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AsiaTIDES: Oligonucleotide and Peptide Research, Technology and Product Development

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Where Global Oligonucleotide and Peptide Leaders Connect to Share Groundbreaking Science and Form Successful Business Collaborations

March 03 - March 05,--> 2015 · Osaka, Japan

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Alternative Language Options:

  • Japanese
  • Taiwanese
  • Chinese
  • Korean
  • English

Agenda

Agenda

Main Conference - Plenary Keynote Presentations - Tuesday, March 3, 2015

7:30
Registration and Coffee/Tea

8:40
Chairman's Remarks
Mark Graham, Executive Director, Cardiovascular Disease Research, Antisense Drug Discovery, Isis Pharmaceuticals Inc.

8:45
Takahiro Ochiya, Ph.D. Transfer of exRNAs in Cancer Metastasis
The existence of circulating miRNAs and exosomes in the blood of cancer patients has raised the possibility that disease-specific miRNAs and exosomes may serve as a novel diagnostic marker. Moreover, exosomes secreted from tumor cells contribute micromanaging tumor microenvironment. We and other groups reported that tumor-derived exosomes mediated tumor angiogenesis and thus facilitated tumor metastasis for distant organs. Here we will discuss importance of transfer of extracellular microRNAs (exRNAs) and their contribution to tumor development and metastasis.
Takahiro Ochiya, Ph.D., Chief, Division of Molecular and Cellular Medicine, National Cancer Center Research Institute, Japan

9:15
Case StudyNew Data
Wendy Wei Qiao Qiu, M.D., Ph.D. Amylin and its Analog: Potential Diagnostic Test and Therapeutic Drug for Alzheimer's Disease
Amylin is a naturally occurring peptide with important functions in the brain, including regulating glucose metabolism. Using Alzheimer's disease (AD) mouse models, we show here the evidence that chronic peripheral treatment with amylin or its analog, pramlintide, reduced the amyloid burden and lowered the concentrations of amyloid-beta peptides (Aβ) in the brain when compared with saline treatment. Further, behavioral tests showed that learning and memory in these mice were improved by the treatment. Despite the fact that amylin and Aβ share a similar secondary structure, our study indicates the therapeutic potential of amylin type peptides for treating Alzheimer's.
Wendy Wei Qiao Qiu, M.D., Ph.D., Associate Professor, Departments of Psychiatry and Pharmacology, Alzheimer's Disease Center, Boston University School of Medicine, USA

9:45
Takanori Yokota, MD, Ph.D. DNA/RNA Heteroduplex Oligonucleotide as a New Class of Oligonucleotide
We developed a short DNA/RNA heteroduplex oligonucleotide (HDO) with a structure different from siRNA and ASO. A 13-14-mer DNA/locked nucleotide acid (LNA) gapmer ASO targeting ApoB mRNA duplexed with a vitamin E (α-tocopherol) conjugated complementary RNA strand (Toc-HDO) achieved the most efficacious gene silencing (effective dose 50 [ED50], 0.038 mg/kg) yet reported using ASOs.
Takanori Yokota, MD, Ph.D., Tokyo Medical and Dental University

10:15
Grand Opening of the Poster and Exhibit Hall with Refreshments
Sponsored by

Main Conference - Concurrent Tracks - Tuesday, March 3, 2015

Oligonucleotide-Based Therapeutics in Preclinical and Clinical Development

11:00
Chairman's Remarks
David L. Lewis, Ph.D., Chief Scientific Officer, Arrowhead Research Corp., USA

11:15
New Data
N-acetyl Galactosamine Conjugation Enhances Potency of Second-Generation Antisense Targeting Hepatocyte Expressed Genes
Second-generation antisense oligonucleotide (ASO) mRNA/protein potency was increased 5-10 fold versus parent following N-acetyl galactosamine (GalNAc3) conjugation of human apolipoprotein C-III, apolipoprotein(a) and angiopoeitin protein-like3 ASOs in transgenic mice. GalNAc3 ASO conjugation should significantly enhance the therapeutic index, reduce cost, and support monthly dosing regimens for hepatocyte mRNA targets.
Mark Graham, Executive Director, Cardiovascular Disease Research, Antisense Drug Discovery, Isis Pharmaceuticals Inc., USA

