TIDES: Oligonucleotide & Peptide...

TIDES: Oligonucleotide &Peptide TherapeuticsApril 30 - May 3, 2017Manchester Grand Hyatt San DiegoSan Diego, CA

Table of ContentsGeneral Information . . . . . . . . . . . . . . .4Agenda at a Glance . . . . . . . . . . . . . . .5Sponsors . . . . . . . . . . . . . . . . . . . . . . . .6Venue Floor Plan . . . . . . . . . . . . . . . . . .7TIDES Advisory Board . . . . . . . . . . . 28

Exhibition InformationPassport Contest . . . . . . . . . . . . . . . . .8Poster Presentations . . . . . . . . . 18, 20Exhibitor List and Floor Plan . . 22, 23Featured Exhibitors . . . . . . . . . . 24, 26

Conference AgendaPre-Conference Workshops . . . 10, 11Monday, May 1 . . . . . . . . . . . . . . . . . 12Tuesday, May 2 . . . . . . . . . . . . . . .14-15Wednesday, May 3 . . . . . . . . . . . .16-17

Event Partner:

Special Thanks to:

Event Guide

4 www.TIDESEvent.com

Admission Admission is limited to persons actively engaged in oligonucleotide and peptide R&D and manufacturing . KNect365 has taken every effort to prohibit admissions to persons not engaged in this area . Children under 18 are not permitted in the Exhibit Hall under any circumstances . Badges and badge holders must be worn at all times while attending the event . Conference badges are non-transferable and lost badges will not be replaced without payment of the full conference registration fee . No exceptions .

Registration Desk Open Sunday Pre-Conference Workshops (third level) 7:15 am - 3:30 pm

Main Registration (lobby level) 4:00 pm - 6:00 pm

Monday Main Registration – 7:45 am - 6:30 pm

Tuesday Main Registration – 7:30 am - 6:30 pm

Wednesday Main Registration (lobby level) 7:00 am - 11:00 am Satellite Registration (second level) 11:00 am-3:15 pm

Exhibit Hall& Poster Viewing Hours Monday 12:30 pm - 6:30 pm

Tuesday 10:00 am - 6:30 pm

Wednesday 7:00 am - 10:55 am

Video/Photo Consent PolicyThis conference is being filmed. When you enter this event, you may be in areas that are being filmed by video or photography.

By entering the event premises, you consent to the filming, display, release, publication, exhibition or reproduction of your image and anything spoken by you to be used for news, Web casts, promotional purposes, telecasts, advertising, inclusion on Web sites, or any other purposes by KNect365 Life Sciences . You release KNect365 Life Sciences and its respective affiliates, employees and representatives, and each and all persons involved from any liability connected with such filming. You have been fully informed of your consent and release .

Program Changes KNect365 does everything in its power to ensure there are no changes to the published program but sometimes circumstances that are beyond our control (i .e . speaker cancellations, speaker changes, etc .) do arise . In the event this does occur, every effort is made to find a suitable replacement. Please Note: The listings and contents of this book are proprietary and cannot be reproduced in part or in whole without permission . Every effort has been made to ensure the accuracy of this Guide . However, KNect365 Life Sciences cannot be held responsible for errors or omissions . Product names may be trademarks or registered trademarks of their companies .

TIDES AppAll registered attendees receive access to the TIDES app, which allows you to view the full attendee list, schedule meetings and create a customized agenda . You should have received an email with your log in details already . If you need assistance, please contact Appsupport@knect365 .com or visit the App help desk onsite by the registration area .

Assistance and Special NeedsOrganizers of the TIDES Conference fully support the Americans with Disabilities Act . If you require assistance of any kind, please inquire at Attendee Registration .

Lost and FoundItems found can be dropped off at Attendee Registration . Items not claimed by the conclusion of the conference will be turned over to building security .

Passport ContestAll registered attendees receive access to the TIDES app, which allows you to view the full attendee list, schedule meetings, win prizes in the passport contest and more . Download the app on your iOs/Android mobile device by searching for ‘TIDES US 2017’ in the app store . All attendees were emailed their log in details before the event . Please visit our team at the registration desk if you need assistance .

General Information

WiFi Network: TIDESPassword: TIDES2017

Join KNect365 Life Sciences’ LinkedIn group and becoming a member of our LinkedIn groups: Oligonucleotide and Peptide Development Professionals

Follow us on Twitter @TIDES365 and tweet using hash tag #TIDES365

Follow us on Twitter @TIDES365 and tweet using hash tag #TIDES365 5

Agenda At-A-Glance

Sunday, April 30, 2017 • Pre-Conference Workshops

8:00 am - 12:00 pm Workshop #1:

Analytical and Purification Strategies for Peptide

Manufacturing (Hillcrest CD)

Workshop #2: Emerging Technologies in Oligonucleotide Based Therapeutics:

Challenges for Manufacturing and Controls of Novel Chemistries (Cortez Hill ABC)

Workshop #3: an Introduction to CRISPR and Genome Editing Applications

(Golden Hill)

Workshop #4: Peptide Manufacturing Risk Assessment at CMOS, A Multi-Product

Environment (Torrey Hills)

1:00 pm - 5:00 pm

Workshop #5: Quality Considerations for

Oligonucleotide Drug Products (Cortez Hill ABC)

Workshop #6: An Introduction to messengerRNA (mRNA)

Therapeutics (Golden Hill)

Workshop #7: Sensitivity Versus Specificity of Modern Bioanalytical Assays and the

Considerations of ADME and PK/PD Studies of Oligonucleotides

(Solana Beach)

Workshop #8: Enhancing Pharmaceutical

Properties Of Peptides (Torrey Hills)

Monday, May 1, 2017 • Main Conference Exhibit Hall & Poster Viewing Hours: 12:30 pm-6:30 pm

8:55 am - 12:30 pm

KEYNOTE/PLENARY SESSION: (Seaport ABC) Edward Kaye, M.D., President, CEO and Chief Medical Officer, Sarepta Therapeutics Marvin Caruthers, Ph.D., Distinguished Professor, Chemistry and Biochemistry, University of Colorado Bradley L. Pentelute, Ph.D., Professor, Chemistry, Massachusetts Institute of Technology

12:30 pm - 1:45 pm Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break Co-Sponsored by Ajinomoto

1:45 pm - 5:00 pm

KEYNOTE/PLENARY SESSION: (Seaport ABC) Michael E. Kopach, Ph.D., Research Advisor, Eli Lilly and Company James R. Heath, Ph.D., Elizabeth W . Gilloon Professor of Chemistry, California Institute of Technology Richard S. Geary, Ph.D., Senior Vice President, Development, Ionis Pharmaceuticals, Inc. Mayra Reyes-Armour, Nusinersen Asset Leader, Pharmaceutical Operations, Biogen

5:00 pm - 6:30 pm

Networking Reception in Poster and Exhibit Hall Sponsored by RiboBio (Grand Hall)

Tuesday, May 2, 2017 • Main Conference Exhibit Hall & Poster Viewing Hours: 10:00 am – 6:30 pm7:45 am - 8:15 am Breakfast Presentations Sponsored by: Polymun Scientific GmbH (Torrey Hills) and Nagase (Golden Hill AB)

Track 1: Oligonucleotide Chemistry Manufacturing and Controls

Track 2: Oligonucleotide Discovery, Preclinical and Clinical

Track 3: Peptide Chemistry Manufacturing and Controls

Track 4: Peptide Discovery, Preclinical and Clinical

8:25 am - 10:00 am

Seaport B Seaport C Seaport A Cortez Hill

Quality of Oligonucleotide Starting Materials (Starts 7:55 am) Oligonucleotide Discovery

and PreclinicalCMC, Quality and Regulatory

Aspects of Peptide APIsPeptide Targets, Peptide Discovery

and SynthesisGalNAc Conjugates: From Starting Materials to Manufacturing

Track 1: Oligonucleotide Chemistry Manufacturing

and Controls

Track 2: Oligonucleotide Discovery, Preclinical

and Clinical

Track 5: mRNA Therapeutics

Track 3: Peptide Chemistry Manufacturing

and Controls

Track 4: Peptide Discovery, Preclinical

and ClinicalSeaport B Seaport C Golden Hill Seaport A Cortez Hill

10:45 am - 12:45 pm

GalNAc Conjugates: From Starting Materials to

Manufacturing (continued)

Oligonucleotide Discovery and Preclinical (continued)

Discovery and Development of mRNA Therapeutics

and Vaccines

CMC, Quality and Regulatory Aspects of Peptide APIs

(continued)

Peptidomimetic and Peptidic Macrocycles: Expanding the

Peptide Drug Modality12:45 pm -

1:55 pm Networking Luncheon in Poster and Exhibit Hall (Grand Hall)

1:55 pm -5:30 pm

Innovative Processes and Synthesis Strategies for

Oligonucleotides

Current Issues in the Nonclinical Development of

Oligonucleotide TherapeuticsmRNA Delivery Strategies

Peptide CMC Case Studies and Strategies

Peptides in the Clinic: Lessons LearnedmRNA Bioanalytical, CMC

and Manufacturing (shared session)

Oligonucleotide Delivery Strategies

mRNA Bioanalytical, CMC and Manufacturing

(shared session in Seaport B)5:30 pm - 6:30 pm Networking Reception in Poster and Exhibit Hall (OPEN TO ALL ATTENDEES) Sponsored by Bachem (Grand Hall)

Wednesday, May 3, 2017 • Main Conference Exhibit Hall & Poster Viewing Hours: 7:00 am – 10:55 am7:00 am - 8:25 am Early Morning Exhibit Viewing and Breakfast in the Poster & Exhibit Hall

8:25 am - 10:00 am PLENARY SESSION: CRISPR and Genome Editing Applications (Harbor G-H)

Track 1: Oligonucleotide Chemistry Manufacturing and Controls




Harbor G Harbor H Harbor E Harbor D

10:55 am - 12:30 pm

Analytical Strategies for Oligonucleotides

CRISPR and Genome Editing Applications Analytical Strategies to Enable

Peptide DevelopmentPeptide Delivery and Formulation

StrategiesPreclinical and Clinical Development of Oligonucleotides

12:30 pm - 1:40 pm Lunch on Your Own

1:40 pm - 3:45 pm

Validation and Regulatory Strategies for Oligonucleotides

Preclinical and Clinical Development of Oligonucleotides Peptide Vaccines and Peptide Immunotherapy (Harbor E)


Event Partner:

Platinum Sponsor:

Gold Sponsors:

Silver Sponsors:

Bronze Sponsors:

Exhibition Hall Grand Opening Sponsor:

Breakfast Presentation Sponsors:

Reception Co-Sponsors:

Refreshment Break Sponsors:

Registration Sponsor:

Lanyard Sponsor:

Tote Bag Sponsor:

Brewery Tour Sponsors:

Media Partners:

Thank You to Our Sponsors


Venue Floor Plan

FRONT DESK

MAIN ENTRANCE

Seaport (Monday -Tuesday)Monday Keynote/Plenary Sessions (ABC)TRACK 1: Oligonucleotide Chemistry Manufacturing and Controls (B)TRACK 2: Oligonucleotide Discovery, Preclinical and Clinical (C)TRACK 3: Peptide Chemistry Manufacturing and Controls (A)

A

BD

C

F

G

H

Second Level

D

AB

C

Third Level

Lobby Level

Hillcrest Workshop 1

Grand HallPoster & Exhibit Hall

Solana BeachWorkshop 7

REGISTRATION

Cortez Hill Workshops 2 & 5 TRACK 4: Peptide Discovery, Preclinical and Clinical (Tuesday)

AB

CTorrey HillsWorkshops 4 & 8

Golden HillWorkshops 3 & 6TRACK 5: mRNA Therapeutics Track

AB

AB

DG

EH

Harbor (Wednesday)Wednesday Keynote/Plenary Sessions (GH)TRACK 1: Oligonucleotide Chemistry Manufacturing and Controls (G)TRACK 2: Oligonucleotide Discovery, Preclinical and Clinical (H)TRACK 3: Peptide Chemistry Manufacturing and Controls (E)TRACK 4: Peptide Discovery, Preclinical and Clinical (D)

AB


Download the ‘TIDES US 2017’ mobile app to participate in the Passport Contest! Use the QR code scanner in the app to collect points which will help you win prizes at the prize drawing at the end of the event . Make sure to visit all of our Passport companies in the exhibit hall and track your progress on our Leaderboard next to our TIDES Sales office in booth #122.

Prizes!Winners will be announced at 10:30 on Wednesday at booth #122 . MUST BE PRESENT TO WIN

• Gift Cards with Values up to $250

• Electronics (tablets, speakers)

• Top Shelf Alcohol

• AND MORE!

How to play:• Download the ‘TIDES US 2017’ App on your iOs/Android mobile

device

• Scan QR codes around the event and on other attendees’ badges to earn POINTS

• Keep track of your score on the leaderboard outside booth #122 or on your mobile app

• The top attendees on the leaderboard will be entered to win one of nearly 20 prizes provided by our Passport Companies!

• Winners will be announced at 10:30am on Wednesday at booth #122 – MUST BE PRESENT TO WIN

• Note: Exhibitors are not eligible to win prizes

1. Scan QR Codes at Participating companies (200 Points each!)

ALMAC. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 702

Asahi Kasei Bioprocess America Inc. . . . . . . . . . 102

Bachem . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 203

Biotage. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606

BioTechLogic . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 407

CEM . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417

ChemGenes. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 410

Corden Pharma. . . . . . . . . . . . . . . . . . . . . . . . . . . . 302

Nagase & Co. Ltd.. . . . . . . . . . . . . . . . . . . . . . . . . . 419

Polymun Scientific . . . . . . . . . . . . . . . . . . . . . . . . . 219

Pyramid Laboratories . . . . . . . . . . . . . . . . . . . . . . 602

QPS Holdings LLC . . . . . . . . . . . . . . . . . . . . . . . . . 307

ST Pharm. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 317

Sussex Research . . . . . . . . . . . . . . . . . . . . . . . . . . 610

Tosoh Bioscience . . . . . . . . . . . . . . . . . . . . . . . . . . 416

United States Pharmacopeia (USP). . . . . . . . . . . 110

2. Scan QR codes at locations around the event! (100 Points each)• Event Registration Desk

• Outside the Keynote/Plenary Room (Seaport ABC)

• Join us Sunday night at the Brewery Tour (sponsored by Thermo Fisher and Bachem) and find a QR code at the bar!

• Check out the Leaderboard next to booth #122 and

scan a code located there!

• Look for a QR code in the Poster area in the Exhibit Hall

3. Update your attendee profile in the mobile app! (50 Points)

4. Tweet using the Event Hashtag! (25 Points each tweet!)Follow us on Twitter @TIDES365 and tweet using hash tag #TIDES365

5. Networking with other attendees! (20 points each)As you meet and networking with your colleagues at the event, don’t forget to scan the QR code on their badge to earn points! No limit on the number of other attendees you can scan .

TIDES PASSPORT CONTEST – PARTICIPATE AND WIN PRIZES!Five Ways to Earn Points and Win!


Pre-Conference Symposia • Sunday, April 30, 2017

Workshop #1: ANALYTICAL AND PURIFICATION STRATEGIES FOR PEPTIDE MANUFACTURING

Full Day Workshop • 8:00am-5:00pm

Hillcrest CDWorkshop Leaders:

Marc Jacob, Ph .D ., Product Manager, Chiral and Preparative Chromatography, PhenomenexTrishul Shah, Associate Director Business Development, PolyPeptide Laboratories Inc .

8:00 Workshop IntroductionMarc Jacob, Ph .D ., Product Manager, Chiral and Preparative Chromatography, PhenomenexTrishul Shah, Associate Director Business Development, PolyPeptide Laboratories Inc .

ANALYTICAL 8:30 A Holistic Quality Control Strategy for Peptide APIs

Tobias Hauck, Ph .D ., Vice President Quality Control, Bachem AG, Switzerland

9:15 NMR Spectroscopy Applied to Peptides and Peptide Impurities – Case StudiesJohan Evenas, Ph.D., Chief Executive Officer, Read Glead Discovery, Sweden

9:45 Refreshment Break 10:00 General Overview of Peptide Analysis by Mass Spectrometry

Jiri Snopek, Ph .D ., Mass Spectrometry Lab Manager, Department of Analytical Development, PolyPeptide Laboratories

10:30 MS Peak Homogeneity: The Use of Mass Spectrometry as a Tool for LC Method Specificity Sumukh Ray, Manager, Method Development Group, Bachem Americas Inc .

11:00 Panel Discussion 12:00 Lunch on your own

PURIFICATION 1:30 High Throughput Purification Process Development of

Synthetic Peptides using a Quality by Design ApproachPer Möller, Purification Scientist, Process Development, PolyPeptide Group, Sweden

2:15 Expanding the Toolbox of Peptide PurificationSteven McIntyre, Senior Team Leader, Almac Group Ltd ., United Kingdom

2:45 Refreshment Break 3:00 Peptide Purification: Bridging the Gap Between Discovery

and DevelopmentRegina Black, Senior Research Investigator II, Chemical and Synthetic Development, Bristol Myers-Squibb

3:30 Downstream Purification Peptide Case Study: The Fuzeon StoryBrad DeHoff, Ph .D ., Director, R&D at CordenPharma GmbH

4:00 Panel Discussion 5:00 Close of Workshop

Cortez Hill ABC Workshop Leaders: G . Susan Srivatsa, Ph .D ., President, ElixinPharma

Fran Wincott, Ph .D ., Wincott and Associates 8:00 Introductory Remarks

G . Susan Srivatsa, Ph .D ., President, ElixinPharmaFran Wincott, Ph .D ., Wincott & Associates, LLC

8:20 Unlocked Nucleomonomer Agent (UNA) Oligomer™ System, Challenges and OpportunitiesNancy Fuselli, Director, Drug Development, Arcturus Therapeutics

9:00 Unique Analytical Challenges Associated with the CMC Development of mRNA Therapeutics Kristian Link, Ph .D ., Senior Manager, Analytical Development, Moderna Therapeutics

9:40 Refreshment Break 10:10 Manufacturing Innovation through Partnership

Eduardo Paredes, Ph .D ., Manager, Oligonucleotides Process Development, Nitto Denko Avecia

10:50 Practical, Quality and Regulatory Considerations to Manufacture API for Phase 1 Clinical StudiesKevin Fettes, Ph .D ., Principal and Founder, FTS Pharma Consulting, LLC


Workshop #2: EMERGING TECHNOLOGIES IN OLIGONUCLEOTIDE BASED THERAPEUTICS: CHALLENGES FOR

MANUFACTURING AND CONTROLS OF NOVEL CHEMISTRIESMorning Half-Day Workshop • 8:00am-12:00pm

Workshop #3: AN INTRODUCTION TO CRISPR AND GENOME EDITING APPLICATIONS

Morning Half-Day Workshop • 8:00am-12:00pm

Golden HillWorkshop Leader: Cecilia Fernández, Ph .D ., Director, Platform Development, Editas Medicine

8:00 Workshop Moderator's Welcome and Opening RemarksCecilia Fernández, Ph .D ., Director, Platform Development, Editas Medicine

8:15 CRISPR-based Genome Editing: Considerations for Therapeutic ApplicationsCecilia Fernández, Ph .D ., Director, Platform Development, Editas Medicine

8:45 Synthetic sgRNaA Enables Highly Efficient and Consistent CRISPR Editing of Cells for Therapeutic ApplicationsKevin Holden, Ph .D ., Head of Synthetic Biology, Synthego

9:15 Tuning the CRISPR Code: Chemically Modified RNAs Enhance CRISPR Genome Editing in Human Primary CellsAyal Hendel, Ph .D ., Principal investigator, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Israel

9:45 Refreshment Break 10:15 Applying Agilent’s CRISPR/Cas Technology and

Services to the Current Market with Emphasis on gRNA Production and Analysis Joe Guiles, Ph .D ., Head of Development, Agilent Technologies, Inc . Blake Unterreiner, Director, Business Development & Customer Relations, Nucleic Acid Solutions Division, Agilent Technologies

10:45 Delivering Genome Editing: Strategies from RNA Therapeutics Muthiah (Mano) Manoharan, Ph .D ., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals


Torrey HillsWorkshop Leaders: Gary Musso, Ph .D ., President, Musso and Associates LLC

Trishul Shah, Associate Director Business Development, PolyPeptide Laboratories Inc .

8:00 Workshop Opening Remarks

8:15 Safe Handling of Peptides: From Oligonucleotides to Peptide Hormones - Quality and Worker Safety Requirements in Multi-Purpose FacilitiesAllan Ader, Ph .D ., Managing Director, SafeBridge Consultants Inc .

8:45 Manufacturing Risk Assessment at API CMOs: How to Reduce Cross Contamination in a Multi-Product Environment Robert Geiger, Ph .D ., Vice President of Quality, AmbioPharm, Inc .

