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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

February 24 - February 26,--> 2016 · Kyoto, Japan

Alt-Languages

Alternative Language Options:

  • Japanese
  • Taiwanese
  • Chinese
  • Korean
  • English

Agenda

Agenda

Main Conference · Plenary Session · Wednesday, February 24, 2016

8:15
Registration and Coffee

9:15
Chairman's Remarks
Paul Watt, Ph.D., Chief Scientific Officer, Phylogica Ltd, Australia

Keynote Presentation

9:20
Shin'ichi Takeda, M.D., Ph.D. Therapeutic Approaches to Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common childhood genetic disease, affecting one in 3,500 newborn boys, causing progressive muscle weakness, heart and respiratory failure and premature death. This disease is caused by the mutations of the DMD gene, and no cure exists for this disease, but a number of promising new molecular therapies are being intensively studied. Among them, exon skipping by antisense oligonucleotides is a novel method to restore the reading frame of the mutated DMD gene, and rescue dystrophin expression. I will present the recent progress of therapy of the disease.
Shin'ichi Takeda, M.D., Ph.D., Director General, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Japan

Keynote Presentation

9:55
Keiichi Masuya, Ph.D. Paradigm Shift in Drug Discovery: Constrained Peptides and Peptide-Based Drug Design
Constrained peptides represent highly valuable chemical matter for use in biological validation, identifying hit/lead molecules for drug discovery, and also directly as peptide therapeutics. Our in-house approach to the optimization of constrained peptides, and how these peptides contribute to hit finding for difficult target classes from a general perspective will be introduced.
Keiichi Masuya, Ph.D., Chief Operating Officer, PeptiDream, Inc., Japan

10:30
Networking Refreshment Break with Exhibit and Poster Viewing

Main Conference · Concurrent Tracks · Wednesday, February 24, 2016

Oligonucleotides in Preclinical and Clinical Development

11:20
Chairman's Remarks
TBA

11:30
Stabilization and Activation of Double-stranded Nucleic Acid Drugs by Artificial Cationic Molecules
We have developed novel cationic oligosaccharides and cationic peptides that selectively bind to A-type RNA/RNA and DNA/RNA duplexes. In this presentation, I would like to describe the synthesis and properties of these unique molecules as carriers and stabilizers of siRNA drugs and antisense DNA/RNA heteroduplexes.
Takeshi Wada, Ph.D., Professor, Department of Medicinal and Life Science, Faculty of Pharmaceutical Sciences, Tokyo University of Science, Japan

12:00
Clinical Efficacy of AZD9150, a Next Generation, Constrained Ethyl Modified Antisense Oligonucleotide Inhibitor of STAT3 - Tumor Cell Autonomous and Immuno-modulatory Activity
AZD9150 is a next generation ASO that has demonstrated robust single agent efficacy in several tumor types in phase I clinical studies. Preclinical and clinical data suggest the activity of AZD9150 is mediated by both tumor cell autonomous effects as well as through modulation of the immunosuppressive microenvironment through the inhibition of STAT3 in tumor-associated immune cell populations.
Youngsoo Kim, Ph.D., Director of Oncology, Ionis Pharmaceuticals, Inc.

12:30
Networking Luncheon with Exhibit and Poster Viewing

2:00
Preclinical Therapeutic Development against Topical Diseases Using Cell Penetrating, Asymmetric RNAi Triggers
Cell penetrating, asymmetric siRNA (cp-asiRNA) is an asymmetric RNAi trigger with simple combination of chemical modifications. cp-asiRNA enters into cells and triggers target gene silencing via RNA interference without the need of delivery vehicle. Preclinical therapeutic development against skin, eye and lung diseases using cp-asiRNA platform will be presented.
Dong-ki Lee, Ph.D., Professor, Sungkyunkwan University and Founder and CEO, OliX Pharmaceuticals, Korea

