tides: oligonucleotide & peptide therapeutics san diego ... · gaining increasing regulatory...

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

David C. Hanley, Ph.D. Executive Director, Technical Operations RADIUS HEALTH, INC.

Abaloparatide: Benchtop to Commercial Manufacture

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

Cell-penetrating Miniproteins

Gregory Verdine, Ph.D., Principal Investigator and Professor, Harvard University, HARVARD MEDICAL SCHOOL, and Founder, FOGPHARMA

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-GLANCE

Sunday, April 30, 2017 • Pre-Conference Workshops

AM Workshop #1: Analytical and Purification Strategies for

Peptide Manufacturing

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

Chemistries

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

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

PMWorkshop #5: Quality Considerations for

Oligonucleotide Drug ProductsWorkshop #6: An Introduction to

messengerRNA (mRNA) Therapeutics

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

of ADME and PK/PD Studies of Oligonucleotides

Workshop #8: Enhancing Pharmaceutical Properties Of Peptides

Monday, May 1, 2017 • Main Conference Exhibit Hall & Poster Viewing Hours: 3:00pm-6:30pm

AM

KEYNOTE/PLENARY SESSION: Edward Kaye, M.D., President, CEO and Chief Medical Officer, Sarepta Therapeutics Marvin Caruthers, Ph.D., Distinguished Professor, Chemistry and Biochemistry, University of Colorado David C. Hanley, Ph.D., Executive Director, Technical Operations, Radius Health, Inc. Bradley L. Pentelute, Ph.D., Professor, Chemistry, Massachusetts Institute of Technology 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 Gregory Verdine, Ph.D., Principal Investigator and Professor, Harvard University, Harvard Medical School, and Founder, FogPharma

PM PLENARY SESSION

Networking Reception in Poster and Exhibit Hall

Tuesday, May 2, 2017 • Main Conference Exhibit Hall & Poster Viewing Hours: 10:00 am – 6:30 pm

Oligonucleotide Chemistry Manufacturing and Controls

Oligonucleotide Discovery, Preclinical and Clinical

Peptide Chemistry Manufacturing and Controls

Peptide Discovery, Preclinical and Clinical

AM

Quality of Oligonucleotide Starting MaterialsOligonucleotide Discovery and Preclinical CMC, Quality and Regulatory Aspects of Peptide

APIs Peptide Targets, Peptide Discovery and SynthesisGalNAc Conjugates: From Starting Materials to Manufacturing



mRNA Therapeutics Peptide Chemistry Manufacturing and Controls


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

Networking Luncheon in Poster and Exhibit Hall

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)

Networking Reception in Poster and Exhibit Hall

Wednesday, May 3, 2017 • Main Conference Exhibit Hall & Poster Viewing Hours: 10:00 am – 1:45 pm





AM

PLENARY SESSION: CRISPR and Genome Editing Applications

Analytical Strategies for OligonucleotidesCRISPR and Genome Editing Applications

Analytical Strategies to Enable Peptide Development Peptide Delivery and Formulation StrategiesPreclinical and Clinical Development

of Oligonucleotides

Networking Luncheon in Poster and Exhibit Hall

PM Validation and Regulatory Strategies for Oligonucleotides

Preclinical and Clinical Development of Oligonucleotides Peptide Vaccines and Peptide Immunotherapy

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 • 9:00am-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

5Register Early for Best Savings • www.TIDESevent.com • 888-670-8200


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

Confirmed Speakers (as of December 19, 2016):

Advancing CRISPR/Cas9 for Therapeutic ApplicationsCecilia Fernández, Ph.D., Director, Platform Development, Editas Medicine

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

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

Additional Speakers to Be Confirmed

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 Facility Design Keith Higgins, Associate, CRB

11:25 Impact of SHE (Safety, Health and Engineering) on Product Containment StrategiesSpeaker TBA

12:00 Close of Workshop

7:15 Workshop Registration

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

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

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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 Workshop

James V. McArdle, Ph.D., President, McArdle & Associates, LLC 1:15 Expectations for Quality in Oligo Drug Products

René 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 Product

Brent 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

Workshop Leader: James Thompson, Ph.D., Head of CMC, Moderna Therapeutics, Inc.

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 Linking PK/ PD with Combinatorial Use of PNA Hybridization for Guide Strand Detection with QuantiGene for EfficacyAnastasia Khvorova, Ph.D., Professor, RNA Therapeutics Institute and Program in Molecular Medicine, 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 bDNA vs. qPCR Method: Is Sensitivity or Specificity More Critical for Support of mRNA Therapeutics?Mark Ma, Ph.D., Senior Director Bioanalytical Development and Biomarker, Alexion New Haven


Workshop #7: SENSITIVITY VERSUS SPECIFICITY OF MODERN BIOANALYTICAL ASSAYS AND THE CONSIDERATIONS OF ADME AND PK/PD STUDIES OF OLIGONUCLEOTIDES


Workshop 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

Additional Speakers: Nozer Mehta, Ph.D, Principal, Peptide Technologies LLCWeijun Shen, Ph.D., Principal Investigator, California Institute for Biomedical Research Christopher Rhodes, Ph.D., President and CEO, Drug Delivery Experts, USAAn Insider's Guide to PEG as a Half-life Extender Christopher Holmes, Ph.D., Industry Consultant

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

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

David C. Hanley, Ph.D. Executive Director, Technical Operations, Radius Health, Inc.

