Nucleic Acid Therapeutics and Targeted Delivery
Mar 09–12, 2026 | Fairmont Banff Springs, Banff, AB, Canada
Scientific Organizers:
Anastasia Khvorova, Eric Vallabh Minikel, Annemieke Aartsma-Rus, and Holly Kordasiewicz
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Mar 09–12, 2026 | Fairmont Banff Springs, Banff, AB, Canada
Scientific Organizers: Anastasia Khvorova, Eric Vallabh Minikel, Annemieke Aartsma-Rus, and Holly Kordasiewicz
Anastasia Khvorova, PhD
University of Massachusetts Medical School
Anastasia Khvorova, PhD is professor in the RNA Therapeutics Institute (RTI) and Program in Molecular Medicine at the University of Massachusetts Chan Medical School, where she established the Nucleic Acid Chemistry Center, which provides leading expertise in RNA chemistry and therapeutic RNA optimization for researchers worldwide. Her current research focuses on delivering nucleic acid-based therapies to the brain and other extrahepatic tissues, work recognized by the 2025 Else Kröner Fresenius Prize for Medical Research. Dr. Khvorova has organized numerous scientific conferences, most recently for the Oligonucleotide Therapeutics Society (2025) and Cold Spring Harbor Laboratory (2025) and organized sessions at the American Association for Cancer Research (2024) annual conference.
Eric Vallabh Minikel, PhD
Broad Institute
Eric Minkel, PhD is Director of Prion Therapeutic Science at the Broad Institute in Cambridge, MA and Assistant Professor of Neurology at Massachusetts General Hospital. He retrained as a biomedical scientist after learning, in 2011, that his wife, Sonia Vallabh, inherited a pathogenic mutation in the PRNP gene from her mother who had died of genetic prion disease. Dr. Minikel’s research spans ASO and siRNA therapies for prion disease in preclinical models, as well as the creation and characterization of biomarkers, mouse models, and genomic and natural history datasets to enable clinical tools to support prion disease treatment.
Annemieke Aartsma-Rus, PhD
Leiden University Medical Center
Annemieke Aartsma-Rus, PhD is a professor of translational genetics at the Leiden University Medical Center in the Netherlands. She is a pioneer in antisense oligonucleotide-mediated exon skipping approaches targeting Duchenne muscular dystrophy (3 of 4 FDA-approved Duchenne oligonucleotide therapies are based on IP she generated). In 2000 she co-founded the Dutch Center for RNA Therapeutics, an academic center focused on developing individualized antisense oligonucleotides for patients with genetic brain and eye diseases. Dr. Aartsma-Rus has organized many scientific conferences including the annual conference of the Oligonucleotide Therapeutics Society and several Cold Spring Harbor Laboratory Nucleic Acid Therapies conferences.
Holly Kordasiewicz, PhD
Ionis Pharmaceuticals
Holly Kordasiewicz, PhD is the Senior Vice President of Neurology at Ionis Pharmaceuticals, where she leads the company’s neurology research and product efforts focused on. identifying disease-modifying RNA therapeutics for severe neurological diseases, which including drugs in clinical trials for Alzheimer’s disease, prion disease and multiple rare neurological diseases. Dr. Kordasiewicz has transitioned 15 medicines from research into clinical development, including Qalsody recently approved for the treatment of SOD1 ALS.