Featured Presentation

2:30
Troels Koch, Ph.D. Activity Determinants of Locked Nucleic Acids
Small structural modifications of antisense oligonucleotides can have significant effects on structure/activity relations. The underlying nature of these subtle relationships cannot be understood from a reductionist view point. We have employed a new modelling strategy to get a better understanding on how the structural units can impact the properties of LNA. It will be presented how new chemical modifications and designs can improve central therapeutic parameters of LNA.
Troels Koch, Ph.D., VP & Head of Research, RNA Therapeutics, Roche Innovation Center Copenhagen, Denmark

3:00
Networking Refreshment Break with Exhibit and Poster Viewing

3:40
Chairman's Remarks
Junichi Koga, Ph.D., Corporate Officer and Global Head of Biologics, Daiichi Sankyo, Japan

3:45
Daiichi-Sankyo's Strategies and Experiences in Biologics focused Oligonucleotide Drug Development
This presentation will discuss Daiichi-Sankyo's progress in the development of biologics especially oligonucleotide drugs. Lessons learned to date, technical challenges experienced and future approaches to progress our oligonucleotides drugs as an important part of our biologics pipeline will be presented.
Junichi Koga, Ph.D., Corporate Officer and Global Head of Biologics, Daiichi Sankyo, Japan

4:15
Recent Progress with RNAi Therapeutics Addressing Hepatic Targets
Chronic hepatitis B infection is a serious liver disease impacting 400 million people worldwide. Clinical manifestations are severe, and an estimated 1 million people die each year from the disease and its complications. With today's medicines, the cure rate for chronic HBV infection is less than 10%. An RNAi therapeutic inhibiting all steps of the HBV life cycle and silencing tolerogenic viral antigens has the potential to achieve a "functional cure."
Laura Sepp-Lorenzino, Ph.D., Vice President, Entrepreneur-in-Residence, Alnylam Pharmaceuticals, USA

4:45
Eteplirsen a Drug for Duchenne Muscular Dystrophy
DMD, a neuromuscular disease, affects ~1:3500 of newborn boys and invariably leads to their untimely death. DMD is most commonly caused by deletions in the DMD gene that disrupt the mRNA reading frame and prevent translation of dystrophin, an essential muscle protein. In ongoing trials, eteplirsen, a morpholino oligomer, has been shown to restore the mRNA reading frame and induce production of dystrophin. This treatment led to significant benefit in the six-minute walk test.
Ryszard Kole, Ph.D., Distinguished Scientist, Sarepta Therapeutics, USA

Peptide Discovery

11:20
Chairman's Remarks
Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion, USA

11:30
Oral Equivalents of Monoclonal Antibodies: Exploitation of Constrained Peptides
This presentation will briefly describe platform technology and focus on optimization of the oral delivery of constrained peptides, that are alternatives to marketed antibodies Stelara and Entyvio, for treatment of IBD. The presentation will describe rodent and monkey data, and criteria for selection of IND candidates which should enter the clinic in 2015 and early 2016.
Mark Smythe, Ph.D., Founder and Vice President, Protagonist Therapeutics, Australia

Drug Delivery Strategies for Peptides

12:00
Delivery Strategies for Peptides in Clinical Development
This presentation will include a brief overview of technologies available for subcutaneous injection replacement, injectable sustained release formulation, half-life extension and non-invasive delivery. The challenges and opportunities for each of these approaches will be clarified. In addition, a pathway for clinical development of a new peptide will be discussed along with strategy and timing for incorporating life cycle options.
Christopher A. Rhodes, Ph.D., President and CEO, Drug Delivery Experts, USA

12:30
Networking Luncheon with Exhibit and Poster Viewing

2:00
Peptide Mediated Delivery of Bioactive Proteins into Cells
Numerous approaches have been reported up to the present time for intracellular delivery. However, approaches are still needed that have improved efficacy in delivering high-molecular-weight proteins into cytosol to obtain enhanced bioactivity. By modifying sequences of natural hemolytic peptides, we have succeeded in creating a peptide, which has a low cytotoxicity but has a high ability to yield a marked cytosolic release of endocytosed proteins.
Shiroh Futaki, Ph.D., Professor, Institute for Chemical Research, Kyoto University, Japan

