriuretic Peptide Brad DeHoff, Ph.D., Director of R&D, CPC, CordenPharma Colorado Audience Interactive Panel Discussion 4:45 Challenges with Novel Technologies for the Analysis and Control of Oligonucleotides Moderator: Matthias Kretschmer, Ph.D., Director, Analytica/CMC, Alnylam Pharmaceuticals, Inc. Panelists: Johannes Hoos, Senior Director Chemical Analytics, NOXXON Pharma AG, Germany Claus Rentel, Ph.D., Executive Director, Analytical Development Quality Control, Isis Pharmaceuticals, Inc. Additional panelists to be announced 5:30 Summer Soirée Networking Cocktail Reception in Poster and Exhibit Hall What better way to kick off Summer 2014 than to relax over cocktails and snacks with fellow attendees? Join us at this summer-themed reception in the poster and exhibit hall, where you can network with attendees and speakers in a relaxed, informal environment. 14 TIDES® Conference & Exhibition Wednesday, May 14, 2014 • Main Conference Oligonucleotide Therapeutics Discovery Peptide Discovery and Development (Ballroom D) (Room 551) 1:55 Chairman’s Remarks Chairman’s Remarks Steven Dowdy, Ph.D., Professor, Cellular and Molecular Medicine, UCSD School of Medicine Discovery and Preclinical Strategies 2:00 Targeted Therapeutic Alternative Splicing Steve Wilton, Ph.D., Director, Foundation Chair in Molecular Therapeutics, Australian Neuromuscular Research Institute, Murdoch University, Australia 2:30 A Novel, Potent, and Safe RNAi Therapeutic Platform Utilizing LUNAR© Delivery Technology Pad Chivukula, Ph.D., Chief Operating Officer, Arcturus Therapeutics, Inc. Tomi Sawyer, Ph.D., Past-Founding Chief Scientific Officer, Aileron Therapeutics; Adjunct Professor, University of Massachusetts and Northeastern University Computational Modeling and Design of Peptides Session Sponsored by: The Role of Computation in Peptide Drug Design: Discovery, Understanding and Design Joseph Audie, Ph.D., CEO and CSO, CMDBioscience Unexpected Structure-permeability Relationships in Cyclic Peptides inspired by Natural Products: Charting Islands of Bioavailability beyond the Rule of 5s Scott Lokey, Ph.D., Professor, Chemistry and Biochemistry, University of California Santa Cruz 3:00 Novel Potent Antisense Oligonucleotides Targeting Transforming Growth Factor beta (TGF-β) Isoforms Michel Janicot, Ph.D., Chief Scientific Officer, Isarna Therapeutics GmbH, Germany A Systematic Approach for Targeting Protein-Protein Interactions Paramjit Arora, Ph.D., Professor, Department of Chemistry, New York University Networking Refreshment Break in Poster and Exhibit Hall 3:30 Discovery and Preclinical Strategies 4:15 The Prognostic and Predictive Utility of Measuring Biomarkers in Serum and Other Bodily Fluids Nelson Chau, Ph.D., Director of Target Identification and Target Assay Group, Regulus Therapeutics Audience Interactive Panel Discussion 4:45 What Constitutes a Good Target for Further Insights for Intracellular Delivery of Cyclic Peptides Thomas Vorherr, Ph.D., Director, Peptide Discovery, Novartis Pharma AG, Switzerland Novel Chemistries for Peptidomimetic Libraries: Diagnostic and Therapeutic Applications Targeting Antigen-Specific Antibodies and B Cells With Synthetic “Antigen Surrogates Oligonucleotide Modalities? • Identifying targets by comparing expression profiles of disease vs. healthy tissues • Credentialing targets with relevant animal models and/or human systems • Coding or non-coding – what are the issues? • Target expression in organs • Targeted effects versus off-target effects? Moderator: Nelson Chau, Ph.D., Director of Target Identification and Target Assay Group, Regulus Therapeutics Panelists: Muthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals Jyoti Chattopaohyaya, Ph.D., Professor, Chemical Biology Program, Cell & Molecular Biology, University of Uppsala, Sweden Nebojsa Janjic, Ph.D., Chief Science Officer, SomaLogic, Inc. Romesh Subramanian, Ph.D., Senior Director, Biology, RaNA Therapeutics 5:30 Intracellular Delivery of Peptides Jumpei Morimoto, Ph.D., Research Associate, The Scripps Research Institute, Scripps Florida Summer Soirée Networking Cocktail Reception in Poster and Exhibit Hall What better way to kick off Summer 2014 than to relax over cocktails and snacks with fellow attendees? Join us at this summer-themed reception in the poster and exhibit hall, where you can network with attendees and speakers in a relaxed, informal environment. www.IBCLifeSciences.com/TIDES15 Thursday, May 15, 2014 • Main Conference Oligonucleotide and Peptide Manufacturing, Technology and Product Development Coffee 7:30 (Ballroom B) (Room 552) 8:25 Chairman’s Remarks Chairman’s Remarks Paul McCormac, Ph.D., Director, Biomanufacturing Sciences Group, Pfizer Global Manufacturing Process Validation: Design, Qualify, Verify 8:30 Unpublished Data Oligonucleotide Drug Substance Manufacturing: Process Development, Validation and the Application of Platform Knowledge Andy McPherson, Ph.D., Associate Director, Process Organic Chemistry, Isis Pharmaceuticals, Inc. 9:00 Innovative Approaches to Multi Sequence Platform Validation Emma Wright, D.Phil., Senior Director, Process Development, Nitto Denko Avecia Inc. 9:30 What are Critical Quality Attributes for Peptide APIs? Anita Y. Szajek, Ph.D., Principal Scientific Liaison, U.S. Pharmacopeia Christopher A. Rhodes, Ph.D., President, Christopher A. Rhodes & Associates LLC Formulation and Delivery Technologies: Enabling Peptide and Oligo Product Opportunities Overview of Oral, Transdermal, Pulmonary, CNS and Nasal Routes Christopher A. Rhodes, Ph.D., President, Christopher A. Rhodes & Associates LLC Unpublished Data Case Study Physicochemical Property and Formulation Risk Assessment for Peptides and Case Studies Dennis H. Leung, Ph.D., Principal Scientist, Basic Pharmaceutical Sciences, Merck Research Laboratories, Merck & Co., Inc. Unpublished Data Case Study Enhanced Stability and Drug Delivery of Peptides with Non-Aqueous Formulations Neelima Phadnis, Ph.D., CMC Director, Xeris Pharmaceuticals, Inc. 10:00 Unpublished Data Case Study A Continuous Verification Approach to Oligonucleotide Process Validation Soumitra S. Ghosh, Ph.D., Former Senior Director, Research, Amylin Pharmaceuticals LLC Jeff Beckvermit, Senior Scientist, Commercial Launch, Agilent Technologies, NASD Networking Refreshment Break in Poster and Exhibit Hall 10:30 11:15 Design of Peptide Therapeutics Exhibiting Dual Pharmacology and Extended Duration of Action Strategy Discussion Forum (Room 555) What’s Changed in our Approach to Process Validation? With the advent of the FDA (2011) and EMEA (2012 draft guidance) the industry has moved from a more event based approach to process validation to incorporating Drug-Coated Microneedle Patch for Rapid Delivery of Peptides Mahmoud Ameri, Ph.D., Director, Pharmaceutical Development, Zosano Pharma 11:45 aspects of lifecycle to their programs. What challenges have been encountered? What Unpublished Data Peptide Hydrogels for Controlled Release Drug Delivery and for Cell Culture Moderator: Paul McCormac, Ph.D., Director, Biomanufacturing Sciences Group, 12:15 Pfizer Global Manufacturing Panelists: Jeffrey Baker, Ph.D., Deputy Director, Office of Biotechnology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration Jeff Beckvermit, Senior Scientist, Commercial Launch, Agilent Technologies, NASD Andy McPherson, Ph.D., Associate Director, Process Organic Chemistry, Isis Pharmaceuticals, Inc. Jeroen van Rheenen, M.Sc., Manager, Chemical Development & Production, Prosensa, The Netherlands René Thürmer, Ph.D., Deputy Head Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany Emma Wright, D.Phil., Senior Director, Process Development, Nitto Denko Avecia Inc. Laurent Jeannin, Ph.D., Operation Manager, Peptide Biomaterials, Peptisyntha SA, Belgium have organizations seen as the benefits? 