Joint with: Precision Genome Engineering
Delivery of Nucleic Acid Therapeutics
Jan 22–25, 2024 | Fairmont Banff Springs, Banff, AB, Canada
Scientific Organizers:
Daniel J. Siegwart and Kathryn Whitehead
In Person
On Demand
Jan 22–25, 2024 | Fairmont Banff Springs, Banff, AB, Canada
Scientific Organizers:
Daniel J. Siegwart and Kathryn Whitehead
Available Formats:
Supported by the Directors' Fund
In Person
On Demand
***Meeting program subject to change.
Available Formats: = In Person = On DemandMerchandise Options
Registration Options
Booking Function
Fundraising
Monday, January 22, 2024
Arrival and Registration
4:00–8:00 PM
Van Horne Foyer
Welcome Mixer
6:00–8:00 PM
Van Horne Foyer
Tuesday, January 23, 2024
Breakfast
7:00–8:00 AM
President's Hall
Welcome and Keynote Address (Joint)
8:00–9:00 AM
Van Horne A/B
Jennifer A. Doudna, HHMI/University of California, Berkeley
Delivering the Future of CRISPR-directed Genome Editing
Delivering the Future of CRISPR-directed Genome Editing
Gene Editing: Past and Future (Joint)
9:00–11:15 AM
Van Horne A/B
Sabin Mulepati, Intellia Therapeutics
Progress of CRISPR Clinical Programs
Progress of CRISPR Clinical Programs
Gerald Schwank, ETH Zurich
In Vivo Base and Prime Editing of Pathogenic Mutations in Mice and Non-Human Primates
In Vivo Base and Prime Editing of Pathogenic Mutations in Mice and Non-Human Primates
Daniel G. Anderson †, Massachusetts Institute of Technology
Nucleic Acid Delivery Systems for RNA Therapy and Genome Editing
Nucleic Acid Delivery Systems for RNA Therapy and Genome Editing
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
Short Talk: Short Talk: In Utero Gene Editing for a Metabolic Liver Disease in Mice and Nonhuman Primates
Michael Ward, NIH
Short Talk: Short Talk: CRISPR Engineering and Phenotyping of a Large Isogenic Set of Neurodegeneration-Related Mutations in Human iPSC Models
Short Talk: Short Talk: CRISPR Engineering and Phenotyping of a Large Isogenic Set of Neurodegeneration-Related Mutations in Human iPSC Models
Coffee Break
9:30–9:50 AM
Van Horne Foyer
Poster Setup
11:15–1:00 PM
Van Horne C
On Own for Lunch
11:15–5:00 PM
Poster Viewing
1:00–10:00 PM
Van Horne C
Workshop 1
2:30–4:30 PM
Van Horne A
Rohan Palanki, University of Pennsylvania
Targeted Ionizable Lipid Nanoparticles for In Utero Gene Editing of Hematopoietic Stem Cells
Targeted Ionizable Lipid Nanoparticles for In Utero Gene Editing of Hematopoietic Stem Cells
Jon Martínez de Lagos Marcos, Universitat Pompeu Fabra
In vivo FiCAT Programmable Gene Writing Platform Based on Lipid Nanoparticles Delivery System
In vivo FiCAT Programmable Gene Writing Platform Based on Lipid Nanoparticles Delivery System
Colton Strong, University of British Columbia
Genetically Engineered Transfusable Platelets Using mRNA-Lipid Nanoparticles
Genetically Engineered Transfusable Platelets Using mRNA-Lipid Nanoparticles
Xin Ye, Genevant Sciences Corp.
A Novel Ligand-siRNA Conjugate Approach to RNAi in Hepatic Stellate Cells
A Novel Ligand-siRNA Conjugate Approach to RNAi in Hepatic Stellate Cells
Debadyuti Ghosh, University of Texas at Austin
Designing Lipid Nanoparticle Formulations for the Respiratory Tract
Designing Lipid Nanoparticle Formulations for the Respiratory Tract
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
Nanoparticle-Mediated Delivery of miR-590-3p Decreases Recurrent GBM Tumor Growth by Inhibits Multiple Oncogenic Nodes
Coffee Available
4:30–5:00 PM
Van Horne Foyer
Delivery of mRNA Therapeutics
5:00–7:00 PM
Van Horne A
Shehnaaz Suliman, ReCode Therapeutics
mRNA and the Lung - Progress in Extrahepatic Delivery
mRNA and the Lung - Progress in Extrahepatic Delivery
Pieter Cullis, University of British Columbia
A Career of Lipid Nanoparticle Development Leading to the Commercial Success of RNA Therapies
A Career of Lipid Nanoparticle Development Leading to the Commercial Success of RNA Therapies
Edward Hennessy, Moderna
Discovery of Novel Ionizable Lipids for LNP-Mediated Delivery of Therapeutic mRNA
Discovery of Novel Ionizable Lipids for LNP-Mediated Delivery of Therapeutic mRNA
Ameya R Kirtane, Brigham and Women's Hospital, Harvard Medical School
Short Talk: Short Talk: Nature-Inspired mRNA-Based Radioprotectants for Improving Cancer Therapy