9:15 Risk Assessment Aspects of Working with Peptides for Drug Product in a Multi-Product EnvironmentMimoun Ayoub, Ph .D ., Director, Global Peptides, Oligonucleotides, Lipids, Carbohydrates & Injectables Platforms, CordenPharma International, Switzerland

9:45 Refreshment Break 10:15 Aspects of Cleaning of Equipment Used for Multi-Product

ManufacturingHenric Byhlin, Global Development Manager, Polypeptide Group, Sweden

10:50 Crisis to Compliance: Reconciling Hazardous Materials in an Operating Facility Steve Tomko, Architectural Discipline Lead, CRB

11:25 Pharmaceutical Supply Chain Initiative Self Assessment Questionnaire (SHE) and Related Health and Safety Initiatives - Challenges to Early Stage Development ProgramsGary Musso, Ph .D ., President, Musso and Associates LLC

12:00 Close of Workshop

Workshop #4: PEPTIDE MANUFACTURING RISK ASSESSMENT AT CMOS, A MULTI-PRODUCT ENVIRONMENT

Morning Half-Day Workshop • 8:00am-12:00pm


Pre-Conference Symposia • Sunday, April 30, 2017

Cortez Hill ABC 1:00 Welcome and Introduction to Workshop

James V . McArdle, Ph .D ., President, McArdle & Associates, LLC

1:15 Expectations for Quality in Oligo Drug ProductsRené Thürmer, Ph .D ., Deputy Head, Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany

2:00 Early Experience with Terminal Sterilization of OligonucleotidesGregory E . Hardee, Ph . D ., Senior Pharmaceutical Consultant, Oligo Development

2:45 Refreshment Break

3:00 Quality Considerations in Developing an Oligo PFS Combination ProductBrent Golec, Assistant Director, Pharmaceutical Development, Ionis Pharmaceuticals

3:45 Pharmaceutical Quality/CMC Aspects of Lipid Nanoparticle Formulations of Nucleic AcidsChris Barbosa, Director of Technology Development, Acuitas Therapeutics

4:30 Panel Discussion


Workshop #5: QUALITY CONSIDERATIONS FOR OLIGONUCLEOTIDE DRUG PRODUCTS

Afternoon Half-Day Workshop • 1:00pm-5:00pm

Golden Hill 1:00 Welcome and Introductions by Workshop Leader

James Thompson, Ph .D ., Head CMC Project Management, Moderna Therapeutics

1:15 Introduction to mRNA TherapeuticsJames Thompson, Ph .D ., Head CMC Project Management, Moderna Therapeutics

2:00 Design and Manufacturing of Chemically Modified mRNA TherapeuticsAnton P . McCaffrey, Ph .D ., Senior Director of Research and Development, Biology, TriLink BioTechnologies, Inc .

3:00 Refreshment Break

3:30 Delivery Strategies for mRNAs Paul Burke, Ph .D ., Principal, Burke Bioventures LLC

4:00 Applications of mRNA Therapeutics Patrick Baumhof, Ph .D ., Vice President Formulation & Delivery, CureVac AG, Germany

4:30 Panel Discussion


Workshop #6: AN INTRODUCTION TO messengerRNA (mRNA) THERAPEUTICS


Solana BeachWorkshop Leader: Laixin Wang, Ph.D., Vice President of Bioanalytical, NovaBioAssays, LLC

1:00 Workshop Opening Remarks 1:15 PK/PD Modeling - Antisense Oligonucleotides (ASO) and

Current StatusYanfeng Wang, Ph .D ., Executive Director, PK & Clinical Pharmacology, Ionis Pharmaceuticals

1:50 PK/PD Profiling of Therapeutic Oligonucleotides: A Combinatorial Method using Peptide Nucleic Acid Hybridization and Quantigene Branched-DNA AssaysBruno Godinho, Ph .D ., Milton-Safenowitz PostDoctoral Fellow, RNA Therapeutics Institute, University of Massachusetts Medical School

2:25 Applications of Hybridization ELISA Assays in Regulated Bioanalysis of Oligonucleotide TherapeuticsSarah Greenlee, Senior Scientist, PK & Clinical Pharmacology, Ionis Pharmaceuticals

3:00 Refreshment Break 3:30 Providing Bioanalytical Support to Advance siRNA Drug

Development from Pre-Clinical to Clinical StagesKirk M . Brown, Ph .D ., Principal Scientist, Bioanalytical Sciences, Alnylam Pharmaceuticals

4:00 Quantigene Branched-DNA Assay as a Robust, Selective and Sensitive Bioanalytical Method in Support of mRNA TherapeuticsNanda Balasubramanian, Development Scientist II, Alexion Pharmaceuticals


Workshop #7: SENSITIVITY VERSUS SPECIFICITY OF MODERN BIOANALYTICAL ASSAYS AND THE CONSIDERATIONS

OF ADME AND PK/PD STUDIES OF OLIGONUCLEOTIDES Afternoon Half-Day Workshop • 1:00pm-5:00pm

Torrey HillsWorkshop Leaders:Ved Srivastava, Ph .D ., Vice President of Peptide Chemistry, Intarcia Therapeutics, Inc . Bruce H . Morimoto, Ph .D ., Executive Director, Applied Translational Medicine, Celerion, Inc .

1:00 Workshop Leaders' RemarksVed Srivastava, Ph .D ., Vice President of Peptide Chemistry, Intarcia Therapeutics, Inc .Bruce H . Morimoto, Ph .D ., Executive Director, Applied Translational Medicine, Celerion, Inc .

1:20 Peptide Stapling and Half-life ExtensionWeijun Shen, Ph .D ., Principal Investigator, California Institute for Biomedical Research

2:10 An Insider's Guide to PEG as a Half-life ExtenderChristopher Holmes, Ph .D ., Industry Consultant

3:00 Refreshment Break 3:30 Recent Advances in Peroral Delivery of Peptide Drugs

Nozer Mehta, Ph .D, Principal, Peptide Technologies LLC

4:20 Half-life Extension Strategies for Peptides as Seen Through the Lens of the GLP-1 ClassChristopher Rhodes, Ph .D ., President and CEO, Drug Delivery Experts, USA


Workshop #8: ENHANCING PHARMACEUTICAL PROPERTIES OF PEPTIDES


JOIN US AT MISSION BREWERYSponsored in Part by:

April 30th 6:00pm - 9:00pm . In the Historic Wonder Bread Building in the East Village where their “mission is great beer” . With over 40 National and International awards under its belt, Mission is considered one of the top craft breweries in San Diego County . Visit Registration Desk to RSVP – Limited Space Available! Buses Depart at 5:45 pm from Kettner Drive Entrance www .missionbrewery .com

12 www.TIDESEvent.com *Schedule subject to change

Main Conference • Keynote/Plenary Session • Monday, May 1, 2017

7:45 Registration and Coffee

8:55 Chairperson’s RemarksVed Srivastava, Ph .D ., Vice President of Chemistry, Intarcia Therapeutics

9:00 Clinical Development of Eteplirsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Edward Kaye, M.D., President, CEO and Chief Medical Officer, Sarepta Therapeutics

9:45 Speaker TBA .

10:30 Networking Refreshment Break

11:00 Synthesis of DNA/RNA on Chips and Biochemical/Biological Activity of New Morpholino DNAsMarvin Caruthers, Ph .D ., Distinguished Professor, Chemistry and Biochemistry, University of Colorado

11:45 Site Selective Antibody Drug Conjugates Enabled by Cysteine Arylation and Native ConjugationBradley L . Pentelute, Ph .D ., Professor, Chemistry, Massachusetts Institute of Technology

12:30 Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break Co-Sponsored by:

1:50 Chairman’s RemarksBob D . Brown, Ph .D ., CSO, SVP Research and Development, Dicerna Pharmaceuticals, Inc .

1:55 SPINRAZA: The Road to ApprovalMayra B . Reyes-Armour, Ph .D ., Director, Asset Lead, Biogen

2:40 Innovations in Antisense RNA Targeting: Driving Value, Targeting the SourceRichard S . Geary, Ph .D ., Senior Vice President, Development, Ionis Pharmaceuticals, Inc .

3:15 Networking Refreshment Break in Poster and Exhibit Hall

Co-Sponsored by:

4:00 Advancing Greener Peptide and Oligonucleotide SynthesisMichael E . Kopech, Ph .D ., Executive Director, Technical Operations, Radius Health, Inc .

4:30 Epitope Targeted Peptide-based Protein Catalyzed Capture AgentsJames R . Heath, Ph .D ., James R . Heath, Ph .D ., Elizabeth W . Gilloon Professor of Chemistry, California Institute of Technology

5:00-6:30 pm Networking Reception in Poster and Exhibit HallJoin fellow attendees and speakers for the TIDES opening night networking reception . Enjoy drinks and appetizers with colleagues while viewing the exhibits and scientific poster sessions.

Sponsored by:

Seaport ABC


Main Conference • Tuesday, May 2, 2017Track 1: Oligonucleotide Chemistry,

Manufacturing and ControlsTrack 2: Oligonucleotide Discovery,

Preclinical and ClinicalTrack 3: Peptide Chemistry

Manufacturing and ControlsTrack 4: Peptide Discovery,

Preclinical and Clinical7:30 Registration and Coffee

Sponsored by:

7:45 -

8:15BREAKFAST PRESENTATION: Large-scale Production of Liposomal Oligonucleotides: Lessons Learned from Successful Product and Process Developments (Torrey Hills) Charalampos Koutsoulas, Ph .D ., Scientific Project Leader, Liposome Technology, Polymun Scientific GmbH, Austria

BREAKFAST PRESENTATION: Liquid-Phase Oligonucleotide Synthesis by Segment Condensation (Golden Hill AB) Masanori Kataoka, Ph.D., Chief Technical Officer, Shikoku Nucleic Acid Chemistry (S-NAC), Japan

Seaport B Seaport C Seaport A Cortez Hill8:25 (7:55) Chairman’s Remarks

Lubomir Nechev, Ph .D ., Vice President, Process Sciences, Alnylam Pharmaceuticals

Chairman’s RemarksDmitry Samarsky, Ph.D., Chief Scientific Officer, Silence Therapeutics, Germany

Chairman’s RemarksAlex Fässler, Ph .D ., COO Europe, Bachem AG, Switzerland

Chairman’s RemarksJohn Nuss, Ph .D ., Vice President, Drug Discovery, Ferring Research Institute

Quality of Oligonucleotide Starting Materials

Oligonucleotide Discovery and Preclinical


Peptide Targets, Peptide Discovery and Synthesis

(8:00) Driving Large Scale Oligonucleotides to Be Commercially ViableLouis Diorazio, Ph .D ., Principal Scientist, Chemical Development, AstraZeneca, United Kingdom

8:30 GalNAc Conjugates: From Starting Materials to Manufacturing

This Is Your Brain on Antisense Oligonucleotides: Distribution, Activity and Application to the Treatment of Severe Neurodegenerative DiseaseEric Swayze, Ph .D ., Vice President, Chemistry, Ionis Pharmaceuticals

High Quality Starting Materials: One Important Key to SuccessDirk Bächle, Ph .D ., Director Quality Control II, Bachem AG, Switzerland