11:45
Optimization of Phosphorothioate Antisense DNA by Controlling the Phosphorous Chirality
This presentation demonstrates that the P-chirality affects nearly all the critical properties of the antisense drugs, from binding affinity to target RNA to metabolic stability and RNase H activity.
Takeshi Wada, Ph.D., Department of Medicinal and Life Science, Faculty of Pharmaceutical Sciences, Tokyo University of Science, and Department of Medical Genome Sciences, Graduate School of Frontier Sciences, The University of Tokyo, Japan

12:15
Luncheon in Poster and Exhibit Hall

1:25
Chairman's Remarks
Dr. Troels Koch, VP & CTO, Roche Innovation Center Copenhagen, Denmark

1:30
Using Dynamic Polyconjugate (DPC) Delivery Technology in RNAi-Based Therapeutics for the Treatment of Liver Disease
We have developed a platform technology called Dynamic Polyconjugate (DPC) for targeted delivery of RNAi trigger molecules. Key to this technology is a reversibly masked endosomolytic polymer. The masking chemistry prevents the polymer from interacting with blood components and non-targeted tissues, but is hydrolyzed in the acidic environment of endosomes allowing activation of the polymer and release of the RNAi-trigger to the cytoplasm of the targeted cell. Attachment of N-acetyl galactosamine moieties to the DPC enables delivery specifically to liver hepatocytes via the asialoglycoprotein receptor. We are currently using this technology in therapeutics designed for the treatment of chronic hepatitis B virus infection and alpha-1 antitrypsin deficiency-associated liver disease.
David L. Lewis, Ph.D., Chief Scientific Officer, Arrowhead Research Corp., USA

2:00
Nucleic Acid Ligands with Protein-like Side Chains and Their Application in Diagnostics and Therapeutics
This presentation discusses a new class of nucleic acid ligands with protein-like side chains, called slow off-rate modified aptamers (SOMAmers). Their broader chemical diversity results in higher success rate of SELEX, expanded range of accessible epitopes, and enhancement in nuclease resistance. These features are well-suited for large-scale proteomics, biomarker discovery and development of new diagnostic and therapeutic agents.
Nebojsa Janjic, Ph.D., Chief Science Officer, SomaLogic, Inc., USA

2:30
New Data
Gene Silencing Oligos: The Next Generation in Gene Silencing
This presentation discusses a unique therapeutic oligonucleotide modality. Gene silencing oligonucleotides (GSOs) are composed of two single stranded oligonucleotides directed against the same target mRNA connected at their 5' ends by a linker. GSOs show greater activity, potency and duration when compared to antisense oligonucleotides (ASOs) directed against the same target. In addition, GSOs mitigate the immune response seen with second generation ASOs and show no obvious signs of organ toxicity after repeat dosing. Significant work went into the SAR of GSOs demonstrates specific unique structural properties and provide opportunities for additional optimization of the drug-liker properties of GSOs. The GSOs represent a significant opportunity in exploding field of oligonucleotide therapeutics. The presentation also includes exploration of the structure/activity relationship and work with the GSOs in various preclinical models.
Walter Strapps, Ph.D., Executive Director of RNA Therapeutics, Idera Pharmaceuticals, USA

Peptide-Based Therapeutics in Preclinical and Clinical Development

11:00
Chairman's Remarks
Jörg Vollmer, Ph.D., Chief Executive Officer, Nexigen GmbH, Germany

11:15
Case StudyNew Data
Design, Optimization and Early Development of the Second Generation Dopamine-Somatostatin Chimeric Ligand
The concept for the design of a novel dopamine-somatostatin chimeric ligand will be described as well as some of the hurdles from Ipsen's first generation project. Clinical reports of enhanced growth hormone suppression with a combination of somatostatin and dopamine indicate that a chimeric molecule will be advantageous in treating endocrinology diseases that result in excess secretion of growth hormone. The progress on a second generation project and some of the strategies employed to overcome the limitations from the first generation lead candidate compound will be highlighted.
Yvonne M. Angell, Ph.D., Director of Peptide and Protein Chemistry, Ipsen Bioscience, Inc., USA

11:45
Case Study
Development of a Novel Peptide Immunotherapeutic for Prostate Cancer
We are developing a second generation immunotherapeutic peptide vaccine for various cancers. The peptide was designed to activate critical components of the cellular immune system in an antigen-specific manner. In a Phase I prostate cancer study, both a robust immune response and correlates of improved overall and progression-free survival were obtained. The same peptide gave encouraging results from a Phase II breast cancer study.
Eric von Hofe, Ph.D., President, Antigen Express Inc., USA