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On Demand
***Meeting program subject to change.
Available Formats: = In Person = On DemandMonday, March 9, 2026
Fundraising
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Registration
4:00–8:00 PM
Van Horne Foyer
Welcome Mixer
6:00–8:00 PM
Van Horne Foyer
Tuesday, March 10, 2026
Breakfast
7:00–8:00 AM
President's Hall
Welcome Remarks (Joint)
8:00–8:10 AM
Van Horne A/B
Keynote Session (Joint)
8:10–9:00 AM
Van Horne A/B
Jennifer A. Doudna, HHMI/University of California, Berkeley
CRISPR Enzyme Evolution and Applications
CRISPR Enzyme Evolution and Applications
Delivery (Joint)
9:00–11:00 AM
Van Horne A/B
Daniel J. Siegwart, University of Texas Southwestern Medical Center
LNP-SORT - Targeted Delivery Through Size and Charge
LNP-SORT - Targeted Delivery Through Size and Charge
Michael A. Fischbach, Stanford University
Engineered Viral-Like Particle for Targeted Delivery of Gene Editors
Engineered Viral-Like Particle for Targeted Delivery of Gene Editors
Leah R Sabin, Regeneron Pharmaceuticals
Antibody-AAV Targeting: Fundamental Principle Behind Achieving BBB Crossing
Antibody-AAV Targeting: Fundamental Principle Behind Achieving BBB Crossing
Coffee Break
9:30–9:50 AM
Van Horne Foyer
Award Recipient Acknowledgement
9:50–9:55 AM
Van Horne A/B
Poster Setup
11:00–1:00 PM
Van Horne C
On Own for Lunch
11:00–5:00 PM
Poster Viewing
1:00–10:00 PM
Van Horne C
Symposia Spotlight 1
2:30–4:30 PM
Van Horne B
Yoshikazu Nakamura †, University of Tokyo
Anti-FGF2 Aptamer Therapy in Achondroplasia
Anti-FGF2 Aptamer Therapy in Achondroplasia
Mathieu Cinier †, Affilogic
Nanofitin Scaffolds for Precision and Versatile Oligonucleotide Delivery
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
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
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
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
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
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
Click-Chemistry-Based Strategy for Modular Ligand Attachment to siRNAs: Toward Tissue-Specific RNAi
Coffee Available
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, RNA Therapeutics Institute, UMass Chan Medical School
Barcoded Libraries for Targeted siRNA Delivery
Barcoded Libraries for Targeted siRNA Delivery
Andrea L. Kasinski, Purdue University and LigamiR Therapeutics
Folate-miRNA Conjugates for Tumor Delivery
Folate-miRNA Conjugates for Tumor Delivery
Steven F. Dowdy, University of California, San Diego
How to Tackle Endosomal Escape
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
Short Talk: Safe and Effective Extrahepatic mRNA Delivery for CRISPR Gene Editing Using Fusogenix Proteolipid Vehicles
Shreyas G Iyer †, Purdue University
Short Talk: Development of a Fully Modified MicroRNA-34a (FM-mir-34a) Cancer Therapeutic: A Translational Research Study
Short Talk: Development of a Fully Modified MicroRNA-34a (FM-mir-34a) Cancer Therapeutic: A Translational Research Study
Social Hour with Dinner
7:00–8:00 PM
President's Hall
Poster Session 1
7:30–10:00 PM
Van Horne C
Wednesday, March 11, 2026
Breakfast
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
Studying Oligonucleotide Distribution after Intrathecal Treatment using Imaging
Shalini Andersson, AstraZeneca R&D
Oligonucleotide Biodistribution after Systemic Treatment
Oligonucleotide Biodistribution after Systemic Treatment
Eunji Chung †, University of Southern California
Short Talk: mRNA Delivery for Genetic Kidney Disease
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
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
Short Talk: Ocular Gene Therapy Using the Proteolipid Vehicle Nucleic Acid Delivery Platform