12:45 Spotlight Presentation The Light Scattering Toolbox for Biophysical Screening and Characterization of X-tide Formulations John C. Champagne, Ph.D., Senior Applications Scientist, Wyatt Technology Corporation Networking Luncheon in Poster and Exhibit Hall IBC’s 7th Annual Sponsored by: Coming in 2015: www.IBCLifeSciences.com/AsiaTIDES 16 TIDES® Conference & Exhibition Thursday, May 15, 2014 • Main Conference Oligonucleotide Therapeutics Discovery Peptide Discovery and Development Coffee 7:30 (Ballroom D) 8:25 Chairwoman’s Remarks (Room 551) Chairman’s Remarks Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel PK/PD and Toxicity of Oligonucleotides Jesse Dong, Ph.D., TIDES Scientific Advisor Peptides in Preclinical and Clinical Development 8:30 Understanding Delivery Barriers of siRNA Therapeutics by Monitoring GPCR-Specific, Lanthionine-Stabilized Agonistic Peptides Metabolic Trafficking Gert N. Moll, Ph.D., Chief Scientific Officer, Lanthio Pharma, The Netherlands Mark Cancilla, Ph.D., Director, PPDM, Merck & Co., Inc. 9:00 Mechanisms of Vascular Toxicity in Monkeys with Antisense Oligonucleotides Kendall S. Frazier, Ph.D., Senior Managing Director of Cellular Pathology, GlaxoSmithKline Engineering Venom Peptides Targeting the Kv1.3 Ion Channel Wilson Edwards, Principal Scientist, Biologics Research, Janssen Research & Development Preclinical and Clinical Oligonucleotides 9:30 Update on Mirror-image Aptamers in Phase IIa Studies Sven Klussmann, Ph.D., Chief Scientific Officer, Noxxon Pharma AG, Germany The Utility of Selective Melanocortin 1 Receptor (MC1R) Agonists in Inflammatory Conditions John Dodd, Ph.D., Vice President, Preclinical Research, Palatin Technologies, Inc. 10:00 The Role of PMO Chemistry in Emerging Infections Diseases: Efficacy and Safety in Hemorrhagic Virus Therapeutics to Potential Treatment of Multi-drug Resistant Bacteria Michael Wong, M.D., Senior Medical Director, Sarepta Therapeutics Neuroprotection in Preclinical Models of Parkinson Disease by the NAPVSIPQ Peptide Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion Networking Refreshment Break in Poster and Exhibit Hall 10:30 11:15 Aptamers and Conjugates Exemplified by UNA- and LNA-based Constructs Screening and Delivery of Peptidic Inhibitors of Protein-protein Interactions Jesper Wengel, Ph.D., Chairman and CSO, RiboTask and Professor, Center Director BioNEC, University of Southern Denmark Bernard Lebleu, Ph.D., Professor of Molecular Biology, University of Montpellier, France 11:45 Multivalent N-Acetylgalactosamine Conjugate Significantly Improves Potency of Antisense Oligonucleotides in Mice Guanylate Cyclase-C Agonists for the Treatment of Gastrointestinal Disorders and Inflammatory Bowel Diseases Thazha P. Prakash, Ph.D., Senior Research Fellow, Isis Pharmaceuticals Kunwar Shailubhai, Ph.D., Chief Scientific Officer, Synergy Pharmaceuticals, Inc. 12:15 Use of RNAi for Treatment of Inner Ear Disorders Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel Successful Drug Discovery and Development Case Studies of Protein Epitope Mimetics Daniel Obrecht, Ph.D., Chief Scientific Officer, Polyphor Ltd., Switzerland 12:45 Networking Luncheon in Poster and Exhibit Hall Sponsored by: www.IBCLifeSciences.com/TIDES17 Thursday, May 15, 2014 • Main Conference Oligonucleotide and Peptide Manufacturing, Technology and Product Development (Ballroom B) (Room 552) 1:55 Chairman’s Remarks Chairman’s Remarks Claus