***Meeting program subject to change.
Available Formats: = In Person
= On Demand
4:00–8:00 PM
Van Horne Foyer
6:00–8:00 PM
Van Horne Foyer
7:00–8:00 AM
President's Hall
Welcome and Keynote Session (Joint)
8:00–9:00 AM
Van Horne A/B
Jennifer A. Doudna, HHMI/University of California, Berkeley
CRISPR Enzyme Evolution and Applications
9:00–11:15 AM
Van Horne A/B
Daniel J. Siegwart, University of Texas Southwestern Medical Center
LNP-SORT - Targeted Delivery Through Size and Charge
Michael A. Fischbach, Stanford University
Engineered Viral-Like Particle for Targeted Delivery of Gene Editors
Leah R. Sabin, Regeneron Pharmaceuticals
Antibody-AAV Targeting: Fundamental Principle Behind Achieving BBB Crossing
Short Talk(s) Chosen from Abstracts
9:30–9:50 AM
Van Horne Foyer
11:15–1:00 PM
Van Horne C
1:00–10:00 PM
Van Horne C
Symposia Spotlight 1: Late-breaking research presentations selected from abstract submissions
2:30–4:30 PM
Van Horne B
Short Talks Chosen from Abstracts
4:30–5:00 PM
Van Horne Foyer
Delivery Systems based on Conjugates, vs. Delivery Systems based on Particles Targeted Nucleic Acid Delivery
5:00–7:00 PM
Van Horne B
Anastasia Khvorova, University of Massachusetts Medical School
Barcoded Libraries for Targeted siRNA Delivery
Andrea L. Kasinski, Purdue University and LigamiR Therapeutics
Folate-miRNA Conjugates for Tumor Delivery
Steven F. Dowdy, University of California, San Diego
How to Tackle Endosomal Escape
Short Talk(s) Chosen from Abstracts
7:00–8:00 PM
President's Hall
7:30–10:00 PM
Van Horne C
7:00–8:00 AM
President's Hall
Distribution of RNA-based Drugs
8:00–11:00 AM
Van Horne B
Brendon E Cook, Biogen
Studying Oligonucleotide Distribution after Intrathecal Treatment using Imaging
Shalini Andersson, AstraZeneca R&D
Oligonucleotide Biodistribution after Systemic Treatment
Short Talk(s) Chosen from Abstracts
9:00–9:20 AM
Van Horne Foyer
11:00–1:00 PM
Van Horne C
1:00–10:00 PM
Van Horne C
Career Roundtable (Joint)
3:00–4:30 PM
Van Horne B
4:30–5:00 PM
Van Horne Foyer
New Approaches to Extrahepatic Delivery
5:00–7:00 PM
Van Horne B
Liqun Wang, Wyss Institute at Harvard University
Brain Delivery of Antibody-Oligonucleotide Conjugates for CNS Diseases
Holly Kordasiewicz, Ionis Pharmaceuticals
Extra Hepatic Delivery of Oligonucleotides
Scarlett Barker, Denali Therapeutics
Delivery of ASOs to the CNS using hTfR Antibodies
June Y Park, Cenos Therapeutics
Micellar RNAi Conjugates for Extrahepatic Delivery
Short Talk(s) Chosen from Abstracts
7:00–8:00 PM
President's Hall
7:30–10:00 PM
Van Horne C
7:00–8:00 AM
President's Hall
Nucleic Acid Chemistry, Structure, Mechanism, Potency
8:00–11:00 AM
Van Horne B
Ken Yamada, University of Massachusetts Chan Medical School
exNA and Ionizable Motifs Towards Next Generation RNA Therapeutics
Anna Marie Pyle, Yale University
Structural Exploration of RNA Drug Function
Punit P. Seth, Alnylam Pharmaceuticals
Chemical Modifications of siRNA and ASOs
Short Talk(s) Chosen from Abstracts
9:00–9:20 AM
Van Horne Foyer
Symposia Spotlight 2: Late-breaking research presentations selected from abstract submissions
2:30–4:30 PM
Van Horne B
Short Talks Chosen from Abstracts
4:30–5:00 PM
Van Horne Foyer
Clinical Innovation in RNA-based Drug Development
5:00–6:45 PM
Van Horne B
Scott Schobel, VICO Therapeutics
ASO Development for Neurodegenerative Diseases
Annemieke Aartsma-Rus, Leiden University Medical Center
N-of1 based ASOs
Eric Vallabh Minikel, Broad Institute
Progress in PrP-Lowering Oligonucleotides for Prion Disease
Scott Johnson, Comanche
Developing RNA-based Drugs for Diseases of Pregnancy: The Preeclampsia Story
Short Talk(s) Chosen from Abstracts
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
6:45–7:00 PM
Van Horne B
7:00–8:00 PM
President's Hall
