Breakfast Roundtable Discussions
Balancing Cost and Risk in Peptide Manufacturing from Early Development through Late Stage clinical trials
Moderator: Dave Garman, PhD, CTO, NoNO, Inc.
- Managing CMC development on limited budget
- Selecting the appropriate manufacturing scales for early development
- Analytical method development and validations – timing and risks vis a vis safety and clinical studies
Measuring and Optimizing Cell Penetration for Peptides and Macrocycles
Moderator: Joshua Kritzer, PhD, Associate Professor, Department of Chemistry, Tufts University
- Advantages and disadvantages of current methods for measuring cell penetration
- Success stories of cell-penetrant peptides and macrocycles, and what can be learned from them
- Emerging guidelines for maximizing cell penetration
Delivery Mechanisms for Oligonucleotide Therapeutics
Moderator: Art Levin, Ph.D., Executive Vice President, Research and Development, Avidity NanoMedicines
- • Driving forward more effective targeted delivery of oligonucleotide drugs
- Topical delivery
- Lipid-enabled delivery
- Polypeptide-based delivery technology
Chemical Modifications of Oligonucleotide Therapeutics
Moderator: Muthiah (Mano) Manoharan, PhD, Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc
- Novel chemical modifications
- Improving delivery
Moderator: Nanna Albaek, PhD, Principal Scientist and Group Leader of New Chemistry, Roche Pharma Research and Early Development, Roche Innovation Center Copenhagen
- Solid phase oligonucleotide synthesis is an established technology. Over the years tremendous improvements have been made, but there is still areas to improve. Which elements of the process would be most beneficial to focus on?
- Oligonucleotide based therapies are constantly developed and this is to a great extend based on new and more advance chemical modifications. These new building blocks will often require customized synthesis methodologies. How are these best handled and what are the challenges e.g. when oligonucleotides with mixture of chemical modifications are synthesised?
- Conjugates are becoming more and more important in RNA therapeutics. What are the challenges we face with the introduction of new conjugate moieties, in this regard consider "in solid phase synthesis" conjugation and "post solid phase synthetic" conjugation.