Rentel, Ph.D., Executive Director, Analytical Development Quality Control, Isis Pharmaceuticals, Inc. Raw Materials and Supply Chain: Assessing and Mitigating Risk with Novel and Traditional Starting Materials 2:00 Novel Expectations for Raw Materials and Supply Chain: Pitfalls and Christopher A. Rhodes, Ph.D., President, Christopher A. Rhodes & Associates LLC Formulation and Delivery Technologies: Enabling Peptide and Oligo Product Opportunities (continued) How to Avoid Them Complex Polymeric Formulations of Oligonucleotides: CMC and Regulatory Experience Brant Zell, Vice President and Global Director, Quality, PolyPeptide Group Jeremy D. Heidel, Ph.D., President, Informulate LLC 2:30 Case Study Assessing and Mitigating Risk in the Raw Material Supply Chain Manufacture of Lipid Nanoparticle Formulations: Novel Characterization Assays for Release Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals, Inc. Uditha deAlwis, Ph.D., Senior Director, Analytical Sciences, Nitto Denko Avecia Inc. Networking Refreshment Break 3:00 3:30 Specialty Amidites: Challenges and Solutions Strategy Discussion Forum (Room 556) Christoph Rosenbohm, Ph.D., Senior Director, Research Operations, Santaris Pharma A/S, Denmark Challenges in CMC Development of Novel Delivery Systems: Functional Excipients, Novel Formulations and Regulatory Expectations Strategy Discussion Forum 4:00 Where Does your Building Block Come From? Suppliers, manufacturers, and drug sponsors all have a stake in ensuring a high quality raw material supply chain that will support commercial API production. This session addresses the various challenges associated with securing a robust supply chain of critical raw materials used in oligonucleotide and peptide manufacturing. Peptides Discussion (Room 554) Moderator: Thomas Meier, Vice President, Production, Bachem AG, Switzerland Panelist representing sponsor companies: Jeroen M. Bezemer, Ph.D., Scientific Director, Small Molecule Process and Product Development, Amgen Panelist representing CMO’s: Brant Zell, Vice President and Global Director, Quality, PolyPeptide Group Panelist representing suppliers: Gary W. Erickson, Ph.D., CEO, CBL Biopharma LLC Marc J. Jacob, Ph.D., Product Manager, Chiral & Preparative Chromatography, Phenomenex Oligonucleotides Discussion (Room 555) Moderator: René Thürmer, Ph.D., Deputy Head Unit Pharmaceutical Biotechnology, Moderator: BfArM - Federal Institute for Drugs and Medical Devices, Germany Claus Rentel, Ph.D., Executive Director, Panelists: Analytical Development Quality Control, Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Isis Pharmaceuticals, Inc. Research, Dicerna Pharmaceuticals Panelists representing sponsor companies: Jeremy Heidel, Ph.D., President, Informulate, LLC Jayant Aphale, Ph.D., MBA, RAC, Dennis H. Leung, Ph.D., Principal Scientist, Basic Pharmaceutical Sciences, Senior Vice President, Technical Merck Research Laboratories, Merck & Co., Inc. Operations, Sarepta Therapeutics Christoph Rosenbohm, Ph.D., Senior Director, Research Operations, Santaris Pharma A/S, Denmark Panelist representing CMO’s: Kalakota Reddy, Ph.D., Development Manager, Nucleic Acids Solutions Division, Agilent Technologies Inc. Hagen Cramer, Ph.D., Director, Process Development, Nitto Denko Avecia Inc. Panelists representing suppliers: John Koterba, Product Manager EMD Solvents, EMD Millipore Yogesh S. Sanghvi, Ph.D., President, Rasayan, Inc. Dr. Andreas Wolter, Site Manager, Sigma-Aldrich Biochemie GmbH, Germany Close of Conference 5:00 18 In large part, the successful development of therapeutics is predicated on the development of safe and effective drug product formulations. This session will examine recent advances in formulation development, including those in the field