Informa

講演者

Confirmed Keynote/Plenary Sessions

Rare Diseases and RNA-Targeted Medicines

Sarah Boyce
President, Akcea Therapeutics

The First-Ever FDA Approval of an RNAi Therapeutic and Other Pipeline Updates

Kevin Fitzgerald, Ph.D.
Senior Vice President and Head of Research, Alnylam Pharmaceuticals

Accelerated Development of an Antisense Oligonucleotide: Milasen

Timothy Yu, M.D., Ph.D.
Attending Physician, Division of Genetics and Genomics, Boston Children's Hospital and Assistant Professor in Pediatrics, Harvard Medical School

Confirmed Oligonucleotide Sessions

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

RNAi Specificity in vivo and New RNAi Platforms
Bob D. Brown, Ph.D., Chief Scientific Officer and Senior Vice President, Research, Dicerna Pharmaceuticals

Anti-FGF2 aptamer RBM-007 in phase I/IIa trials for wet AMD
Yusuf Ali, Ph.D., CEO, RIBOMIC USA

TANGO - Targeted-Augmentation of Nuclear Gene Output - for the Treatment of Genetic Diseases
Huw Nash, Ph.D., Chief Operating Officer and Chief Business Officer, Stoke Therapeutics

From Stereopurity to Precision Medicine: Optimizing the Properties of Antisense Nucleic Acid Therapeutics
Chandra Vargeese, Ph.D., Senior Vice President, Head of Drug Discovery, WAVE Life Sciences

Enhancing Therapeutic Profile of ASOs Using Gap-modifications
Punit Seth, Ph.D., Vice President, Medicinal Chemistry, Ionis Pharmaceuticals

The Oligonucleotide Agent BC 007 Neutralizes Agonistic Autoantibodies Directed Against β1-Adrenoceptors - Data of Clinical Phase 1 Trial
Annekathrin Haberland, Ph.D., Director Regulatory Affairs, Berlin Cures GmbH, Germany

Preclinical Discovery of Oligonucleotides
Session Chair: Shuling Guo, Ph.D., Executive Director, Exploratory and Genetic Diseases Drug Discovery, Ionis Pharmaceuticals

Case Study of Oligonucleotide Preclinical Development
Aimee Jackson, Ph.D., Director of Target Development, miRagen Therapeutics

Preclinical Development of Antibody-Oligonucleotide Conjugates (AOC)
Arthur A. Levin, EVP Research and Development, Avidity Biosciences

Oligonucleotide Manufacturing and Analytical
Session Co-Chairs: Doug Brooks, Ph.D., Consultant, Matapalo Pharma Consulting

Services and Claus Rentel, Ph.D., Executive Director Analytical Development QC
Ionis Pharmaceuticals

CMC Perspectives on Oligonucleotides
Vidhya Gopalakrishnan, Ph.D., Senior Vice President, Pharmaceutical Development, Quark Pharmaceuticals, Inc.

Case Study: Lessons Learned from an IND/IMPD Submission
Jennifer Lockridge, SVP, Program Development, Dicerna Pharmaceuticals, Inc.

Regulatory Perspectives and Points to Consider in Making Successful Oligonucleotide IND/CTA Submissions
Paul Manley, President and Principal Consultant, Orvieto Consulting

HILIC Chromatography for Oligonucleotide Analysis
Zach Parsons, Scientist, Avecia

Pre-PPQ Analytical Readiness
Ed Huber, Scientist, Avecia

Hot Topics in Oligonucleotides
Session Chair:  Arthur A. Levin, EVP Research and Development, Avidity Biosciences

Quality Development in European Early Access Approaches (PRIME) 
Rene Thurmer, Ph.D., Deputy Head, Unit Pharmaceutical Biotechnology, BfArM Federal Institute for Drugs and Medical Devices, Germany (Invited)

Control of Oligonucleotide Drugs: Product Quality Attribute Assessment to Ascertain Criticality
Robert Duff, Principal Scientist, Amgen

Successful Millimole gRNA Production & Highly Resolving Purity Test Methods
Joe Guiles, Ph.D., Head of Development, Nucleic Acid Solutions Division, Agilent Technologies, Inc

Presentation Title TBA
Daisuke Takahashi, Ph.D., Senior Principal Researcher, Ajinomoto Co. Inc., Japan

Confirmed Peptide Sessions

Genetically Encoded Selection of Novel Cyclic and Bicyclic Architectures
Ratmir Derda, Ph.D., Associate Professor, Department of Chemistry, University of Alberta, Canada

A Personalized Neoantigen Vaccine
Jesse Z. Dong, Ph.D., Vice President, Peptide Chemistry, Neon Therapeutics 

LY3298176, A Novel Dual GIP and GLP-1 Receptor Agonist for the Treatment of Type 2 Diabetes Mellitus: From Discovery to Clinical Proof of Concept
Axel Haupt, M.D., Senior Medical Director and Head of Early Phase Clinical R&D, Eli Lilly (Invited)

Peptide Leads and Modulators of Cellular Proteins
Session Chair: Rami Hannoush, Principal Scientist & Group Leader, Genentech 

Individualized/Personalized Neo-Antigens
Session Chair: Trishul Shah, Director, Business Development, North America, PolyPeptide Laboratories

Peptide Drug Discovery and Development
Session Chair: Mimoun Ayoub, Ph.D., Director and Head of North American and Emerging Markets, CordenPharma International, Switzerland

Peptide Aggregation, Gelation, and Injection Site Precipitation: Challenges, Tools, and Solutions
Steven Prestrelski, Ph.D., Chief Scientific Officer, Xeris Pharmaceuticals, Inc.

