tides: oligonucleotide & peptide therapeutics san diego...

TIDES: Oligonucleotide &Peptide Therapeutics

April 30 - May 3, 2017Manchester Grand Hyatt San DiegoSan Diego, CA

THE LARGEST MEETING TO ACCELERATE OLIGONUCLEOTIDE AND PEPTIDE PRODUCTS FROM EARLY DISCOVERY TO LATE-STAGE DEVELOPMENT & COMMERCIALIZATION

Industry Experts Present Innovative Strategies to Fast-Track Product Development

Marvin Caruthers, Ph.D. Distinguished Professor, Chemistry and Biochemistry UNIVERSITY OF COLORADO

Bradley L. Pentelute, Ph.D. Professor, Chemistry, MASSACHUSETTS INSTITUTE OF TECHNOLOGY

Clinical Development of PMOs to Treat Duchenne Muscular Dystrophy

Edward Kaye, M.D. President, CEO and Chief Medical Officer SAREPTA THERAPEUTICS

Synthesis of DNA/RNA on Chips and Biochemical/Biological Activity of New Morpholino DNAs

Site Selective Antibody Drug Conjugates Enabled by Cysteine Arylation and Native Conjugations

Event Partner: Register Early and Save www.TIDESevent.com

James R. Heath, Ph.D Elizabeth W. Gilloon Professor of Chemistry, CALIFORNIA INSTITUTE OF TECHNOLOGY

Michael E. Kopach, Ph.D. Research Advisor, ELI LILLY AND COMPANY

Advancing Greener Peptide and Oligonucleotide Synthesis

Epitope Targeted Peptide-based Protein Catalyzed Capture Agents

850+ OLIGONUCLEOTIDE AND PEPTIDE SCIENTISTS AND EXECUTIVES

Fast-track your oligonucleotide and peptide research to the clinic and beyond by collaborating with leading pharma, biotechs, academia and solution providers from North America, Asia and Europe.

125 CASE STUDIES AND NEW DATA PRESENTATIONS

90+EXHIBITORS Connect with leading manufacturing, technologies, and service providers to drive your promising therapeutic towards commercial success.

40+PEER-SUBMITTED POSTERSStay at the forefront of oligonucleotide and peptide innovation by accessing cutting-edge data from fellow attendees.

TIDES IS THE GLOBAL MEETING PLACE AND THE NUMBERS SPEAK FOR THEMSELVES

For up-to-date program information and new abstracts, visit: www.TIDESevent.com 2

“TIDES is our main event for peptides and oligo delivery. Anyone working in this market in either development or supply must be there” Abby Thompson, CordenPharma

“An excellent combination of networking opportunities and learning about oligonucleotide and peptide drug discovery and development”Mimoun Ayoub, CordenPharma International

Use the TIDES conference app to contact any of the 850+ TIDES speakers, attendees, exhibitors and sponsors before, during or after the TIDES conference. Use the app to schedule a meeting, browse the attendee list or view the updated conference agenda. More details and login information for the TIDES conference app will be provide to all registered attendees.

CONNECT WITH FELLOW TIDES ATTENDEES AND AUTOMATICALLY SCHEDULE MEETINGS WITH THETIDES CONFERENCE APP

NEW THIS YEAR! 2 TRACKS ON mRNA AND CRISPR THERAPEUTICSLearn industry best practices for overcoming delivery and regulatory challenges, advance mRNA and CRISPR therapeutics into Phase 1, 2, and 3 clinical trials, and optimize the properties of your complex molecules.

NEW: Non-clinical Development of Oligonucleotides Session

Covering the entire spectrum of oligonucleotide and peptide development and production

3Register Early for Best Savings • www.TIDESevent.com • 888-670-8200

AGENDA AT-A-GLANCESunday, April 30, 2017 • Pre-Conference Workshops 8:00 am - 12:00 pm Workshop #1:

Analytical and Purification Strategies for Peptide

Manufacturing (Cortez Hill ABC)

Workshop #2: Emerging Technologies in Oligonucleotide Based Therapeutics: Challenges for Manufacturing and Controls

of Novel Chemistries (Hillcrest CD)

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

(Hillcrest CD)

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 Torrey Hill Cortez Hill

Quality of Oligonucleotide Starting Materials Oligonucleotide Discovery and Preclinical

