.jpg)
.jpg)
.jpg)
AsiaTIDES: Oligonucleotide and Peptide® Research, Technology and Product Development
Agenda
Monday, February 22, 2010
8:00
Conference Registration Begins
Tutorial #1:
Analysis and Bioanalysis of Oligonucleotides in Support of Drug Development
Tutorial Leader: G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
The tutorial will lead off with a review of US and European regulatory expectations for the analysis of oligonucleotides (antisense, siRNA, DNA decoys, aptamers, etc.). Assuring product quality through raw material testing, in process control and quality control testing of single strand and siRNA oligonucleotides in a manufacturing environment will be addressed. Special topics will include analysis of oligonucleotides in complex formulations and biological matrices, technology transfer/validation as well as a case study of a siRNA drug product analysis.
9:00
Analysis of Oligonucleotides: US and European Regulatory Expectations
The complex and diverse nature of oligonucleotides currently in development (antisense, DNA decoys, siRNA, aptamers, etc.) 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 entering or in clinical development.
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
The complex and diverse nature of oligonucleotides currently in development (antisense, DNA decoys, siRNA, aptamers, etc.) 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 entering or in clinical development.
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
9:35
Analysis of Single Strand Oligonucleotides
Established and state-of-the-art analytical techniques utilized to control the quality of single strand oligonucleotides will be discussed in this presentation. Approaches to purity and assay determination during QC release testing will further be emphasized through a thorough understanding of typical single strand oligonucleotide physico-chemical properties and their implications on the respective test specifications.
Ipsita Roymoulik, Ph.D., Analytical Development Group Leader, Aveica OligoMedicines, USA
Established and state-of-the-art analytical techniques utilized to control the quality of single strand oligonucleotides will be discussed in this presentation. Approaches to purity and assay determination during QC release testing will further be emphasized through a thorough understanding of typical single strand oligonucleotide physico-chemical properties and their implications on the respective test specifications.
Ipsita Roymoulik, Ph.D., Analytical Development Group Leader, Aveica OligoMedicines, USA
10:10
Special Considerations for siRNA
This presentation will deal with analytical methods for siRNA. The monitoring and control of various production steps as well as the final product QC release will be discussed. All methods will by explained by showing results of an example sequence. In detail, analytical methods to analyse critical starting materials, the crude product, product after purification and hybridization will be presented and discussed. Finally, final QC release tests, including sequencing and duplex mass determination will be further elucidated.
Hüseyin Aygun, Ph.D., Chief Scientific Officer, BioSpring GmbH, Germany
This presentation will deal with analytical methods for siRNA. The monitoring and control of various production steps as well as the final product QC release will be discussed. All methods will by explained by showing results of an example sequence. In detail, analytical methods to analyse critical starting materials, the crude product, product after purification and hybridization will be presented and discussed. Finally, final QC release tests, including sequencing and duplex mass determination will be further elucidated.
Hüseyin Aygun, Ph.D., Chief Scientific Officer, BioSpring GmbH, Germany
10:45
Networking Refreshment Break with Poster and Exhibit Viewing
11:15
Case
Study
Analysis of Complex Stable Nucleic Acid Lipid Particle (SNALP) FormulationsStudy
SNALP are specialized lipid nanoparticles that fully encapsulate and systemically deliver a variety of nucleic acid molecules such as siRNA. Pre-clinical in vivo studies have shown SNALP to be effective in delivering nucleic acid to the cytoplasm of target cells to enable the desired therapeutic effect while minimizing systemic toxicity. siRNA-SNALP products are currently being evaluated in clinical studies. This presentation will focus on the development of methods used to accurately and completely describe SNALP and ensure consistent quality of the clinical products.
Nancy Fuselli, Director, Quality Control and Analytical Development, Tekmira Pharmaceuticals Corporation, Canada
11:50
Analytical Approaches to Determine Stability, PK Properties and Biodistribution of siRNAs
Various approaches to analyze RNA, especially siRNA, from biological samples with LC-MS and IEX-HPLC will be presented. An AEX-HPLC based approach and its braod application to a variety of biological samples will be discussed in more detail. A major focus of this presentation will be the recovery of the analyte molecules for different applications.
Ingo Roehl, Ph.D., Associate Director, Analytical Chemistry, Roche Kulmbach GmbH, Germany
Various approaches to analyze RNA, especially siRNA, from biological samples with LC-MS and IEX-HPLC will be presented. An AEX-HPLC based approach and its braod application to a variety of biological samples will be discussed in more detail. A major focus of this presentation will be the recovery of the analyte molecules for different applications.
Ingo Roehl, Ph.D., Associate Director, Analytical Chemistry, Roche Kulmbach GmbH, Germany
12:25
Case
Study
Analytical Development for Early-Stage siRNA Products: A Case StudyStudy
Quark Pharmaceuticals has three siRNAs in the clinic: two that are administered via intravitreal injection for ocular indications and one that is administered systemically for kidney diseases. This tutorial will describe the analytical methods used for quality control testing and stability monitoring of API/drug product. The strengths and weaknesses of these methods will be summarized, and a case study of how the performance of these methods has impacted the development process will be provided.
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
1:00
Close of Tutorial
Tutorial #2:
Scaling up Oligonucleotide Manufacturing Process
Tutorial Leader: Lubomir Nechev, Ph.D., Senior Director, Process Chemistry, Alnylam Pharmaceuticals, Inc., USA
The tutorial will focus on the scale-up challenges of the critical steps in the oligonucleotide manufacturing process. The points of view of drug developers, oligonucleotide contract manufacturers, as well as synthesis hardware manufacturers will be presented. The effects of different modifications and scale on the manufacturing process as well as techniques for developing scaled-down process models will be discussed.
9:00
Introduction to the Tutorial
Lubomir Nechev, Ph.D., Senior Director, Process Chemistry, Alnylam Pharmaceuticals, Inc., USA
Lubomir Nechev, Ph.D., Senior Director, Process Chemistry, Alnylam Pharmaceuticals, Inc., USA
9:10
A History of DNA/RNA Synthesizers
Hear information on early synthesizers, the leaders and their challenges on how and why some of the synthesizers were built. Look back at some of the first DNA synthesizers sold commercially, as well as the technical challenges due to limited off the shelf available hardware. Move forward with DNA synthesis today and where it is heading.
Jeffrey Christopher Strauss, Vice President, BioAutomation Corp., USA
Hear information on early synthesizers, the leaders and their challenges on how and why some of the synthesizers were built. Look back at some of the first DNA synthesizers sold commercially, as well as the technical challenges due to limited off the shelf available hardware. Move forward with DNA synthesis today and where it is heading.
