Nucleic Acid Therapeutics and Targeted Delivery (L2)
March 9-12, 2026
| Fairmont Banff Springs, Banff, AB, Canada
Anastasia Khvorova, Eric Vallabh Minikel, Annemieke Aartsma-Rus and Holly Kordasiewicz
Scholarship Deadline: Nov. 21, 2025 | Abstract Deadline: Feb. 18, 2026 | Early Registration Deadline: Jan. 9, 2026
| 4:00–8:00 PM |
Registration |
Van Horne Foyer |
| 6:00–8:00 PM |
Welcome Mixer |
Van Horne Foyer |
| 7:00–8:00 AM |
Breakfast |
President's Hall |
| 8:00–8:10 AM |
Welcome Remarks (Joint) |
Van Horne A/B |
| 8:10–9:00 AM |
Keynote Session (Joint) |
Van Horne A/B |
| |
Jennifer A. Doudna, HHMI/University of California, Berkeley
CRISPR Enzyme Evolution and Applications |
|
| 9:00–11:00 AM |
Delivery (Joint) |
Van Horne A/B |
| |
Daniel J. Siegwart, University of Texas Southwestern Medical Center
LNP-SORT - Targeted Delivery Through Size and Charge |
|
| |
Leah R Sabin, Regeneron Pharmaceuticals
A Modular, Antibody-Based AAV Retargeting Approach For In Vivo Gene Delivery to the CNS |
|
| 9:30–9:50 AM |
Coffee Break |
Van Horne Foyer |
| 9:50–9:55 AM |
Award Recipient Acknowledgement |
Van Horne A/B |
| 11:00–1:00 PM |
Poster Setup |
Van Horne C |
| 11:00–5:00 PM |
On Own for Lunch |
|
| 1:00–10:00 PM |
Poster Viewing |
Van Horne C |
| 2:30–4:30 PM |
Symposia Spotlight 1 |
Van Horne B |
| |
Yoshikazu Nakamura, RIBOMIC and University of Tokyo
Anti-FGF2 Aptamer Therapy in Achondroplasia |
|
| |
Mathieu Cinier, Affilogic
Nanofitin Scaffolds for Precision and Versatile Oligonucleotide Delivery |
|
| |
Brian Xi, University of Chicago
Lipid Nanoparticle-Mediated Spatiotemporal Modulation of NF-kB Activation to Treat Atherosclerosis |
|
| |
Justine M Aelvoet, Ghent University
A Pre-targeted Nanomedicine Strategy for In Vivo CD8+ T-cell Engineering |
|
| |
Simone Carneiro, Ludwig-Maximilians-University Munich
Pulmonary Lipid Nanoparticle Delivery of CRISPR/Cas9 Targeting KRAS G12S Mutations in a Lung Cancer Model |
|
| |
Maaike De Cock, Ghent University
Making the Cut: CD4-Targeted Lipid Nanoparticles for CRISPR-Cas9-Mediated HIV Cure |
|
| |
Kenan Ozcan, Purdue University
Targeted Delivery of Chemically Modified microRNA-34a using 5-Methyltetrahydrofolate Improves Cytosolic RNA Delivery and Therapeutic Efficacy in Folate Receptor α-Overexpressing Tumors |
|
| |
Julia Rädler, Karolinska Institutet
Click-Chemistry-Based Strategy for Modular Ligand Attachment to siRNAs: Toward Tissue-Specific RNAi |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–7:00 PM |
Delivery Systems based on Conjugates, vs. Delivery Systems based on Particles Targeted Nucleic Acid Delivery |
Van Horne B |
| |
Anastasia Khvorova, RNA Therapeutics Institute, UMass Chan 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 |
|
| |
Douglas Brown, Entos Pharmaceuticals
Short Talk: Safe and Effective Extrahepatic mRNA Delivery for CRISPR Gene Editing Using Fusogenix Proteolipid Vehicles |
|
| 7:00–8:00 PM |
Social Hour with Dinner |
President's Hall |
| 7:30–10:00 PM |
Poster Session 1 |
Van Horne C |
Wednesday, March 11, 2026
| 7:00–8:00 AM |
Breakfast |
President's Hall |
| 8:00–10:30 AM |
Distribution of RNA-based Drugs |
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 |
|
| |
Eunji Chung, University of Southern California
Short Talk: mRNA Delivery for Genetic Kidney Disease |
|
| |
Jingxuan Liu, City Therapeutics
Short Talk: Peptide Ligands Enable Efficient and Cell-type Specific Delivery of siRNAs to the CNS and Eye |
|
| |
Ping Wee, Entos Pharmaceuticals
Short Talk: Ocular Gene Therapy Using the Proteolipid Vehicle Nucleic Acid Delivery Platform |