2:30
Case StudyNew Data
Biodegradable Silica Based Delivery of Therapeutic Peptides
Biodegradable silica is a powerful drug delivery matrix for sustained release of parenteral therapeutics. Encapsulation of peptides in nanoporous silica provides an effective tool to administer peptides in a controlled manner even for several months. The presentation describes the basics of silica technology with several case studies.
Lasse Leino, Ph.D., Adjunct Professor and CEO, DelSiTech Ltd., Finland

3:00
Networking Refreshment Break with Exhibit and Poster Viewing

3:40
Chairman's Remarks
Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion, USA

3:45
Phylomer Derived Cell Penetrating Peptides Facilitate More Efficient Delivery of Peptides and Proteins to the Cytoplasm
Phylomer peptide libraries have been screened for new cell penetrating peptides for delivery of macromolecules and nanoparticles into cells. A novel genetic screen known as the 'endosome escape trap' enables the isolation of rare CPP's which more efficiently deliver their cargoes to the cytoplasm. Some of these cell penetrating Phylomers can be targeted to particular cell types. Phylogica has developed a variety of functional assays to determine the extent of cytoplasmic delivery of peptide and protein cargoes to the cytoplasm or nucleus. These assays have shown Phylomer CPP's to be 37-160 times more efficient than TAT which can be active in vivo. These tools are now being applied in screens targeting transcription factor oncoproteins such as cMyc NMyc and STAT5 as well as for more efficient intracellular delivery of protein toxin conjugates.
Paul Watt, Ph.D., Chief Scientific Officer, Phylogica Ltd, Australia

4:15
An Improved Transdermal Patch of Teriparatide Using Ionic Liquid Transdermal System (ILTS)
Teriparatide is a recombinant parathyroid hormone used in the treatment of osteoporosis and approved as a subcutaneous injection. A transdermal patch which delivers teriparatide is being developed using Ionic Liquid Technology. A series of in vitro rat skin permeation tests and in vivo rat PK studies have been conducted which led to selection of a formulation which demonstrated a temporary increase in the concentration of teriparatide in the blood. Additionally, dermal irritation tests confirmed that there was no dermal irritation to the rat skin.
Tatsuro Moriyoshi, Researcher, R&D Department, MEDRx Co. Ltd., Japan

Panel Discussion

4:45
Drug Delivery Systems and Strategies
Moderator: Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion, USA

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

6:45
Networking Dinner in Kyoto
Join fellow attendees in a fantastic networking and dining opportunity at a local restaurant in beautiful Kyoto. Space is limited and an additional fee applies. Please indicate when you register if you plan to join the dinner.

Main Conference · Plenary Session · Thursday, February 25, 2016

8:30
Registration and Coffee

8:55
Chairman's Remarks
William S. Marshall, Ph.D., President and CEO, miRagen Therapeutics, USA

The Innovation and Investment Landscape in Japan and China

9:00
The Japan Landscape of Investment and Innovation in Oligonucleotide and Peptide Development
INCJ is a public-private investment fund which provides financial and management support for next-generation businesses including innovative drug development. This presentation will provide an overview of the investment and innovation landscape in Japan as well as discuss the future potential of and opportunities in oligonucleotide and peptide drug development in Japan.
Koichi Ashida, Executive Managing Director, Innovation Network of Japan, Japan

9:30
Development and Production of Therapeutic Oligonucleotides in China
There is a growing interest towards oligonucleotide-based therapeutics among drug developers in China. The field, however, still remains at relatively early stages. This presentation will summarize current status of oligonucleotide therapeutic programs. It will also outline the situation with the oligonucleotide manufacturing capabilities in China. Finally, a summary of the recently introduced by CFDA changes to the overall drug development process and procedures in the country will be presented.
Bill Zhang, Ph.D., President, Guangzhou RiboBio Co. Ltd., China