of liposomal, polymer and nanoparticle-based delivery vehicles. Strategies aimed at mitigating or eliminating the toxicity associated with oligonucleotide and peptide formulations will be presented. Recommendations for designing studies to determine potential toxicities of novel and functional excipients will be discussed. TIDES® Conference & Exhibition Thursday, May 15, 2014 • Main Conference Oligonucleotide Therapeutics Discovery Peptide Discovery and Development (Ballroom D) (Room 551) 1:55 Chairman’s Remarks Chairman’s Remarks David Brown, Ph.D., Director of Research, Mirna Therapeutics, Inc. Paul Watt, Ph.D., Chief Scientific Officer, Phylogica Ltd., Australia Endosomal Escape, Peptide Targeting, and Intracellular Delivery Preclinical and Clinical Oligonucleotides 2:00 Preclinical and Clinical Development of a miRNA-Based Cancer Therapy The Endosome Escape Trap: a Tool to Discover More Efficient Cell Penetrating Peptides David Brown, Ph.D., Director of Research, Mirna Therapeutics, Inc. Paul Watt, Ph.D., Chief Scientific Officer, Phylogica Ltd., Australia 2:30 Nucleic Acid Ligands with Enhanced Chemical Diversity for Delivery of Proteins into Live Human Cells: Virus-inspired Strategies and Therapeutic Opportunities Development of Novel Therapeutics Nebojsa Janjic, Ph.D., Chief Science Officer, SomaLogic, Inc. Jean-Philippe Pellois, Ph.D., Associate Professor, Department of Biochemistry and Biophysics, Texas A&M University Networking Refreshment Break 3:00 3:30 RNA Therapeutics to Selectively Activate Protein Expression Romesh Subramanian, Ph.D., Senior Director, Biology, RaNA Therapeutics p28, A First-in-class, First-in-human, Non-HDM2-mediated Peptide Inhibitor of p53 Ubiquitination Craig W. Beattie, Ph.D., Professor, Surgical Oncology, Department of Surgery, University of Illinois at Chicago 4:00 Clinical Development of RXI-109 to Prevent Dermal Scarring Karen G. Bulock, Ph.D., Vice President, Research, RXi Pharmaceuticals Creation of Brain-penetrant Small Molecules, Peptides and Proteins by Peptide Conjugation Jean Lachowicz, Ph.D., Chief Scientific Officer, Angiochem, Inc., Canada 4:30 Close of Oligonucleotide Therapeutics Discovery CNS Delivery of Peptide Therapeutics Mansoor M. Amiji, Ph.D., Distinguished Professor and Chairman, Department of Pharmaceutical Sciences, School of Pharmacy, Northeastern University 5:00 Close of Conference See You Next Year! 2015 May 12-15, 2014 Rhode Island Convention Center • Providence, RI Check www.IBCLifeSciences.com/TIDES for next year’s date and location www.IBCLifeSciences.com/TIDES19 EXHIBIT HALL MAP 20 TIDES® Conference & Exhibition P O O P P P P O P P P O O P O P O O P P P P O P P O P O P O P P P O P O P O O O O O P O O P O P O P O P O O P P O P O P O O O P Poster and Exhibit Hall Hours Tuesday, May 13 • 3:15 pm - 6:45 pm Wednesday, May 14 • 10:30 am - 6:30 pm Thursday, May 15 • 10:00 am - 2:00 pm O : Oligo-Focused P : Peptide-Focused 335 434 Resource Center AAPPTec . . . . . . . . . . . . . . . . . . . . . . . . . . . 330 Agilent . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 309 AIC/Tedia . . . . . . . . . . . . . . . . . . . . . . . . . . . 233 Ajinomoto Althea, Inc. . . . . . . . . . . . . . . 109 Almac . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 334 Ambio Pharm, Inc. . . . . . . . . . . . . . . . . . . 117 American Peptide Company . . . . . . . . 103 Asahi Kasei Bioprocess . . . . . . . . . . . . . . 206 Bachem . . . . . . . . . . . . . . . . . . . . . . . . . . . . 403 BCN Peptides . . . . . . . . . . . . . . . . . . . . . . . 419 Berry & Assoc. Inc . . . . . . . . . . . . . . . . . . . 213 BioAutomation . . . . . . . . . . . . . . . . . . . . . 307 Biopeptek, Inc. . . . . . . . . . . . . . . . . . . . . . 