Jeffrey Christopher Strauss, Vice President, BioAutomation Corp., USA
9:50
Challenges in the Preparation and Analysis of Conjugated Oligonucleotide Drug Candidates
Methods, standard as well as novel, used to prepare oligonucleotide conjugates with large and small molecules will be discussed. LCMS based methods used to analyze conjugated and or conjuagatable impurities will be presented. Small molecule derivatization and enzymatic techniques to determine impurities in PEG derivatized oligos, which are too large to be analyzed by standard LCMS techniques, will be discussed. Our recent work on characterization of conjugatable impurities formed during deprotection will also be shown.
Kenneth W. Hill, Ph.D., Principal Scientist, Agilent Technologies, USA
Methods, standard as well as novel, used to prepare oligonucleotide conjugates with large and small molecules will be discussed. LCMS based methods used to analyze conjugated and or conjuagatable impurities will be presented. Small molecule derivatization and enzymatic techniques to determine impurities in PEG derivatized oligos, which are too large to be analyzed by standard LCMS techniques, will be discussed. Our recent work on characterization of conjugatable impurities formed during deprotection will also be shown.
Kenneth W. Hill, Ph.D., Principal Scientist, Agilent Technologies, USA
10:30
Networking Refreshment Break with Poster and Exhibit Viewing
11:00
QbD Development Strategies for Oligonucleotide Therapeutics
The application of pharmaceutical development strategies described in ICH Q8, such as Design of Experiments (DoE), requires the use of accurate small scale process models. The tutorial will discuss the challenges involved in developing representative process models for the manufacture of oligonucleotide API from a scale up and scale down perspective and demonstrate their use in the application of DoE to oligonucleotide development.
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
The application of pharmaceutical development strategies described in ICH Q8, such as Design of Experiments (DoE), requires the use of accurate small scale process models. The tutorial will discuss the challenges involved in developing representative process models for the manufacture of oligonucleotide API from a scale up and scale down perspective and demonstrate their use in the application of DoE to oligonucleotide development.
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
11:40
Unique Challenges Associated with Large Scale Manufacturing of Different RNA Sequences
Increasing numbers of RNA based therapeutics are being developed. Many of these sequences contain 2' modifications or mixed backbones with phosphorothioate and phosphodiester linkages. Chemical modifications and sequence specific effects must be considered when developing the manufacturing process. The impact of various RNA sequences and modifications on the development of solid phase synthetic cycles and chromatographic purifications will be discussed.
Kathryn L. Ackley, Ph.D., Director of Operations, Girindus., USA
Increasing numbers of RNA based therapeutics are being developed. Many of these sequences contain 2' modifications or mixed backbones with phosphorothioate and phosphodiester linkages. Chemical modifications and sequence specific effects must be considered when developing the manufacturing process. The impact of various RNA sequences and modifications on the development of solid phase synthetic cycles and chromatographic purifications will be discussed.
Kathryn L. Ackley, Ph.D., Director of Operations, Girindus., USA
12:20
Effect of Scale on Oligonucleotide Manufacturing Process
The changes in the oligonucleotide manufacturing process needed to accommodate the requirement and the specifics of different manufacturing scales will be discussed. The challenges of scaling up and the equipment used in different steps of the process will be presented.
Lubomir Nechev, Ph.D., Senior Director Process Chemistry, Alnylam Pharmaceuticals, Inc., USA
The changes in the oligonucleotide manufacturing process needed to accommodate the requirement and the specifics of different manufacturing scales will be discussed. The challenges of scaling up and the equipment used in different steps of the process will be presented.
Lubomir Nechev, Ph.D., Senior Director Process Chemistry, Alnylam Pharmaceuticals, Inc., USA
1:00
Close of Tutorial
AsiaTIDES® attendees may also attend the plenary session of the concurrent conference, Drug & Biologics Japan, as an optional session Monday morning. The session is titled:
"Innovation in Japan - Is it Still Alive? Strategies for Open Innovation to Enhance Productivity and Creativity."
Please visit for more information.
1:00
Networking Luncheon with Poster and Exhibit Viewing and Registration for Main Conference
Monday, February 22, 2010
Tutorials | Main Conference: Monday | Main Conference: Tuesday | Main Conference: Wednesday
1:45
Chairperson's Remarks
Yoshikazu Nakamura, Ph.D., Professor, Department of Basic Medical Science, Institute of Medical Science, University of Tokyo; Founder, Ribomic, Japan
Yoshikazu Nakamura, Ph.D., Professor, Department of Basic Medical Science, Institute of Medical Science, University of Tokyo; Founder, Ribomic, Japan
Keynote Presentations
2:00
Therapeutic Applications of Dicer Substrate siRNAsThis presentation describes the use of Dicer substrate RNAs (dsiRNAs) for the treatment of HIV infection in a humanized mouse model for HIV infection. In addition we have developed dual targeting dsiRNAs for cancer treatment, in which each strand selectively targets a different message to trigger synthetic lethality in cancer treatment. Dicer substrate siRNAs present a unique platform for a variety of RNAi applications.
John J. Rossi, Ph.D., Chairman, Lidow Family Endowed Research; Professor, Molecular Biology, Beckman Research Institute of City of Hope, USA
Presentation supported by Dicerna Pharmaceuticals
2:45
Defying Difficult Diseases: Biomolecule-based Medicinal ScienceBased on the substrate transition state, we designed and synthesized novel classes of inhibitors of aspartic proteases such as renin, HIV protease, malarial plasmepsin II, ß-secretase (BACE1) containing the hydroxymethylcarbonyl (HMC) isostere. On the basis of our study with the "O-acyl isopeptide method", we developed novel photo- and pH-triggered "click" peptides that readily convert to the native amyloid ß peptide 1-42 upon activation.
Yoshiaki Kiso, Ph.D., Professor & Chairman, Department of Medicinal Chemistry, Kyoto Pharmaceutical University, Japan
3:30
Networking Refreshment Break with Poster and Exhibit Viewing
Keynote Presentations
4:00
RNAi and Peptide Intellectual Property Landscapes: Early and EvolvingShort nucleic acids (RNAi and oligonucleotides) and peptides share the fundamental feature that they are a fragment of a longer molecule and possess regulatory function. Is there important IP to consider for such molecules? What has granted in the US, Europe, and Japan? Why do the country IP landscapes differ? Early RNAi patent filings include Fire, Tuschl I/II, Kreutzer, Glover, Rossi. How do the early patents affect the current market?