Proteomics Methods to Discover Bioactive Peptides and Small Proteins Alan Saghatelian, Ph .D ., Professor, Dr . Frederik Paulsen Chair, Salk Institute for Biological Studies

GalNAc Solid Support as Starting Material for the Oligonucleotide Conjugate Manufacturing ProcessRoumen Radinov, Ph .D ., Associate Director, Process Chemistry, Alnylam Pharmaceuticals

9:00 Conjugation of Oligonucleotides under Non-aqueous ConditionsChristoph Rosenbohm, Ph .D ., VP and Head of Discovery Operations, Roche Innovation Center Copenhagen, Denmark

Modified Aptamers As a New Generation of Nucleic Acid TherapeuticsDan Drolet, Ph .D ., Senior Director, Pharmacology, SomaLogic

Characterization of Starting Resins and Resin PropertiesBrad DeHoff, Ph .D ., Director, R&D at CordenPharma GmbH

Recombinant Expression of Protein and Peptide Therapeutics with Multiple Non-Natural Amino AcidsJerod Ptacin, Ph .D ., Principal Scientist, Synthorx

9:30 Solid vs. Solution Phase GalNAc/Oligonucleotide ConjugationAndrew Rodriguez, Ph .D ., Assistant Director, Process Organic Chemistry, Ionis Pharmaceuticals

Reduced in vivo Tumor Grown Using CAR T-cells Treated ex vivo with PD-1 Targeting sd-rxRNA®Karen Bulock, Ph .D ., Vice President, Research, RXi Pharmaceuticals

Regulatory Change Control for Peptide Starting Materials: A Case Study Gerhard Haas, Ph .D ., Vice President, Quality Assurance and Regulatory Affairs, Bachem AG, Switzerland

The Application of Assembly Line Methodologies to the Development and Manufacture of Complex Synthetic OligomersKimon Roussopoulos, Ph .D ., Head of Research and Development, Swedish Biomimetics 3000 Ltd ., United Kingdom

10:00Networking Refreshment Break in Poster and Exhibit Hall Sponsored by:

Track 1: Oligonucleotide

Chemistry, Manufacturing and Controls


and ClinicalTrack 5: mRNA Therapeutics Track 3: Peptide Chemistry

Manufacturing and ontrolsTrack 4: Peptide Discovery,

Preclinical and Clinical

Seaport B Seaport C Golden Hill Seaport A Cortez Hill

GalNAc Conjugates: From Starting Materials to

Manufacturing (continued)

Oligonucleotide Discovery and Preclinical (continued)

Discovery and Development of mRNA Therapeutics

and VaccinesChairperson: Örn Almarsson, Ph .D .,

Head of Formulations, Moderna Therapeutics


(continued)

(10:45) Protein Engineering to Improve the Expression of Protein-Intein Fusions for Development of Protein-Peptide ConjugatesAlex Jacobitz, Postdoctoral Fellow, Amgen

10:45 GalXC cGMP Manufacturing ExperienceEmma Wright, D .Phil, Senior Director, Manufacturing, Dicerna Pharmaceuticals

Locked Nucleic Acid: Enabling RNA TherapeuticsMaj Hedtjærn, Scientist, Roche Innovation Center Copenhagen, Denmark

Preclinical and Clinical Development of mRNA Therapeutics Örn Almarsson, Ph .D ., Head of Formulations, Moderna Therapeutics

Downstream Process Controls and Aspects of the API Solid StateJon Holbech Rasmussen, Ph .D ., Director Global Development, PolyPeptide Group, Sweden

Peptidomimetic and Peptidic Macrosycles: Expanding the

Peptide Drug Modality

11:15 Strategies for Scaling, Optimizing and Understanding the Manufacture of GalNAc-Conjugated OligonucleotidesChris Shaffer, Associate Director, Pharmaceutical Development, Regulus Therapeutics

RNAi Structures in Animals: An RNAi Platform and Preclinical UpdateBob D . Brown, Ph .D ., CSO, SVP Research and Development, Dicerna Pharmaceuticals

Cancer Immunotherapy: Individualized mRNA-based Vaccines for the Treatment of Cancer Matthias Miller, Ph .D ., Project Manager, BioNTech AG, Germany

Critical Quality Attributes for Activated PEGsRaymond Behrendt, Ph .D ., Project Manager R&D, Drug Delivery Compounds – Functionalized PEGs, Merck, Switzerland

Development of Cytotoxic Bicyclic Peptide Drug Conjugates and Applications in Molecular Targeted Cancer TherapyDan Teufel, Ph .D ., Head of Chemistry, Bicycle Therapeutics Limited, United Kingdom

11:45 PANEL DISCUSSION: Qualification of Impurities for OligonucleotidesModerator: Lubomir Nechev, Ph .D ., Alnylam Panelists: Claus Rentel, Ph .D ., Ionis; René Thürmer, Ph .D ., BfArM, Mike Webb, Atlantic Healthcare, , Louis Diorazio, Ph .D ., AstraZeneca

RNAi Based Human Therapeutics: From Discovery to ApplicationsMuthiah (Mano) Manoharan, Ph .D ., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals

Overview of RaNA's mRNA Up-regulation Platform and mRNA TechnologyThomas McCauley, Ph .D ., Chief Scientific Officer, RaNA Therapeutics

Implementation of Appropriate cGMPs for Pre-Clinical Through Commercial Drug SubstanceGary Erickson, Ph .D ., CEO, CBL Biopharma LLC

Development of Macrocyclic Peptides for the Treatment of Complement-Mediated DisordersAlonso Ricardo, Ph .D ., Senior Vice President, Research, Ra Pharmaceuticals

12:15 Spotlight Presentation Oligonucleotides Targeting Periostin Ameliorates Pulmonary FibrosisEsteban Gabazza, M .D ., Ph .D ., Professor in Medicine and Chairman, Department of Immunology, Mie University Graduate School of Medicine, Japan

Ex vivo and in vivo Modification of Dendritic Cells with mRNAKris Thielemans, M .D ., Ph .D ., Chief Scientific Officer, eTheRNA immunotherapies, Belgium

Etelcalcetide: Overcoming Challenges in the Commercialization of a Late Development In-licensed Peptide ProgramAsher Lower, Ph .D ., Senior Scientist, Pivotal Drug Substance Process Development, Amgen

Invention of Grazoprevir: A Hepatitis C Virus NS3/4a Protease InhibitorJohn A . McCauley, Ph .D ., Director, Medicinal Chemistry, Merck Research Laboratories

Sequence Fidelity of Chemical Oligonucleotide SynthesisHüseyin Aygün, Ph .D ., Chief Scientific Officer, BioSpring GmbH, Germany

12:45 Networking Luncheon in Poster and Exhibit Hall


Main Conference • Tuesday, May 2, 2017

Track 1: Oligonucleotide Chemistry, Manufacturing

and Controls


and ClinicalTrack 5: mRNA Therapeutics Track 3: Peptide Chemistry

Manufacturing and ontrolsTrack 4: Peptide Discovery,

Preclinical and Clinical

Seaport B Seaport C Golden Hill Seaport A Cortez Hill1:55 Chairman’s Remarks

Emma Wright, D .Phil, Senior Director, Manufacturing, Dicerna Pharmaceuticals

Chairman’s RemarksArthur Levin, Ph .D ., Executive Vice President R&D, Avidity Biosciences

Chairman’s RemarksNigel Horscroft, Vice President Development RNArt, CureVac AG, Germany

Chairman’s RemarksRené Thürmer, Ph .D ., Deputy Head, Unit Pharmaceutical Biotechnology, BfArM – Federal Institute for Drugs and Medical Devices, Germany

(1:45) Chairman’s RemarksWaleed Danho, Ph .D ., Distinguished Research Leader and Consultant

Innovative Processes and Synthesis Strategies for

Oligonucleotides


Oligonucleotide TherapeuticsmRNA Delivery Strategies Peptide CMC Case Studies

and StrategiesPreclinical and Clinical

Peptides: Lessons Learned

2:00 Liquid Phase Oligonucleotide Synthesis with Membrane Separation for Efficient Large Scale ManufacturingPiers Gaffney, Ph .D ., Engineering Chemist, Imperial College London, United Kingdom

Strategies to Optimize Oligonucleotide Safety in Drug Discovery and Candidate SelectionFranz Schuler, Ph .D ., Head Drug Disposition and Safety, Pharmaceutical Sciences, Roche Pharma Research and Early Development, Roche Innovation Center Basel, F . Hoffmann-La Roche Ltd ., Switzerland

Targeted mRNA Delivery to the Liver for Intracellular Enzyme Replacement Therapy (i-ERT)Michael Houston, Ph .D ., Chief Scientific Officer, PhaseRx

Ferring QbD Approach to Peptide APIs in DevelopmentMette Bryder, M .D ., QbD Facilitator, Ferring Pharmaceuticals A/S, Denmark

(1:50) Half-Life Extended NaV1.7 Inhibitory Peptide-Antibody Conjugate Biodistributes to NerveJustin Murray, Ph .D ., Senior Scientist, Hybrid Modality Engineering, Therapeutic Discovery, Amgen

(2:15) Discovery of Selepressin and Merotocin, Two linical Candidates from Neurohypophyseal Family of HormonesKazimierz Wisniewski, Ph .D ., Senior Scientist II, Ferring Research Institute

2:30 New Purification Process for ASOsRobert Gronke, Ph .D ., Senior Principal Scientist, Biogen

Cobitolimod, a Novel Immunomodulatory Oligonucleotide Entering Clinical Phase IIb for Local Treatment of Active Ulcerative ColitisChristine Dieterich Johansson, Ph .D ., Senior Project Manager CMC and Pre-clinical, InDex Pharmaceuticals, Sweden

Therapeutic mRNA Delivery: Into Hepatocytes and BeyondAdrien Weingärtner, Ph .D ., Group Leader mRNA, Silence Therapeutics, Germany

Regulatory Perspectives on Characterization of Quality Attributes of Therapeutic Peptides René Thürmer, Ph .D ., Deputy Head, Unit Pharmaceutical Biotechnology, BfArM - Federal Institute for Drugs and Medical Devices, Germany

(2:40) Setmelanotide for the Treatment of Rare Monogenic Forms of Obesity: The MC4-PathwayLex Van der Ploeg, Ph .D ., Chief Scientific Officer, Rhythm Pharmaceuticals3:00 AJIPHASE®: Novel Solution

Phase Approach for OligonucleotidesHideaki Sato, General Manager, GeneDesign Inc ., a part of Ajinomoto Group, Japan

Assessment of the Effects of 2′-Methoxyethyl Antisense Oligonucleotides on Platelet Count in Cynomolgus NHPsScott Henry, Ph .D ., Vice President of Nonclinical Development, Ionis Pharmaceuticals

RNAntibody® - A Potent mRNA Technology for Therapeutic AntibodiesNigel Horscroft, Vice President Development RNArt, CureVac AG, Germany

Peptide Synthesis Using Our Coupling Agent AllessanCap® with Case StudyMimoun Ayoub, Ph .D ., Director, Global Peptides, Oligonucleotides, Lipids, Carsbohydrates & Injectables Platforms, CordenPharma, Switzerland

(3:05) Elastin-Like Polypeptide Biopolymers Enhance the Pharmacology of Therapeutic PeptidesJim Ballance, Ph .D ., VP Research & Scientific Affairs, PhaseBio Pharmaceuticals, Inc .