Keynote Presentation

10:00
Masafumi Matsuo, M.D., Ph.D. Exon-skipping Therapy Advancing Oligonucleotide Drug Development
This presentation will describe continued efforts in our laboratory to exploit exon-skipping oligonucleotides to treat a variety of diseases. Current progress and ongoing challenges of exon-skipping will be discussed. An update on the learnings from clinical studies in DMD patients will also be presented.
Masafumi Matsuo, M.D., Ph.D., Professor, Department of Physical Therapy, Kobe Gakuin University, Japan

10:30
Networking Refreshment Break with Exhibit and Poster Viewing

Main Conference · Concurrent Tracks · Thursday, February 25, 2016

Oligonucleotides in Preclinical and Clinical Development (continued)

11:10
Chairperson's Remarks
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany

11:15
Mirror-image Oligonucleotide Aptamers Inhibiting the Chemokine CXCL12/SDF-1 - From Identification to Phase IIa Data
SDF-1/CXCL12 is a pro-angiogenic chemokine that plays a major role in homing processes of stem cells and malignant stem cells. Furthermore, the chemokine also interacts with two receptors, i.e. CXCR4 and CXCR7. We have identified a high affinity mirror-image oligonucleotide (NOX-A12, olaptesed pegol) that neutralizes SDF-1's interactions with both receptors. The compounds showed intriguing efficacy in different oncology models. Currently, the substance is profiled in two Phase IIa studies in patients with multiple myeloma and chronic lymphocytic leukemia.
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany

11:45
DNA/RNA Heteroduplex Oligonucleotide as a Novel Concept of Therapeutic Oligonucleotide
We develop a short DNA/RNA heteroduplex oligonucleotide (HDO) with a structure different from double-stranded RNA used for short interfering RNA and single-stranded DNA used for ASO. A DNA/lockednucleotide acid gapmer duplex with an a-tocopherol-conjugated complementary RNA (Toc-HDO) is significantly more potent at reducing the expression of the targeted mRNA in liver compared with the parent single-stranded gapmer ASO. Toc-HDO also improves the phenotype in disease models more effectively. In addition, the high potency of Toc-HDO results in a reduction of liver dysfunction observed in the parent ASO at a similar silencing effect.
Takanori Yokota, M.D., Ph.D., Professor, Neurology & Neurological Science, Tokyo Medical and Dental University, Japan

12:15
Networking Luncheon with Exhibit and Poster Viewing

1:30
Case Study
From Concept to Clinic: MRG-106, aLNA-antimiR® Targeting microRNA-155 for Hematological Malignancies
microRNA-155 is a product of the bic oncogene and its overexpression has been implicated as a poor prognostic indicator in a variety of hematological malignancies. We identified MRG-106 through a rational screening approach demonstrating target engagement in human cancer cells in vitro via passive cellular uptake. Additional preclinical studies revealed a favorable PK/PD and safety profile for the candidate. Our translational development strategy is based on early mechanistic proof of concept and progressive de-risking in man.
William S. Marshall, Ph.D., President and CEO, miRagen Therapeutics, USA

2:00
Oligonucleotides in Oncology and Conjugation Strategies
Abstract not available at time of print.
Bob Brown, Ph.D., CSO and SVP, Research, Dicerna Pharmaceuticals, USA

Oligonucleotide CMC and Analytical Strategies

2:30
AJIPHASE®: Improved Solution-Phase Technology for Large Scale Oligonucleotides Manufacturing
Synthetic oligonucleotide ranging from few-kilograms to several ton will be required in the near future especially as some antisense drugs. Recently, we have reported a unique solution-phase technology AJIPHASE® for DNA/RNA-type and morpholino-type oligonucleotide synthesis which can solve an issue of procurement in large volume. This presentation will describe the technical updates of AJIPHASE® technology and the quality comparison to the solid-phase
Kunihiro Hirai, Researcher, Research Institute for Bioscience Products & Fine chemicals, Ajinomoto Co. Inc., Japan