Kathleen M. Williams, Ph.D., J.D., Partner, Intellectual Property, Edwards Angell Palmer & Dodge LLP, USA
4:45
Synthesis and Biological Activity of the Nucleic Acids and Their AnalogsThe lecture will focus on biochemical activities of boranephosphonate oligonucleotides, phosphonoacetate oligonucleotide, and new methods for synthesizing DNA and RNA.
Marvin H. Caruthers, Ph.D., Distinguished Professor, Chemistry and Biochemistry, University of Colorado, USA
5:30
Networking Cocktail Reception with Poster and Exhibit Viewing
Reception Co-Sponsored by
and
and
Tuesday, February 23, 2010
Tutorials | Main Conference: Monday | Main Conference: Tuesday | Main Conference: Wednesday
Oligonucleotide-Based Therapeutics
Updates on Oligonucleotide-Based Therapeutics in Preclinical Development
8:30
Chairperson's Remarks
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, Inc., USA
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, Inc., USA
8:45
Lead Generation for microRNA Modulators
microRNAs regulate targets by binding to 3'UTRs with sequence complementarity to the microRNA seed region. The ability of microRNAs to target multiple transcripts enables fine-tuning of gene networks as a novel approach to therapy, but also presents unique challenges for lead candidate selection and development of microRNA therapeutics. We will discuss how we are addressing some of these unique challenges.
Bal Bhat, Ph.D., Senior Director, Medicinal Chemistry, Regulus Therapeutics
microRNAs regulate targets by binding to 3'UTRs with sequence complementarity to the microRNA seed region. The ability of microRNAs to target multiple transcripts enables fine-tuning of gene networks as a novel approach to therapy, but also presents unique challenges for lead candidate selection and development of microRNA therapeutics. We will discuss how we are addressing some of these unique challenges.
Bal Bhat, Ph.D., Senior Director, Medicinal Chemistry, Regulus Therapeutics
9:15
Intratumoral Knockdown of the Oncogene KRAS with DsiRNA
Dicer-substrate siRNAs (DsiRNAs) are capable of triggering potent and long lasting RNA interference effects. DsiRNAs targeting the oncogene KRAS have been used in vitro and in vivo to reduce KRAS expression to nearly undetectable levels in some normal tissues and in tumors. In vivo results focusing on KRAS-expressing orthotopic human xenograft tumor models, including both solid and hematological tumors, will be presented.
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, Inc.
Dicer-substrate siRNAs (DsiRNAs) are capable of triggering potent and long lasting RNA interference effects. DsiRNAs targeting the oncogene KRAS have been used in vitro and in vivo to reduce KRAS expression to nearly undetectable levels in some normal tissues and in tumors. In vivo results focusing on KRAS-expressing orthotopic human xenograft tumor models, including both solid and hematological tumors, will be presented.
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, Inc.
9:45
microRNA as a Novel Modality for Cancer Therapy
RNAi offers possible platform to identify potential drug candidates to any target even in undruggable or not amenable to conventional drugs. This paper introduces the examples of siRNAs and microRNAs delivery into tumors with metastasis in animal models, and discusses the potential there of these miRNAs as diagnostic and prognostic markers and as targets/molecules for molecular cancer therapy.
Takahiro Ochiya, Ph.D., Head, Section for Studies on Metastasis, National Cancer Center Research Institute, Japan
RNAi offers possible platform to identify potential drug candidates to any target even in undruggable or not amenable to conventional drugs. This paper introduces the examples of siRNAs and microRNAs delivery into tumors with metastasis in animal models, and discusses the potential there of these miRNAs as diagnostic and prognostic markers and as targets/molecules for molecular cancer therapy.
Takahiro Ochiya, Ph.D., Head, Section for Studies on Metastasis, National Cancer Center Research Institute, Japan
10:15
Networking Refreshment Break with Poster and Exhibit Viewing
10:45
Preclinical and First Clinical Data from Mirror-Image RNA Oligonucleotides
Functional mirror-image oligonucleotides, also called Spiegelmers® or L-aptamers, can be identified to bind and inhibit targets conceptually similar to antibodies. They have a direct mode of action and tremendous biostability. NOXXON's lead candidates are NOX-E36 and NOX-A12, targeting the chemokines MCP-1 and SDF-1, respectively. Data will be presented on NOX-E-36, which is in Phase I, and NOX-A12, which has completed regulatory tox and safety studies.
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany
Functional mirror-image oligonucleotides, also called Spiegelmers® or L-aptamers, can be identified to bind and inhibit targets conceptually similar to antibodies. They have a direct mode of action and tremendous biostability. NOXXON's lead candidates are NOX-E36 and NOX-A12, targeting the chemokines MCP-1 and SDF-1, respectively. Data will be presented on NOX-E-36, which is in Phase I, and NOX-A12, which has completed regulatory tox and safety studies.
Sven Klussmann, Ph.D., Chief Scientific Officer, NOXXON Pharma AG, Germany
11:15
Scientific
Highlight
Stereocontrolled Synthesis and Properties of Backbone-Modified OligonucleotidesHighlight
We have developed an oxazaphospholidine method for the stereocontrolled synthesis of phosophorothioate DNA and RNA. The method has been successfully applied to the synthesis of stereoregulated boranophosphate DNA. Quite recently, the method has also been used for the first synthesis of stereoregulated H-phosphonate DNA, which is useful as a versatile intermediate for the synthesis of P-chiral backbone-modified DNA analogs.
Takeshi Wada, Ph.D., Department of Medical Genome Sciences, The University of Tokyo, Japan
11:45
Technology Workshop Presentation Opportunity Available
IBC's Technology workshops offer supplier and service companies the opportunities to present product and service offers directly to the audience at the conference. For more information on presenting a technology workshop at this meeting, please contact us.
IBC's Technology workshops offer supplier and service companies the opportunities to present product and service offers directly to the audience at the conference. For more information on presenting a technology workshop at this meeting, please contact us.
12:15
Networking Luncheon with Poster and Exhibit Viewing
Updates on Oligo-Based Therapeutics in Clinical Development
1:30
Chairperson's Remarks
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
1:45
Anti-Fibrosis Therapy by siRNA
Fibrosis is caused by overabundant deposition of collagen in damaged tissue. Various severe diseases, including liver cirrhosis, idiopathic lung fibrosis, myelofibrosis, are categorized as fibrosis, however, there is no radical therapy for them. We will report a novel therapy for liver cirrhosis by siRNA for collagen chaperone HSP47 with targeting delivery system.