3:30Networking Refreshment Break in Poster and Exhibit Hall Sponsored by:

mRNA Bioanalytical, CMC and Manufacturing (shared session)


Oligonucleotide Therapeutics (continued)

mRNA Bioanalytical, CMC and Manufacturing

(shared session in Seaport B)

Peptide CMC Case Studies and Strategies (continued)

Peptides in the Clinic: Lessons Learned (continued)

4:15 Therapeutic mRNA: Manufacturing and Regulatory ConsiderationsAndreas Kuhn, Ph .D ., Vice President RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH, Germany

Selection of Well-Tolerated GalNAC-siRNAs by Screening for RNAi-mediated Off-Target Effects in Rodent Toxicity StudiesMaja Janas, Ph .D ., Senior Scientist, Investigative Toxicology, Nonclinical Safety and Bioanalysis, Alnylam Pharmaceuticals

Therapeutic mRNA: Manufacturing and Regulatory ConsiderationsAndreas Kuhn, Ph .D ., Vice President RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH, Germany

Single to Multiple Disulfide Bridge-rich Peptides: A Perpetually Evolving Approach in Process Development and Scale-UpEl Djouhar Rekai, Ph .D ., Head of Operation Products, PolyPeptide Group, Belgium

Discovery and Development of Novel Macrocycle Drugs: Outer Membrane Protein Targeting Antibiotics (OMPTA), A New Class with a Novel Mode of ActionDaniel Obrecht, Ph .D ., Chief Scientific Officer, Co-Founder, Polyphor Ltd ., Switzerland

Oligonucleotide Delivery Strategies

4:45 Case Study of Bioanalytical Approaches for Assessing Biodistribution of Modified mRNA TherapeuticsNeil Henderson, Ph .D ., Associate Principal Scientist, Nucleotide Bioanalysis Group, AstraZeneca, United Kingdom

Recent Advances in the Design and Delivery of microRNA-based TherapeuticsCharles Allerson, Ph .D ., Director, Chemistry, Regulus Therapeutics

Case Study of Bioanalytical Approaches for Assessing Biodistribution of Modified mRNA TherapeuticsNeil Henderson, Ph .D ., Associate Principal Scientist, Nucleotide Bioanalysis Group, AstraZeneca, United Kingdom

Insights from PolyPeptide Group: Impurities in Peptide ManufacturingJon Holbech Rasmussen, Ph .D ., Director Global Development, PolyPeptide Group, Sweden

Dasiglucagon (ZP4207) Single-Dose Rescue Treatment for Acute, Severe Hypoglycemia Events, Phase II UpdateAndy Parker, Ph .D ., CSO & SVP, Head of Research & External Innovation, Zealand Pharma A/S, Denmark

5:15 Biochemical, Biophysical and Cell-based Characterization of mRNA, a Novel Drug ModalityKristian Link, Ph .D ., Senior Manager, Analytical Development, Moderna Therapeutics

Enhancing ASO Activity in Extra-Hepatic TissuesPunit Seth, Ph .D ., Vice President, Medicinal Chemistry, Ionis Pharmaceuticals

Biochemical, Biophysical and Cell-based Characterization of mRNA, a Novel Drug ModalityKristian Link, Ph .D ., Senior Manager, Analytical Development, Moderna Therapeutics

LC-MS, A Key Technique to Understanding the Fate of Impurities in Ferring’s Synthetic Therapeutic PeptidesJörgen Kjellgren Sjögren, Ph .D ., Senior Research Scientist, Product Development and Drug Delivery, Ferring Pharmaceuticals A/S, Denmark

Clinical Development of Elamipretide for Rare and Common DiseasesHazel H . Szeto, M .D ., Ph .D ., Scientific Founder, Stealth Biotherapeutics

5:30Networking Reception in Poster and Exhibit Hall (OPEN TO ALL ATTENDEES) Sponsored by


Main Conference • Wednesday, May 3, 2017

Track 1: Oligonucleotide Chemistry, Manufacturing and Controls




7:00 - 8:25

Early Morning Exhibit Viewing and Breakfast in the Exhibit (Grand Hall)

Plenary Session: CRISPR and Genome Editing Applications (Harbor GH)8:25 Chairman’s Remarks Firoz Antia, Ph .D ., Director, Chemical Process R&D, Biogen

8:30 in vivo Gene Delivery and Genome Editing: How Advances in siRNA and mRNA Delivery Enable in vivo CRISPR/Cas9 Delivery Daniel G . Anderson, Ph .D ., Associate Professor, Department of Chemical Engineering, David H . Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

9:00 Taking Advantage of Modified RNAs to Improve the CRISPR System Considering Off-target Effects and in vivo StrategiesThazha P . Prakash, Ph .D ., Director, Medicinal Chemistry, Ionis Pharmaceuticals

9:30 High-resolution Interrogation of the Human Noncoding Genome Using Pooled CRISPR ScreensNeville Sanjana, Ph .D ., Core Faculty Member, New York Genome Center, Assistant Professor, Department of Biology, New York University

10:00 Networking Refreshment Break and Last Chance for Poster and Exhibit Viewing

Harbor G Harbor H Harbor E Harbor D10:55 Chairman’s Remarks

Paul Metz, Principal Consultant, Metz Biotechnology Consulting, LLC

Chairman’s RemarksTroels Koch, Ph .D ., VP & Head of Research, RNA Therapeutics, Roche Innovation Center Copenhagen, Denmark

Chairman’s RemarksFanyu Meng, Ph .D ., Principal Scientist, Analytical Research and Development, Merck Research Labs

Chairman’s RemarksChristopher Rhodes, Ph .D ., President and CEO, Drug Delivery Experts, USA

Analytical Strategies for Oligonucleotides

CRISPR and Genome Editing Applications

Analytical Strategies to Enable Peptide Development

Peptide Delivery and Formulation Strategies

11:00 Evolution of the Reference Standard with Product Development CycleConnie Cho, Associate Director, CMC Analytical Development, Regulus Therapeutics

Advancing CRISPR Technologies for Therapeutic ApplicationChristopher Wilson, Ph .D ., Director of Lead Finding, Editas Medicine

Analytical Strategy for Continuous Manufacture Process Development for PeptidesAlicia Zeng, Ph .D ., Scientist, Process Development, Amgen

Tailoring Peptides for Less Frequent Dosing and Non-invasive DeliveryJesper Lau, Ph .D ., Vice President, Protein and Peptide Chemistry, Global Research, Novo Nordisk A/S, Denmark

11:30 Application of ESI-MS/MS for Oligonucleotide Sequencing Methods Edward Huber, Ph .D ., Director, Analytical Development/QC, Nitto Denko Avecia

Chemical Modification of Guide RNAs for SpyCas9 and AsCpf1 CRISPR NucleasesMark Behlke M.D., Ph.D., Chief Scientific Officer, Integrated DNA Technologies

2D-LC as an On-line Desalting Tool Allowing Peptide Identification Directly from MS Unfriendly HPLC MethodsHao Luo, Ph .D ., Associate Principal Scientist, Merck & Co .

Sublingual Delivery of Peptides and Small Proteins: Exenatide, Insulin and IL-2 As ExamplesYves Decadt, Chief Executive Officer, BioLingus, Switzerland

12:00 Validation of Analytical Methods for Oligonucleotide TherapeuticsClaus Rentel, Ph .D ., Executive Director Analytical Development QC, Ionis Pharmaceuticals

Preclinical and Clinical Development of Oligonucleotides

Application of High Resolution MS for the Analysis and Characterization of PeptidesMarion King, Ph .D ., Analytical Development Manager, Ipsen Manufacturing Ireland Ltd .

Formulation Approaches to Overcome Barriers to Oral Delivery of MacromoleculesRonak Savla, Ph.D., Scientific Affairs Manager, Catalent Psharma Solutions

A New Dimension in LNA TherapeuticsTroels Koch, Ph .D ., VP & Head of Research, RNA Therapeutics, Roche Innovation Center Copenhagen, Denmark

12:30 Lunch on Your Own

June 5-7, 2017Paradise Point ResortSan Diego, CA

DRIVING CREATIVE PROTEIN ENGINEERING & DESIGN TO SUCCESSFULLY TRANSFORM PROMISING NEW MOLECULES INTO DIFFERENTIATED PRODUCTS

proteintherapeuticsevent.com

Next GenerationProtein Therapeutics & Bioconjugates Summit


Main Conference • Wednesday, May 3, 2017

Track 1: Oligonucleotide Chemistry, Manufacturing and Controls




Harbor G Harbor H Harbor E1:40 Chairman’s Remarks

Paul Metz, Principal Consultant, Metz Biotechnology Consulting, LLC

Chairman’s RemarksElena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel

Chairman’s RemarksMimoun Ayoub, Ph .D ., Director, Global Peptides, Oligonucleotides, Lipids, Carbohydrates & Injectables Platforms, CordenPharma, Switzerland

Validation and Regulatory Strategies for Oligonucleotides

Preclinical and Clinical Development of Oligonucleotides Peptide Vaccines and Peptide Immunotherapy

1:45 QbD Approaches to Oligonucleotide Process DevelopmentGill Turner, Ph .D ., QbD Lead, Product Development & Supply, GlaxoSmithKline, United Kingdom

Update on the Development of Oligonucleotides at QuarkElena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel

The Galinpepimut-S (GPS) Clinical Development Program in Immuno-oncology (IO): Novel Approach Using a Mixture of Heteroclitic Peptides for Epitope Immunization against the Wilms Tumor-1 (WT1) Protein across Several Tumor TypesNicholas J. Sarlis, M.D., Ph.D., Chief Medical Officer & Senior VP, Sellas Life Sciences Group

2:15 The Application of DoE to Oligonucleotide Synthesis – Generating Process UnderstandingBen Andrews, Ph.D., Scientific Investigator, API Chemistry, GlaxoSmithKline, United Kingdom

Clinical Development of AST-005, A Topically-applied Antisense Spherical Nucleic Acid for the Treatment of PsoriasisWeston Daniel, Ph .D ., Director of Program Management, Exicure, Inc .