107 BioSpring GmbH . . . . . . . . . . . . . . . . . . . 230 Biotage . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 303 BioTechLogic, Inc. . . . . . . . . . . . . . . . . . . . 228 Buchiglas USA . . . . . . . . . . . . . . . . . . . . . . 335 California Peptide . . . . . . . . . . . . . . . . . . 333 C.A.T. GmbH & Co KG . . . . . . . . . . . . . . . 116 Carbosynth LLC . . . . . . . . . . . . . . . . . . . . . 423 CAS . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 331 CEM . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 216 Cepia Sanofi . . . . . . . . . . . . . . . . . . . . . . . . 402 Certified Scientific Instruments, Inc . 413 Chemgenes . . . . . . . . . . . . . . . . . . . . . . . . 315 Corden Pharma International . . . . . . . 302 CPC Scientific . . . . . . . . . . . . . . . . . . . . . . . 225 CS Bio Co. . . . . . . . . . . . . . . . . . . . . . . . . . . 203 Daiso Co. Ltd. . . . . . . . . . . . . . . . . . . . . . . . 431 EMD Millipore . . . . . . . . . . . . . . . . . . . . . . 425 Exiqon . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 129 GE Healthcare . . . . . . . . . . . . . . . . . . . . . . 102 Gene Design, Inc. . . . . . . . . . . . . . . . . . . . 119 Glen Research . . . . . . . . . . . . . . . . . . . . . . 406 Halocarbon Products . . . . . . . . . . . . . . . 112 Honeywell Burdick & Jackson . . . . . . . 324 Hongene Biotechnology Ltd. . . . . . . . 229 Hybio Pharmaceutical . . . . . . . . . . . . . . 328 Intavis, Inc. . . . . . . . . . . . . . . . . . . . . . . . . . 126 Irvine Pharmaceutical Services, Inc. . 120 JenKem Technology USA . . . . . . . . . . . 108 Kinovate Life Sciences, Inc. . . . . . . . . . . 224 Lonza . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 312 Luxembourg Biotechnologies . . . . . . 333 Mallinkrodt Pharmaceuticals . . . . . . . 210 Metkinen Chemistry . . . . . . . . . . . . . . . . 329 Nagase America Corporation . . . . . . . 135 National Institute of Pharmaceutical . 424 Nitto Denko Avecia Inc. . . . . . . . . . . . . . 215 NOF Corporation . . . . . . . . . . . . . . . . . . . 417 135 234 131 230 129 334 333 432 435 330 331 430 431 228 229 328 329 428 429 125 224 225 324 325 424 425 123 222 223 323 422 423 233 126 120 118 119 116 117 216 215 315 419 417 114 112 111 109 210 213 312 211 309 413 411 108 106 102 107 105 103 206 207 306 202 203 302 305 404 303 402 407 403 Entrance Entrance Novasep . . . . . . . . . . . . . . . . . . . . . . . . . . . . 429 NSF Pharmalytica . . . . . . . . . . . . . . . . . . . 404 PCAS BioMatrix, Inc. . . . . . . . . . . . . . . . . 111 Peptide Machines Inc. . . . . . . . . . . . . . . 428 Phenomenex . . . . . . . . . . . . . . . . . . . . . . . 306 PolyPeptide Group . . . . . . . . . . . . . . . . . 202 Precision NanoSystems . . . . . . . . . . . . . 434 Prime Syntheses . . . . . . . . . . . . . . . . . . . . 211 Protein Technologies . . . . . . . . . . . . . . . 435 Pyramid Laboratories, Inc. . . . . . . . . . . 325 Quanta BioDesign . . . . . . . . . . . . . . . . . . 323 307 406 P O P P O P P O O O P O P P RiboBio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 234 ScinoPharm . . . . . . . . . . . . . . . . . . . . . . . . 123 ShengNuo Peptide . . . . . . . . . . . . . . . . . 207 Sigma-Aldrich . . . . . . . . . . . . . . . . . . . . . . 223 ST Pharm . . . . . . . . . . . . . . . . . . . . . . . . . . . 422 Sussex Research . . . . . . . . . . . . . . . . . . . . 305 Thermo Scientific . . . . . . . . . . . . . . . . . . . 411 Tosoh Bioscience LLC . . . . . . . . . . . . . . . 222 TriLink Biotechnologies, Inc. . . . . . . . . 407 Waters Corporation . . . . . . . . . . . . . . . . . 