12:15
Luncheon in Poster and Exhibit Hall

1:25
Chairman's Remarks
Eric von Hofe, Ph.D., President, Antigen Express Inc., USA

1:30
PeptiDream; PDPS and the Discovery & Development of Constrained Peptide Therapeutics
PeptiDream's proprietary Peptide Discovery Platform System (PDPS) has proven highly effective at identifying constrained lead peptide candidates, in which such hits exhibit excellent therapeutic properties directly from selections themselves. In addition, advanced screening systems have been established to allow for the rapid assessment of those hits without the need for laborious chemical synthesis, significantly shortening the time from selections to the identification of hit constrained peptides that exhibit the desired therapeutic candidate profile.
Patrick C. Reid, Chief Scientific Officer, PeptiDream Inc., Japan and USA

2:00
Case StudyNew Data
Targeting Key Signaling Pathways of Cancer Stem Cells with Intracellular Peptide Therapeutics
One of the challenges in today's treatment of cancer is tumor recurrence and spread caused by the existence of subpopulations within a tumor with distinct tumor-initiating powers, the cancer stem cells. Peptide therapeutics targeting intracellular protein-protein interactions are an attractive alternative to conventional therapies to inactivate signaling pathways in cancer stem cells. Nexigen's NexiTides target such pathways and have powerful in vitro anti-cancer stemness activity and are effective in xenografted and orthotopic models of tumor growth and metastases.
Jörg Vollmer, Ph.D., Chief Executive Officer, Nexigen GmbH, Germany

2:30
Case StudyNew Data
High Affinity Bicyclic Peptides: Application to Payloads in Oncology
The Bicycle technology is based on repertoires of peptides displayed on the surface of bacteriophages which can be modified with organochemical scaffolds to create a diverse array of constrained peptides. These repertoires have been extensively used for iterative selections to identify high affinity binding peptides for a wide array of targets, including receptors, interleukins and proteases. The bicycle peptides show antibody-like properties such as low to sub-nanomolar affinities and exquisite selectivity, but in a 100-fold smaller, chemically synthesized format. We showed that these small entities extravasate and penetrate tumors much more rapidly and efficiently than antibodies, thus delivering high concentrations of payloads in a very short time. These features allow efficient tumor killing while drastically minimizing systemic toxin exposure. Bicycle Therapeutics will present in vivo POC data from its pre-clinical programs demonstrating the power of its technology to deliver cytotoxic payloads to tumor cells.
Christophe Bonny, Ph.D., Chief Scientific Officer, Bicycle Therapeutics, United Kingdom

3:00
Networking Reception in Poster and Exhibit Hall

Oligonucleotide-Based Therapeutics in Preclinical and Clinical Development (continued)

Novel LNA and siRNA Developments

3:40
Chairman's Remarks
Patrick Y. Lu, Ph.D., President & CEO, Sirnaomics, Inc., USA

Featured Presentation

3:45
New Data
LNA Therapeutics: A Technology Perspective
The field of LNA and RNA therapeutics is undergoing rapid development. New trends in the field influence future developments and further progress will be linked to a reformulation of older concepts. It will be shown that, in general, the way forward will benefit from a fresh view of the technology space. It will be illustrated how the recent progresses in the field has enabled renewed interest in the technology and in the deal space.
Dr. Troels Koch, VP & CTO, Roche Innovation Center Copenhagen, Denmark

4:15
Case StudyNew Data
Advancing Novel siRNA Therapeutic Products in Both China and USA
Sirnaomics has developed an enriched product pipeline including siRNA therapeutic STP705 (Cotsiranib®) for Hypertrophic Scar prevention and reduction, STP702 for "Resistent-Proof" influenza therapeutics, and STP909 for HPV and cervical cancer treatment. The company is pushing two siRNA therapeutic programs into clinical study in China and USA with support of manufacturing capability, technical strength and financial resources.
Patrick Y. Lu, Ph.D., President & CEO, Sirnaomics, Inc., USA