Translating B Cell Epitope Peptide-Based Cancer Vaccines to the Clinic and Future Combination ImmunotherapiesPravin T .P . Kaumaya, Ph .D ., Professor, Division of Vaccine Development/Peptide & Protein Engineering Laboratory, The Ohio State University Medical Center and the James Comprehensive Cancer Center

2:45 CMC Strategies for Managing Late Stage Development of Global OligonucleotidesVidhya Gopalakrishnan, Ph .D ., Senior Vice President, Pharmaceutical Development, Quark Pharmaceuticals

Therapeutic microRNA Modulation: From Concept to the ClinicWilliam S . Marshall, Ph .D ., President and CEO, miRagen Therapeutics

CMC Strategy for a Clinically Approved “Nanoparticle-in-oil Based Vaccine Delivery System (Water Free) with an Effective, Long Lasting and Durable Immune Responses against “Multi Peptide Vaccine” Encoding T- Cell for Treatment of Ovarian Cancer Immunotherapy (IO) and B-cell Epitope for RSV (Infectious Disease (ID)Leeladhar Sammatur, Vice President, Product Development and Manufacturing, Immunovaccine, Inc .

3:15 Scaling up for Commercial Supply of Alicaforsen and Treating Patients Janette Thomas, Ph .D ., Director of International Operations, Atlantic Healthcare plc, United KingdomMike Webb, Ph .D ., Vice President of Manufacturing, Atlantic Healthcare plc, United Kingdom

Oligonucleotide Clinical Case StudyPatrick Haslett, M .D ., Executive Director of Clinical Research, Alnylam Pharmaceuticals

Development of Peptide Vaccines for Cancer: Overcoming the Challenges for InnovationYasuhide Uejima, Ph .D ., General Manager, Quality Assurance & Pharmaceutical Development, GreenPeptide Co ., Ltd ., Japan

3:45 Close of Conference

TIDES: Oligonucleotide &Peptide Therapeutics

May 7-10, 2018Hynes Convention CenterBoston, MA

SEE YOU NEXT YEAR!

June 5-7, 2017Paradise Point ResortSan Diego, CA


Poster Presentations

Dedicated Poster Viewing HoursMonday, May 1 5:30 pm - 6:30 pmTuesday, May 2 10:00 am-10:45 amWednesday, May 3 10:00 am-10:55 am

Exhibit & Poster Hall Viewing HoursMonday, May 1: 12:30 pm - 6:30 pmTuesday, May 2: 10:00 am - 6:30 pmWednesday, May 3: 7:00 am - 10:55 am

BIOANALYTICAL STRATEGIES AND TECHNOLOGIESB1 Synthetic Challenges: Using Size-Exclusion Chromatography for Annealing Monitoring Nadya Brady Nitto Denko Avecia Inc.

B2 Aptamer-based Molecular Imaging using Complementary Oligonucleotide Platform Jun Young Park Yonsei University Health System

CRISPR THERAPEUTICS - DEVELOPMENT STRATEGIES AND CASE STUDIESC1 Towards More Reproducible CRISPR-related Discovery and Screening Results via

Synthetic Single Guide RNAsJohn Walker Synthego

CROSS-PLATFORM DRUG DELIVERY TECHNOLOGIES FOR OLIGOS, PEPTIDES AND OTHER MOLECULESCR2 Delivery of mRNA using the Viromer Technology Steffen Panzner Lipocalyx

MESSENGERRNA (mRNA) THERAPEUTICS - DEVELOPMENT STRATEGIES AND CASE STUDIESM1 Considerations for the Design and cGMP Manufacturing of mRNA Therapeutics Craig Dobbs TriLink BioTechnologies

M2 mRNA-based Therapeutics for Intractable Diseases and Regenerative Medicine Keiji Itaka Tokyo Medical and Dental University

NONCLINICAL (TOX, PK) DEVELOPMENT STRATEGIES AND CASE STUDIESN1 Tritium Radiolabeled Antisense Oligonucleotides for ADME Studies Jon Bloom Pharmaron

OLIGONUCLEOTIDE DISCOVERY TECHNOLOGIES AND DESIGN STRATEGIESOD1 Novel Achiral Building Blocks and Solid Supports for Preparation of

Multi-labeled OligonucleotidesKhirud Gogoi A M Chemicals LLC

OD2 Optimization of Spherical Nucleic Acids (SNAs) for Improved Therapeutic Properties Rich Kang Exicure, Inc.

OD3 Defined Multimeric Oligonucleotides for Enhanced Therapeutic Effect Jonathan Miles Brown MPEGLA LLC

OD4 Molecular Targets of Short Activating RNAs: Characterizing Antisense lncRNAs of CEBPA Ryan Setten City of Hope

OD5 Therapeutic Approach of Synthetic SINEUPs RNA: That Can Enhance Translation Activities Of Hepatocyte Nuclear Factor 4 Alpha

Hazuki Takahashi RIKEN

OLIGONUCLEOTIDE MANUFACTURING, CMC AND ANALYTICAL CASE STUDIES & TECHNOLOGIESOM1 Method Development and Evaluation of the Techniques for Oligonucleotide

Concentration DeterminationRobert J. Duff Amgen, Inc

OM2 Improving MS Sensitivity through the Reduction of Metal Salt Adducts in IP-RPLC/MS Oligonucleotide Analyses

Joe Fredette Waters Corporation

OM3 Quantitation and Characterization of Polysorbate-80 in Protein Formulations by HPLC and Charged Aerosol Detection

Yingying Lee Catalent Pharma Solutions

OM4 Forced Degradation Light Studies of Synthetic Phosphorothioate Oligonucleotides Megan Pearce Ionis Pharmaceuticals

OM5 An Investigation into Oligonucleotide Quadruplex Formation using Size Exclusion Chromatography

Melissa Sato Amgen, Inc.

OM6 Demonstration of Complete Control of Oligonucleotide Drug Substance Rachel Orr GSK

OLIGONUCLEOTIDE THERAPEUTICS: PRECLINICAL AND CLINICAL DEVELOPMENTOT1 The Clinical Candidate MTL-CEBPA Leads to Significant Reduction in Ascites and

Improvement in Overall Survival in a CCl4-induced Liver Failure ModelRobert Habib MiNA Therapeutics

OT2 Synthesis and Biological Properties of Chemically-Modified Dsrnas Possessing Ligands Such as Saccharides and Peptides

Yukio Kitade Aichi Institute of Technology

OT3 Derepressing Muscleblind Expression by miRNA Inhibitors Ameliorates Myotonic Dystrophy-Like Phenotypes

Beatriz Llamusi University of Valencia

OT4 Therapeutic Oligo Quality: Profiling and Controlling for Raw Material Impurities Indra Pal Thermofisher

OT5 Harnessing the Biology of Vitamin D to Enable Sustained Delivery of Peptides and Proteins

Tarik Soliman Extend Biosciences

OT6 Long-circulating Polymeric Nanoparticles for Efficient siRNA Delivery in Cancer Therapy Jiyeon Son Samyang Biopharmaceuticals Corp.

Poster presentations will be on display at all times during Exhibit Hall viewing hours . Poster presenters may stand by their posters at any time, but to ensure that attendees are able to meet poster presenters at specific times during the conference, we have designated the following days/times as “Dedicated Poster Viewing” Hours . We ask all poster presenters to stand by their posters during the dedicated hours listed above to make it easy for attendees to “find” you. We will be promoting these dedicated poster viewing hours during the conference to facilitate better attendee and poster presenter interactions and discussions .


Poster Presentations (continued)

OT7 Conditional-siRNAs: a New Approach to Targeted Molecular Therapy Si-ping Han California Institute of Technology and City of Hope Comprehensive Cancer Center

OT8 In vivo Delivery of Asymmetric Gene-silencing RNAs Targeting CTNNB1 and PD-L1 Show a Broad Spectrum of Potent Anti-tumor Activities in Preclinical Cancer Models

Yuan Gao Boston Biomedical Inc., Sumitomo Dainippon Pharma Global Oncology

PEPTIDE DISCOVERY TECHNOLOGIES AND DESIGN STRATEGIESPD1 Diversity Oriented Synthesis, Characterization and Anti-Cancer Applications

of Peptide NucleolipidsNiki Rana Seton Hall University

PD2 PhcrTx1, a Novel Marine Peptide Acting on Acid-Sensing Ion Channels. Isolation and Characterization.

Armando AlexeiRodríguez Alfonso

Hannover Medical School

PD3 Analysis of Global Protein Conformational Changes in Solution by Combined Approach of IMS–MS with Size-Exclusion Chromatography and Differential Hydrogen–Deuterium Exchange

Christopher Strulson Merck & Co., Inc.

PD4 ORBIT Peptide Display—High Throughput Selection of Peptides with Biological Function Alex Batchelor Orbit Discovery Ltd

PD5 HTS Identification of a Nav1.7 Blocker from Animal Venoms Aude-Marie Alem Smartox Biotechnology

PEPTIDE MANUFACTURING, CMC AND ANALYTICAL CASE STUDIES & TECHNOLOGIESPM1 Development of Grazoprevir Manufacturing Route Qinghao Chen Merck & Co. Inc.

PM2 Merrifield Reloaded: Peptide Chemistry Goes Green Harald Kolmar Technische Universität Darmstadt

PM3 Chromatographic Improvement in Peptides Separations Using the Pulverized Monoliths Column

Riichi Miyamoto Kyoto University, SNG

PM4 Novel Application of Standard Chromatographic Tests of Stationary Phase Characterization in Industrial Scale HPLC Processes

Imre Sallay Osaka Soda Co., ltd

PM5 Little Science – Big Difference in Peptide Purification Processes Oscar Rebolledo DAISO Fine Chem USA, Inc.