Eliza A Filipiak †, Karolinska Institutet
Short Talk: Lipid-Peptide Linker Design for Enhanced Therapeutic Delivery of Oligonucleotides
Short Talk: Lipid-Peptide Linker Design for Enhanced Therapeutic Delivery of Oligonucleotides
Coffee Break
9:00–9:20 AM
Van Horne Foyer
Poster Setup
11:00–1:00 PM
Van Horne C
On Own for Lunch
11:00–5:00 PM
Poster Viewing
1:00–10:00 PM
Van Horne C
Career Roundtable (Joint)
3:00–4:30 PM
Van Horne B
Coffee Available
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
Brain Delivery of Antibody-Oligonucleotide Conjugates for CNS Diseases
Holly Kordasiewicz, Ionis Pharmaceuticals
Extra Hepatic Delivery of Oligonucleotides
Extra Hepatic Delivery of Oligonucleotides
Scarlett Barker, Denali Therapeutics
Delivery of ASOs to the CNS using hTfR Antibodies
Delivery of ASOs to the CNS using hTfR Antibodies
June Y Park, Cenos Therapeutics
Micellar RNAi Conjugates for Extrahepatic Delivery
Micellar RNAi Conjugates for Extrahepatic Delivery
Social Hour with Dinner
7:00–8:00 PM
President's Hall
Poster Session 2
7:30–10:00 PM
Van Horne C
Thursday, March 12, 2026
Breakfast
7:00–8:00 AM
President's Hall
Nucleic Acid Chemistry, Structure, Mechanism, Potency
8:00–11:00 AM
Van Horne B
Speaker to be Announced
Ken Yamada, University of Massachusetts Chan Medical School
exNA and Ionizable Motifs Towards Next Generation RNA Therapeutics
exNA and Ionizable Motifs Towards Next Generation RNA Therapeutics
Anna Marie Pyle, Yale University
Structural Exploration of RNA Drug Function
Structural Exploration of RNA Drug Function
Punit P. Seth, Alnylam Pharmaceuticals
Chemical Modifications of siRNA and ASOs
Chemical Modifications of siRNA and ASOs
Karin Broennimann †, Weizmann Institute of Science
Short Talk: N1-Methylpseudouridine Directly Modulates Translation Dynamics
Short Talk: N1-Methylpseudouridine Directly Modulates Translation Dynamics
Coffee Break
9:00–9:20 AM
Van Horne Foyer
On Own for Lunch
11:00–5:00 PM
Symposia Spotlight 2
2:30–4:30 PM
Van Horne B
Barney Hill †, University of Oxford
Accurately Modelling RNase H-mediated Antisense Oligonucleotide Efficacy and Toxicity
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
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
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
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
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
Development of a Dual Action mRNA Therapeutic to Treat Atherosclerosis
Addison C Hill †, East Tennessee State University
Targeting Hepatitis B Virus: Synthetic gRNA/Cas12a and gRNA/Cas9 Ribonucleoproteins as a Novel Therapeutic
Targeting Hepatitis B Virus: Synthetic gRNA/Cas12a and gRNA/Cas9 Ribonucleoproteins as a Novel Therapeutic
Michael Siroky †, Scripps Research
Engineering AAV to encapsidate RNA
Engineering AAV to encapsidate RNA
Coffee Available
4:30–5:00 PM
Van Horne Foyer
Clinical Innovation in RNA-based Drug Development
5:00–7:00 PM
Van Horne B
Scott Schobel, VICO Therapeutics
ASO Development for Neurodegenerative Diseases
ASO Development for Neurodegenerative Diseases
Annemieke Aartsma-Rus, Leiden University Medical Center
N-of1 based ASOs
N-of1 based ASOs
Eric Vallabh Minikel, Broad Institute
Progress in PrP-Lowering Oligonucleotides for Prion Disease
Progress in PrP-Lowering Oligonucleotides for Prion Disease
Scott Johnson, Comanche
Developing RNA-based Drugs for Diseases of Pregnancy: The Preeclampsia Story
Developing RNA-based Drugs for Diseases of Pregnancy: The Preeclampsia Story
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
7:00–7:15 PM
Van Horne B
Social Hour with Dinner
7:15–8:00 PM
President's Hall
Cash Bar
8:00–11:00 PM
Entertainment
8:00–11:00 PM
Friday, March 13, 2026
Departure
12:00–11:59 PM
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