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AsiaTIDES: Oligonucleotide and Peptide® Research, Technology and Product Development
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Agenda
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8:30
Registration and Coffee/Tea
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Tutorial #1
Quality by Design (QbD) / In Process Control (IPC) Requirements and Analytical Methods to Control Product Quality
Tutorial Leaders:
9:00
Start of Tutorial
Learn about analytical methods development for In Process Control during manufacturing and correlation with final product release testing. Understand the basics of Quality by Design (QbD) to control product quality. Implications to chemistry manufacturing and controls (CMCs) both initially and at later stages of product life cycle will be discussed. Instructors will present:
Plus guest presenters:
Regulatory Expectations for CMC for Peptides in Europe
Dr. René Thürmer, Deputy Head Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany
Regulatory Considerations on Setting Impurity Specification for Peptide Drug Products
Duu-Gong Wu, Ph.D., Director Regulatory Affairs/Senior Consultant, Global CMC Global Regulatory Affairs, Pharmaceutical Product Development, LLC (PPD); Former CMC Deputy Division Director, US FDA
10:20
Networking Refreshment Break
12:30
Close of Tutorial
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Tutorial #2
Approaches to Successful Delivery of Oligonucleotides in vivo: Chemical Modifications and DDS (Drug Delivery Systems)Tutorial Leader: Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China
9:00
Principles of Oligonucleotide In Vivo Delivery
Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China
9:40
Local Delivery: Lessons Learned from 'Self-Delivering' RNAi Compounds
Karen G. Bulock, Ph.D., Vice President, Research, RXi Pharmaceuticals, USA
10:20
Networking Refreshment Break
10:50
Size Matters, Gymnosis - Breakthrough Design, Chemistries And Applications
Troels Koch, Ph.D., Vice President and CTO, Santaris Pharma A/S, Denmark
11:30
Lipid Nanoparticles for Oligonucleotide Delivery: From Basic Principles to the Clinic
Peter Lutwyche, Ph.D., Senior Vice President, Pharmaceutical Development, Tekmira Pharmaceuticals Corporation, Canada
12:00
Chairman's Concluding Remarks and Discussion
Dmitry Samarsky, Ph.D., Executive Vice President, Technology Development, RiboBio, China
12:30
Close of Tutorial
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Lunch will be served for tutorial attendees.
Tutorials: Tuesday | Main Conference: Tuesday | Wednesday | Thursday
12:00
Registration for Main Conference Begins
1:30
Chairman's Remarks
Bob D. Brown, Ph.D., CSO, Senior Vice President, Research, Dicerna Pharmaceuticals, USA
1:40
Oligonucleotide Therapeutics: The Third Platform for Drug DevelopmentIn the two decades since the birth of oligonucleotide therapeutics, the field has seen a series of innovations in platform chemistries, delivery systems, and in the types of applications. The scope of targets has expanded from mRNA to include miRNA, lncRNA, DNA, and proteins. Recent years have seen dramatic improvements in potency and therapeutic index, and the progression of increasing numbers of compounds into late stage clinical development. Oligonucleotides are poised to become generally recognized, along with small molecules and biotherapeutics, as the third platform for drug development. Arthur M. Krieg, M.D., Chief Executive Officer, RaNA Therapeutics, USA Unpublished Data
2:20
Development of Lipid Nanoparticle (LNP) Based Delivery System of siRNA Targeting Extrahepatic Tumor
LNP is the most advanced delivery system of siRNA, however, its application has been limited in liver targeting and there are big hurdles to target tumor, especially in extrahepatic region. We recently developed a new LNP-based delivery system which can highly distribute into tumor and achieve target mRNA knock down. In this presentation, we discuss the hurdles and strategy to overcome based on our experience. Junichi Enokizono, Senior Scientist, Pharmacokinetic Research Laboratory, Kyowa Hakko Kirin Co., Ltd., Japan
3:00
Networking Refreshment Break
3:20
Defying Difficult Diseases: Protease Inhibitors andthe Click Peptide Concept Using O-Acyl IsopeptidesAll living organisms on Earth use the same biomolecular system, namely DNAs, RNAs and proteins/peptides consist of twenty types of L-α-amino acid. Drug discovery for many intractable diseases, such as AIDS, malaria, hypertension, diabetes and Alzheimer's disease, is related by intermolecular interactions between drugs and proteins, so called 'enzyme/receptor'. This lecture highlights the importance of peptide chemistry for the development of drugs for difficult disease. Yoshiaki Kiso, Ph.D., Professor, Laboratory of Peptide Science, Nagahama Institute of Bio-Science and Technology, Japan
4:00
The Journey Together: Omontys (Peginesatide Injection) - From Bench to BedsideOmontys (Peginesatide Injection) is a novel synthetic PEGylated peptidic ESA recently approved in the US for the treatment of anemia due to CKD in adult patients on dialysis. Highlights of the strong partnership of Affymax and Takeda in the journey of Omontys from bench to approval is presented. Ping Qiu, M.D., Senior Medical Director, Clinical Science, Takeda Global Research and Development Inc., USA
5:30
Close of AsiaTIDES Day One Sessions
6:30
Co-Sponsored by
Networking Dinner Join fellow attendees in a fantastic networking and dining opportunity at Kamadoka Restaurant Kamadoka is a traditional Izakaya restaurant particularly known for Kamameshi (traditional flavored rice) and Yakitori (spit-roasted chicken.) 