PEPTIDE THERAPEUTICS: PRECLINICAL AND CLINICAL DEVELOPMENTPT1 Using Compliant-Ready Software for Synthetic Peptide Impurity Tracking and

Reporting with Added Mass Detection for Improved Confidence in ResultsBrooke Koshel Waters Corporation

PT2 Solution and Solid-State NMR Investigation of Stabilizing and Destabilizing Interactions in Peptide Formulations

Yongchao Su Merck Research Laboratories

PT3 Therapeutic Effect of Anti-Inflammatory Peptide from Human Beta Defensin-3 for the Treatment of Arthritis

Gook Jin Yoon Seoul National University

PT4 Selective Osteogenesis by a Synthetic Calcium Accumulating Peptide for the Treatment of Osteoporosis

Dong Woo Yin NIBEC

PT5 Human Gastrointestinal Stability of Peptides for Oral Delivery Vipul Yadav Intract Pharma Limited

PEPTIDE VACCINES, PEPTIDE COCKTAILS AND PEPTIDE-BASED IMMUNOTHERAPIESPV1 Reversed Phase Liquid Chromatography Using Surrogate (Additional)

Stationary PhasesMohmed Anwer Neuland Laboratories Inc

PV2 B7H6 Derived Peptides Trigger TNF-a Secretion in Lymphocytic NK92-MI cells. Mariana Phillips Seton Hall University

REGULATORY STRATEGIES & CONSIDERATIONS FOR PEPTIDES AND OLIGONUCLEOTIDESR1 Antisense Oligonucleotides Internally Labeled With Peptides Show Improved

Target Recognition and Stability to Enzymatic DegradationMaria Taskova University of Southern

Denmark

OTHERO1 Optimized Synthesis of Therapeutic MK2 Inhibitor Cell Penetrating Peptide: Reduction in

Synthesis Time Using Parallel Automated SynthesisJames Cain Gyros Protein Technologies

O2 Studies on Uptake of Oligonucleotide-Lipid Conjugates Györgyi Ferenc Hungarian Academy of Sciences, Biological Research Centre, Szeged

O3 Enhanced Peptide and Oligonucleotide Purification via Novel Orthogonal, Doped Reverse Phase Chromatography

Jurgen Machielse Zeochem AG

O4 Improved Chemical and Enzymatic Synthesis of Threose Nucleic Acid (TNA) Sujay Sau University of California Irvine

O5 Exploring Potential for Oral Delivery of Monoclonal Antibodies for Local Treatment of Inflammatory Bowel Disease

Vipul Yadav Intract Pharma Limited

OLIGONUCLEOTIDE THERAPEUTICS: PRECLINICAL AND CLINICAL DEVELOPMENT (continued)


Exhibit Hall Map

aapptec . . . . . . . . . . . . . . . . . . . . . . . . . . 605Advanced Analytical Technologies . . 116Advanced ChemTech (A Division of CreoSalus) . . . . . . . . . . . 704Advion, Inc . . . . . . . . . . . . . . . . . . . . . . . . 717Agilent Technologies, Inc . . . . . . . . . . . 310AIC, Inc . . . . . . . . . . . . . . . . . . . . . . . . . . . . 502Ajinomoto Co ., Inc . . . . . . . . . . . . . . . . . 316Almac Group . . . . . . . . . . . . . . . . . . . . . . 702AM Chemicals . . . . . . . . . . . . . . . . . . . . . 714AmbioPharm, Inc . . . . . . . . . . . . . . . . . . 216Asahi Kasei Bioprocess America Inc . 102Avanti Polar Lipids, Inc . . . . . . . . . . . . . . 607Bachem Americas, Inc . . . . . . . . . . . . . . 203

BCN Peptides S .A . . . . . . . . . . . . . . . . . . 403BioAutomation . . . . . . . . . . . . . . . . . . . . 313Biocomma Biotech Co ., Ltd . . . . . . . . . 404BioSpring . . . . . . . . . . . . . . . . . . . . . . . . . 507Bio-Synthesis, Inc . . . . . . . . . . . . . . . . . . 707Biotage . . . . . . . . . . . . . . . . . . . . . . . . . . . 606BioTechLogic . . . . . . . . . . . . . . . . . . . . . 407Bio-Works . . . . . . . . . . . . . . . . . . . . . . . . . 719Carbosynth LLC . . . . . . . . . . . . . . . . . . . 402CEM . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417ChemGenes . . . . . . . . . . . . . . . . . . . . . . . 410CMIC, Inc . . . . . . . . . . . . . . . . . . . . . . . . . . 217Corden Pharma . . . . . . . . . . . . . . . . . . . 302

CPC Scientific . . . . . . . . . . . . . . . . . . . . . 303CS Bio . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212DAISO Fine Chem USA, Inc . . . . . . . . . . 519Eurofins Genomics . . . . . . . . . . . . . . . . 324Exiqon . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311GE Healthcare BioSciences . . . . . . . . . 107GeneDesign, Inc . . . . . . . . . . . . . . . . . . . 318Glen Research Corporation . . . . . . . . . 213Granlen, Inc . . . . . . . . . . . . . . . . . . . . . . . . 516Guangzhou Ribobio Co ., Ltd . . . . . . . . 319Gyros . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Hamari Chemicals, USA Inc . . . . . . . . . 705Honeywell Research Chemicals . . . . 211

Exhibit & Poster Hall Viewing HoursMonday, May 1: 12:30 pm - 6:30 pmTuesday, May 2: 10:00 am - 6:30 pmWednesday, May 3: 7:00 am - 10:55 am


Exhibit Hall Map

Hongene Biotechnology Ltd . . . . . . . . . 517Hybio Pharmaceutical . . . . . . . . . . . . . 518Intavis . . . . . . . . . . . . . . . . . . . . . . . . . . . . 418Interchem/Anygen Co . . . . . . . . . . . . . . 706Interchim Inc . . . . . . . . . . . . . . . . . . . . . . 711Intertek . . . . . . . . . . . . . . . . . . . . . . . . . . . 603JenKem Technology USA Inc . . . . . . . . 604Kinovate Life Sciences Inc . . . . . . . . . . 406LGC . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 117MilliporeSigma . . . . . . . . . . . . . . . . . . . . 503Nagase & Co . Ltd . . . . . . . . . . . . . . . . . . . 419Neuland Laboratories . . . . . . . . . . . . . . 703Nitto Avecia . . . . . . . . . . . . . . . . . . . . . . . 510

NOF Corporation . . . . . . . . . . . . . . . . . . 616Novasep . . . . . . . . . . . . . . . . . . . . . . . . . . 210PerkinElmer . . . . . . . . . . . . . . . . . . . . . . . 618Phenomenex . . . . . . . . . . . . . . . . . . . . . . 207Polymun Scientific . . . . . . . . . . . . . . . . . 219PolyPeptide Group . . . . . . . . . . . . . . . . . 202PPD . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105Pyramid Laboratories . . . . . . . . . . . . . . 602QPS Holdings LLC . . . . . . . . . . . . . . . . . 307Quanta BioDesign . . . . . . . . . . . . . . . . . 112Sai Life Sciences . . . . . . . . . . . . . . . . . . . 716 ShenZhen JYMed Technology Co ., Ltd 118ST Pharm . . . . . . . . . . . . . . . . . . . . . . . . . 317

Sunresin New Materials Co ., Xi'an . . . 712Sussex Research . . . . . . . . . . . . . . . . . . 610Syngene International Ltd . . . . . . . . . . 612Synthego . . . . . . . . . . . . . . . . . . . . . . . . . 106Thermo Fisher Scientific . . . . . . . . . . . 306Tosoh Bioscience . . . . . . . . . . . . . . . . . . 416TriLink BioTechnologies . . . . . . . . . . . . 305United States Pharmacopeia (USP) . . 110USV Private Ltd . . . . . . . . . . . . . . . . . . . . 206Waters Corporation . . . . . . . . . . . . . . . . 708Wave Life Sciences . . . . . . . . . . . . . . . . CS4Yield Engineering Systems . . . . . . . . . 710Zeochem AG . . . . . . . . . . . . . . . . . . . . . . 218


Featured Exhibitors

Booth #310Trusted Solutions for Oligo Therapeutics . Agilent's Nucleic Acid Solutions Division

(www.Agilent.com) offers industry leading experience to efficiently advance your lead oligo candidates from the clinic to market with a common goal of patient health and safety . Agilent has experience with all classes of oligo APIs and supports customers from toxicology through commercialization . With Agilent, you always have peace of mind by partnering with a company that has the financial resources, stability and vision required for long-term success. Contact us at: [email protected] and find out for yourself why the world's most revolutionary biotech companies and their Big Pharma partners are choosing Agilent Technologies to develop and commercialize their oligo APIs . www.agilent.com/chem/nucleicacid

Booth #502AIC has been supporting all phases of the synthesis industry with high quality reagents for more than 25 years . We are one of the largest suppliers of Activators

and PADS reagent in the US . We offer Tedia High Purity Solvents and Ancillary Reagents, Thermo Fisher Phosphoramidites, Link Technologies Specialty Phosphoramidites, and EMP Biotech Zetasep Products under exclusive US agreements . www.aicma.com/atd

Booth #316AJINOMOTO offers oligonucleotides and peptides manufacturing service named

AJIPHASE® . AJIPHASE® · Realizes the concept of solid phase in liquid phase by using ""Anchor"" instead of Resin .• Achieves cost reduction and steady supply in large scale• We have completed a peptide manufacturing under GMP using AJIPHASE®

and also successfully manufactured oligonucleotides in several Kg scale using AJIPHASE® .

http://www.ahs.ajinomoto.com/products/bio/bio_4.html

Booth #702Almac Group is a leading contract development and manufacturing organisation, with over 40

years’ experience, providing an extensive range of integrated services to over 600 biopharma companies globally . Services range from R&D, biomarker discovery and development, API manufacture, formulation development, clinical trial supply, IRT technology (IVRS/IWRS) through to commercial-scale manufacture . www.almacgroup.com

Booth #216AmbioPharm, Inc . (APi) is a full-service peptide manufacturing company headquartered in North Augusta,

SC . In cGMP facilities in the USA and Shanghai, China, we develop highly efficient processes for manufacturing small to very large scale Active Pharmaceutical Ingredients used in New Chemical Entities and generic peptides by clients worldwide .) ambiopharm.com

Booth #102Asahi Kasei Bioprocess is the established global market leader in virus removal filtration,

with Planova filter range having an unrivalled 25-year history of trusted use in the production of biotherapeutics . Asahi Kasei Bioprocess is also providing other innovative products: Cellufine™ chromatography resins, BioCradle™ microcarriers and equipment (inline buffer dilution system, filtration skid, chromatography system) . Asahi Kasei Bioprocess seeks to deliver the very best in not only the products and technologies we manufacture, but also in the technical support swe provide through our worldwide technical support network of specialists . http://www.ak-bio.com

Booth #203Bachem provides comprehensive services to the pharma and biotech industries .

It specializes in the development of innovative, efficient manufacturing processes and the reliable production of peptide-based APIs . The group has a global reach with more experience and know-how than any other company in the industry . Bachem . Pioneering Partner for Peptides .www.bachem.comwww.bachem.com/customers/api-new-chemical-entities/

Booth #313For over 17 years, BioAutomation has been the industry leader in producing the most flexible

and highest quality DNA/RNA synthesizers on the market . With this expertise in mind, we now offer high purity amidites for a wide range of synthesis chemistries, as well as solvents, columns and desiccant trap packs, for the Oligo market . www.bioautomation.com

Booth #707Bio-Synthesis, a biology CRO company can help by partnering with you in developing and manufacturing all

your synthetic biology projects . Bio-Synthesis offers leadership and commitment in delivering quality products with over 30 years of experience in producing synthetic peptides, highly modified oligonucleotides, sophisticated chimeras, BNA, siRNA coupled to other molecules, molecular biology products and biomolecule conjugates for all your diagnostic and therapeutic development along with complete technical support . All products are manufactured under ISO quality standards in our facilities located in Texas, USA .

