2024 Coming Soon…

Tuesday, March 14, 2023

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.

Oligonucleotide Discovery and Delivery

IN-PERSON BREAKOUT DISCUSSION: Integrating Innovative Technologies into Oligo Therapeutics Discovery and Delivery
June Park, PhD, CEO, siRNAgen Therapeutics
Gerard Platenburg, CSO and Co-Founder, ProQR Therapeutics

  • What are emerging innovative technologies in oligo R&D, focusing on in particular:
    • AI/machine learning
    • Delivery strategies
    • Safety screening
  • Challenges in the adoption and implementation of innovative technologies
  • Strategies to keep up with the rapidly evolving field
  • What’s next in oligo discovery and delivery innovation?

IN-PERSON BREAKOUT DISCUSSION: From Early Discovery and the Clinic to Collaborations Between Big Pharma and Small Biotechs
Marvin Caruthers, PhD, Distinguished Professor, Biochemistry, University of Colorado

Developments from Early Discovery Through to Late-Stage Clinical Programs

  • How do you know your discovery should move toward the clinic
  • Patents
  • Steps toward the clinic-biology/biochemistry/chemistry, cell biology, animal studies, toxicology, and who/how to carry out a clinical trial

Strategic Collaboration Between Big Pharma and Small Biotechs

  • How do you define a strategic collaboration-from the perspective of big pharma and small biotech (i.e. why a collaboration)
  • Conditions for a strategic/successful collaboration

Oligonucleotide CMC and Regulatory Strategies

IN-PERSON BREAKOUT DISCUSSION: Evaluating CMC and Analytical Strategies
Mike Webb, PhD, Founder and CEO, Mike Webb Pharma; Former Vice-President of API Chemistry & Analysis at GSK

  • Do we need an ICH guideline for the development of oligonucleotides?
  • How should we address diastereogmer control in phosphorothioated oligonucleotides?
  • Avoiding lyophilisation and providing drug substance in solution, a better approach to compounding?
  • A single industry specification and methods for amadite impurities

Emerging mRNA Therapeutics

Paloma H. Giangrande, PhD, Vice President, Platform and Discovery Sciences Biology, Wave Life Sciences
Mark Kay, MD, PhD, Dennis Farrey Family Professor of Pediatrics and Genetics, Department of Pediatrics and Genetics, Stanford University
Robert Mabry, PhD, CSO, Orna Therapeutics
Drew Weissman, MD, PhD, Roberts Family Professor in Vaccine Research, Department of Infectious Diseases; Director of the Penn Institute for RNA Innovation and Professor, Department of Medicine, University of Pennsylvania

  • Challenges with mRNA delivery
  • Innovative mRNA modifications
  • Emerging mRNA formats (circular, replicating etc.)
  • Tackling mRNA safety


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2024 Conference Programs

Oligo Discovery & Delivery