Space is limited and additional fee applies. Please indicate when you register if you plan to join the dinner.
Tutorials: Tuesday | Main Conference: Tuesday | Wednesday | Thursday
8:30
Registration and Coffee/Tea
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Updates on Oligonucleotide-Based Therapeutics in Preclinical and Clinical Development
8:55
Chairman's Remarks
William S. Marshall, Ph.D., President and Chief Executive Officer, miRagen Therapeutics, Inc., USA
9:00
microRNA Targeting for Cardiovascular Disease Intervention
Alterations in the expression of specific microRNAs have been observed in several cardiovascular disease models and human clinical samples, providing an exciting potential for therapeutic intervention. Genetic deletion and synthetic antimiR dosing have demonstrated that the inhibition of certain microRNA's is sufficient to produce beneficial outcomes in relevant disease models. An update on our latest preclinical studies is presented. William S. Marshall, Ph.D., President and Chief Executive Officer, miRagen Therapeutics, Inc., USA Unpublished Data
9:30
Development of NF-kB Decoy ODN Coated PTA Balloon Catheter for Suppressing Restenosis of Blood Vessels
We have developed decoy ODN against NF-kB, an essential transcription factor for inflammation and adhesion, to treat various inflammatory diseases. Currently, we initiate a clinical trial of NF-kB decoy ODN coated PTA balloon catheter for arteriovenous fistula stenosis in hemodialysis shunts. The presentation will include the preclinical data and clinical trial outline of the novel drug coated PTA balloon catheter for suppressing restenosis of blood vessels. Shinya Ihara, Director, Pharmaceutical Research, R&D, AnGes MG, Inc., Japan
10:00
Challenges for Preclinical and Clinical Development of siRNA Therapeutics in China
To catch the advancements of clinical development in siRNA therapeutics playing field, Sirnaomics is pushing its two siRNA therapeutic product candidates, STP705 for Scarless Skin Wound Healing and STP601 for Diabetic Ocular disease, for clinical studies in China. Working closely with the Chinese pharma sponsors, international CMC vendors, local pre-clinical and clinical CROs, and China's SFDA, the company has overcome many challenges and hurdles for getting regulatory approval for clinical study of siRNA therapeutics in China. Patrick Lu, Ph.D., Founder, President and CEO, Sirnaomics, US and China
10:30
Networking Refreshment Break in Poster and Exhibit Hall
Updates on Oligonucleotide-Based Therapeutics in Preclinical Development
11:15
Therapeutic Development Using Second Generation RNAi Triggers with Enhanced Cellular Delivery
Hear about novel RNAi trigger structures with improved delivery properties over conventional siRNA. A tripodal RNA structure complexed with cationic delivery vehicles that can trigger multi-target gene silencing with enhanced delivery in vitro and in vivo is presented. A chemically modified asymmetric RNA with cell penetrating ability and its therapeutic development are described. Dong-ki Lee, Ph.D., Principal Investigator, Global Research Laboratory for RNAi Medicine; Associate Professor, Chemistry, Sungkyunkwan University; Founder, BioMolecular Therapeutics, Inc., South Korea Unpublished Data
11:45
Case
High Potency in vivo Gene Silencing Activity via Chemically-Modified siRNAsStudy Investigating chemistry for enhancing siRNA gene silencing in vivo is very important for the development of siRNA-based therapeutic applications. To this end, the following is presented: (1) the novel modification chemistry for synthesizing modified siRNAs, (2) the biophysical properties of modified siRNAs, and (3) the high potency gene silencing activity in vitro and in vivo of modified-siRNAs. Xianbin Yang, Director, AM Biotechnologies, USA
12:15
Networking Luncheon in Poster and Exhibit Hall
Updates on Oligonucleotide-Based Therapeutics in Clinical Development
Session Sponsored by:
1:30
Chairwoman's Remarks
Pamela A. Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals, USA Unpublished Data
1:45
Case
