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 |
| |
* Anastasia Khvorova, RNA Therapeutics Institute, UMass Chan Medical School
Session Chair |
|
| |
* Jonathan S. Weissman, Whitehead Institute, HHMI, and MIT
Session Chair |
|
| |
Jennifer A. Doudna, HHMI/University of California, Berkeley
CRISPR Enzyme Evolution and Applications |
|
| 9:00–11:00 AM |
Delivery (Joint) |
Van Horne A/B |
| |
* Charles Gersbach, Duke University
Session Chair |
|
| |
Daniel J. Siegwart, University of Texas Southwestern Medical Center
Development of Signal Peptide Engineered Nucleic Acid Design (SEND) mRNAs and Ionizable Phospholipid (iPhos) Lipid Nanoparticles (LNPs) that Control Localization of Protein Therapeutics |
|
| |
Anastasia Khvorova, RNA Therapeutics Institute, UMass Chan Medical School
Barcoded Libraries for Targeted siRNA Delivery |
|
| |
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 |
| |
* Annemieke Aartsma-Rus, Leiden University Medical Center
Session Chair |
|
| |
* Scott Schobel, VICO Therapeutics
Session Chair |
|
| |
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 |
|
| |
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 miRNAs Using a Folate Metabolite Increases 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 |
| 5:00–6:30 PM |
Delivery Systems based on Conjugates, vs. Delivery Systems based on Particles Targeted Nucleic Acid Delivery |
Van Horne B |
| |
* Ken Yamada, University of Massachusetts Chan Medical School
Session Chair |
|
| |
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
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 |
| |
* Punit P. Seth, Alnylam Pharmaceuticals
Session Chair |
|
| |
* Eric Vallabh Minikel, Broad Institute
Session Chair |
|
| |
Brendon E Cook, Biogen
Studying Oligonucleotide Distribution after Intrathecal Treatment using Imaging |
|
| |
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 |
|
| 8:50–9:20 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 |
|
| |
Ying Tam, Acuitas Therapeutics
Chief Scientific Officer |
|
| |
Zachary Zappala, Vertex Pharmaceuticals
Director |
|
| |
Ralph J Garippa, Memorial Sloan Kettering Cancer Center
Director |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–6:45 PM |
New Approaches to Extrahepatic Delivery |
Van Horne B |
| |
* Samir EL Andaloussi, Karolinska Institutet
Session Chair |
|
| |
* Annemieke Aartsma-Rus, Leiden University Medical Center
Session Chair |
|
| |
Liqun Wang, Wyss Institute at Harvard University
Brain Delivery of Antibody-Oligonucleotide Conjugates for CNS Diseases |
|
| |
Vadim Dudkin, Souffle Therapeutics
Novel Ligands for Cell Selective Gene Silencing |
|
| |
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 |
| 8:00–10:30 AM |
Nucleic Acid Chemistry, Structure, Mechanism, Potency |
Van Horne B |
| |
* Anastasia Khvorova, RNA Therapeutics Institute, UMass Chan Medical School
Session Chair |
|
| |
* Steven F. Dowdy, University of California, San Diego
Session Chair |
|
| |
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 |
|
| |
Ruben Warkentin, Yale University
Short Talk: Advancing circRNA Biotechnologies through an Optimized Group I Intron Platform |
|
| |
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 |
| |
* Leah R Sabin, Regeneron Pharmaceuticals
Session Chair |
|
| |
* Annemieke Aartsma-Rus, Leiden University Medical Center
Session Chair |
|
| |
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 |
| |
* Anastasia Khvorova, RNA Therapeutics Institute, UMass Chan Medical School
Session Chair |
|
| |
* Brendon E Cook, Biogen
Session Chair |
|
| |
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 |