CMC, Quality and Regulatory Aspects of Peptide APIs

Peptide Targets, Peptide Discovery and SynthesisGalNAc Conjugates: From Starting Materials

to Manufacturing Track 1: Oligonucleotide Chemistry

Manufacturing and ControlsTrack 2: Oligonucleotide Discovery,

Preclinical and ClinicalTrack 5:

mRNA TherapeuticsTrack 3: Peptide Chemistry Manufacturing and Controls


Seaport B Seaport C Golden Hill Seaport A Cortez Hill10:45 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 Modality

12:45 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 Learned

mRNA 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 (Invitation Only) 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

Track 2: Oligonucleotide Discovery, Preclinical and Clinical

Track 3: Peptide Chemistry Manufacturing and Controls


Harbor HI Harbor G Harbor E Harbor D

10:55 am - 12:30 pm Analytical Strategies for Oligonucleotides

CRISPR and Genome Editing ApplicationsAnalytical Strategies to Enable

Peptide Development Peptide 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)

For up-to-date program information and new abstracts, visit: www.TIDESevent.com

SUNDAY, APRIL 30, 2017 • PRE-CONFERENCE WORKSHOPS • CHOOSE ONE FULL DAY WORKSHOP OR 2 HALF-DAY WORKSHOPS

Workshop #1: ANALYTICAL AND PURIFICATION STRATEGIES

FOR PEPTIDE MANUFACTURINGFull Day Workshop • 8:00am-5:00pm

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

Confirmed Speakers:

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 APIsTobias 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 SpectrometryJiri 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 Development

Regina 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

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

Fran Wincott, Ph.D., Wincott and Associates

The definition of an oligonucleotide therapeutic has greatly expanded in the past few years with the introduction of novel technologies such as messenger RNAs and CRISPR/CAS9 systems. Furthermore, even the more established oligonucleotide modalities (antisense, siRNA, aptamers, microRNA, etc.) have seen a dramatic increase in the complexity of drug candidates being promoted into preclinical and clinical development. These oligonucleotides often have significant chemical modifications requiring specialty starting materials as well as technical innovations in process development, analytical chemistry, manufacturing and controls. This places extraordinary demands on both sponsor companies and contract service organizations to meet regulatory expectations while ensuring patient safety, often under aggressive timelines. This workshop will present case studies of CMC development of emerging technologies from the perspective of sponsors, CMO and Quality Assurance.

8:00 Introductory RemarksG. 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


10:10 Manufacturing Innovation through PartnershipEduardo 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


7:15 Workshop Registration

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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

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

This workshop will provide a high level primer on the use of CRISPR and genome editing technologies in both research and therapeutic applications. An overview of the discovery, preclinical, clinical, CMC, manufacturing and regulatory considerations for CRISPR and genome editing will be discussed. The current challenges and opportunities of CRISPR and genome editing technologies will also be presented.

Topics to be Discussed:

• Creative Chemical Modifications and Synthesis Approaches

• Considerations for Safety, Quality, Regulatory and Off-target Effects

• Advances in Genome Editing Technologies

• Delivery Strategies

• What Can Be Learned from Oligonucleotides and mRNA?

• Applications of CRISPR in Research and Therapeutics

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 ApplicationssCecilia Fernández, Ph.D., Director, Platform Development, Editas Medicine

8:45 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

9:15 Synthetic sgRNA Enables Highly Efficient and Consistent CRISPR Editing of Cells for Therapeutic ApplicationsKevin Holden, Ph.D., Head of Synthetic Biology, Synthego


10:15 Applying Agilent's CRISPR/Cas Technology and Services to the Current Market with Emphasis on gRNA Production and AnalysisJoe 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 TherapeuticsMuthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals

11:15 Panel Discussion

12:00 Close of Workshop

Workshop Leaders:Gary Musso, Ph.D., President, Musso and Associates LLCTrishul Shah, Associate Director Business Development, PolyPeptide Laboratories Inc.Peptides are being developed for a diverse set of clinical uses and have a variety of potencies. Because processes for manufacture of peptides use common platforms, peptides are often prepared in multi-use (non-dedicated) environments. Multi- use facilities for non-commercial manufacture present several risk assessment issues that are gaining increasing regulatory scrutiny. Key topics include dedicated vs multiuse, cleaning validation, occupational containment, facilities design, quality aspects of control strategies.

