Delivery of Nucleic Acid Therapeutics (Q7)
January 22-25, 2024
| Fairmont Banff Springs, Banff, AB, Canada
Daniel J. Siegwart and Kathryn Whitehead
Scholarship Deadline: Nov. 3, 2023 | Abstract Deadline: Nov. 3, 2023 | Early Registration Deadline: Nov. 22, 2023
| 4:00–8:00 PM |
Arrival and Registration |
Van Horne Foyer |
| 6:00–8:00 PM |
Welcome Mixer |
Van Horne Foyer |
Tuesday, January 23, 2024
| 7:00–8:00 AM |
Breakfast |
President's Hall |
| 8:00–9:00 AM |
Welcome and Keynote Address (Joint) |
Van Horne A/B |
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Jennifer A. Doudna, HHMI/University of California, Berkeley
Delivering the Future of CRISPR-directed Genome Editing |
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| 9:00–11:15 AM |
Gene Editing: Past and Future (Joint) |
Van Horne A/B |
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Sabin Mulepati, Intellia Therapeutics
Progress of CRISPR Clinical Programs |
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Gerald Schwank, ETH Zurich
In Vivo Base and Prime Editing of Pathogenic Mutations in Mice and Non-Human Primates |
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Daniel G. Anderson †, Massachusetts Institute of Technology
Nucleic Acid Delivery Systems for RNA Therapy and Genome Editing |
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William Peranteau, The Children's Hospital of Philadelphia
Short Talk: Short Talk: In Utero Gene Editing for a Metabolic Liver Disease in Mice and Nonhuman Primates |
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Michael Ward, NIH
Short Talk: Short Talk: CRISPR Engineering and Phenotyping of a Large Isogenic Set of Neurodegeneration-Related Mutations in Human iPSC Models |
|
| 9:30–9:50 AM |
Coffee Break |
Van Horne Foyer |
| 11:15–1:00 PM |
Poster Setup |
Van Horne C |
| 11:15–5:00 PM |
On Own for Lunch |
|
| 1:00–10:00 PM |
Poster Viewing |
Van Horne C |
| 2:30–4:30 PM |
Workshop 1 |
Van Horne A |
| |
Rohan Palanki, University of Pennsylvania
Targeted Ionizable Lipid Nanoparticles for In Utero Gene Editing of Hematopoietic Stem Cells |
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Jon Martínez de Lagos Marcos, Universitat Pompeu Fabra
In vivo FiCAT Programmable Gene Writing Platform Based on Lipid Nanoparticles Delivery System |
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Colton Strong, University of British Columbia
Genetically Engineered Transfusable Platelets Using mRNA-Lipid Nanoparticles |
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Xin Ye, Genevant Sciences Corp.
A Novel Ligand-siRNA Conjugate Approach to RNAi in Hepatic Stellate Cells |
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| |
Debadyuti Ghosh, University of Texas at Austin
Designing Lipid Nanoparticle Formulations for the Respiratory Tract |
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Hernando Lopez-Bertoni, Johns Hopkins School Of Medicine
Nanoparticle-Mediated Delivery of miR-590-3p Decreases Recurrent GBM Tumor Growth by Inhibits Multiple Oncogenic Nodes |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–7:00 PM |
Delivery of mRNA Therapeutics |
Van Horne A |
| |
Shehnaaz Suliman, ReCode Therapeutics
mRNA and the Lung - Progress in Extrahepatic Delivery |
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Pieter Cullis, University of British Columbia
A Career of Lipid Nanoparticle Development Leading to the Commercial Success of RNA Therapies |
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Edward Hennessy, Moderna
Discovery of Novel Ionizable Lipids for LNP-Mediated Delivery of Therapeutic mRNA |
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Ameya R Kirtane, Brigham and Women's Hospital, Harvard Medical School
Short Talk: Short Talk: Nature-Inspired mRNA-Based Radioprotectants for Improving Cancer Therapy |
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Yen Anh Nguyen, UMass Chan Medical School
Short Talk: Short Talk: Developing Lipid Nanoparticle Formulations for mRNA Delivery in the Brain |
|
| 7:00–8:00 PM |
Social Hour with Lite Bites |
President's Hall |
| 7:30–10:00 PM |
Poster Session 1 |
Van Horne C |
Wednesday, January 24, 2024
| 7:00–8:00 AM |
Breakfast |
President's Hall |
| 8:00–11:00 AM |
Delivery of siRNA and ASOs |
Van Horne A |
| |
Anastasia Khvorova, University of Massachusetts Medical School
A Divalent siRNA Chemical Scaffold for Potent and Sustained Modulation of Gene Expression throughout the Central Nervous System |
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Millicent Sullivan, University of Delaware
Collagen-assisted Gene Therapy for Regenerative Medicine |
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Yoon Yeo, Purdue University
Nucleic Acid and Oligonucleotide Delivery for Activating Innate Immunity in Cancer Immunotherapy |
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Marino Zerial, Max Planck Institute of Molecular Cell Biology and Genetics
Harnessing Endocytosis to Improve the Delivery of Oligonucleotide-Based Drugs |
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Adam Marks, Mount Sinai Hospital