Update on RXI-109, an RNAi Compound in Clinical Development to Reduce Dermal ScarringStudy RXI-109 is the first member of a novel class of RNAi compounds, termed 'self-delivering' or sd-rxRNA, to enter into clinical development. RXI-109 targets connective tissue growth factor (CTGF), a key regulator of fibrosis and scar formation, and is initially being developed to reduce or inhibit dermal scarring. Safety and tolerability data from a Phase 1 dose escalation clinical trial of intradermal administration of RXI-109 at incision sites is presented. Pamela A. Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals, USA Unpublished Data
2:15
Case
Dbait - New Approach in Cancer Therapy by Jamming DNA Repair Signaling to Overcome Tumor's Resistance to Conventional Cancer Therapies: From Concept to ClinicStudy Enhanced DNA repair activity in tumor cells confers resistance to treatment. A first-in-class oligonucleotide therapeutic that mimics DNA double-strand breaks has been developed. It acts by jamming DNA damage signaling and thereby inhibits DNA repair. This presentation will describe its mechanism of action and proofs of concept, animal toxicology and pharmacokinetics data, and preliminary data in the first-in-human trial of DT01- 1st drug candidate of Dbait. Jiang Shen Sun, Ph.D., CEO, DNA Therapeutics, France Unpublished Data
2:45
Efficacy of Treating HBV Infection by Combining the Nucleic Acid Polymer REP 9AC' with Pegasys™ or Zadaxin™
REP 9AC' is a nucleic acid polymer which clears serum hepatitis B surface antigen (HBsAg) in patients with chronic hepatitis B (HBV). Removal of HBsAg allows the existing host immune response to actively target HBV infection. Removal of HBsAg also appears to enhance the activity of immunotherapeutic polypeptides like thymosin alpha-1 and pegylated interferon alpha-2a. Combination treatment may produce a durable immunological control of HBV infection in most patients. Andrew Vaillant, Ph.D., Vice President, Chief Scientific Officer, REPLICor Inc., Canada
3:15
Networking Refreshment Break in Poster and Exhibit Hall
Unpublished Data
4:00
Development of Liposomally Formulated siRNA for RNAi-Based Therapy: Atu027 for Therapeutic Application in Oncology
Atu027 refers to a liposomally formulated siRNA targeting PKN3 expression in the vascular endothelium. In this case, siRNA molecules become interspersed with a liposomal moiety resulting in lipoplex particles which become internalized by endothelical cells of the vasculature after systemic administration. Proof-of-concept experiments on mouse tumor xenografts suggest profound inhibition of tumor progression and particularly of metastasis. Final results of this Phase I study in patients with advanced solid tumors are presented. Joerg Kaufmann, Ph.D., Vice President, Research, Silence Therapeutics AG, Germany
4:30
First Clinical Phase IIa Data of the Mirror-Image RNA Oligonucleotide NOX-A12
NOX-A12 is an RNA-Spiegelmer) that inhibits the pro-angiogenic chemokine SDF-1 (stromal cell-derived factor-1). SDF-1 is involved in homing processes of hematopoietic as well as malignant stem cells. After preclinical characterization, NOX-A12 was shown to mobilize stem cells in healthy volunteers. Currently, NOX-A12 is evaluated in an open label study in patients suffering from chronic lymphatic leukemia (CLL) and multiple myeloma (MM) in combination with chemotherapy. Sven Klussmann, Ph.D., Chief Scientific Officer, Noxxon Pharma AG, Germany Featured Presentation
5:00
Miravirsen Clinical Update - First MicroRNA Drug for Hepatitis CShort, single stranded LNA oligonucleotides delivered systemically as naked molecules are able to potently and safely inhibit therapeutically attractive miRNAs in a range of tissues in experimental animals. The presentation will provide an update on the use of LNA oligonucleotides in miRNA therapeutics with particular emphasis on Miravirsen, an LNA-antimiR targeting miRNA-122 that is being developed for the treatment of hepatitis C viral (HCV) infection. Henrik Ørum, Ph.D., Chief Scientific Officer, Santaris Pharma A/S, Denmark
5:30
Networking Reception in Exhibit and Poster Hall
Co-Sponsored by
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Updates on Peptide-Based Therapeutics in Preclinical and Clinical DevelopmentUpdates on Peptide-Based Therapeutics and Vaccines in Discovery and Preclinical Development
