Pre-Conference Day
Pre-Conference Day
- Martin Deetz, PhD - Senior Technical Laureate, DuPont
- Lars Geiger, PhD - Global Director of Project Management Drug Substance, Evonik Health Care
- Yoshitaka Nemoto - Vice President R&D, PeptiStar
The purpose of this workshop is to introduce scientists to therapeutic oligonucleotides. This workshop will discuss the different types of therapeutic oligonucleotides and how they work in the body to treat disease. Difference modalities such as SiRNA, ASO, MiRNA and Aptamer will be covered. The key factors for the design of the molecules will be described. The workshop will also discuss the challenges of delivery to the appropriate tissue and into the appropriate cell, and the strategies currently employed to address these. The toxicology, metabolism and clearance in the body will be covered. Finally, the workshop will discuss formulation options and how the drug substances and drug products are manufactured and controlled.
- Mike Webb, Ph.D. - Founder and CEO, Mike Webb Pharma
- G. Susan Srivatsa, PhD - President, ElixinPharma
- Fran Wincott, PhD - President, Wincott & Associates, LLC
- Claus Rentel, PhD - Vice President, Analytical Development/QC, Ionis Pharmaceuticals, Inc.
- Keith Bowman - Vice President of Process Development, Wave Life Sciences
- Lubomir Nechev, PhD - Senior Vice President CMC Development, Alnylam Pharmaceuticals, Inc.
- Bao Cai, PhD - Executive Director, Process Development, Sarepta Therapeutics
- Rubina Parmar, PhD - Vice President, Chemistry and Delivery Sciences, Intellia Therapeutics
- Cecilia Fernández, Ph.D. - VP of Strategic Planning and Operations, Chroma Medicine
Scientists will be introduced to the technology of gene editing in this workshop. Participants in the workshop will learn about the different gene editing technologies available and how they work for therapeutic purposes. In this workshop, we will cover the delivery technologies available for delivering these gene editing technologies to appropriate tissues. Some of the other questions to be considered in this workshop include: What are the recent improvements and advances in genome editing technologies? What are some strategies for handling off-target detection and mitigation? What is the current status, as well as the challenges and risks of in vivo gene editing beyond the liver? How are companies handling precision targeted integration of large genetic cargo? What are the latest tools for DMPK and (bio)analytics of gRNAs? What are the current regulatory perspectives and guidances in the current landscape of gene edited products?
- Rubina Parmar, PhD - Vice President, Chemistry and Delivery Sciences, Intellia Therapeutics
- David Bryson, PhD - Director, Gene Editing, Beam Therapeutics
- Jeff Hussmann, PhD - Scientist, Prime Medicine
- Cecilia Fernández, Ph.D. - VP of Strategic Planning and Operations, Chroma Medicine
- Tyson Moyer, PhD - Principal Scientist, Korro Bio
- Felix Krupp, PhD - Project Lead GMP & Large-Scale Production, BioSpring GmbH
- Matteo Villain, PhD - Vice President and Global Peptides Technical Lead, Piramal Pharma Solutions
- Jamie Brugnano, Ph.D. - Director of Regulatory Affairs, Bachem Americas
- Lisa Caralli - Sr. Director of Scientific Advisory, Pharmaceutics, Catalent Pharma Solutions
- Jean-Marc Poudrel, PhD - Head of Regulatory Affairs, PolyPeptide Group
- Ashli Simone - Technical Product Specialist, MOBILion Systems
- Priska Frei, Ph.D. - Scientist QC, Bachem AG
- Derek Gauntlett - Director, Process Chemistry, Codexis
- Lilit Avagyan - Team Leader Customized Solutions, Knauer
- Matthew Stone, PhD - Advanced Workflow Specialist, SCIEX
- Kathryn Ackley, PhD - CMC Consultant Specializing in Oligonucleotides, Independent Consultant
- Peter Korytko, Ph.D. - President, Preclinical GPS
- Kathryn Ackley, PhD - CMC Consultant Specializing in Oligonucleotides, Independent Consultant
- Judy Carmody, Ph.D. - Founder and Principal Consultant, Carmody Quality Solutions, LLC
The workshop will focus on the specific requirements for control that are common to all therapeutic oligonucleotides. For example, common impurities from solid-state synthesis especially those which come from the starting materials, the synthetic process and degradation products. The issues of determining water in hygroscopic products. Issues with assays for both single and double stranded oligonucleotides. In addition, we will discuss how establishing an ongoing control strategy is important to determine and monitor critical quality attributes that affect the drug product and the key aspects of specification setting across the phases of development. The workshop will also touch on risk assessment in late phase quality by design approaches and the role of analysis in determining critical process parameters and their relationship to critical quality attributes.
- Mike Webb, Ph.D. - Founder and CEO, Mike Webb Pharma
- Marc Jacob, PhD - Executive Director of Business Development, SK pharmteco
- Martin Gilar, PhD - Scientific Fellow, Separations R&D, Waters Corporation
- Jean-Noel Lemercier, PhD - Associate Director, Chemistry, Editas Medicine
- Kaizhang He, Ph.D. - Director of Process Chemistry, Agilent Technologies
- Joe Guiles, PhD - Head of Chemical Development, Agilent Technologies
- Garrett Rettig, Ph.D. - Senior Director of Product Development, Integrated DNA Technologies
- Luis Santos, Ph.D. - Director, Non-viral Delivery, mRNA and LNP Product, Prime Medicine
- Gary Musso, PhD - President, Musso and Associates LLC
- Samrat Sisodia, Ph.D. - Vice President RA and QA, Palatin Technologies
- Aileen Ryan - Senior Regulatory Advisor, Prometrika, LLC
- Gary Musso, PhD - President, Musso and Associates LLC
- Laurin Melzig, PhD - Director R&D Process Development, Bachem AG