3:00
Networking Refreshment Break with Exhibit and Poster Viewing

3:30
Small Scale Manufacturing of Novel Oligonucleotides
Abstract not available at time of print.
Huihe (Julia) Zhu, Ph.D., Group Leader, Small Scale Oligo Unit, Nitto Denko Avecia

4:00
Analysis of Oligonucleotide and RNAi Molecules using UPLC/HRMS and Routine Mass Detection Workflows
RNAi drugs needs to be well characterized to satisfy regulatory requirements and minimize possible adverse affect on the safety and efficacy. High resolution of ultra performance liquid chromatography (UPLC) coupled with mass spectrometry analysis is a powerful tool for analysis of biopharmaceutical drugs such as RNAi oligonucleotides. This presentation will focus on a direct UPLC/MS(MS) workflow for RNAi characterization.
Scott Berger, Ph.D., Senior Manager, Pharmaceutical Business, Waters Corporation Waters Corporation

4:30
GalNac Conjugated LNA Oligonucleotides
The conjugation of targeting moieties to oligonucleotides has seen much increasing research efforts. This talk will present examples of conjugation of LNA oligonucleotide from a CMC prespective and the changes in properties induced hereby.
Christoph Rosenbohm, Ph.D., Senior Director Research Operations, Roche Innovation Center Copenhagen, Denmark

5:00
Close of Day Two

Peptide Discovery, Preclinical and Clinical

11:10
Chairperson's Remarks
El Djouhar Rekaï, Head of Peptide Products Operation, Lonza, Belgium

11:15
Cystine Knot Peptides as Templates in Drug Design
Cyclotides are ultra-stable cystine-knot containing peptides that have great potential as templates in drug design. There are now 15 published examples of cyclotide-based drug leads for conditions including cancer, cardiovascular disease, autoimmune disease and pain, amongst others. This presentation will describe the design strategies and future possibilities for cyclotide-based drug design. Of particular note is the ability of cyclotides to penetrate cells and modulate intracellular targets, including protein-protein interactions.
David Craik, Ph.D., Professor of Biomolecular Structure, Institute for Molecular Bioscience, University of Queensland, Australia

11:45
New Broad Activities of CBP501 in Tumor Cells and the Tumor Microenvironment Uncovered by the Results of Phase II Clinical Studies
CBP501, a synthetic dodecapeptide, completed two Phase II clinical studies for malignant pleural mesothelioma and NSCLC. In the NSCLC study, survival was significantly prolonged in a patient population with normal WBC. These results led to studies revealing new broad effects of calmodulin modulation by CBP501 in tumor cells and the tumor microenvironment. CBP501 continues to hold promise as a drug candidate in the paradigm-shifted immune oncology drug development world.
Takumi Kawabe, M.D., Ph.D., President & CEO, CanBas Co. Ltd., Japan

12:15
Networking Luncheon with Exhibit and Poster Viewing

1:30
Case Study
Macrocycles Addressing Challenging Therapeutic Targets – An Underexploited Structural Class
Macrocycles provide diverse functionality and stereochemical complexity in a conformationally pre-organized ring structure. This can result in high affinity and selectivity for challenging intra- and extracellular protein targets while preserving excellent bioavailability. Polyphor has developed two proprietary technologies for the generation of fully synthetic macrocycles for target-based as well as phenotypic screening. An automated and highly streamlined medicinal chemistry optimization process to advance initial hits to clinical candidates in rapid iterative cycles is in place. This presentation will focus on some representative case studies.
Daniel Obrecht, Ph.D., Chief Scientific Officer, Co-Founder, Polyphor Ltd., Switzerland Polyphor

Peptide CMC and Analytical Strategies

2:00
Improved Chemical and Physical Stability in Liquid Formulations – How to Design Stable Glucagon Analogues
Through a series of iterative changes in the native sequence of glucagon we present an optimized glucagon analogue (ZP-GA-1) suitable for long term storage as a liquid formulation with a pharmacokinetic and pharmacodynamic profile of native glucagon. ZP-GA-1 exhibits superior solubility (>25 mg/ml) at neutral pH relative to native glucagon and improved chemical and physical stability.
Lise Giehm, Ph.D., Senior Scientist, Pharmaceutical Development, Zealand Pharma A/S, Denmark