4:45
New Data
Locked Nucleic Acid: Enabling RNA Therapeutics
RNA therapeutics have over the past decade developed from concept, clinic to market. However, the development has been hampered by a too simplistic approach to the underlying drug discovery principles resulting in unnecessary attrition rates during development. The lessons learned have resulted in new concepts to conduct drug discovery leading to increases in the productivity of RNA therapeutics. Locked Nucleic Acids are recognized as a preferred chemistry for RNA therapeutics and this will be presented in the context of lesson learned and LNA drug discovery case stories.
Dr. Bo Rode Hansen, Vice President, Drug Discovery & Alliance, Roche Innovation Center Copenhagen, Denmark

5:15
New Data
DT01: A First-in-Class DNA Repair Inhibitor Against Advanced Cancers, From Concept to Clinic
Enhanced DNA repair activity in tumors confers resistance to treatment. A first-in-class oligonucleotide therapeutics that mimics DNA double-strand breaks (named "Dbait") has been developed. It acts by jamming DNA damage signaling that disorganizes DNA repair system and thereby inhibits DNA repair. This presentation will describe its mechanism of action and preclinical proofs of concept, animal toxicology and pharmacokinetics data, and preliminary data in the first-in-human trial of DT01 - 1st drug candidate of Dbait.
Professor Jian-Sheng Sun, CEO, DNA Therapeutics SA, France

5:45
End of Day One

Peptide-Based Therapeutics in Preclinical and Clinical Development (continued)

3:40
Chairman's Remarks
Patrick C. Reid, Chief Scientific Officer, PeptiDream Inc., Japan and USA

3:45
Kisspeptin and its Agonist Analogs: From Discovery to Clinical Studies
Abstract not available.
Hisanori Matsui, Principal Scientist (Pharmacology), Extra Value Generation & General Medicine Drug Discovery Unit, Pharmaceutical Research Division, Takeda Pharmaceutical Company, Ltd., Japan

4:15
FRONTIER: A Phase 3, Pre-Hospital Peptide Therapeutic Trial for Acute Ischemic Stroke
Abstract Not Available.
Dave Garman, Ph.D., Technology Officer, NoNo Inc., Canada

4:45
MicroAntibodes: Generation of Molecular-targeting Peptides by Directed Evolution in Phage-Displayed Libraries of Conformationally Constrained Peptides
To enable new applications where antibodies show some limitations, we have developed an alternative-binding molecule with non-immunoglobulin domain. The molecule is a helix-loop-helix peptide, which is stable against enzyme degradations in vivo and is too small to be non-immunogenic. Here, we introduce the molecular-targeting peptides termed "microantibodes" that show antibody-like functions, high affinity and high specificity for the targeted proteins.
Ikuo Fujii, Ph.D., Professor Graduate School of Science, Osaka Prefecture University

5:15
New Data
Cryptides and Their Accumulative Signaling Mechanisms for Novel Therapeutic Targets
Many fragmented peptides are produced during maturation and degradation processes of endogenous proteins but their physiological and/or pathological roles are not well elucidated yet. We discovered novel bioactive peptides derived from various mitochondrial proteins that efficiently activated neutrophils and named such functional peptides hidden in protein structures "cryptides." Here I will discuss about such cryptides and their signaling mechanisms for possible therapeutic targets.
Hidehito Mukai, Ph.D., Principal Investigator, Laboratory of Peptide Science and Associate Professor, Graduate School of Bio-Science, Nagahama Institute of Bio-Science and Technology, Japan

5:45
End of Day One

5:15
Networking Reception in Exhibit and Poster Hall
Co-sponsored by

Main Conference - Wednesday, March 4, 2015

8:00
Networking Coffee and Tea

DDS (Drug Delivery Systems and Strategies for Peptides and Oligonucleotides)

8:55
Chairman's Remarks
Bruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

9:00
New Data
Lipid Nanoparticles for Therapeutic Delivery of miRNA-Targeting Oligonucleotides
miRNA dysregulation plays a critical role in tumor development and resistance to therapy. miR mimics and antimiR oligos have potential therapeutic applications. A series of lipid nanoparticle formulations have been developed for therapeutic delivery of these oligonucleotide agents in leukemia and in lung cancer. We have shown in animal models that miR-29b mimics can inhibit xenograft tumor growth and extend the survival of leukemia engrafted mice. Lipid nanoparticles loaded with antimiR-21 have been shown to effectively modulate miR-21 targets in vitro and in vivo, and to inhibit tumor growth in a xenograft model. Lipid nanoparticle-based delivery of miR-modulating oligos has great promise for clinical translation for cancer therapy.
Robert J. Lee, Ph.D., Professor of Pharmaceutics, Division of Pharmaceutics, OSU College of Pharmacy, The Ohio State University, USA