Topics to be Discussed:

• OEL Banding

• Regulatory Requirements for Cleaning Validation

• Multi-use facilities- Design and Support Considerations

• Cleaning Validation

• Risk Assessment and Containment Strategies for Peptide Drug Products

Confirmed Speakers: 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


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


7:15 Workshop Registration

Workshop #4: PEPTIDE MANUFACTURING RISK ASSESSMENT AT CMOS,

A MULTI-PRODUCT ENVIRONMENTMorning Half-Day Workshop • 8:00am-12:00pm

6

This workshop will focus on quality expectations for oligonucleotide drug products. A number of new guidances have emerged from the FDA and the EMA recently, and this workshop will explore the application of the underlying quality concerns in those guidances to oligonucleotide drug products. The case studies chosen will span early development through marketed products, in all cases maintaining a focus on the oligonucleotide.Topics to be Discussed:

• Emerging Regulatory Expectations Regarding Quality of Drug Products

• Terminal Sterilization

• Combination Products• Co-formulated Products

• Complex Drug Products

1:00 Welcome and Introduction to WorkshopJames 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


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



Register Early for Best Savings • www.TIDESevent.com • 888-670-8200

Workshop #5: QUALITY CONSIDERATIONS FOR OLIGONUCLEOTIDE DRUG PRODUCTS

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

This workshop will provide a high level primer on the development of messengerRNA (mRNA) therapeutics. An overview of the discovery, preclinical, clinical, CMC, manufacturing and regulatory considerations for mRNA therapeutics will be discussed. The current challenges and opportunities of these complex molecules will also be presented. Topics to be discussed include:

• The Anatomy of an mRNA

• mRNA Chemistries

• Manufacturing Strategies for mRNA

• Control Strategies for mRNA

• Applications of mRNA as Therapeutics and Vaccines

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.


3:30 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


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




Workshop 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


3:30 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-5:00pmWorkshop 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. Peptides offer notable advantages as drugs including their high biological activity, high specificity and low toxicity. However, peptides also present challenges in drug development. (1) short half-life because they are quickly broken down by proteolytic enzymes; (2) inadequate drug-like pharmaceutical properties such as solubility and chemical and physical stability (3) the polar, chemical nature of peptides prevent them from getting past physiological barriers or membranes, and (4) need to be parenterally delivered (via injection) because oral administration would lead to their degradation in the digestive tract. This workshop will focus on cutting-edge, novel technologies to optimize peptides as therapeutics. Case studies will illustrate the use of these platform technologies to enhance the pharmaceutic properties of peptides. Topics to be Discussed:

• Stabilizing Peptide Backbone • Half-life Extension Technologies • Creative Formulation Approaches

• Strategies to Improve Peptide Delivery • Other Approaches to Improve Peptide Properties

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


3:30 Recent Advances in Peroral Delivery of Peptide DrugsNozer 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



MONDAY, MAY 1, 2017 • KEYNOTE/PLENARY SESSION

9:00Edward Kaye, M.D. President, CEO and Chief Medical Officer, Sarepta Therapeutics

Clinical Development of Eteplirsen for the Treatment of Duchenne Muscular Dystrophy (DMD)DMD is a rare, X-linked disease that results in progressive muscle loss and premature death. DMD is primarily caused by whole exon deletions in the dystrophin gene resulting in an out-of-frame shift of the dystrophin mRNA reading frame that prevents production of functional dystrophin protein. PMO eteplirsen was recently granted accelerated approval for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. The approval and lessons learned will be discussed along with next-generation PMOs designed to improve upon delivery and efficacy.

7:45 Registration and Coffee

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

11:00Marvin Caruthers, Ph.D. Distinguished Professor, Chemistry and Biochemistry, University of Colorado

Synthesis of DNA/RNA on Chips and Biochemical/Biological Activity of New Morpholino DNAsWe have synthesized imidoamidate DNA having two equivalent nitrogen groups at the internucleotide, nonbonding positions and a large number of morpholino/thiomorpholino DNAs. These analogues were tested for biological activity. In collaboration with Agilent Technologies, we have developed synthesis methods for preparing DNA/RNA containing up to 300 nucleotides per segment. Applications include biological research on genomes, RNA for CRISPR/Cas9 applications, and data storage. The lecture will focus on these research areas.