Yasunobu Tanaka, Ph.D., Manager, Hokkaido Laboratory, Corporate Business Development, Nitto Denko Corporation, Japan
Fibrosis is caused by overabundant deposition of collagen in damaged tissue. Various severe diseases, including liver cirrhosis, idiopathic lung fibrosis, myelofibrosis, are categorized as fibrosis, however, there is no radical therapy for them. We will report a novel therapy for liver cirrhosis by siRNA for collagen chaperone HSP47 with targeting delivery system.
Yasunobu Tanaka, Ph.D., Manager, Hokkaido Laboratory, Corporate Business Development, Nitto Denko Corporation, Japan
2:15
Phase I Evaluation of Stable Nucleic Acid Lipid Particles Containing Anti-ApoB siRNA
Stable Nucleic Acid Lipid Particles (SNALP) that potently silence the conventionally non-druggable target, apolipoprotein B, have been shown to protect mice from developing atherosclerotic lesions in preclinical models of cardiovascular disease. Findings of clinically relevant RNAi-mediated gene silencing support the evaluation of ApoB-SNALP in hypercholesterolemic patients. Early results from a Phase I clinical trial will be presented.
Ian MacLachlan, Ph.D., Executive Vice President & Chief Scientific Officer, Tekmira Pharmaceuticals Corp., Canada
Stable Nucleic Acid Lipid Particles (SNALP) that potently silence the conventionally non-druggable target, apolipoprotein B, have been shown to protect mice from developing atherosclerotic lesions in preclinical models of cardiovascular disease. Findings of clinically relevant RNAi-mediated gene silencing support the evaluation of ApoB-SNALP in hypercholesterolemic patients. Early results from a Phase I clinical trial will be presented.
Ian MacLachlan, Ph.D., Executive Vice President & Chief Scientific Officer, Tekmira Pharmaceuticals Corp., Canada
2:45
RNA Silencing: Realization and Novel Concepts
Executing the therapeutic potential of RNA silencing is of great interest. A single stranded approach using short, stable and highly potent oligonucleotides is the most direct strategy. Locked Nucleic Acid (LNA) represents such a direct strategy, and LNA can equally well be used for mRNA and microRNA silencing. The latest silencing concepts and mechanisms will be presented, together with the status of our LNA R&D programs.
Troels Koch, Ph.D., Vice President, Research, Santaris Pharma, Denmark
Executing the therapeutic potential of RNA silencing is of great interest. A single stranded approach using short, stable and highly potent oligonucleotides is the most direct strategy. Locked Nucleic Acid (LNA) represents such a direct strategy, and LNA can equally well be used for mRNA and microRNA silencing. The latest silencing concepts and mechanisms will be presented, together with the status of our LNA R&D programs.
Troels Koch, Ph.D., Vice President, Research, Santaris Pharma, Denmark
3:15
Networking Refreshment Break and Last Chance for Poster and Exhibit Viewing
3:45
Development of Decoy Oligonucleotide Drug
We have developed decoy ODN against NFkB, an essential transcription factor for inflammation and adhesion. Currently, Phase III trial to treat atopic dermatitis will be scheduled. In addition, clinical trials of decoy eluting medical device will be initiated soon. In this session, together with the present clinical situation of NFkB decoy ODN, our recent modified decoy, to overcome the short half life, low efficiency of uptake, and degradation by endocytosis, will be introduced..
Ryuichi Morishita, Ph.D., Professor, Dept. of Clinical Gene Therapy, Graduate School of Medicine, Osaka University; Founder, Board Member, AnGes MG, Inc., Japan
We have developed decoy ODN against NFkB, an essential transcription factor for inflammation and adhesion. Currently, Phase III trial to treat atopic dermatitis will be scheduled. In addition, clinical trials of decoy eluting medical device will be initiated soon. In this session, together with the present clinical situation of NFkB decoy ODN, our recent modified decoy, to overcome the short half life, low efficiency of uptake, and degradation by endocytosis, will be introduced..
Ryuichi Morishita, Ph.D., Professor, Dept. of Clinical Gene Therapy, Graduate School of Medicine, Osaka University; Founder, Board Member, AnGes MG, Inc., Japan
4:15
Clinical Development of Synthetic siRNAs for Ocular and Renal Diseases
Quark Pharmaceuticals has a robust clinical development pipeline of synthetic siRNAs that includes two siRNAs administered via intravitreal injection for ocular indications and one siRNA administered systemically for two renal indications. This talk will describe the design and manufacture of these synthetic siRNAs, and provide an overview of the clinical experience from the Phase I safety studies and an update on the status of the Phase 2 studies.
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
Quark Pharmaceuticals has a robust clinical development pipeline of synthetic siRNAs that includes two siRNAs administered via intravitreal injection for ocular indications and one siRNA administered systemically for two renal indications. This talk will describe the design and manufacture of these synthetic siRNAs, and provide an overview of the clinical experience from the Phase I safety studies and an update on the status of the Phase 2 studies.
James D. Thompson, Ph.D., Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc., USA
4:45
Development of the REG1 Anticoagulation System for Acute Coronary Syndromes
REG1 is an anticoagulation system consisting of the aptamer-based anticoagulant, RB006, and its specific control agent, RB007. The REG1 system has been evaluated in 3 phase 1 studies and a phase 2a feasibility study in PCI. This presentation will include:
REG1 is an anticoagulation system consisting of the aptamer-based anticoagulant, RB006, and its specific control agent, RB007. The REG1 system has been evaluated in 3 phase 1 studies and a phase 2a feasibility study in PCI. This presentation will include:
- Overview of the Regado drug-control agent technology
- Overview of the REG1 System
- Summary of the clinical findings to date
5:15
Close of Tuesday Sessions
Worldwide Advances in Peptides & Diagnostics
Updates on Peptide-Based Therapeutics in Preclinical and Clinical Development
8:30
Chairperson's Remarks
Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USA
Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USA
8:45
Case
Study
AL-309: A Preclinical Case Study for Peripheral NeuropathiesStudy
AL-309 is an all D-amino peptide derived from Activity-Dependent Neurotrophic Factor (ADNF). In preclinical models, AL-309 demonstrated protection of peripheral nerve damage after chemotherapy- or diabetes-induced neuropathy as well as an improvement in the pain response which develops in these models. The pharmacology, pharmacokinetics and preliminary toxicology suggest that AL-309 is an interesting drug candidate for the prevention of peripheral neuropathies.
Bruce H. Morimoto, Ph.D., Vice President, Drug Development, Allon Therapeutics Inc., Canada
9:15
Medical Applications of Peptides with Angiogenesis or Antimicrobial Properties
We have been extensively investigating medical application of antimicrobial peptides with various pharmacological activities. A review will be provided of recent developments in this field and an overview of preclinical data regarding the projects: 1) medical application of antimicrobial peptide, and 2) utilization of antimicrobial peptide with angiogenesis property as wound healing agent.