Short Talk: Short Talk: MicroRNA-Targeted Control of Messenger RNA Therapeutic Expression and Immunity |
|
| 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) |
|
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Anastasia Khvorova, University of Massachusetts Medical School
Professor |
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Marino Zerial, Max Planck Institute of Molecular Cell Biology and Genetics
Group Leader |
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Blythe Duke Sather, Tune Therapeutics
VP, Head of Research |
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Hyongbum Henry Kim, Yonsei University College of Medicine
Principal Investigator |
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Michael Ward, NIH
Senior Investigator |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–7:00 PM |
Delivery of DNA |
Van Horne A |
| |
Angela K. Pannier, University of Nebraska-Lincoln
Nonviral DNA Delivery to Human Stem Cells |
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Kazunori Kataoka, University of Tokyo
Self-Assembled Polymeric Nanocarriers for Delivery of Nucleic Acid Therapeutics |
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Zhenpeng Qin, University of Texas at Dallas
Short Talk: Short Talk: Focused-Ultrasound Mediated Gene Delivery to Brain Endothelium Without Blood-Brain Barrier Opening |
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Joanna Yang, Johns Hopkins University
Short Talk: Short Talk: Development of Plasmids for Transcriptionally Targeted Immunological Reprogramming of Hepatocellular Carcinoma with Polymeric Nanoparticles |
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Douglas Brown, Entos Pharmaceuticals
Short Talk: Short Talk: Durable DNA-based Gene Therapy Using the Non-Viral Fusogenix Proteolipid Vehicle Platform |
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Melissa Hanson, Bristol Myers Squibb
Short Talk: Short Talk: Development of Novel Triple-Payload Lipid Nanoparticles for Autologous Ex-Vivo Gene Editing |
|
| 7:00–8:00 PM |
Social Hour with Lite Bites |
President's Hall |
| 7:30–10:00 PM |
Poster Session 2 |
Van Horne C |
Thursday, January 25, 2024
| 7:00–8:00 AM |
Breakfast |
President's Hall |
| 8:00–9:00 AM |
Keynote Address (Joint) |
Van Horne A/B |
| |
Feng Zhang, Broad Institute of MIT and Harvard University
Mammalian Retrovirus-Like Protein PEG10 Based Delivery Systems for mRNA Delivery and Genome Engineering |
|
| 9:00–9:20 AM |
Coffee Break |
Van Horne Foyer |
| 9:00–11:00 AM |
Delivery of Gene Editors (Joint) |
Van Horne A/B |
| |
Kathryn Whitehead, Carnegie Mellon University
Lipid Nanoparticles for mRNA Delivery: The Exquisite Effects of Chemistry on Biological Function |
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Daniel J. Siegwart, University of Texas Southwestern Medical Center
Selective Organ Targeting (SORT) Lipid Nanoparticles (LNPs) for Tissue-Specific Gene Editing |
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Christopher Baehr, UC Berkeley
Short Talk: Short Talk: Engineered Neuron Targeting CRISPR RNPs for Improved Genome Editing in the Brain |
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Hyukjin Lee, Ewha Womans University
Short Talk: Short Talk: Development of Immune Modulating Ionizable Lipids and Their Lipid Nanoparticle Formulation for mRNA Vaccines and Therapeutics |
|
| 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 |
Workshop 2 |
Van Horne A |
| |
Wangweerawong Apiwat, LNP -Lipid Chemistry
Lipid Nanoparticle-Mediated RNA Delivery to Hematopoietic and T-Cells in Humanized Mice and Non-Human Primates |
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| |
Won Jong Kim, POSTECH
Polymer Nanoparticles for mRNA Delivery |
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Emma Kang, UBC
mRNA-LNP Engineering of Donor Platelets is Compatible with Current Blood Banking Practices |
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| |
Will Dowell, The University of Vermont
Hallmarks of mRNA/LNP Immunogenicity for the Rational Design of Vaccines and Therapeutics |
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Blake Lash, MIT
Engineering Domesticated Retroelements for Therapeutic mRNA Delivery |
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Tetiana Korzun, OHSU
Probing Reactogenicity Mechanisms for Safer mRNA LNP Formulations |
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| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–6:45 PM |
Applications of Nucleic Acid Delivery |
Van Horne A |
| |
Niren Murthy, University of California, Berkeley
Acid degradable lipid nanoparticles efficiently deliver mRNA to multiple organs in vivo |
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| |
Hyongbum Henry Kim, Yonsei University College of Medicine
Application of Prime Editing to the Correction of Mutations and Phenotypes in Adult Mice with Liver and Eye Diseases |
|
| |
Norbert Pardi, University of Pennsylvania
Delivery of mRNA Vaccines |
|
| 6:45–7:00 PM |
Meeting Wrap-Up: Outcomes and Future Directions (Organizers) |
Van Horne A |
| 7:00–8:00 PM |
Social Hour with Lite Bites |
President's Hall |
| 7:30–10:00 PM |
Poster Session 3 |
Van Horne C |
