2025 Breakout Discussions

WEDNESDAY MARCH 12, 2025: 2:40 – 3:25 pm

Breakout discussions provide an opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges. These will take place IN-PERSON ONLY.

TABLE 1: Targeted Delivery of Oligonucleotides
Jim Weterings, PhD, Vice President, Head of Oligonucleotide Therapeutics, Bonito Biosciences

Extrahepatic opportunities & progress
Current targeting ligand portfolio
Conjugate chemistry: Linker chemistry and oligonucleotide chemistry

TABLE 2: Trending: How Companies Get Acquired
Moderator: Renee Williams, Founder and Managing Partner, Williams Biotech Consulting
This discussion will look at the following trends as it relates to acquisitions of biotechs over the past years:

Deal volume and size
Preclinical vs clinical
Stage of Financing
Time to exit vs valuation
Top strategic acquirers

TABLE 3: Laying the Groundwork for a Successful IND or CTA
Moderator: Michael Hellerstein, Head of Operations, Vaxxinity

Phase-appropriate development: Planning for evolution and improvement of CMC systems to meet regulatory requirements
Planning for success: Design of process and analytical development programs to meet regulatory requirements
How much is too much? How to balance the need for control against the need for flexibility
The expected unexpected: Proactively dealing with gaps in the data

TABLE 4: Analytical Characterisation and Quantitation of Impurities in Single and Double Stranded Oligonucleotides
Moderator: Mike Webb, PhD, Founder and CEO, Mike Webb Pharma; Former Vice President, API Chemistry & Analysis, GSK

How to control impurity families with different 2' chemistries and limited chromtographic separation
Orthogonal chromatographic methods versus orthogonal detection (UV and MS)
Data requirements for controlling CQA's for batch analysis versus measuring CQA outputs during process characterisation experiments
How to leverage prior knowledge for process characterisation

TABLE 5: Tackling Challenges with mRNA Delivery
Moderators:
Charles Chen, PhD, Senior Scientist, Advanced Drug Delivery, Pharmaceutical Sciences, AstraZeneca Pharmaceuticals, R&D
Ekkehard Leberer, PhD, Professor of Biochemistry, Technical University of Munich; Senior Consultant, ELBIOCON; Advisor, Neuway Pharma
Dan Peer, PhD, Professor & Director, Laboratory of Precision Nanomedicine; Vice President for Research, Tel Aviv University

Innovative mRNA formulations and delivery approaches
Improving selectivity and efficiency of delivery
Delivery across the blood-brain-barrier
Targeting specific cell types for treating various diseases
Low immunogenicity mRNA formulations

TABLE 6: Overcoming Translational Challenges in RNA Therapeutics Development
Moderators:
Vikram Agarwal, PhD, Head of mRNA Platform Design Data Science, mRNA Center of Excellence, Sanofi
Arthur Krieg, MD, Founder, President and Acting CEO/CSO, Zola Therapeutics

Optimizing innovative chemistries and modifications
Strategies for regulating mRNA expression
Addressing biodistribution and tissue specificity
Uses and limitations of mouse models in RNA therapeutics development
What properties are most important to optimize? Are there tradeoffs?
Defining the safety profile

TABLE 7: Designing and Optimizing New RNA Modalities as Therapeutics
Moderators:
William Kiesman, PhD, Chief Technology Officer, Alltrna
Edo Kon, PhD, Director of Business Development, RiboX Therapeutics
Li Li, PhD, Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical School

Innovative circular RNAs and their applications
Optimizing tRNA design and delivery
Developing Self-amplifying and programmable RNAs
Establishing criteria for modality selection, application and success