8:55
Chairman's Remarks
Shawn Lee, Ph.D., President and CEO, CPC Scientific, USA and China Featured Presentation
9:00
Stapled Peptide Drugs: Transforming Nature's α−Helix to Breakthrough MedicinesStapled Peptides are a promising breakthrough therapeutic modality for the treatment of many diseases by modulating intracellular or extracellular α−helical protein-protein interactions and expand drug target space. Aileron's Stapled Peptide integrates drug design, synthetic chemistry, structural biology, target binding, cell biology, pharmacokinetics, metabolism and in vivo pharmacology. Aileron's advancement of Stapled Peptides and translation to the clinic highlights the dual MDM2/MDMX inhibitor ATSP-7041 and GHRH agonist ALRN-5281. Tomi K. Sawyer, Ph.D., Chief Scientific Officer and Senior Vice President, Aileron Therapeutics, Inc., USA
9:30
Phage Displayed Phylomer Libraries for the Discovery of Custom Scaffolds Suitable for Intracellular or Extracellular Targets
Phylomers are a new class of peptide, derived from fragments of biodiverse microbial genomes. Phylomer libraries can also be used to identify new cell penetrating peptides for delivery of macromolecules into cells. Some of these cell penetrating Phylomers are specific for particular cell types. Phylomers themselves can be active against intracellular targets in vivo and can be used to discover and validate new targets. Richard Hopkins, Ph.D., Vice President, Research, Phylogica, Australia Updates on Peptide-Based Therapeutics in Preclinical DevelopmentUnpublished Data
10:00
PeptiDream's PD Platform: Innovative Peptide Discovery Platform
Peptides represent an exciting and rapidly growing therapeutic class of medicines with significant potential, but there are still challenges in stability and delivery. The aim of PeptiDream, a privately-held biopharmaceutical company based in Tokyo, Japan, is to address some of these issues by producing libraries of highly diverse, stable, and potent non-standard macrocyclic peptides that have potential to be first-in-class, based on its proprietary PD (peptide discovery) Platform. Patrick C. Reid, Ph.D., Chief Scientific Officer, PeptiDream Inc., Japan
10:30
Networking Refreshment Break in Poster and Exhibit Hall
11:15
Direct Selection of Cyclic Peptidomimetics from In Vitro Display Libraries
Peptides offer high potency and target selectivity but display poor pharmacokinetics and bioavailability. Modifications aimed at improving these properties are typically added after a candidate sequence is identified, often reducing potency and rarely delivering high quality clinical leads. By incorporating non-natural side chains and N-substituted amino acids into cyclic peptide libraries, Ra Pharma's Extreme Diversity™ platform allows direct selection of cyclic peptidomimetics from libraries of up to 100 trillion members. Douglas A. Treco, Ph.D., President and CEO, Ra Pharmaceuticals, Inc., USA
11:45
Peptide Antibody Conjugates: From Half Life Extension to Multifunctional Biologics
The presentation focuses on chemical strategies for generating peptide antibody conjugates, including the description of a novel site directed antibody conjugation method developed at Pfizer. The presentation also covers highlights of relevant preclinical data supporting the development of peptide antibody conjugates. Abhijit "Abhi" Bhat, Ph.D., Head, Covx Chemistry, Pfizer, USA
12:15
Networking Luncheon in Exhibit and Poster Hall
Updates on Peptide-Based Therapeutics and Vaccines in Clinical Development
1:30
Chairman's Remarks
Jesse Z. Dong, Ph.D., Vice President, Compound Discovery, IPSEN, USA Unpublished Data
1:45
Case
Evaluation of DPX-Survivac, a Multi-Peptide Vaccine Targeting the Broadly Expressed Tumor Antigen Survivin, in Patients with Ovarian CancerStudy DPX-Survivac is a peptide based cancer vaccine that targets the tumor-associated antigen survivin. The vaccine contains peptides that bind a majority of HLA-A haplotypes (A1, A2, A3, A24) allowing for its broad utility in cancer patients. The vaccine is formulated in the adjuvanting platform DepoVax which has the capacity to significantly enhance the immunogenicity of peptide based tumor associated antigens. A phase I/II trial was designed to evaluate the safety, immunogenicity and efficacy of the vaccine in ovarian cancer patients. Marc Mansour, Ph.D., CSO and COO, Immunovaccine Inc., Canada Unpublished Data