Abaloparatide: Benchtop to Commercial ManufactureTypical of start-up biotech, abaloparatide-SC development featured rich preclinical and clinical programs, while CMC development was initially constrained. Following clinical success, the complex abaloparatide CMC development program was rapidly accelerated to achieve development, regulatory and commercial supply goals. We tell our story of a synthetic peptide, injectable combination product, and partnership with 3 CMOs, to share the learnings of a strong team that overcame great challenges to deliver a timely, cost-effective and high quality product.

10:30 Networking Refreshment Break

12:30 Sponsored Spotlight Luncheon Presentations AvailableA limited number of opportunities are available for sponsored spotlight luncheon presentations during this session. If your company has an oligonucleotide or peptide technology or application that you are interested in sharing during a luncheon presentation, please contact Patrick Shaughnessy at [email protected] or call 857-504-6684


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


2:25Gregory Verdine, Ph.D.Principal Investigator and Professor, Harvard University, Harvard Medical School, and Founder, FogPharma

Cell-penetrating MiniproteinsCell-penetrating miniproteins (CPMPs) are a new drug modality that combines the cell-penetrating power of small molecules with the versatile target-engagement ability of biologics. These synthetic polypeptides comprising a synthetically hyperstabilized protein target-engagement domain have demonstrated the ability, in cells, in animal models, and now in human patients to target disease drivers heretofore considered difficult or impossible to drug. This talk will focus on the promising efforts to drug intractable transcription factors using CPMPs.

3:45Michael 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.

1:45 Chairman’s Remarks

1:50 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.

3:00 Grand Opening of Poster and Exhibit Hall and Networking Refreshment Break

Co-Sponsored by:

4:15James 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.

4:45 An Analysis of Recent Failures of Oligonucleotide Clinical Trials Due to Safety IssuesSpeaker TBA

5:15-6:30pm 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:

TUESDAY, MAY 2, 2017


Oligonucleotide Chemistry, Manufacturing and Controls




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 DevelopmentsCharalampos Koutsoulas, Ph.D., Scientific Project Leader, Liposome Technology, Polymun Scientific GmbH, Austria

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®Pamela Pavco, Ph.D., Chief Development Officer, 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:



mRNA Therapeutics Peptide Chemistry Manufacturing and Controls


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)

(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 Matthew Stanton, Ph.D., Vice President, Head of Chemistry, 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 Björn-Philipp Kloke, Ph.D., Head of IVAC Technology Platform Engineering, BioNTech AG, Germany

Development of Analytical Methods for Quality Control of PEG DerivativesMichael J. Roberts, Ph.D., Sales and Scientific Consultant, NOF America Corporation

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 (Invited)

Preclinical and Clinical Development of OligonucleotidesMuthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals

Stabilized Non-immunogenic messenger RNA (SNIM® RNA) for Transcript TherapyCarsten Rudolph, Ph.D., CEO and President, Ethris GmbH, Germany

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 ProgramSheng Cui, Ph.D., Senior Scientist, Drug Substance Technologies, 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

10

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 Remarks 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

Discovery of Selepressin and Merotocin, Two Clinical 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

Speaker TBA 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

Setmelanotide for the Treatment of Rare Monogenic Forms of Obesity: The MC4-PathwayLex Van der Ploeg, Ph.D., Chief Scientific Officer, Rhythm Pharmaceuticals

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 Therapeutics (continued)

mRNA Bioanalytical, CMC and Manufacturing (shared session)

Peptide CMC Case Studies and Strategies (continued)

Peptides in the Clinic: Lessons Learned (continued)

4:15 Case Study of Bioanalytical Approaches for Assessing Biodistribution of Modified mRNA TherapeuticsNeil Henderson, Ph.D., Associate Principal Scientist, Nucleotide Bioanalysis Group, AstraZeneca, United Kingdom

Non-Clinical Studies on RevusiranMichael Placke, Ph.D., Senior Vice President, Nonclinical Development, Alnylam Pharmaceuticals

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

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., SwitzerlandOligonucleotide Delivery Strategies

4:45 Biochemical, Biophysical and Cell-based Characterization of mRNA, A Novel Drug ModalityKristian Link, Ph.D., Senior Manager, Analytical Development, Moderna Therapeutics

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

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

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

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

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

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 Sponsored by


Schedule Subject to Change

WEDNESDAY, MAY 3, 2017





7:00 Coffee and Registration

7:45 - 8:15

Characterization and Purity Analysis of Synthetic ASOs by Low Resolution LCMSJoseph P. Nawrocki, Ph.D., Director, Biophysical Characterization and Analysis, Catalent Pharma SolutionsAndrew Argo, Senior Associate Scientist, Catalent Pharma Solutions

Plenary Session: CRISPR and Genome Editing Applications8:25 Chairman’s Remarks

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 StrategiesMeghdad Rahdar, Ph.D., Scientist, 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 in Poster and Exhibit Hall

10:40 Chairman’s Remarks Chairman’s Remarks 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

10:45 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

11:15 Application of ESI-MS/MS for Oligonucleotide Sequencing Methods Edward Huber, Ph.D., Director, Analytical Development/QC, Nitto Denko AveciaRanasinghe Gangani Silva, Ph.D., Senior Scientist, 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

11:45 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

12:15 Networking Luncheon in Poster and Exhibit Hall


12

WEDNESDAY, MAY 3, 2017





1:40 Chairman’s Remarks Chairman’s Remarks 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

Peptide Vaccine Presentation Title TBAJesse Dong, Ph.D., Vice President, Peptide Chemistry, Neon Therapeutics

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

Innovations in Antisense RNA Targeting: Driving Value, Targeting The SourceRichard S. Geary, Ph.D., Senior Vice President, Development, Ionis 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


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