Shunsuke Sami, Ph.D., Executive Vice President, Drug Discovery Research, AnGes MG, Inc., Japan
Hideki Tomioka, Manager, Pharmaceutical Research Group, AnGes MG, Inc., Japan
We have been extensively investigating medical application of antimicrobial peptides with various pharmacological activities. A review will be provided of recent developments in this field and an overview of preclinical data regarding the projects: 1) medical application of antimicrobial peptide, and 2) utilization of antimicrobial peptide with angiogenesis property as wound healing agent.
Shunsuke Sami, Ph.D., Executive Vice President, Drug Discovery Research, AnGes MG, Inc., Japan
Hideki Tomioka, Manager, Pharmaceutical Research Group, AnGes MG, Inc., Japan
9:45
Stapled Peptides: Leveraging the a-Helix to Create a New Class of Targeted Therapeutics
Hydrocarbon "stapled" versions of the BH3-domain alpha-helices of BID and BIM, pro-apoptotic BCL-2 family members, possess potent and specific in vitro and in vivo anti-cancer activities for a broad diversity of solid and liquid tumors. AILERON is completing pre-clinical testing of BH3 Stapled Peptides for IND submission and is developing additional Stapled Peptide therapeutics for oncology, metabolic diseases, immunological disorders and viral infections.
Huw W. Nash, Ph.D., Vice President, Corporate Development, Aileron, Inc., USA
Hydrocarbon "stapled" versions of the BH3-domain alpha-helices of BID and BIM, pro-apoptotic BCL-2 family members, possess potent and specific in vitro and in vivo anti-cancer activities for a broad diversity of solid and liquid tumors. AILERON is completing pre-clinical testing of BH3 Stapled Peptides for IND submission and is developing additional Stapled Peptide therapeutics for oncology, metabolic diseases, immunological disorders and viral infections.
Huw W. Nash, Ph.D., Vice President, Corporate Development, Aileron, Inc., USA
10:15
Networking Refreshment Break with Poster and Exhibit Viewing
10:45
Case
Study
Development of Terlipressin as an Orphan Drug in the USStudy
An overview of the status of peptides as active pharmaceutical ingredients (APIs) will be presented. As a recent case study, challenges related to manufacturing and regulatory aspects of the development of Terlipressin, which is currently approved in the EU and South East Asia and pending approval as an orphan drug in the US, will be discussed.
Robert Hagopian, Director, Business Development, PolyPeptide Group, USA
11:15
Update on Hematide™, a New Peptidic ESA being Evaluated for the Treatment of Anemia
We have discovered a novel synthetic peptide-based compound (Hematide) that is a potent erythropoiesis-stimulating agent (ESA). A novel linker-spacer couples the peptide portion to PEG and prolongs its duration of action. In partnership with Takeda, Hematide is in global Phase 3 trials to establish the safety and efficacy after administration every four weeks in predialysis, dialysis, and PRCA patients.
Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USA
We have discovered a novel synthetic peptide-based compound (Hematide) that is a potent erythropoiesis-stimulating agent (ESA). A novel linker-spacer couples the peptide portion to PEG and prolongs its duration of action. In partnership with Takeda, Hematide is in global Phase 3 trials to establish the safety and efficacy after administration every four weeks in predialysis, dialysis, and PRCA patients.
Christopher P. Holmes, Ph.D., Executive Director, Chemistry, Affymax, Inc., USA
11:45
Scientific
Highlight
RaPID (Random Peptide Integrated Discovery) System under Reprogrammed Genetic CodeHighlight
RaPID (Random Peptide Integrated Discovery) System is a new emerging technology that enables the expression of non-standard peptides using the translation machinery and rapid discovery of unique peptide drug leads. Particularly, when it is coupled with a cell-free display system, referred to as RaPID display, such peptides can be selected from a library with more than 100 billions of complexity. The lecture describes recent development of this technology.
Hiroaki Suga, Ph.D., Professor, Chemical Biology and Biotechnology Lab, The University of Tokyo, Japan
12:15
Networking Luncheon with Poster and Exhibit Viewing
1:30
Chairperson's Remarks
Jesse Z. Dong, Ph.D., Senior Director, Medicinal Chemistry, Ipsen, USA
Jesse Z. Dong, Ph.D., Senior Director, Medicinal Chemistry, Ipsen, USA
1:35
GLP-1 Analogue Taspoglutide: From Discovery to Clinical Development
Taspoglutide, a human GLP-1 analogue, is currently in phase 3 clinical development for the treatment of type 2 diabetes. The peptide is designed to be stable against enzymatic degradation and compatible with sustained-release formulations. It is administrated as a novel sustained-release formulation intended to be used as once weekly subcutaneous injection. The molecule design, chemistry, clinical safety, tolerance and efficacy data will be discussed.
Raffaella Balena, M.D., Ph.D., Clinical Science Leader, F. Hoffmann-La Roche Ltd, Switzerland
Jesse Z. Dong, Ph.D., Senior Director, Medicinal Chemistry, Ipsen, USA
Taspoglutide, a human GLP-1 analogue, is currently in phase 3 clinical development for the treatment of type 2 diabetes. The peptide is designed to be stable against enzymatic degradation and compatible with sustained-release formulations. It is administrated as a novel sustained-release formulation intended to be used as once weekly subcutaneous injection. The molecule design, chemistry, clinical safety, tolerance and efficacy data will be discussed.
Raffaella Balena, M.D., Ph.D., Clinical Science Leader, F. Hoffmann-La Roche Ltd, Switzerland
Jesse Z. Dong, Ph.D., Senior Director, Medicinal Chemistry, Ipsen, USA
2:15
A Bivalent Influenza Peptide Conjugate Vaccine Based on Highly Conserved Epitopes
Developing improved influenza virus vaccines that provide broader and more durable protection is an important, international public health objective. We have developed peptide-conjugate vaccines based on the M2 and HA proteins that are highly conserved and provide protection from disease against heterologous influenza infections in preclinical studies. These data and results from an initial clinical trial will be reviewed.
John W. Shiver, Ph.D., Vice President, Worldwide Basic Research Franchise Head, Vaccines, Merck Research Laboratories, USA
Developing improved influenza virus vaccines that provide broader and more durable protection is an important, international public health objective. We have developed peptide-conjugate vaccines based on the M2 and HA proteins that are highly conserved and provide protection from disease against heterologous influenza infections in preclinical studies. These data and results from an initial clinical trial will be reviewed.