2:15
Development of Novel Cancer Vaccine Using Peptide Cocktail Targeting Oncoantigen and Tumor Angiogenesis
Peptide cocktails, multiple epitope peptides containing vaccines, are a promising strategy for cancer immunotherapy. C01, a novel peptide cocktail vaccine, targets oncoantigen and tumor angiogenesis. Tumor specific vaccine and anti-tumor angiogenesis vaccine showed synergistic anti-tumor effect in vivo. Dual anti-tumor actions of C01 are expected to show strong antitumor activity. We have performed multicenter, Phase III, randomized, placebo controlled trials against pancreatic cancer. Takuya Tsunoda, M.D., Ph.D., President and CEO, OncoTherapy Science, Inc., Japan
2:45
Dual and Triple Acting Peptides for Metabolic Disease
We have observed exciting results in our dual and triple agonist program, representing novel approaches to the treatment of diabetes, with potential disease modifying properties, as well as obesity. Our observations further substantiate the therapeutic rationale for dual and triple agonists as a future new treatment option for patients with type 2 diabetes and/or obesity with the potential to alter the chronic progression of the T2DM disease, indicating also a possible role in the treatment of Type 1 diabetes. Christian Grondahl, DVM, Ph.D., DMSc, Executive Vice President, Chief Scientific Officer, Head of Research & Development, Zealand Pharma A/S, Denmark
3:15
Networking Refreshment Break in Poster and Exhibit Hall
Updates on Peptide-Based Therapeutics in Clinical DevelopmentUnpublished Data
4:00
Case
Development of a Novel Analog of Human PTHrP to Treat OsteoporosisStudy BA058 is a novel peptide being developed as a bone anabolic agent to treat osteoporosis. In a Phase 2 clinical trial BA058, delivered by daily SC injection, demonstrated rapid and potent bone building effects, with a limited effect on bone resorption. A Phase 3 bone fracture prevention study is currently enrolling. Additionally, transdermal BA058 is also in clinical development and has the potential to enhance patient convenience and compliance. Gary Hattersley, Ph.D., Senior Vice President, Preclinical Development, Radius Health, Inc., USA
4:30
Stapled Growth Hormone Releasing Hormone as a Promising Peptide Therapeutic
We have applied the Stapled Peptide technology to human growth hormone releasing hormone (GHRH). For appropriate patient populations, GHRH is expected to be more physiologically relevant, safer, and better tolerated than GH. In preclinical models, the stapled GHRH, ALRN-5281, has improved drug-like properties relative to short-acting GHRH analogs, including a longer plasma half-life and drug exposures to enable potential once-weekly dosing. We anticipate initiation of the phase 1 program in 1H13. Hubert C. Chen, M.D., Vice President, Clinical Development, Aileron Therapeutics, Inc., USA Featured Presentation
5:00
The Development of Linaclotide for the Treatment of Chronic Functional Gastrointestinal DisordersLinaclotide recently received US Food and Drug Administration approval for the once-daily treatment of adults suffering from irritable bowel syndrome with constipation (IBS-C) or chronic idiopathic constipation (CIC). The orally delivered peptide is a first-in-class guanylate cyclase type‐C agonist and acts locally in the intestine with minimal systemic exposure. In nonclinical studies, linaclotide was shown to reduce intestinal pain and accelerate gastrointestinal transit. The development program for linaclotide will be summarized. Angelika Fretzen, Ph.D., Vice President, Pharmaceutical Chemistry and Development, Ironwood Pharmaceuticals, USA
5:30
Networking Reception in Exhibit and Poster Hall
Co-Sponsored by
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Tutorials: Tuesday | Main Conference: Tuesday | Wednesday | Thursday
8:30
Networking Coffee and Tea