Booth #410ChemGenes Corporation, an ISO 9001 certified

company established in 1981, is the industry leader in manufacturing oligonucleotide synthesis reagents and has consistently provided the highest quality phosphoramidites and solid supports in the market . Our facility, just outside of Boston/Cambridge Massachusetts USA, is setup for bulk manufacturing of therapeutic grade phosphoramidite and solid support DNA/RNA synthesis products for GMP grade oligonucleotide manufacturing . Additionally, ChemGenes carries the widest variety of modified phosphoramidites and supports currently used in oligonucleotide synthesis including Microarray Technology, Oligonucleotide Therapeutics, Oligonucleotide Based Probes and other areas of Nucleic Acid research . ChemGenes remains devoted to providing you with invaluable customer service and comprehensive technical support .www.chemgenes.com

Booth #302CordenPharma is your full-service CDMO for APIs, Drug Products and Packaging .

Through cGMP facilities organized under Technology Platforms - Peptides, Oligonucleotides, Lipids, & Carbohydrates - Injectables - Highly Potent & Oncology - Small Molecules - Antibiotics - CordenPharma experts translate complex processes at any stage of development into high-value products . Visit www .cordenpharma .com or Contact Us http://www .cordenpharma .com/contact-us/ www.cordenpharma.com

Booth #617Formed by the merger of Gyros AB and

Protein Technologies, Inc ., Gyros Protein Technologies AB is a provider of peptide synthesis and bioanalytical tools for discovery, development and manufacturing of biotherapeutics . The Company combines Gyrolab’s nanoliter-scale immunoassay innovation with Protein Technologies’ expertise in peptide synthesizers scaling from 0 .001-200 mmol . www.gyros.com

continued on page 26


Featured Exhibitors

Booth #211Honeywell Burdick & Jackson is a leading manufacturer of high-purity solvents and DNA/RNA reagents . But that isn’t all – our growing portfolio now includes other trusted brands like Fluka®, Chromasolv® and Hydranal® . Our packaging solutions, ranging from lab scale through production, provide our customers with added value, increased efficiency and enhanced safety. labchemicals-honeywell.com

Booth #517Hongene Biotechnology Ltd . was established in

1998, and using cutting edge technology, is a dedicated manufacturer in the fields of nucleosides, nucleotides and phosphoramidites in China. Hongene is continuously developing new products in this area of science . Our experts are also happy to offer CRO/CMO services and custom synthesis of these and related products essential for your R&D, helping to bring your products to market more efficiently. www.hongene.com

Booth #503Sigma-Aldrich and EMD Millipore have joined

forces to create MilliporeSigma, the U .S . life science business of Merck KGaA, Darmstadt, Germany . With 19,000 employees and 72 manufacturing sites worldwide, MilliporeSigma’s portfolio spans more than 300,000 products enabling scientific discovery. At TIDES, we’ll be discussing our combined portfolio of an extensive line of products, from Proligo Reagents® to production-scale organic synthesis . sigma-aldrich.com/proligo

Booth #419Nagase introduces unique technologies for

peptide and nucleotide area . One is Liquid-Phase Oligonucleotide Synthesis by Segment Condensation and another is Maruoka Catalyst® which can create UNAA (Unnatural amino acid). LPOSSC brings high output and less purification loss . See you on Day 3 at Breakfast Presentation by Mr . Kataoka .www.nagase.co.jp

Booth #510With over 20 years of experience in

oligonucleotide development and production, and over 1000 sequences manufactured, Nitto Denko Avecia Inc . is a leader in the advancing oligo therapeutic market . Our mission is to continue to build value for our customers as they progress through drug development into commercialization . www.avecia.com

Booth #219Polymun Scientific is a family-owned company in Klosterneuburg, Austria, founded in 1992 . The

company's core activities are contract development and GMP production of biopharmaceuticals and liposomal formulations of active pharmaceutical ingredients as well as vaccine antigens - for clinical studies and the market . www.polymun.com

Booth #202The PolyPeptide Group is a leading provider of proprietary GMP, generic and non-GMP

peptides and peptide conjugates for the pharmaceutical, cosmetic, and biotech industries as well as basic research . With 6 GMP facilities located across 3 continents, the Group supports peptide projects at all scales from pre-clinical through to commercialization . www.polypeptide.com

Booth #602PYRAMID is a contract aseptic manufacturing, Lyophilization and analytical service provider for sterile

injectable drugs . PYRAMID provides expertise in formulation and process development, aseptic filling for vials and syringes, as well as Lyophilization applications for Clinical and Commercial products . PYRAMID has established a reputation of exceptional performance, integrity and quality . www.pyramidlabs.com

Booth #112The Quanta BioDesign dPEG® product line is a unique technology platform, single MW PEGs, which

can be tailored to meet specific physical and chemical requirements in a broad array of diagnostic and therapeutic applications . Chemistry applications which incorporate dPEG® products include conjugations, chemical modifications, cross linking, modification of biological therapeutics. www.quantabiodesign.com

Booth #319Guangzhou RiboBio Co ., Ltd . is a leading

oligonucleotide-based biotech enterprise in China with the world-class R&D team and the cutting-edge technologies . The company develops and supplies innovative DNA/RNA, RNAi and miRNA products, as well as provides diverse oligonucleotide related R&D services. RiboBio, the first and unique cGMP production base in China with the CFDA drug production license, established a comprehensive oligonucleotide manufacturing facility, and equipped with the state-of-the-art instruments and advanced techniques . www.ribobio.com

Booth #306Thermo Fisher Scientific is a developer and

manufacturer of standard and customized phosphoramidites (including TheraPure® ™ and DyLight™ phosphoramidites) used in the production of oligonucleotides and modified specialty nucleic acids for research, diagnostics and therapeutic applications. Thermo Fisher Scientific is your industrial partner of choice offering over 25 years of manufacturing experience, a mature quality system and a cross functional project team approach . Our Milwaukee-based facility is dedicated to meeting the growing needs of the market .

Booth #708Waters Corporation creates business

advantages for laboratory-dependent organizations by delivering scientific innovation to enable customers to make significant advancements. Waters helps customers make profound discoveries, optimize laboratory operations, deliver product performance, and ensure regulatory compliance with a connected portfolio of separations and analytical science, laboratory informatics, mass spectrometry, as well as thermal analysis . www.waters.com

Booth #CS4WAVE Life Sciences is a genetic medicine company focused on stereopure nucleic acid therapies for patients impacted by rare diseases . We are utilizing our proprietary synthetic

chemistry platform to design and develop these therapeutics . WAVE’s pipeline spans multiple oligonucleotide modalities including antisense, exon-skipping and single-stranded RNAi . www.wavelifesciences.com


Firoz Antia, Ph.D., Director, Chemical Process R&D, Biogen

Mimoun Ayoub, Ph.D., Director, Global Peptides and Injectables, Corden Pharma, Switzerland

Fabrizio Badalassi, Ph.D., Director, Chemical Development, Ferring Pharmaceuticals, Denmark

Neil H. Baine, Ph.D., President, Neil Baine Consulting, LLC and Past Executive Director, Scinovo, Preclinical Development, GlaxoSmithKline Pharmaceuticals

Doug Brooks, Ph.D., Vice President, Manufacturing Development, Ophthotech Corporation

Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals

David Brown, Ph.D., Director, Discovery and in vivo Research, Mirna Therapeutics, Inc.

Waleed Danho, Ph.D., Consultant

Jesse Z. Dong, Ph.D., Vice President, Peptide Chemistry, Neon Therapeutics

Alex Fässler, Ph.D., COO Europe, Bachem AG, Switzerland

Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, Quark Pharmaceuticals, Israel

Chris Holmes, Ph.D., Former Vice President, Chemical and Pharmaceutical Development, Regado Biosciences

Troels Koch, Ph.D., VP & Head of Research, RNA Therapeutics, Roche Innovation Center Copenhagen, Denmark

Jesper Lau, Vice President, Protein & Peptide Chemistry, Novo Nordisk A/S

David Litzinger, Ph.D., President, David Litzinger and Associates, LLC

Jennifer Lockridge, Ph.D., Vice President, Program Development, Dicerna, Pharmaceuticals

John Mayer, Ph.D., Principal Research Scientist, Novo Nordisk

Muthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals, Inc.

Michael McGinley, Senior Product Manager, BioSeparations, Phenomenex

Fanyu Meng, Ph.D., Principal Scientist, Analytical Research and Development, Merck Research Labs

Bruce H. Morimoto, Ph.D., Executive Director, Applied Translational Medicine, Celerion

Gary F. Musso, Ph.D., President, Musso and Associates LLC

Lubomir V. Nechev, Ph.D., Vice President, Process Sciences, Alnylam Pharmaceuticals

John M. Nuss, Ph.D., Vice President, Drug Discovery, Ferring Research Institute

Pamela Pavco, Ph.D., Chief Development Officer, RXi Pharmaceuticals

El Djouhar Rekai, Ph.D., Head of Operation Products, PolyPeptide Group, Belgium

Claus Rentel, Ph.D., Executive Director, Analytical Development/Quality Control, Ionis Pharmaceuticals, Inc.

Christopher A. Rhodes, Ph.D., President and CEO, Drug Delivery Experts

Joel Richard, Ph.D., Senior Vice President, Peptides, Head of CMC Dreux Site, IPSEN, France

Christoph Rosenbohm, Ph.D., VP and Head of Discovery Operations, Roche Innovation Center Copenhagen, Denmark

Dmitry Samarsky, Ph.D., Chief Scientific Officer, Silence Therapeutics, Germany

Tomi Sawyer, Ph.D., Distinguished Scientist & Head, Peptide Drug Discovery and Innovative Technologies, Merck Research Laboratories

Trishul Shah, Associate Director, Business Development, North America, PolyPeptide Laboratories

Ved Srivastava, Ph.D., Vice President of Chemistry, Intarcia Therapeutics, Inc.

G. Susan Srivatsa, Ph.D., President, ElixinPharma

James Thompson, Ph.D., Head of CMC, Moderna Therapeutics

Blake Unterreiner, Business Development Manager, Nucleic Acid Solutions Division, Agilent Technologies, Inc.

Sridhar Vaddeboina, Ph.D., Vice President, Analytical Development, Nitto Denko Avecia

Paul Watt, Ph.D., Chief Scientific Officer, Phylogica Ltd., Australia

Fran Wincott, Ph.D., President, Wincott & Associates, LLC

TIDES 2017 Advisory Board


DRIVING THE FUTURE OF THE LIFE SCIENCE INDUSTRY

Hynes Convention Center September 25-28, 2017 • Boston, MA

BiotechWeekBoston.com

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TIDES: Oligonucleotide & Peptide...

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