9:45

Lex Van der Ploeg, Ph.D.Chief Scientific Officer, Rhythm Pharmaceuticals

Setmelanotide for the Treatment of Rare Monogenic Forms of Obesity: The MC4-Pathway

10:30 Networking Refreshment Break

12:30 Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break

Co-Sponsored by:


11:45Bradley L. Pentelute, Ph.D. Professor, Chemistry, Massachusetts Institute of Technology

Site Selective Antibody Drug Conjugates Enabled by Cysteine Arylation and Native ConjugationHere we report a robust bioconjugation method using cysteine arylation. This chemistry enables site-specific conjugation at cysteine residues within peptides, proteins, and antibodies. Our two developed approaches use either perfluoroaryl-cysteine SNAr chemistry or organometallic palladium reagents. This work led to the discovery of a self-labeling four-residue sequence that enables regioselective conjugation at only one cysteine residue within an intact antibody containing natural amino acids. Recently, we discovered a new approach for the native conjugation of complex natural products such as vancomycin onto peptides and proteins without the introduction of linkers or chemical handles.

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MONDAY, MAY 1, 2017 • KEYNOTE/PLENARY SESSION


4:00Michael E. Kopech, Ph.D. Executive Director, Technical Operations, Radius Health, Inc.

Advancing Greener Peptide and Oligonucleotide Synthesis Peptide and Oligonucleotide products are an area of significant growth within the biopharma industry. For example, there are approximately 140 peptide based active pharmaceutical ingredients (API’s) in clinical trials and more than 400 in pre-clinical development. However, the current state of the art in peptide and oligonucleotide syntheses utilize primarily legacy technologies, with little focus on green chemistry and engineering. Waste generated from current peptide and oligonucleotide processes ranges from 3,000-15,000 kg/kg API (10-50 mer products) with multiple usages of highly hazardous reagents and solvents. Contributing to the poor environmental profile is the pervasive and extensive use of chromatography to produce peptide and oligonucleotide products with required quality attributes. In 2016, the ACS Pharmaceutical Roundtable identified the need for greener syntheses for peptide and oligonucleotide products as a key research priority. This presentation will describe the newly formed ACS Pharmaceutical Roundtable Greener Peptide, Oligonucleotide and ADC’s project team initial activities in these important areas.

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

1:55 Mayra B. Reyes-Armour, Ph.D.Director, Asset Lead, Biogen

SPINRAZA: The Road to Approval Spinal Muscular Atrophy (SMA) is a serious, debilitating, and life-threatening disease characterized by degeneration of the motor neurons in the anterior horn of the spinal cord, resulting in atrophy of the voluntary muscles of the limbs and trunk. There was no treatment for this disease until SPINRAZA was approved late last year by the FDA. The road to SPINRAZA's approval including high level clinical results and CMC strategy will be discussed.

2:40Richard S. Geary, Ph.D.Senior Vice President, Development, Ionis Pharmaceuticals, Inc.

Innovations in Antisense RNA Targeting: Driving Value, Targeting the SourceSecond generation 2’-MOE and cEt modified antisense therapeutics have a well understood PK/PD profile. We have shown that activity is not limited to the liver and mechanisms are not limited to mRNA reduction. A mature, growing and innovative development Ionis pipeline update coupled with improvements in chemistry and innovation in cell targeted delivery and mechanism of action will be discussed.

3:15 Networking Refreshment Break in Poster and Exhibit Hall

Co-Sponsored by:

4:30James R. Heath, Ph.D.James R. Heath, Ph.D., Elizabeth W. Gilloon Professor of Chemistry, California Institute of Technology

Epitope Targeted Peptide-based Protein Catalyzed Capture AgentsI will discuss the all synthetic platform technology of protein catalyzed capture agents (PCCs) that we have developed over the past few years. In particular, I will focus on methods for the high-throughput production of these ligands, and methods for engineering these ligands for low picoM-type affinities and in vivo applications.

