Pre-Conference Workshops (am) & Main Conference Day 1 Plenary (pm)
Workshop Overview:
The landscape of oligonucleotide manufacturing is evolving to meet the requirements of a robust pipeline of innovative oligonucleotide therapeutics being developed around the world. From solid-phase to enzymatic and hybrid synthesis approaches, many new technologies are emerging to address issues related to sustainability, scalability and efficiency in oligo manufacturing. Along with these new technologies come more complex challenges within process development, CMC and establishment of not yet considered regulatory pathways. This workshop will explore the current state-of-the-industry for these novel synthesis approaches with an emphasis on the CMC challenges, strategies and opportunities that will be needed for successful next-generation oligonucleotide therapeutic manufacturing.
- Thomas Rupp - Vice President, CMC Strategy, Axolabs Berlin GmbH
- Chris Oswald - Founder, Owner, and Principal Consultant, Coswald Consulting LLC
Workshop Overview:
The landscape of oligonucleotide manufacturing is evolving to meet the requirements of a robust pipeline of innovative oligonucleotide therapeutics being developed around the world. From solid-phase to enzymatic and hybrid synthesis approaches, many new technologies are emerging to address issues related to sustainability, scalability and efficiency in oligo manufacturing. Along with these new technologies come more complex challenges within process development, CMC and establishment of not yet considered regulatory pathways. This workshop will explore the current state-of-the-industry for these novel synthesis approaches with an emphasis on the CMC challenges, strategies and opportunities that will be needed for successful next-generation oligonucleotide therapeutic manufacturing.
- Thomas Rupp - Vice President, CMC Strategy, Axolabs Berlin GmbH
- Chris Oswald - Founder, Owner, and Principal Consultant, Coswald Consulting LLC
- Bruce Morimoto, PhD - Independent Consultant, TIDES Advisor
- El Djouhar Rekaï, PhD - Process & Analytical Development Director at PolyPeptide Group, PolyPeptide Group
Quality by Design (QbD) enables the consistent manufacture of products with the intended quality through science- and risk-based process design. Although the concept is well established, its application is not mandatory and the extent of implementation varies among companies. In particular, industry-wide approaches to understanding peptide active pharmaceutical ingredient (API) manufacturing processes using liquid phase peptide synthesis (LPPS) are still under development.
To deepen our process understanding, we adopted the process characterization approach reported by Roche. This presentation describes our case study of LUNA18 process and discusses how process characterization can support the understanding of peptide API manufacturing processes. Based on the lessons learned, we also propose future directions for establishing more systematic and broadly applicable strategies for peptide synthetic platform development.
- Haruka Kawabata - Scientist, API Process Development Department, Chugai Pharmaceutical Co., Ltd.
- Marc Jacob, PhD - Head of Strategic Development, Chromatography Products, YMC America
- Matt Quinn - Director CMC, Analytical Development, Verdiva Bio
The evolution of oligonucleotide manufacturing mirrors the rapid advancement of biotechnology itself, transitioning from niche, small-scale research synthesis to highly automated, industrialized production platforms capable of delivering therapeutic oligonucleotides at commercial scale. This presentation examines key advances in chemistry, downstream processing, and automation, and explores emerging innovations, including enzymatic synthesis, and sustainable manufacturing strategies that are reshaping the future of oligonucleotide production.
- James Powell - VP, RNAi CMC and Boulder Site Head, Novo Nordisk
We developed PulmoRNA®, an inhaled siRNA platform enabling efficient, durable pulmonary gene silencing with favorable safety. Using this platform, we generated a dual-targeting siRNA that
simultaneously silences IL-33 and TSLP, demonstrating sustained anti-inflammatory efficacy in preclinical models. A clinical candidate has been selected, and studies supporting clinical development are underway.
- Junichi Nezu, PhD - Member of the Board and Executive Managing Officer, Mochida Pharmaceutical Co., Ltd
This lecture discusses the most recent advances occurring in the Suga laboratory for de novo discovery of highly constrained macrocyclic peptides.
- Hiroaki Suga, PhD - Professor of Chemistry, School of Science, University of Tokyo
Backbone-cyclized α-conotoxin Vc1.1 is a potent GABAB receptor agonist and analgesic lead for neuropathic pain. We describe two yeast-based systems for its direct recombinant production. In Pichia pastoris, a secreted fusion of the conotoxin precursor and asparaginyl ligase enabled pH-triggered cyclization and correct folding. In Saccharomyces cerevisiae, surface-displayed ligase cyclized secreted Vc1.1 precursors directly in culture. These complementary strategies provide efficient recombinant routes for producing disulfide-rich, backbone-cyclized therapeutic peptides.
- David Craik, PhD - Professor of Biomolecular Structure, University of Queensland