9:30
Case Study
Intranasal Drug Delivery: Drug Development Considerations
Intranasal route of administration is attractive for small molecules, peptides, oligonucleotides and even proteins. The nasal epithelium has a large surface and is highly vascularized which can result in rapid absorption and drug onset. The nasal route represents a non-invasive means of delivery and is amenable to peptide drugs as it avoids gastric degradation and hepatic first-pass metabolism. This presentation will go through drug product characterization and highlight aspects which are unique to nasal drugs. The challenges with nonclinical dose administration, determination of the maximum tolerated dose, and methods for pharmacokinetic evaluation will be discussed using case examples.
Bruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

10:00
Networking Refreshment Break

10:40
Chairman's Opening Remarks
Bruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

10:45
Case StudyNew Data
Block Copolymer-Based Nanosystems for siRNA and Oligonucleotide Delivery
This presentation features supramolecular nanosystems assembled from charged block copolymers for siRNA and oligonucleotide delivery. Focus will be on the systemic delivery systems aiming to target intractable cancer, including pancreatic cancer. Tumor penetrability of nanosystems will be a critical point to be discussed particularly in this presentation.
Kazunori Kataoka, Ph.D., Professor, Department of Bioengineering, School of Engineering, University of Tokyo

11:15
New Data
Probing Structural Requirement of Triantennary N-Acetylgalactosamine for Hepatic Targeting of Antisense Oligonucleotides
A summary of our effort to identify simpler GalNAc clusters with specific emphasis on lowering molecular weight and ease of synthesis for Gapmer antisense therapeutics will be presented.
Thazha Prakash, Ph.D., Senior Research Fellow, Isis Pharmaceuticals, USA

11:45
Late-Breaking Presentation

12:15
Networking Luncheon in Poster & Exhibit Hall

1:25
Chairwoman's Remarks
Paula Lorence, VP Business Development & Project Management, Nitto Denko Avecia, USA

Featured Presentation: Manufacturing and Analytical Strategies for Peptides and Oligos

1:30
Purification of Synthetic Oligodeoxynucleotides and Peptides Using a Readily Scalable Catching by Polymerization Approach
Synthetic oligonucleotides and peptides have received increasing attention for drug discovery but their purification is very expensive. To lower the purification costs, we have developed an innovative catching by polymerization approach where a simple polymerizable group is attached to either the failure sequences or the full-length sequences but not to both and purification is achieved by simple washing and extraction process.
Durga P. Pokharel, Ph.D., Post-Doctoral Research Fellow, Michigan Technological University, USA

Main Conference - Concurrent Tracks

Manufacturing and Analytical Strategies for Oligonucleotides

2:00
Rapid Synthesis of Oligonucleotides for Research and Preclinical Studies
Abstract not available.
Rowshon Alam, Group Leader, SSOU, Nitto Denko Avecia, Inc., USA and Japan
Tatsuya Konishi, Process Development, Group Leader, Nitto Denko Avecia, Inc., USA and Japan

2:30
First Manufacturing Results from the Next Generation OligoProcess Synthesizer
Dr. Huseyin Aygün, Ph.D., CSO, BioSpring GmbH, Germany

3:00
Networking Refreshment Break

3:30
Late-Breaking Presentation from Agilent
Abstract not available.

4:00
AJIPHASE®; Novel & Practical Solution Phase Oligonucleotides Synthesis for Large Scale Manufacturing
AJINOMOTO's original liquid-phase technology AJIPHASE® initially developed for peptide manufacturing has been successfully applied to oligonucleotide synthesis.This presentation will describe the novel and practical liquid-phase approach for synthesis or manufacturing of two types of oligonucleotides, DNA/RNA-type oligonucleotides and Morpholino oligonucleotides. Several examples of liquid-phase oligonucleotide synthesis had been reported in the literatures; however, most of them were fit only for short length oligos in terms of yield and quality. Here, we developed 'one-pot' elongation cycle and optimized several factors of the reaction condition to obtain highly pure oligonucleotide in a large scale.
Satoshi Katayama and Daisuke Takahashi, Research Institute for Bioscience Products & Fine Chemicals, Ajinomoto Co. Inc., Japan