John W. Shiver, Ph.D., Vice President, Worldwide Basic Research Franchise Head, Vaccines, Merck Research Laboratories, USA
2:45
Clinical Development for Peptide-Based Therapeutic Cancer Immunotherapy Targeting Oncoantigen and Tumor Angiogenesis
Oncoantigen and tumor angiogenesis are crucial targets for therapeutic cancer immunotherapy. Basic data showed potent antitumor effect using epitope peptides derived from target-molecules. We have performed several clinical trials, including the pivotal study against pancreatic cancer and Phase II clinical trial against cholangiocellular carcinoma. Data will be demonstrated in detail and future potential for therapeutic immunotherapy will be discussed.
Takuya Tsunoda, M.D., Ph.D., Chief Operating Officer, OncoTherapy Science, Inc., Japan
Oncoantigen and tumor angiogenesis are crucial targets for therapeutic cancer immunotherapy. Basic data showed potent antitumor effect using epitope peptides derived from target-molecules. We have performed several clinical trials, including the pivotal study against pancreatic cancer and Phase II clinical trial against cholangiocellular carcinoma. Data will be demonstrated in detail and future potential for therapeutic immunotherapy will be discussed.
Takuya Tsunoda, M.D., Ph.D., Chief Operating Officer, OncoTherapy Science, Inc., Japan
3:15
Networking Refreshment Break and Last Chance for Poster and Exhibit Viewing
Updates on Diagnostics in Development
Chairperson: Peter Haima, Ph.D., Director, IVD Services, EUROGENTEC-group, USA
3:45
New SNPs Typing Method Based on Abasic Site-Containing Probe DNA and Fluorescent Ligands
A new SNPs typing method is proposed based on abasic site (AP site)-containing DNAs (AP-DNA) and fluorescent ligands. At an AP site in an AP-DNA/target DNA duplex, fluorescent ligands can recognize target nucleobases selectively. Using two ligands with different color emission, allele genotyping is demonstrated using PCR amplified products without any pre-treatment.
Norio Teramae, Ph.D., Professor, Chemistry, Tohoku University, Japan
A new SNPs typing method is proposed based on abasic site (AP site)-containing DNAs (AP-DNA) and fluorescent ligands. At an AP site in an AP-DNA/target DNA duplex, fluorescent ligands can recognize target nucleobases selectively. Using two ligands with different color emission, allele genotyping is demonstrated using PCR amplified products without any pre-treatment.
Norio Teramae, Ph.D., Professor, Chemistry, Tohoku University, Japan
4:15
Development of a New GMP TAQ Polymerase for Improved PCR Performance
PCR is the method of choice for many diagnostic applications. A new hot-start method was developed that improves the efficiency, specificity, yield and sensitivity of the PCR reaction. HOT Diamond TAQ™ is manufactured according to a GMP-Pharma process; resulting in an exhaustively characterized, lot-to-lot reproducible enzyme, with extremely low residual DNA content.
Peter Haima, Ph.D., Director, IVD Services, EUROGENTEC-group, USA
PCR is the method of choice for many diagnostic applications. A new hot-start method was developed that improves the efficiency, specificity, yield and sensitivity of the PCR reaction. HOT Diamond TAQ™ is manufactured according to a GMP-Pharma process; resulting in an exhaustively characterized, lot-to-lot reproducible enzyme, with extremely low residual DNA content.
Peter Haima, Ph.D., Director, IVD Services, EUROGENTEC-group, USA
4:45
Modified dNTPs for improved PCR Performance: An Innovative Use of Nucleoside Protecting Groups
PCR is widely applied in high specificity detection assays. This presentation describes an approach in which the dNTPs, an essential PCR component, are modified with thermolabile dNTP protecting groups for application to Hot Start activation schemes in PCR. Studies describe the development of this technology, including the proof of principle experiments that identified the optimal temperature-sensitive 3'-nucleoside protecting group.
Natasha Paul, Ph.D., Senior Staff Scientist, R&D, TriLink BioTechnologies, Inc., USA
PCR is widely applied in high specificity detection assays. This presentation describes an approach in which the dNTPs, an essential PCR component, are modified with thermolabile dNTP protecting groups for application to Hot Start activation schemes in PCR. Studies describe the development of this technology, including the proof of principle experiments that identified the optimal temperature-sensitive 3'-nucleoside protecting group.
Natasha Paul, Ph.D., Senior Staff Scientist, R&D, TriLink BioTechnologies, Inc., USA
5:15
Close of Tuesday Sessions
6:00
Networking Dinner in Tokyo
Join fellow attendees and speakers for a memorable evening out in Tokyo. Space is limited and an additional fee applies. Please check box on registration page to join the dinner.
Join fellow attendees and speakers for a memorable evening out in Tokyo. Space is limited and an additional fee applies. Please check box on registration page to join the dinner.
Wednesday, February 24, 2010
Tutorials | Main Conference: Monday | Main Conference: Tuesday | Main Conference: Wednesday
Delivery Challenges and Solutions for Oligos and Peptide Therapeutics
8:30
Chairperson's Remarks
Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc., USA
Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc., USA
8:45
Chemical Strategies for Achieving Systemic Delivery of siRNAs
An update will be presented on our progress toward systemic delivery of small interfering RNAs (siRNAs) using various chemical approaches.
Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc., USA
An update will be presented on our progress toward systemic delivery of small interfering RNAs (siRNAs) using various chemical approaches.
Muthiah Manoharan, Ph.D., Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc., USA
9:15
RNAi Chemical and Structural Optimization for Therapeutic Development
Nucleotide chemistry is being used to improve therapeutic oligonucleotide properties such as potency, stability and specificity. Moreover, we have developed a new class of modified RNAi compound, self-delivering rxRNA (sd-rxRNA™), that is characterized by spontaneous cellular and tissue uptake. This translates to dramatically enhanced in vivo cellular uptake locally following direct administration and to liver following intravenous administration. Kinetics and mechanism of cellular uptake as well as preclinical development of sd-rxRNA™ will be discussed.
Pamela A. Pavco, Ph.D., Vice President, Pharmaceutical Development, RXi Pharmaceuticals, USA
Nucleotide chemistry is being used to improve therapeutic oligonucleotide properties such as potency, stability and specificity. Moreover, we have developed a new class of modified RNAi compound, self-delivering rxRNA (sd-rxRNA™), that is characterized by spontaneous cellular and tissue uptake. This translates to dramatically enhanced in vivo cellular uptake locally following direct administration and to liver following intravenous administration. Kinetics and mechanism of cellular uptake as well as preclinical development of sd-rxRNA™ will be discussed.
