2027 Plenary Keynote Sessions

11:25 am Delivery of Oligonucleotide Conjugates: Beyond the Liver
Shalini Andersson, PhD, Vice President Nucleic Acid Therapeutics, Discovery Sciences, AstraZeneca
Efficient delivery remains a central challenge for oligonucleotide therapeutics targeting tissues beyond the liver. Selective, productive uptake in relevant cells is essential to realize their full potential. Advances in delivery approaches of oligonucleotides can significantly improve distribution, cellular uptake, and potency in extrahepatic tissues. These targeted approaches can expand the druggable target space, improve safety, reduce dose requirements and cost of goods, and enable development of precisely targeted oligonucleotide medicines.

11:55 am Engineering Delivery of RNA and Genome Editors
Daniel Anderson, PhD, Professor, Chemical Engineering, Massachusetts Institute of Technology
Nucleic acids have enormous potential to provide therapies for the prevention, treatment, or even cure of human diseases. However, these powerful drug molecules must be delivered inside of cells in vivo, in a safe and effective manner, to provide therapy. The diversity of RNA drugs continues to expand, including relatively short single and double-stranded constructs such as siRNA and guide RNA, as well as longer protein coding forms such as mRNA and circular RNA. I will describe the development and engineering of delivery systems for these different RNA payloads to treat a range of organ systems and diseases.

12:25 pm PANEL DISCUSSION: Strategies and New Modalities for Optimizing Oligo Delivery
PANEL MODERATOR:
Dmitry Samarsky, PhD, CSO & Board Member, ARNAgen Therapeutics

PANELISTS:
Shalini Andersson, PhD, Vice President Nucleic Acid Therapeutics, Discovery Sciences, AstraZeneca
Daniel Anderson, PhD, Professor, Chemical Engineering, Massachusetts Institute of Technology
Mano Manoharan, PhD, Distinguished Scientist & Senior Vice President, Innovation Chemistry, Alnylam Pharmaceuticals
Chandra Vargeese, PhD, CTO & Head, Platform Discovery Sciences, Wave Life Sciences
Vadim Dudkin, PhD, Founding CTO, Souffle Therapeutics

Topics Include:

• Advances in Antibody-Oligonucleotide Conjugates (AOCs) and Peptide-Oligonucleotide Conjugates (POCs)
• Moving beyond the liver: efficient delivery to muscle, brain, lung, and more
• Search for the next GalNAc: finding highly expressed, rapidly internalizing and recycling receptors
• Cell-specific delivery: targeting specific cell populations in the tumor, brain, muscle etc.
• Engineering molecular shuttles across the Blood-Brain Barrier (BBB)
• Smarter linker chemistry: optimizing stability and payload release capabilities
• Optimization of payload ratio, conjugation sites, and multivalent architectures​