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Manufacturing and Analytical Development for Peptides and Oligos
8:55
Co-Chairs' Remarks
Robert Hagopian, Director, Business Development, PolyPeptide Laboratories, USA Mehrdad Tabrizi, Ph.D., Executive Chairman, Avecia, USA, Vice President, Business Development, Avecia, USA Spotlight Presentation
9:00
Case
cGMP Manufacturing of a Conjugated OligonucleotideStudy Oligonucleotides are frequently conjugated to small molecules to enhance pharmacokinetics. These small molecules usually have characteristics that differ significantly from the oligonucleotide. These differences result in unique challenges during synthesis, purification and analysis of the conjugated oligonucleotide. A case study discussing these challenges and how they were overcome is presented. Thomas Rupp, Technical Advisor, Avecia, USA Sponsored by 
Spotlight Presentation
9:25
Differential Scanning Calorimetry (DSC) as a Tool to Characterize Oligonucleotides in Solution
Oligonucleotides as therapeutic agents are usually used as dissolved compounds for in vivo application. So far it is less known how these formulations behave under different conditions like temperature and salt as well as at higher concentrations. In this presentation differential scanning calorimetry (DSC) is used to characterize such oligonucleotide based formulations. Hüseyin Aygün, Dr.phil.nat., Chief Scientific Officer, BioSpring GmbH, Germany Sponsored by 
Spotlight Presentation
9:50
Testing Methodology for Identification of Universal Linker on Solid Support
UnyLinker is the most widely used universal linker in oligonuleotide synthesis which eliminates the need to have several different types of supports loaded with nucleotides. From a quality perspective, it is important to ensure the identity of the first unit loaded onto a solid support prior to initiating synthesis. The UnyLinker molecule on a solid support can be released under basic conditions to allow confirmation of identity. The addition of ammonium hydroxide to release the linker from the support introduces hydrolysis species into the sample. Identification by mass of distinguishable main peaks in the UnyLinker hydrolysis sample were obtained by LC-MS and the peaks can be used to establish positive identity of UnyLinker on support. Tatsuya Konishi, Process Development Group Leader, Avecia, USA Sponsored by
Spotlight Presentation
10:15
A Rapid 2-D UHPLC UV/MS Method for Debottlenecking the Oligo Purification Process
In-process analysis of fractions during oligonucleotide purification often creates a bottleneck in manufacturing. Sample preparation and analysis of fractions can put the process on hold for two days or more. An automated 2-D UHPLC UV/MS analytical method will be presented that combines high pH / high salt sample preparation with rapid quantification of N+ and N- oligonucleotide impurities. Method validation, bridging studies and plant throughput improvements will be discussed. Paul Metz, Senior Director, Manufacturing Operations, Agilent Technologies, Inc. Sponsored by 
10:40
Networking Refreshment Break in Poster and Exhibit Hall
11:15
The Use of Fmoc-Pseudoproline Dipeptides in the Synthesis of Large Peptides
An unbalanced three fragment hybrid solution/solid phase synthesis utilizing FMOC chemistry for the synthesis of Exendin-4 will be discussed. The unbalanced fragment approach contains one fragment that is substantially longer than the other two fragments. The positive impact of pseudoprolines on the longer fragment quality and yield is presented. In addition, the benefits of the precipitation of Exendin-4 at large scale (i.e. advantages of precipitation vs lyophilization) are discussed. Brad DeHoff, Ph.D., Director, R&D, Corden Pharma Colorado, USA Unpublished Data
11:45
Analytical Methods in Peptide Manufacturing: From Starting Materials to Drug Substances
Valid and reliable analytical methods are essential for peptide API manufacturing process development and control. We will present the development and application of a new HR-MAS-NMR method for quality control of solid supports for SPPS, technologies established at Bachem to quantify and control aggregation of peptide APIs, application of DSC for lyophilization process development and large-scale manufacturing of a peptide API with defined particle size distribution for market supply. Guenther Loidl, Ph.D., Vice President R&D, Bachem AG, Switzerland Unpublished Data