2:30
Process Development, Commercial Manufacturing and Quality Control for Generic Peptide API Atosiban
The latest developments in the large scale manufacturing of synthetic peptides will be presented. The talk will consider CMC challenges, process development and manufacturing aspects for large scale SPPS and prep. HPLC purification.
Daniel Samson, Ph.D., Senior Director API Manufacturing, Bachem AG, Switzerland Bachem - Pioneering Partner for Peptides

3:00
Networking Refreshment Break with Exhibit and Poster Viewing

3:30
Case Study
Life-cycle Management in Peptide Manufacturing
As technologies for the production of synthetic peptides evolve, the chemical processes developed and validated 15 or 20 years ago often need to be optimized for safety, productivity and cost effectiveness. As a leader in the custom manufacturing of synthetic peptides, Lonza is putting emphasis on the life-cycle management of its products in an increasingly competitive environment. This case study of a generic peptide will present the activities involved to support such a program, including process and analytical development, process qualification and validation as well as the regulatory filing strategy. The benefits of such a program will be described, both for the CMO and for the customer.
Jean-Marc Poudrel, Ph.D., Program Manager, Lonza Braine SA, Belgium

4:00
Development of a Large Scale Process for the Manufacture of a Small Cyclic Peptide API
Abstract not available at time of print.
Jon Holbech Rasmussen, Ph.D., Director Global Development, PolyPeptide Group, Sweden PolyPeptide Group

4:30
Close of Day Two

Main Conference · Plenary Session · Friday, February 26, 2016

8:45
Registration and Coffee

9:10
Chairman's Remarks
Osamu Sam Sato, Executive Director, R&D Planning Department, Daiichi Sankyo, Japan

Keynote Presentation

9:15
George Nakayama Challenge of Daiichi-Sankyo to be a Global Pharma Innovator: Updated Directions
This presentation will discuss the challenge of Daiichi-Sankyo to become one of the world's leading innovators in pharmaceutical drug business and R&D with a diverse drug portfolio. This talk will give an overview of Daiichi-Sankyo's strategic business and scientific strategies, including its vision in the field of oligonucleotide drug development.
George Nakayama, Representative Director, President & CEO, Daiichi Sankyo, Japan

Featured Presentation

9:45
Jesper Lau, Ph.D. Semaglutide, An Acylated GLP-1 Analog Suitable for Once-Weekly Administration
Following the successful development of the first once-daily GLP-1 analog liraglutide, we aimed to discover analogs suitable for once-weekly dosing through acylation with fatty acids enabling binding to serum albumin in vivo. This technology avoids the need for substantially increased molecular weight by fusion to a large inactive protein, or marked additives in the formulation requiring larger needle sizes. The fatty acid "sidechain" was one of the key features to secure the combination of high albumin affinity and high GLP-1R potency. Several parameters were investigated such as length and type of fatty acid as well as the linking chemistry between the fatty acid and GLP-1. Semaglutide has a peptide backbone with two substitutions compared to human GLP-1 (8Aib, 34Arg) and is derivatized with a fatty acid "sidechain" at lysine 26. Semaglutide is in phase 3 clinical trial for treatment of diabetes.
Jesper Lau, Ph.D., Vice President, Diabetes Protein and Peptide Chemistry, Novo Nordisk A/S, Denmark

10:15
Refreshment Break

Featured Presentation

10:45
Richard Holslag Life and Times of an Acquisition
This presentation will address challenges in 1) Courtship before acquisition 2) Integration after acquisition 3) Preparing and submitting an NDA and MAA while being acquired 4) Development of regulatory strategy and 5) Aspects to manage CMOs relationships.
Richard Holslag, Vice President, Oligo Manufacturing, BioMarin Nederland B.V., The Netherlands