5:00-6:30pmNetworking 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:

TUESDAY, MAY 2, 2017


Oligonucleotide Chemistry, Manufacturing and Controls

Oligonucleotide Discovery, Preclinical and Clinical

Peptide Chemistry Manufacturing and Controls

Peptide Discovery, Preclinical and Clinical

7:30 Registration and Coffee Sponsored by:

7:45 - 8:15

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

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

8:25 (7:55) Chairman’s RemarksLubomir 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 CMC, Quality and Regulatory Aspects of Peptide APIs

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 StudiesGalNAc 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

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


Oligonucleotide Discovery, Preclinical and Clinical mRNA Therapeutics Peptide Chemistry

Manufacturing and ControlsPeptide Discovery,

Preclinical and ClinicalGalNAc Conjugates:

From Starting Materials to Manufacturing (continued)

Oligonucleotide Discovery and Preclinical (continued)

Discovery and Development of mRNA Therapeutics and Vaccines Chairperson: Örn Almarsson, Ph.D., Head of

Formulations, Moderna Therapeutics

CMC, Quality and Regulatory Aspects of Peptide APIs (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 Modality11:15 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

11

TUESDAY, MAY 2, 2017 (continued)


Oligonucleotide Discovery, Preclinical and Clinical mRNA Therapeutics Peptide Chemistry

Manufacturing and ControlsPeptide Discovery,

Preclinical and Clinical12:45 Networking Luncheon in Poster and Exhibit Hall

1:55 Chairman’s RemarksEmma 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 Remarks Chairman’s RemarksWaleed Danho, Ph.D., Distinguished Research Leader and Consultant

Innovative Processes and Synthesis Strategies for Oligonucleotides

Current Issues in the Nonclinical Development of

Oligonucleotide TherapeuticsmRNA Delivery Strategies Peptide CMC Case Studies

and StrategiesPeptides in the Clinic:

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

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

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

Discovery of Selepressin and Merotocin, Two Clinical Candidates from Neurohypophyseal Family of HormonesKazimierz Wisniewski, Ph.D., Senior Scientist II, Ferring Research Institute

3:00 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, Carbohydrates & Injectables Platforms, CordenPharma, Switzerland

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)

Current Issues in the Nonclinical Development of

Oligonucleotide Therapeuticss (continued)

mRNA Bioanalytical, CMC and Manufacturing (shared session)

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 Strategies4:45 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

5:30 Networking Reception in Poster and Exhibit Hall Sponsored by


Schedule Subject to Change

WEDNESDAY, MAY 3, 2017

Oligonucleotide Chemistry Manufacturing and Controls




7:00 - 8:25 Early Morning Exhibit Viewing and Breakfast in the Exhibit

Plenary Session: CRISPR and Genome Editing Applications8:25 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

10:55 Chairman’s RemarksPaul 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 Pharma SolutionsA New Dimension in LNA Therapeutics

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

12:30 Lunch on Your Own


13

WEDNESDAY, MAY 3, 2017

Oligonucleotide Chemistry Manufacturing and Controls




1:40 Chairman’s RemarksPaul 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 ClinicChristina Dalby, Ph.D., Director, Chemistry & Biochemistry, 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

Emerging Therapeutic Profile of RNA Interference against Hepatic TargetsPatrick 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


SHARE AND VIEW CUTTING-EDGE RESEARCH IN THE POSTER HALLShare your company’s research with 850+ global oligonucleotide and peptide professionals by presenting a poster during the event. Any registered attendee may present a poster. Submit your poster online at www.TIDESevent.com no later than March 31, 2017.

“Good way to understand what is happening in our market place” Christy Campbell, Thermo Fisher Scientific


MEET THE PEOPLE BEHIND THE PRODUCTS & GET THE ANSWERS YOU NEED

For more information on how you can connect with key buyers at TIDES 2017, contact: Patrick Shaughnessy (Companies A-N) at [email protected] or call 857-504-6684

Aimee Croke (Companies O-Z): +1.857.504.6697 [email protected]