Peptide CMC, Quality and Manufacturing
Session Chair: Alex Fassler, Ph.D., COO, Bachem Holding AG, Switzerland

Modular CMC Development Concepts
Tanja Huth, Scientist, Bachem

Lumicell Case Study Elucidating the Supply Chain and CMC Planning
Dan Harris, Head of Supply Chain, Lumicell

CMC Approach to Bringing Two Suppliers On-line: Process Alignment, Filing Strategy and Supply Chain for the PEG
Najib Maslouh, Vice President of Manufacturing and Technical Operations, Apellis

Introducing Green and Sustainable Solvents in Liquid Phase Peptide Manufacturing Processes 
El Djouhar Rekai, Ph.D., Head of Development & Manufacturing Process, PolyPeptide Group, Belgium

Quality Development in European Early Access Approaches (PRIME) 
Rene Thurmer, Ph.D., Deputy Head, Unit Pharmaceutical Biotechnology, BfArM Federal Institute for Drugs and Medical Devices, Germany (Invited)

Cost Efficient Peptide Purification via ZEOsphere DRP Mixed-Mode Chromatography
Jurgen Machielse, Business Development Director Spherical Gels, Zecohem, Switzerland 

Confirmed mRNA & Delivery Sessions

Novel mRNA Immunotherapies
Robert Jabulowsky, Ph.D., Deputy Head of Project Management, BioNTech AG, Germany

Inhibition and Degradation of Drug Targets Using bioPROTAC mRNAs - A Novel Approach with Broad Therapeutic Potential 
Anthony Partridge, MSD Singapore

The State of the Art in mRNA Delivery, LNP-Based delivery and Hepatic Lung Delivery
Pad Chivukula, Ph.D., Chief Scientific Officer and COO, Arcturus Therapeutics, Inc.

Polymer-based mRNA Delivery
Daniel Siegwart, Professor, UT Southwestern

mRNA Delivery
John Androsavic, Scientist, Translate Bio (Invited) 

Safe Delivery of Nucleic Acid Cargoes Using Anionic Carriers
Steffen Panzner, Managing Director, Lipocalyx GmbH, Germany

Overcoming the Hurdle of Subcutaneous Delivery of mRNA in LNP to Enable Treatment of Chronic Diseases
Shalini Andersson, Ph.D., Senior Director Drug Metabolism & Pharmacokinetics, CVMD Innovative Medicines, AstraZeneca, Sweden

CMC Strategies for mRNA Therapeutics
Session Chair: Jim Thompson, Ph.D., Head CMC Project Management, Moderna Therapeutics

Presentation Title TBA
Andreas Kuhn, Ph.D., Vice President RNA Biochemistry & Manufacturing, BioNTech RNA Pharmaceuticals GmbH, Germany

Presentation Title TBA
Nigel Horscroft, Area Head, Molecular Therapy, CureVac AG, Germany

Confirmed Delivery Sessions

Novel Technologies for the Delivery of Macromolecular Therapeutics
Session Chair: Stephen T. Spagnol, Ph.D., Associate Principal Scientist, Sterile Formulation Sciences, Pharmaceutical Sciences, Merck Research Labs, Merck & Co., Inc.

Extra Hepatic Delivery of Oligonucleotides
Session Chair: Muthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery, Alnylam Pharmaceuticals

Design and Development of Lipid Nanoparticles for mRNA Vaccines
Marian Gindy, Ph.D., Executive Director, Pharmaceutical Sciences, Merck Research Laboratories

Development of Novel Polymeric Delivery Vehicles
Theresa M. Reineke, Ph.D., Distinguished McKnight University Professor, Department of Chemistry, University of Minnesota

Optimization of Novel Polymeric Delivery Vehicles by Chemical Evolution
Ernst Wagner, Ph.D., Chair, Professor and Chair, Pharmaceutical Biotechnology, Department of Pharmacy, Ludwig Maximilians University, Germany

Charge-altering Releasable Transporters (CARTs) - Novel Delivery Vehicle and Other New Data
Paul Wender/Robert Waymouth, Professors, Stanford University

Rapidly Identifying Nanoparticles for Non-hepatocyte RNA Delivery by Testing Thousands in vivo Using DNA Barcodes
James Dahlman, Ph.D., Assistant Professor, Biomedical Engineering, Georgia Institute of Technology and Emory School of Medicine

Confirmed CRISPR Therapeutics Sessions

CRISPR Therapeutics 
Session Co-Chairs: Cecilia Fernandez, Ph.D., Senior Director, Platform Strategy and Operations, Editas Medicine and Rubina Parmar, Ph.D., Director, Chemistry, Intellia Therapeutics

Novel Cas12a Mutants with Improved Activity and Expanded PAM Recognition Domain
Mark Behlke, M.D., Ph.D., Chief Scientific Officer, Integrated DNA Technologies