Regulatory Perspectives on Oligonucleotide and Peptide Impurities

11:15
Regulatory Considerations and Challenges on Impurity Issues during Different Phases of Oligonucleotide and Peptide Drug Development
Due to their diverse therapeutic activities, peptides and oligonucleotides may be regulated under different regulations in the US, for example, as chemical drugs or biological products, with different requirements for each. ICH changes to the peptide guidance have also made it very challenging for companies to deal with regulatory issues such as impurities, during the different phases of development. The presentation will discuss 1) the effects of organization and process changes in the US on the review and approvals of oligonucleotide and peptide products, 2) the unique regulatory issues associated with impurities of oligonucleotides and peptides and 3) common mistakes and how to manage potential impurity problems during different phases of drug development, for example, during site scale-up and site changes.
Duu-Gong Wu, Ph.D., Director/Senior Consultant, PPD Regulatory Consulting and Former Deputy Division Director, Division of New Drug Chemistry, US FDA/CDER, USA

11:45
Close of AsiaTIDES Main Conference

Post-Conference Workshops · Friday, February 26, 2016

Workshop #1

Current Status of Oligonucleotide Drug Delivery Approaches and Systems (DDS)

Workshop Moderator: Dmitry Samarsky, Ph.D., SVP of Technology and Global Business Development, OliX Pharmaceuticals, South Korea

RNAi and antisense oligonucleotides promise to become the third major therapeutic modality (besides small molecules and biologics), offering an opportunity to target virtually any disease causing gene. Such promise, however, requires addressing the challenge of delivering oligonucleotide-based therapeutic compounds to the tissues/organs originating the disorders, as well as inside the cells producing the disease phenotype. This workshop will dissect the delivery issue into smaller sub-challenges, review those in deeper detail and offer some examples of successful solutions.

Additional topics to be discussed include:

  • Why oligos bio-distribute predominantly to a small group of organs (mostly liver)?
  • If endothelial barriers prevent from broader bio-distribution, what can be done about it?
  • Is tissue/organ penetration an additional challenge/barrier for oligo drug delivery?
  • How do oligonucleotides cross cellular membrane barriers?
  • Should we consider oligos as a class or individual compounds when it comes to delivery?
  • How big of a factor for choosing delivery approaches is the manufacturing component?

Distinguished Participants
Troels Koch, Ph.D., VP & Head of Research, RNA Therapeutics, Roche Innovation Center Copenhagen, Denmark
William Marshall, Ph.D., President & CEO, miRagen Therapeutics, Inc., USA
Laura Sepp-Lorenzino, Ph.D., Vice President, Entrepreneur-in-Residence, Alnylam Pharmaceuticals, USA
Takanori Yokota, M.D., Ph.D., Professor, Neurology & Neurological Science, Tokyo Medical and Dental University, Japan

Workshop #2

Peptide Formulations: Regulatory Considerations for Early Clinical Research

Workshop Moderators:
Christopher A. Rhodes, Ph.D., President and CEO, Drug Delivery Experts
Bruce Morimoto, Ph.D., Executive Director Applied Translational Medicine, Drug Development Services, Celerion

This workshop will discuss lifecycle strategies for peptides and peptide formulations from late stage research to early clinical development and beyond. Formulation challenges of novel and complex peptide structures will be presented along with dosage form strategies in early stage development and techniques for lifecycle planning. The workshop will include a detailed case study on buccal delivery of peptides with an emphasis on the early stage strategies needed to establish feasibility of the project. Additional peptide case studies from early to late stage development along with challenges encountered and lessons learned will also be presented.

Additional topics to be discussed include:

  • What goes into a US Investigational New Drug (IND) application?
  • What research or formulation development work is needed?
  • What toxicology work is needed?
  • Simple versus complex formulations and delivery options and life-cycle management

Post-Conference Workshop Schedule

12:00
Networking Luncheon for Workshop Attendees

1:00
Workshop Opening Remarks

2:30
Refreshment Break

4:00
Close of Workshop

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