12:15
Case
Advantages of High Resolution Mass Spectrometry in Peptide SynthesisStudy Recent developments in mass spectrometry (MS), in terms of resolution and accuracy, have led to improved characterization of peptides and unprecedented confidence in the identification of related impurities. From molecular formula determination to direct sequencing of large peptides, this presentation will demonstrate the value of high-end MS with real case data from Lonza R&D laboratories. David Cosquer, Mass Spectrometry Specialist, Analytical Development, Lonza, Belgium
12:45
Networking Luncheon in Poster and Exhibit Hall
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DDS (Drug Delivery Systems) for Peptides and Oligonucleotides
8:55
Chairman's Remarks
Troels Koch, Ph.D., Vice President and CTO, Santaris Pharma A/S, Denmark
9:00
Delivery and Efficacy of Dicer Substrate siRNAs (DsiRNAs) Using EnCore Lipid Nanoparticles in Liver, Lung, and Prostate Tumor Models
Lipid nanoparticle delivery of RNAi in vivo often requires specific conditions to yield effective target knockdown. We have developed nanoparticles (EnCore) that are robust, straightforward to produce, and well tolerated, and that achieve robust delivery in multiple xenograft models of liver, prostate, and lung cancer, all representing significant tumor burdens. We use EnCore to deliver DsiRNAs targeting MYC, beta-catenin and genes, in single and combination regimens, to maximize antitumor efficacy. Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals, USA Unpublished Data
9:30
Dynamic Polyconjugate (DPC) Technology for Targeted siRNA Delivery
Dynamic Polyconjugate (DPC) technology is a polymer-based siRNA delivery system. We find that co-injection, without complexation, of a hepatocyte-targeted DPC polymer with cholesterol-conjugated siRNA (chol-siRNA) results in >95% knockdown of liver target genes. This is achieved at chol-siRNA doses >500-fold lower than those required when chol-siRNA is injected alone. DPC technology is used in an RNAi-based therapeutic under development for the treatment of chronic hepatitis B virus infection. David L. Lewis, Ph.D., Vice President, Biology, Arrowhead Madison, USA
10:00
Progress in the Development of LNP Delivered siRNA Products
The modular lipid nanoparticle (LNP) siRNA delivery platform has been used to confirm RNAi mediated efficacy in several preclinical models of oncology, infectious and metabolic disease. At least seven products based on Tekmira's LNP technology have entered clinical trials providing increasingly robust proof of clinical activity in multiple disease areas. Ongoing formulation development efforts, informed by this clinical experience, continue to produce LNP formulations with substantially increased potency and reduced toxicity, opening up new product development opportunities in new therapeutic areas. Ian MacLachlan, Ph.D., Executive Vice President and Chief Scientific Officer, Tekmira Pharmaceuticals Corp., Canada
10:30
Networking Refreshment Break in Poster and Exhibit Hall
Unpublished Data
11:15
XTEN: A Protein-Based Polymer for Tumor Targeting of Drug Conjugates with Precisely Controlled Chemical Compositions
Amunix has developed XTEN, a protein-based polymer that mimics the biophysical properties of PEG, for half-life extension of attached therapeutics, and the clinical development of two XTENylated products is in progress. Different classes of drugs can be coupled to XTEN through amino and/or thiol groups included in the XTEN sequence. Here, we describe XTEN conjugates that combine cytotoxic payloads with peptides for tumor-specific targeting to enable delivery of potent anti-cancer drugs to specific locations. Volker Schellenberger, Ph.D., Chief Scientific Officer, Amunix, Inc., USA
11:45
Case