EXHIBITORS with booth numbers

aapptec . . . . . . . . . . . . . . . . . . . . . . . . . . . 605Advanced Analytical Technologies . . . 116Advanced ChemTech (A Division of CreoSalus) . . . . . . . . . . . . 704Advion, Inc. . . . . . . . . . . . . . . . . . . . . . . . . 717Agilent Technologies, Inc. . . . . . . . . . . . 310AIC, Inc.. . . . . . . . . . . . . . . . . . . . . . . . . . . . 502Ajinomoto Co., Inc. . . . . . . . . . . . . . . . . . 316Almac Group . . . . . . . . . . . . . . . . . . . . . . . 405AM Chemicals. . . . . . . . . . . . . . . . . . . . . . 714AmbioPharm, Inc. . . . . . . . . . . . . . . . . . . 216Asahi Kasei Bioprocess America Inc. . 102Avanti Polar Lipids, Inc.. . . . . . . . . . . . . . 607Bachem Americas, Inc.. . . . . . . . . . . . . . 203BCN Peptides S.A. . . . . . . . . . . . . . . . . . . 403BioAutomation . . . . . . . . . . . . . . . . . . . . . 313Biocomma Biotech Co., Ltd . . . . . . . . . . 404Biopeptek, Inc.. . . . . . . . . . . . . . . . . . . . . . 713BioSpring . . . . . . . . . . . . . . . . . . . . . . . . . . 507Bio-Synthesis, Inc. . . . . . . . . . . . . . . . . . . 707Biotage . . . . . . . . . . . . . . . . . . . . . . . . . . . . 606BioTechLogic . . . . . . . . . . . . . . . . . . . . . . 407Bio-Works. . . . . . . . . . . . . . . . . . . . . . . . . . 719Carbosynth LLC . . . . . . . . . . . . . . . . . . . . 402CEM. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 417ChemGenes. . . . . . . . . . . . . . . . . . . . . . . . 410CMIC, Inc.. . . . . . . . . . . . . . . . . . . . . . . . . . 217Corden Pharma . . . . . . . . . . . . . . . . . . . . 302CPC Scientific . . . . . . . . . . . . . . . . . . . . . . 303CS Bio . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 212DAISO Fine Chem USA, Inc.. . . . . . . . . . 519Eurofins Genomics . . . . . . . . . . . . . . . . . 106Exiqon. . . . . . . . . . . . . . . . . . . . . . . . . . . . . 311GE Healthcare BioSciences . . . . . . . . . . 107GeneDesign, Inc. . . . . . . . . . . . . . . . . . . . 318Glen Research Corporation . . . . . . . . . . 213Granlen, Inc.. . . . . . . . . . . . . . . . . . . . . . . . 516Guangzhou Ribobio Co., Ltd . . . . . . . . . 319Gyros. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 617Hamari Chemicals, USA Inc. . . . . . . . . . 705Honeywell Research Chemicals . . . . . 211

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 . . . . . . . . . . . . . . . . . . . . . . . . 510NOF Corporation . . . . . . . . . . . . . . . . . . . 616Novasep . . . . . . . . . . . . . . . . . . . . . . . . . . . 210NSF Health Services . . . . . . . . . . . . . . . . 115PerkinElmer . . . . . . . . . . . . . . . . . . . . . . . . 618Phenomenex. . . . . . . . . . . . . . . . . . . . . . . 207Polymun Scientific . . . . . . . . . . . . . . . . . . 219PolyPeptide Group. . . . . . . . . . . . . . . . . . 202PPD . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 105Pyramid Laboratories . . . . . . . . . . . . . . . 602QPS Holdings LLC . . . . . . . . . . . . . . . . . . 307Quanta BioDesign . . . . . . . . . . . . . . . . . . 112ShenZhen JYMed Technology Co., Ltd 118ST Pharm. . . . . . . . . . . . . . . . . . . . . . . . . . 317Sunresin New Materials Co., Xi'an . . . . 712Sussex Research . . . . . . . . . . . . . . . . . . . 610Syngene International Ltd. . . . . . . . . . . 612Synthego . . . . . . . . . . . . . . . . . . . . . . . . . . 505Thermo Fisher Scientific . . . . . . . . . . . . 306Tosoh Bioscience. . . . . . . . . . . . . . . . . . . 416TriLink BioTechnologies . . . . . . . . . . . . . 305United States Pharmacopeia (USP). . . 110USV Private Ltd. . . . . . . . . . . . . . . . . . . . . 206Vetter Pharma. . . . . . . . . . . . . . . . . . . . . . 103Waters Corporation . . . . . . . . . . . . . . . . . 708Wave Life Sciences . . . . . . . . . . . . . . . . . CS4Yield Engineering Systems . . . . . . . . . . 710Zeochem AG . . . . . . . . . . . . . . . . . . . . . . . 218

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