4:30
You Have Developed a Process - Now What? Key Elements & KPIs to Consider When Setting Up Successful Technology Transfers at Different Stages of the Product Lifecycle
Pre-tech transfer activities are critical in ensuring success of the tech transfer process and clear, specific communication is essential before, during, and after the tech transfer process. You must have the appropriate skill sets and competencies in place with all of your upstream / downstream partners so that you have understood any constraints and agreed on the scope of the work and acceptable parameters months before the tech transfer actually begins.
Jayant Aphale, Ph.D., MBA, Senior Vice President, Technical Operations, Sarepta Therapeutics, USA

5:00
Forced Degradation of Phosphorothioate Oligonucleotides
Forced degradation studies are an important component of drug development. Forced degradation studies may be used to determine the inherent stabilities of drug substances and drug products and to develop an understanding of degradation pathways and degradation products. The main degradation pathways, the structures of degradation products and the inherent stabilities of synthetic phosphorothioate oligonucleotides exposed to a variety of physical and chemical insults will be presented.
Daniel Capaldi Ph.D., Vice President, Analytical and Process Development, Isis Pharmaceuticals, Inc., USA

5:30
Close of Day 2

Manufacturing and Analytical Strategies for Peptides

2:00
Large Scale Manufacturing of Synthetic Peptides
Latest development in large scale manufacturing of synthetic peptides will be presented. The talk will consider both chemical processes as well as engineering aspects of large scale SPPS and downstream manufacturing. A case study will be discussed.
Daniel Samson, Ph.D., Director API Manufacturing, Bachem, Switzerland

2:30
Characterization of Degradation Impurities in API or Formulated drugs: Example of a beta-Aspartic Isomer Identification by Complementary Analytical Methods
ADeep characterization of degradation impurities is a challenge in the analytical laboratory. In this real-life case study, we supported a customer for the characterization of a drug product. An unexpected degradation impurity was detected and identified, with the help of orthogonal HPLC methods, small scale purification, Edman sequencing, and mass spectrometry sequencing.
Didier Monnaie, Analytical Development Group Leader, or David Cosquer, Mass Spectrometry Specialist, Analytical Development, Lonza, Belgium

3:00
Networking Refreshment Break

3:30
Quantifying Sequence and Structural Features of Protein-RNA Interactions
We quantified the contribution of both sequence- and structure-based features as indicators of RNA-binding propensity using a machine-learning approach. Several novel and modified features enhanced the accuracy of residue-level RNA-binding propensity beyond what has been reported previously. We constructed a web server called aaRNA that implements the proposed method and demonstrate its use in identifying putative RNA binding sites.
Singling Li, Ph.D., Specially Appointed Researcher, Systems Immunology Laboratory, Immunology Frontier Research Center (IFReC), Osaka University, Japan

4:00
Methods for Peptide Thioester Preparation
Chemical ligation technique is widely used for protein synthesis. The ligation methods, such as thioester method and native chemical ligation, have been developed based on the use of peptide thioesters as building blocks. We have developed methods for preparation of the peptide thioesters based on an intramolecular N to S acyl shift reaction at a thiol containing residue of peptides.
Turo Kawakami, Ph.D., Associate Professor, Institute for Protein Research, Osaka University, Japan

4:30
Late-Breaking Presentation

5:00
Molecular Hiving Technology as a Strategy for the Manufacturing of Peptides
Molecular Hiving Technology™ is innovative, hydrophobic tagged Liquid Phase Peptide Synthesis (LPPS) that integrates the key advantages of both LPPS and Solid Phase Peptide Synthesis (SPPS). Sekisui Medical has been developing this technology since 2010. Recent progress has allowed Sekisui to carry out a synthesis with a substantial increase in crude purity and reduction in consumables. This has resulted in faster lead times, a more efficient downstream process and considerable reduction in costs. Examples of this will be given in the presentation. This presentation will also include case studies (all unpublished data) for the synthesis of synthetically challenging oligopeptides (e.g. C-terminal modified cyclized). All candidates were synthesized obtaining a high yield and crude purity, followed by an excellent final purity after the purification process.
Barry O'Connor, Research Scientist, Sekisui Medical Co Ltd., Japan

5:30
Close of Day 2

Main Conference - Thursday, March 5, 2015

8:00
Networking Coffee and Tea

Regulatory Considerations for Peptides and Oligonucleotides

8:55
Chairman's Remarks
David T. Lin, Ph.D., Senior Consultant, Biologics Consulting Group, Inc., USA