Pamela A. Pavco, Ph.D., Vice President, Pharmaceutical Development, RXi Pharmaceuticals, USA
9:45
Planning for Success: Scale Up and Manufacturing of Stable Nucleic Acid Lipid Particles for Phase I and Beyond
Stable Nucleic Acid Lipid Particle (SNALP) products containing siRNA payloads are currently being evaluated in several clinical and preclinical studies. This presentation will describe the scale up, manufacturing and characterization of these products to enable initial clinical trials and will also provide an overview of strategies to further develop the platform for late-stage clinical trials and, ultimately, commercialization.
Peter Lutwyche, Ph.D., Vice President, Pharmaceutical Development, Tekmira Pharmaceuticals Corporation, Canada
Stable Nucleic Acid Lipid Particle (SNALP) products containing siRNA payloads are currently being evaluated in several clinical and preclinical studies. This presentation will describe the scale up, manufacturing and characterization of these products to enable initial clinical trials and will also provide an overview of strategies to further develop the platform for late-stage clinical trials and, ultimately, commercialization.
Peter Lutwyche, Ph.D., Vice President, Pharmaceutical Development, Tekmira Pharmaceuticals Corporation, Canada
10:15
Networking Refreshment Break
10:45
Mimicking Endogenous Peptide Secretion Using Dry Powder Inhalation
Technosphere® dry powder formulations in combination with a novel inhalation device comprise a versatile peptide delivery platform that mimics the pharmacokinetics of intra-arterial injection and is characterized by very rapid systemic drug absorption. This physiologic delivery profile provides advantages over the same drugs administered by injection and has applicability over a broad range of therapeutics agents including insulin, GLP-1, PYY, OXM, and PTH.
Andrea Leone-Bay, Ph.D., Vice President, Pharmaceutical R&D, MannKind Corporation, USA
Technosphere® dry powder formulations in combination with a novel inhalation device comprise a versatile peptide delivery platform that mimics the pharmacokinetics of intra-arterial injection and is characterized by very rapid systemic drug absorption. This physiologic delivery profile provides advantages over the same drugs administered by injection and has applicability over a broad range of therapeutics agents including insulin, GLP-1, PYY, OXM, and PTH.
Andrea Leone-Bay, Ph.D., Vice President, Pharmaceutical R&D, MannKind Corporation, USA
Intellectual Property Panel Discussion
11:15
Market Landscape: Demystifying Intellectual Property for Oligos and Peptides around the World
Panelists will discuss the most pressing issues in intellectual property for oligos and peptides in an audience interactive discussion. Topics to be discussed:
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, USA
Panelists:
Steven Lee Highlander, Partner, Fulbright and Jaworski L.L.P., USA
Antonio Maschio, Ph.D., Partner, Edwards Angell Palmer & Dodge UK LLP, United Kingdom
Kathleen M. Williams, Ph.D., J.D., Partner, Intellectual Property, Edwards Angell Palmer & Dodge LLP, USA
Panelists will discuss the most pressing issues in intellectual property for oligos and peptides in an audience interactive discussion. Topics to be discussed:
- Challenges involved with filing for patents and avoiding intellectual property disputes.
- Specific examples/case studies on how patent disputes are being dealt with in the genomics area.
- Peptides vs. oligos, how does IP landscape differ? Is there overlap?
Bob D. Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals, USA
Panelists:
Steven Lee Highlander, Partner, Fulbright and Jaworski L.L.P., USA
Antonio Maschio, Ph.D., Partner, Edwards Angell Palmer & Dodge UK LLP, United Kingdom
Kathleen M. Williams, Ph.D., J.D., Partner, Intellectual Property, Edwards Angell Palmer & Dodge LLP, USA
Manufacturing and Analytical Development for Peptides and Oligos
Session Sponsor:
8:30
Co-Chairpersons' Remarks
Steven J. Prestrelski, Ph.D., M.B.A., Vice President, Pharmaceutical R&D, Amylin Pharmaceuticals, Inc., USA
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
Steven J. Prestrelski, Ph.D., M.B.A., Vice President, Pharmaceutical R&D, Amylin Pharmaceuticals, Inc., USA
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
8:45
Case
Study
The Oligonucleotide Development JourneyStudy
A discussion of oligonucleotide development strategies and requirements through clinical phases, including the application of targeted process development, control of process changes and development of design history, raw materials sourcing tactics and the development of process controls and specifications. The presentation will include key examples to illustrate the areas covered.
Emma Wright, D.Phil., Director of Process Development, Avecia OligoMedicines, USA
9:15
The Challenges of Supply Chain Management with an Emerging Technology
The speaker will explore the numerous issues related to managing an uninterrupted supply of clinical material for a development company expanding the scope of the fundamental technology. Some of the topics will be: changing raw material requirements; limited visibility for forecasted production; flexible inventory management; capacity planning for an evolving product portfolio.
David Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, Inc., USA
The speaker will explore the numerous issues related to managing an uninterrupted supply of clinical material for a development company expanding the scope of the fundamental technology. Some of the topics will be: changing raw material requirements; limited visibility for forecasted production; flexible inventory management; capacity planning for an evolving product portfolio.
David Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, Inc., USA
9:45
Case
Study
Cost-Effective Synthesis of siRNA and Aptamers Using CEM as the 2'-hydroxyl Protecting Group: From Bulk-Scale CEM Amidites to OligoribonucleotidesStudy
We have manufactured all four 2'-O-(2-cyanoethoxymethyl) (2'-O-CEM) amidites on a multi-kilogram scale and successfully used them for the synthesis of RNA oligonucleotides. Data will be presented demonstrating that CEM chemistry significantly improves the efficiency of manufacturing of RNA oligonucleotides with or without modified nucleotides by comparison with TBDMS chemistry. The syntheses of 21mer to 58mer RNA and modified aptamers from micrograms to grams for basic research and preclinical development have been accomplished.
Kyeong Eun Jung, Ph.D., Director, Research Center, Samchully Pharm. Co. Ltd., Korea
10:15
Networking Refreshment Break
10:45
Exenatide Once Weekly: A New Therapy for Type 2 Diabetes
Exenatide Once Weekly (EQW) is a long-acting formulation of exenatide, the active ingredient in Byetta® (exenatide injection) based on the Medisorb technology from Alkermes, Inc. EQW has the potential to be a diabetes therapeutic with superior efficacy, tolerability and patient convenience. This presentation will provide an overview of aspects of drug development including formulation development, scale-up and commercial manufacturing and results of clinical studies.
