What`s Inside... - IBC Life Sciences

Transcription

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