|
| |
Samir EL Andaloussi, Karolinska Institutet
Short Talk: Engineered Extracellular Vesicles Enable Efficient Gene Editing in the CNS |
|
| 9:00–9:20 AM |
Coffee Break |
Van Horne Foyer |
| 11:00–1:00 PM |
Poster Setup |
Van Horne C |
| 11:00–5:00 PM |
On Own for Lunch |
|
| 1:00–10:00 PM |
Poster Viewing |
Van Horne C |
| 3:00–4:30 PM |
Career Roundtable (Joint) |
Van Horne B |
| |
Kusumika Mukherjee, Cell Press
Senior Scientific Editor |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–7:00 PM |
New Approaches to Extrahepatic Delivery |
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 |
|
| |
Matt Simon, Denali Therapeutics
Delivery of ASOs to the CNS using hTfR Antibodies |
|
| |
Zhanna Druzina, Aro Biotherapeutics
Short Talk: A Novel Platform to Discover Targeting Proteins for siRNA Delivery into Immune Cells |
|
| 7:00–8:00 PM |
Social Hour with Dinner |
President's Hall |
| 7:30–10:00 PM |
Poster Session 2 |
Van Horne C |
| 7:00–8:00 AM |
Breakfast |
President's Hall |
| 8:00–11:00 AM |
Nucleic Acid Chemistry, Structure, Mechanism, Potency |
Van Horne B |
| |
Ken Yamada, University of Massachusetts Chan Medical School
exNA and Ionizable Motifs Towards Next Generation RNA Therapeutics |
|
| |
Punit P. Seth, Alnylam Pharmaceuticals
Chemical Modifications of siRNA and ASOs |
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| |
Ruben Warkentin, Yale University
Short Talk: Advancing circRNA Biotechnologies through an Optimized Group I Intron Platform |
|
| |
Karin Broennimann, Weizmann Institute of Science
Short Talk: N1-Methylpseudouridine Directly Modulates Translation Dynamics |
|
| |
Riccardo Panella, Aalborg University
Short Talk: Antisense Oligonucleotide-Mediated Inhibition of MicroRNA-22 Leads to Selective and Sustained Loss of Fat Mass and Enhances Efficacy of GLP1-RA in Obese Mice and Nonhuman Primates |
|
| |
Nabanita De, City Therapeutics
Short Talk: Small RNAs, Big Potential: The Therapeutic Promise of cityRNAs |
|
| 9:00–9:20 AM |
Coffee Break |
Van Horne Foyer |
| 11:00–5:00 PM |
On Own for Lunch |
|
| 2:30–4:30 PM |
Symposia Spotlight 2 |
Van Horne B |
| |
Barnaby Hill, University of Oxford
Accurately Modelling RNase H-mediated Antisense Oligonucleotide Efficacy and Toxicity |
|
| |
Masahiro Ohara, UMass Chan Medical School
Development of Antisense Oligonucleotides Targeting STMN2 for Sporadic ALS |
|
| |
Vicente Planells-Palop, Metagenomi
CRISPR/Cas-Mediated APOC3 Knockout as a One-Time Treatment for Severe Hypertriglyceridemia |
|
| |
Caroline D Doherty, Mayo Clinic
In vivo Selection of Anti-glioblastoma DNA Aptamers for Targeted Toxin Delivery using Orthotopic Patient-derived Xenograft Models |
|
| |
Mariah Hassert, University of Iowa
mRNA Vaccination Overcomes Hemozoin-mediated Antigen Uptake Defects to Efficiently Prime CD8+ T Cell Responses against Liver-stage Malaria |
|
| |
Isabella Hetherington, University of South Florida
Development of a Dual Action mRNA Therapeutic to Treat Atherosclerosis |
|
| |
Michael Siroky, Scripps Research
Engineering AAV to encapsidate RNA |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–7:00 PM |
Clinical Innovation in RNA-based Drug Development |
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 |
|
| |
Rajeshwar Motheram, Comanche Biopharma
Developing RNA-based Drugs for Diseases of Pregnancy: The Preeclampsia Story |
|
| 7:00–7:15 PM |
Meeting Wrap-Up: Outcomes and Future Directions (Organizers) |
Van Horne B |
| 7:15–8:15 PM |
Social Hour with Dinner |
President's Hall |
| 8:15–9:15 PM |
Entertainment |
President's Hall |