114 O O O O O O O O P P P P SCIENTIFIC ADVISORY BOARDS For Oligonucleotide and Peptide Manufacturing Technology and Product Development Track Kathryn L. Ackley, Ph.D., RAC, Senior Director Cincinnati Operations, Nitto Denko Avecia Inc. Firoz Antia, Ph.D., CMC Team Director, Pharmaceutical Operations and Technology, Biogen Idec Gary F. Musso, Ph.D., President, Musso and Associates LLC Lubomir V. Nechev, Ph.D., Vice President, Process Chemistry, Alnylam Pharmaceuticals, Inc. El Djouhar Rekaï, Head of Peptide Products Operation, Lonza, Belgium Mimoun Ayoub, Ph.D., Vice President, Global Peptides and Injectables, Corden Pharma Claus Rentel, Ph.D., Executive Director, Analytical Development Quality Control, Isis Pharmaceuticals Neil H. Baine, Ph.D., Past Executive Director, Scinovo, Preclinical Development, GlaxoSmithKline Pharmaceuticals (Retired) Christopher A. Rhodes, Ph.D., President, Christopher A. Rhodes & Associates LLC Doug Brooks, Ph.D., Vice President, Manufacturing Development, Ophthotech Corporation Christoph Rosenbohm, Ph.D., Senior Director, Research Operations, Santaris Pharma A/S, Denmark Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals Trishul Shah, Key Account Manager, Business Development, North America, PolyPeptide Laboratories Alex Fässler, Ph.D., President and COO, Bachem Americas Inc. G. Susan Srivatsa, Ph.D., President, ElixinPharma Peter Hoffmann, Ph.D. James D. Thompson, Ph.D., Head, Process Sciences, Moderna Therapeutics, Inc. David Litzinger, Ph.D., President, David Litzinger and Associates, LLC John P. Mayer, Ph.D., Senior Research Scientist, Department of Chemistry, Indiana University Paul McCormac, Ph.D., Director, Biomanufacturing Sciences Group, Pfizer Global Manufacturing David Tumelty, Ph.D., Director of Chemistry, New England Peptide Blake Unterreiner, Business Development Manager, Nucleic Acid Solutions Division, Agilent Technologies, Inc. Fran Wincott, Ph.D., President, Wincott & Associates, LLC Michael McGinley, Bioseparations Product Manager, Phenomenex Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion For Oligonucleotide Therapeutics Discovery Track For Peptide Discovery and Development Track David Brown, Ph.D., Director of Research, Mirna Therapeutics, Inc. Jesse Dong, Ph.D., TIDES Scientific Advisor Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel Tomi Sawyer, Ph.D., Past-Founding CSO, Aileron Therapeutics and Adjunct Professor, University of Massachusetts and Northeastern University Troels Koch, Ph.D., Vice President, Research and CTO, Santaris Pharma A/S, Denmark Muthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc. Pamela Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals 22 TIDES® Conference & Exhibition Peter Timmerman, Ph.D., Chief Scientific Officer, Pepscan Therapeutics, The Netherlands Thomas Vorherr, Ph.D., Director Peptide Discovery, Novartis Pharma AG, Switzerland Paul Watt, Ph.D., CEO, Phylogica Ltd., Australia POSTER PRESENTATIONS 1 Towards the Implementation of Novel Computation Tools in CMDInventus for Understanding and Solving ADME/Tox Issues in Peptide-Based Drug Discovery Alexander Bayden CMD Bioscience 2 Analytical Separation of Oligonucleotide Derivatives Using Strong Anion Exchange and C18 Reversed Phase Chromatography Atis Chakrabarti Tosoh Bioscience, LLC 3 Determination of Glucagon in Human Plasma by LC/MS/MS John Chappell CPR Pharma Services 4 The Development, Validation and Application of Various Biophysical Modules in CMDInventus to Enable Structure-Based Peptide Drug Design and Discovery David Diller CMD Bioscience 5 GLP Quantitation of Peptides in Biological Matrices by LC-MS/MS Andrew Dinan CPR Pharma Services Pty Ltd 6 Ion-Pairing Systems for Reversed-Phase Chromatography of Oligonucleotides Ananya Dubey Waters 7 Developing Fit for Purpose Manufacturing Processes Alastair Hay Almac 8 The Chemical Synthesis of Long and Highly