Conference Synopsis

Day 1 Stream 1 – Oligonucleotide Therapeutics R&D

• IP issues & Venture Capital landscape
• Regulatory challenges / FDA experiences
• Strategies to bring an Oligonucleotide drug to market: Phase specific strategies; Accelerating development
• Bioanalytical and Clinical Pharmacology issues
• Predicitivity of animal models
• Immunogenicity assessment
• Conjugation Strategies: Achieving successful development; Linkers, ligands, tethers
• Case Studies – Oligonucleotide Program Progress Updates: Antisense, RNAi, siRNA, Aptamers, MicroRNA, Gapmers, Decoy, Immunostimulatory

Day 1 Stream 2 - Peptide Therapeutics R&D

• Peptide screening and optimisation: Screening technologies; Novel approaches to modify peptides; Matuation - optimisation of peptides; Use of next generation sequencing  to optimise peptides
• Conjugation strategies: Peptide Drug Conjugates; Half life extension /  “ylation and –idations”
• Case Studies – Peptide Program Progress Updates: Intracellular targets; Extracellular targets; GPCR agonists; Hormone receptor agonists; Macrocycle peptides

Day 2 Stream 1 - Advances In Process Development & Manufacturing For Oligonucleotide And Peptides

• Choosing a manufacture partner / CMO outsourcing strategies
• Successful strategies for manufacture
• Advances in process development & manufacturing
• Scale up and optimization of the manufacture process
• Manufacture of conjugated oligonucleotides
• Oligo and peptide synthesis
• QbD principles for validation of manufacturing processes
• Overcoming CMC challenges
• Aggregation
• Regulatory guidelines for Oligonucleotide & peptide quality
• Impurity thresholds, specifications and acceptance criteria
• Panel Discussion - Methods to manage process change during development and scale up
• Panel Discussion  - Overcoming specification and impurity challenges

Day 2 Stream 2 - Oligonucleotide & Peptide Delivery Strategies

• Achieving a successful delivery system
• Novel advances & future perspectives: Designing alternative less invasive routes of delivery
• Oligonucleotide delivery: Lipid Nanoparticle (LNP) Delivery; RNAi Delivery; siRNA Delivery
• Peptide Delivery: Intracellular delivery tissue or organ-specific targeting; Addressing endosomal escape; Designing alternative less invasive routes of delivery
• Formulation and specification issues
• Panel Discussion -  Overcoming delivery problems

Neil Gibson, Chief Scientific Officer, Regulus Therapeutics Brett P. Monia, VP, Drug Discovery Research, Isis Pharmaceuticals Klaus Giese, Chief Scientific Officer Silence, Therapeutics AG Aaron Sato, Vice President of Research, Sutro Biopharma Inc. Roel Q.J. Schaapveld, CEO, InteRNA Technologies Dinesh Patel, President & CEO, Protagonist Therapeutics Giles Campion, CMO and SVP, R&D, Prosensa Mark Currie, Senior Vice President, Chief Scientific Officer, and President of R&D, Ironwood Pharmaceuticals David Lewis, Vice President, Biology, Arrowhead Research Corporation Keith James, President, Ferring Research Institute & Senior Vice-President R&D, Ferring Pharmaceuticals Volker Schellenberger, President and CEO, Amunix Inc Christophe Bonny, CSO, Bicycle Therapeutics Pamela Pavco, Chief Development Officer, RXi Pharmaceuticals Hanjo Hennemann, CSO, Nexigen, Germany Paul Watt, CSO, Phylogica Ltd Helmut Thomas, SVP Research and Preclinical Development, Tranzyme Pharma Inc. Elena Feinstein, Chief Scientific Officer, Quark Pharmaceuticals Peter Timmerman, Chief Technology Officer Pepscan David Suhy, SVP R&D, Tacere Therapeutics, a Benitec Biopharma Company Ron Swanson, Senior Director, Biologics Research, Janssen R&D David Blakey, Chief Scientist, Oncology iMED, AstraZeneca Steven F. Dowdy, Professor, Dept. of Cellular & Molecular Medicine, UCSD School of Medicine Claus Rentel, Executive Director, Analytical Development/ Quality Control, Isis Pharmaceuticals Stephen Carl, Associate Director, Formulation, Enteris BioPharma Muthiah (Mano) Manoharan, Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc. Michael Hodges, Chief Medical Officer, Santaris Pharma Bob Brown, CSO and SVP, Research, Dicerna Pharmaceuticals Jonas Ekblom, President & CEO, Pergamum AB Jayant Aphale, Senior Vice President, Technical Operations, Sarepta Therapeutics Alonso Ricardo, Director of Chemistry, Ra Pharmaceuticals, Inc Shahid Uddin, Head of Formulation, MedImmune, UK Satoshi Nakanishi, Director, Biologics Research Laboratories, Kyowa Hakko Kirin Co., Ltd. Dong-ki Lee, Principal Investigator, Global Research Laboratory for RNAi Medicine, Associate Professor, Department of Chemistry, Sungkyunkwan University, Founder, BioMolecular Therapeutics, Inc., South Korea Derek Maclean, Director, Process Engineering, Amgen Wendy ‎ Rodrigueza VP, Product Development, ProNAI