Steven J. Prestrelski, Ph.D., M.B.A., Vice President, Pharmaceutical R&D, Amylin Pharmaceuticals, Inc., USA
Exenatide Once Weekly (EQW) is a long-acting formulation of exenatide, the active ingredient in Byetta® (exenatide injection) based on the Medisorb technology from Alkermes, Inc. EQW has the potential to be a diabetes therapeutic with superior efficacy, tolerability and patient convenience. This presentation will provide an overview of aspects of drug development including formulation development, scale-up and commercial manufacturing and results of clinical studies.
Steven J. Prestrelski, Ph.D., M.B.A., Vice President, Pharmaceutical R&D, Amylin Pharmaceuticals, Inc., USA
11:15
DSP Challenges in Peptides: Getting the Right Things Done
HPLC typically accounts for 20-50% of peptide production costs, forcing manufacturers to find trade-offs between yield and productivity, while keeping quality, robustness and flexibility. Several case studies will be discussed in which Lonza has tackled this challenge by introducing new process development and optimization tools, including impurity tracking, DoE techniques, and numerical modeling. Strategies for the integration of these tools into QbD methods will also be offered.
Alessandro Butté, Ph.D., Group Leader Peptides R&D, Lonza AG, Switzerland
HPLC typically accounts for 20-50% of peptide production costs, forcing manufacturers to find trade-offs between yield and productivity, while keeping quality, robustness and flexibility. Several case studies will be discussed in which Lonza has tackled this challenge by introducing new process development and optimization tools, including impurity tracking, DoE techniques, and numerical modeling. Strategies for the integration of these tools into QbD methods will also be offered.
Alessandro Butté, Ph.D., Group Leader Peptides R&D, Lonza AG, Switzerland
11:45
Applying Manufacturing Sciences to Increase Productivity and Profitability
Process capability is adversely affected by system variation, which, in turn, renders the process to be irreproducible and products to be out of specifications, and eventually increases manufacturing costs. By using Six Sigma approach to systematically identify and to eliminate the root cause of variability in liquid chromatography processes, reduced manufacturing cost along with improved product quality can be realized.
Michael Li, Ph.D., Manager, Process Sciences, Asahi Kasei TechniKrom, USA
Process capability is adversely affected by system variation, which, in turn, renders the process to be irreproducible and products to be out of specifications, and eventually increases manufacturing costs. By using Six Sigma approach to systematically identify and to eliminate the root cause of variability in liquid chromatography processes, reduced manufacturing cost along with improved product quality can be realized.
Michael Li, Ph.D., Manager, Process Sciences, Asahi Kasei TechniKrom, USA
12:15
Networking Luncheon
Main Conference - Plenary Session
Investment and Licensing Opportunties
1:30
Innovation Showcase
Four emerging drug development and technology companies will give 15-minute updates on their business plan, technology, and partnering objectives. For more information on how to present in the Innovation Showcase, please see guidelines on the website or contact Ellen C. King of IBC Life Sciences at eking@ibcusa.com
Co-Chairs:
Gary Carter, Business Development Manager, Nucleic Acids Solutions Division, Agilent Technologies Inc., USA
David Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, Inc., USA
Four emerging drug development and technology companies will give 15-minute updates on their business plan, technology, and partnering objectives. For more information on how to present in the Innovation Showcase, please see guidelines on the website or contact Ellen C. King of IBC Life Sciences at eking@ibcusa.com
Co-Chairs:
Gary Carter, Business Development Manager, Nucleic Acids Solutions Division, Agilent Technologies Inc., USA
David Konys, Vice President, Manufacturing Operations, Alnylam Pharmaceuticals, Inc., USA
2:30
Networking Refreshment Break
Regulatory Updates: Strategies for Navigating the Approval Pathway
3:00
Chairperson's Remarks
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
3:15
Regulatory Considerations in the Development of RNAi Therapeutics
Regulatory issues in the development of small interfering RNA (siRNA) as therapeutics will be discussed from the perspective of nonclinical pharmacology and toxicology as well as chemistry, manufacturing and controls (CMC). Experience with FDA as well as with European and Canadian authorities in regulatory filings to enable Phase 1 and Phase 2 clinical trials will be shared.
Saraswathy (Sara) V. Nochur, Ph.D., Vice President, Regulatory Affairs, Alnylam Pharmaceuticals, Inc., USA
Regulatory issues in the development of small interfering RNA (siRNA) as therapeutics will be discussed from the perspective of nonclinical pharmacology and toxicology as well as chemistry, manufacturing and controls (CMC). Experience with FDA as well as with European and Canadian authorities in regulatory filings to enable Phase 1 and Phase 2 clinical trials will be shared.
Saraswathy (Sara) V. Nochur, Ph.D., Vice President, Regulatory Affairs, Alnylam Pharmaceuticals, Inc., USA
3:45
Considerations for the CMC Development of Oligonucleotides in Complex Delivery Systems
Recent advances in oligonucleotide-based therapeutics include the development of complex formulations for targeted delivery to various sites. Utilisation of these novel systems for the delivery of complex oligonucleotides presents exceptional challenges for characterisation and control. This presentation will provide strategies for addressing the regulatory expectations for assuring quality and performance of these delivery systems in support of preclinical and clinical development.
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
Recent advances in oligonucleotide-based therapeutics include the development of complex formulations for targeted delivery to various sites. Utilisation of these novel systems for the delivery of complex oligonucleotides presents exceptional challenges for characterisation and control. This presentation will provide strategies for addressing the regulatory expectations for assuring quality and performance of these delivery systems in support of preclinical and clinical development.
G. Susan Srivatsa, Ph.D., President, ElixinPharma, USA
4:15
Development of Peptide-Based Cancer Vaccines up to Phase II - Regulatory Challenges and Opportunities
IMA901 for the treatment of renal cell carcinoma and IMA910 for the treatment of colorectal cancer are two product candidates currently developed in two ongoing phase II cancer vaccine trials in Europe. Both immunotherapy candidates consist of 10 and 13 HLA-restricted tumor-associated peptides (TUMAPs), respectively. We will report on regulatory challenges and opportunities in this "multi-peptide multi-national multi-center" clinical development setting.
Peter Lewandrowski, Ph.D., Director and Head of CMC, immatics biotechnologies GmbH, Germany
IMA901 for the treatment of renal cell carcinoma and IMA910 for the treatment of colorectal cancer are two product candidates currently developed in two ongoing phase II cancer vaccine trials in Europe. Both immunotherapy candidates consist of 10 and 13 HLA-restricted tumor-associated peptides (TUMAPs), respectively. We will report on regulatory challenges and opportunities in this "multi-peptide multi-national multi-center" clinical development setting.
Peter Lewandrowski, Ph.D., Director and Head of CMC, immatics biotechnologies GmbH, Germany
4:45
Close of AsiaTIDES®