9:00
US Regulatory Development and Review Perspectives for New and Generic Peptide Drugs
This session will provide a regulatory overview of the requirements for new and generic peptide drugs. The presentation will include a discussion of the review process in FDA for peptide products with respect to a new peptide as compared to a generic peptide product. Regulatory requirements and expectations between new and generic peptide products are similar so differences will be discussed. To understand these regulatory requirements case studies will be presented that outline development strategies for new dosage forms and delivery systems.
David Lin, Ph.D., MBA, Senior Consultant, Biologics Consulting Group, Inc. (former acting Division Director, Office of New Drug Chemistry, CDER, U.S. FDA), USA
Duu-Gong Wu, Ph.D., Director, Regulatory Consulting/Senior Consultant, PPD, USA (former Deputy Division Director, Office of New Drug Chemistry, CDER, U.S. FDA, USA)

10:00
Update on the Regulatory Expectations for the Quality of Oligonucleotide Products
The complex and diverse nature of oligonucleotides currently in development is posing unprecedented challenges in meeting the regulatory requirements for drug quality. This presentation provides a historical overview and evolution of the US, European and other regulatory agencies' review practices of oligonucleotide products. Also highlighted are emerging trends in the industry to meet current expectations for the characterization and quality control of oligonucleotide drug candidates from preclinical to late stage clinical development.
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA

10:30
Oligonucleotide Therapeutics One Step Closer
Oligonucleotide therapeutics continues to advance toward approval and yet there still remain some critical questions. The early challenges have been met and now to obtain more approvals it will be necessary to address new issues. Identifying these issues and their solutions is the key to future regulatory approvals.
Art Levin, Ph.D., Executive Vice President, Research and Development, Avidity NanoMedicines, USA

11:00
AsiaTIDES Closes and Post-Conference Workshops Begin

Post-Conference Workshops - Thursday, March 5, 2015

Workshop #1

Impurities in Oligonucleotides: Sources, Analysis and Control

Workshop Leaders:
Thomas Rupp, Thomas Rupp Consulting, Germany
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA

With the market approval of three oligonucleotide drugs and the advancement of a myriad of oligonucleotide programs to late stage development, there is an increased interest on the topic of impurities in synthetic oligonucleotides. Due to their inherent complexity, synthetic polymers such as peptides and oligonucleotides are specifically excluded from the scope of the ICH Q3A impurities guideline. This workshop will present current regulatory strategies for the control of impurities through discussions of the sources, methods of analysis and control.

11:00
Welcome and Opening Remarks
Thomas Rupp, Thomas Rupp Consulting, Germany
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA

11:15
Practical Considerations for the Implementation of Quality Criteria for Amidite Starting Materials
Speaker TBD

12:00
Networking Lunch

1:15
Impurities Formed During Synthesis and Downstream Processing
Thomas Rupp, Thomas Rupp Consulting, Germany

2:00
Application of Risk Assessment in Development of Testing Strategies for Non-Oligonucleotide Impurities
Emma Wright, Nitto Denko, USA

2:45
CMC Considerations for the Control of Impurities in Oligonucleotides
G. Susan Srivatsa, Ph.D., ElixinPharma, USA

3:30
Safety Considerations for the Control of Impurities in Oligonucleotides
Mike Templin, SNBL, USA

4:15
General Discussion and Close of Workshop

Workshop #2

Current Practices in Peptide Therapeutics

This course will cover the main topics of manufacturing, formulation and regulatory strategies for peptide therapeutics.

11:00
Evolution of Peptide API Specifications from IND to NDA
Robert Hagopian, Director of Business Development, PolyPeptide Group, USA

11:30
Scale-Up Manufacturing Case Study: Considerations of Transition from Solid-Phase to Solution-Phase Synthesis
Bruce Morimoto, Executive Director, Applied Translational Medicine, Celerion, USA

12:00
Networking Lunch

1:15
Development of a Stable Lyophilized Peptide Formulation
Dave Garman, Ph.D., Technology Officer, NoNo Inc., Canada

1:45
Regulatory Trends and Strategy for Peptide Products
Duu-Gong Wu, Ph.D., Director of Regulatory Consulting/Senior Consultant, Global Regulatory Consulting, PPD, USA

2:00
General Discussion and Close of Workshop

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