Arginine-Rich Cell-Penetrating Peptides and their Application to Intracellular DeliveryStudy Arginine-rich cell-penetrating peptides (CPPs) are attractive as novel means for intracellular delivery of bioactive molecules. In this presentation, factors that affect the methods of internalization of arginine-rich CPPs are summarized. Our approaches to improve the internalization efficiency of arginine-rich CPPs, together with examples that show the potentials of these peptides as delivery tools for cancer therapy, are also introduced. Shiroh Futaki, Ph.D., Professor, Institute for Chemical Research, Kyoto University, Japan
12:15
Case
Development of siRNA Skin Delivery Technologies - A Case Study of the Rare Inherited Skin Disorder Pachyonychia CongenitaStudy ~7,000 rare disorders affect an estimated 6-8 percent of the population. The missing link for translation of siRNA therapeutics for rare disorders is efficient delivery to target tissues in a patient friendly manner. We have developed/manufactured a topical delivery system that uses protrusions of polyvinyl alcohol (PADs) to deliver siRNA cargo to skin. The ability of PADs to deliver functional siRNA are described as well as the upcoming clinical trial. Roger L. Kaspar, Ph.D., TransDerm, Inc.; Department of Pediatrics, Stanford School of Medicine, USA
12:45
Networking Luncheon in Poster and Exhibit Hall
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Main Conference - Plenary Session - Thursday, February 28, 2013Tutorials: Tuesday | Main Conference: Tuesday | Wednesday | Thursday Business Considerations and Intellectual Property
1:55
Chairman's Remarks
Mehrdad Tabrizi, Ph.D., Executive Chairman, Avecia, USA Featured Presentation
2:00
The Excaliard Story: From Start-up through Clinical Success and Acquisition by Pfizer
Strategies for funding, running and selling a virtual antisense company are discussed. Specific examples of preclinical and clinical results are used to illustrate how company value was generated. Nicholas M. Dean, Ph.D., Founder & Former CSO, Excaliard Pharmaceuticals, USA
2:40
The Business of RNAi Therapeutics in 2013
In 2013, the RNAi Therapeutics industry has emerged with renewed vigor following 2-3 years of scientific doubt and investor neglect. This was largely the result of the clinical validation of previous investments in RNAi trigger delivery. This talk will discuss business development strategies during this period, with a particular emphasis on the roles of intellectual property versus technical enablement. The outlook will feature promising technologies and product candidates. Dirk Haussecker, D. Phil, Assistant Professor, Medical Biotechnology, Dongguk University, South Korea
3:10
Networking Refreshment Break in Exhibit and Poster Hall and Final Chance to See Exhibits and Posters
OSWG and JPMA Joint Session: Preclinical and Clinical Safety Assessment of Oligonucleotides
3:50
Co-Chairs' Remarks
Arthur A. Levin, Ph.D., Executive Vice President R&D, Miragen Therapeutics, USA Kazuto Watanabe, Ph.D., Principal Scientist, Safety Assessment Department, Chugai Pharmaceutical Co. Ltd.; Topic Leader, ICH S6(R1) EWG, JPMA, Japan
4:00
OSWG and JPMA Joint Session: Topic for Consideration and Debate: Nonclinical Safety Assessment of Oligonucleotides (Provisional)
Kazuchika Takagaki, Ph.D., Manager, Discovery Research Labs Tsukuba, Nippon Shinyaku Co., Ltd., Japan
4:30
Discussion and Debate on Nonclinical Safety Assessment
5:00
Clinical Experiences with Oligonucleotide Therapeutics and the Productivity of Animal Models for Human Responses
Arthur A. Levin, Ph.D., Executive Vice President R&D, Miragen Therapeutics, USA Featured Presentation
5:30
Special Issues in Regulating Oligonucleotides and Peptides Regarding CMC
This presentation provides an overview about the regulatory landscape for oligonucleotides and peptides. Issues related to the pharmaceutical quality of oligonucleotides and peptides are highlighted. Typical deficiencies in regulatory applications are discussed. Since early and open communication with Regulatory Agencies can significantly reduce time to market for a new drug product, ways of interaction are exposed. Dr. René Thürmer, Deputy Head Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany
6:00
Close of AsiaTIDES
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