Modified RNA using 2’-ACE Chemistry Kaizhang He GE Healthcare 9 Systematic Optimization of Separation Conditions for the Isolation of Synthetic Peptides Jo-Ann Jablonski Waters Corporation 10 HPLC Enantioseparation Of N-Fmoc Amino Acids Using Polysaccharide-Based Chiral Stationary Phases Under Reversed Phase Conditions Marc Jacob Phenomenex 11 Cancer-targeted Delivery of siRNA in HepG2 Hepatocellular Carcinoma Stesha Joseph Seton Hall University 12 Development of miRNA-modulating Drug for Alzheimer’s Disease Kris Choi ST Pharm 13 Development of siRNA of Human Noxin, an Anti-Apoptotic Protein in Response to DNA Damage of A549 Non-Small Cell Lung Carcinoma Kyeong Eun Jung ST Pharm 14 A High Capacity 150Å Reversed-Phase Silica Gel for the Purification of Oligonucleotides Audrey Kelleman Grace 15 A Novel Potent Antimicrobial Peptide, SHAP-19 as a Potential Topical Agent Da Jung Kim KAIST 16 Synthesis, Characterization and Inhibitory Activity of Peptide Mimics of the Insulin Receptor Tyrosine Kinase Lathamol Kurian Seton Hall University 17 A Novel Cell-Penetrating Peptide, BR2, for the Efficient Delivery of a scFv into Cancer Cells Young Woong Lee Korea Advanced Institute of Science and Technology 18 Inhibition of HIv-1 Subtype c by RNA Aptamers Specific to gp120 Grace London Council for Scientific and Industrial Research (CSIR) 19 Effect of Triazine Impurity in NMI Capping Reagent on Oligonucleotides Part 2: Evidence for T+85 Adduct Sandra Lorenz Honeywell Burdick & Jackson 20 EdU an Alternative to BrdU for Cell Proliferation Assays Antonio Manetto baseclick GmbH 21 Therapeutic Implications of Controlling P-chirality in Phosphorothioate Oligonucleotides Meena Meena WaVe Life Sciences 22 Development of a Novel Bioassay Using mRNA Transcription as a Readout Pedro Morales Catalent Pharma Solutions 23 Efficient RNA silencing with LNA™ antisense oligonucleotides Henrik Pfundheller Exiqon A/S 24 Effective Inhibition of Tumor Growth Using Elastin-Like Polypeptide Delivery of Small Interfering RNA Thomas Primiano PeptiMed 25 Pharmacological Characterization of a PEGylated, Disulfide-rich Anti-IL-6 Peptide for the Treatment of Rheumatoid Arthritis and Other Inflammatory Disorders Sheila Ranganath Ironwood Pharmaceuticals, Inc. 26 Optimizing Oligonucleoside Synthesis Using HybCPG Solid Supports Marc Rothstein Prime Synthesis, Inc. 27 Characterization of X-tides by Light Scattering: Aggregation, Self-Association, Liposome Delivery and Protein Binding John Champagn Wyatt Technology Corp. 28 The Differential Role of Clustering of Glycosaminoglycans for the Uptake of Amphipathic and Arginine-Rich Cell-Penetrating Peptides Rike Wallbrecher Radboud University Medical Centre 29 New Reagent for Minimizing Aspartimide Formation in Peptides Lisa Wang EMD Millipore 30 Charged Surface Hybrid C18 for Lab Scale Peptide Purification and High Resolution Analyses Bill Warren Waters Corp 31 Enhanced Peptide Drug Properties through Chemical N-Glycosylation: Somatostatin Derivatives with Native-Like Activity and Prolonged Half-life Naoki Yamamoto GlyTech, Inc. 32 An Evaluation of Bifunctional Linkers for Use in Polymer Conjugates: Coupling Efficiencies and Stability Studies Yuyan Zhao Pfizer Inc. 33 Application of In Vitro V(D)J Recombination to Generate Peptide-Grafted Antibodies to G Protein-Coupled Receptors Michael Gallo Innovative Targeting Solutions Inc. 24 TIDES® Conference & Exhibition 26 TIDES® Conference & Exhibition 28 TIDES® Conference & Exhibition www.nagase.co.jp/pharma/english Notes _______________________________________________________________________________________________ _______________________________________________________________________________________________ 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