Short Talk: Short Talk: Nature-Inspired mRNA-Based Radioprotectants for Improving Cancer Therapy
Yen Anh Nguyen, UMass Chan Medical School
Short Talk: Short Talk: Developing Lipid Nanoparticle Formulations for mRNA Delivery in the Brain
Short Talk: Short Talk: Developing Lipid Nanoparticle Formulations for mRNA Delivery in the Brain
Social Hour with Lite Bites
7:00–8:00 PM
President's Hall
Poster Session 1
7:30–10:00 PM
Van Horne C
Wednesday, January 24, 2024
Breakfast
7:00–8:00 AM
President's Hall
Delivery of siRNA and ASOs
8:00–11:00 AM
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
A Divalent siRNA Chemical Scaffold for Potent and Sustained Modulation of Gene Expression throughout the Central Nervous System
Millicent Sullivan, University of Delaware
Collagen-assisted Gene Therapy for Regenerative Medicine
Collagen-assisted Gene Therapy for Regenerative Medicine
Yoon Yeo, Purdue University
Nucleic Acid and Oligonucleotide Delivery for Activating Innate Immunity in Cancer Immunotherapy
Nucleic Acid and Oligonucleotide Delivery for Activating Innate Immunity in Cancer Immunotherapy
Marino Zerial, Max Planck Institute of Molecular Cell Biology and Genetics
Harnessing Endocytosis to Improve the Delivery of Oligonucleotide-Based Drugs
Harnessing Endocytosis to Improve the Delivery of Oligonucleotide-Based Drugs
Adam Marks, Mount Sinai Hospital
Short Talk: Short Talk: MicroRNA-Targeted Control of Messenger RNA Therapeutic Expression and Immunity
Short Talk: Short Talk: MicroRNA-Targeted Control of Messenger RNA Therapeutic Expression and Immunity
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
Anastasia Khvorova, University of Massachusetts Medical School
Professor
Professor
Marino Zerial, Max Planck Institute of Molecular Cell Biology and Genetics
Group Leader
Group Leader
Blythe Duke Sather, Tune Therapeutics
VP, Head of Research
VP, Head of Research
Hyongbum Henry Kim, Yonsei University College of Medicine
Principal Investigator
Principal Investigator
Michael Ward, NIH
Senior Investigator
Senior Investigator
Coffee Available
4:30–5:00 PM
Van Horne Foyer
Delivery of DNA
5:00–7:00 PM
Van Horne A
Angela K. Pannier, University of Nebraska-Lincoln
Nonviral DNA Delivery to Human Stem Cells
Nonviral DNA Delivery to Human Stem Cells
Kazunori Kataoka, University of Tokyo
Self-Assembled Polymeric Nanocarriers for Delivery of Nucleic Acid Therapeutics
Self-Assembled Polymeric Nanocarriers for Delivery of Nucleic Acid Therapeutics
Zhenpeng Qin, University of Texas at Dallas
Short Talk: Short Talk: Focused-Ultrasound Mediated Gene Delivery to Brain Endothelium Without Blood-Brain Barrier Opening
Short Talk: Short Talk: Focused-Ultrasound Mediated Gene Delivery to Brain Endothelium Without Blood-Brain Barrier Opening
Joanna Yang, Johns Hopkins University
Short Talk: Short Talk: Development of Plasmids for Transcriptionally Targeted Immunological Reprogramming of Hepatocellular Carcinoma with Polymeric Nanoparticles
Short Talk: Short Talk: Development of Plasmids for Transcriptionally Targeted Immunological Reprogramming of Hepatocellular Carcinoma with Polymeric Nanoparticles
Douglas Brown, Entos Pharmaceuticals
Short Talk: Short Talk: Durable DNA-based Gene Therapy Using the Non-Viral Fusogenix Proteolipid Vehicle Platform
Short Talk: Short Talk: Durable DNA-based Gene Therapy Using the Non-Viral Fusogenix Proteolipid Vehicle Platform
Melissa Hanson, Bristol Myers Squibb
Short Talk: Short Talk: Development of Novel Triple-Payload Lipid Nanoparticles for Autologous Ex-Vivo Gene Editing
Short Talk: Short Talk: Development of Novel Triple-Payload Lipid Nanoparticles for Autologous Ex-Vivo Gene Editing
Social Hour with Lite Bites
7:00–8:00 PM
President's Hall
Poster Session 2
7:30–10:00 PM
Van Horne C
Thursday, January 25, 2024
Breakfast
7:00–8:00 AM
President's Hall
Keynote Address (Joint)
8:00–9:00 AM
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
Mammalian Retrovirus-Like Protein PEG10 Based Delivery Systems for mRNA Delivery and Genome Engineering
Coffee Break
9:00–9:20 AM
Van Horne Foyer
Delivery of Gene Editors (Joint)
9:00–11:00 AM
Van Horne A/B
Kathryn Whitehead, Carnegie Mellon University
Lipid Nanoparticles for mRNA Delivery: The Exquisite Effects of Chemistry on Biological Function
Lipid Nanoparticles for mRNA Delivery: The Exquisite Effects of Chemistry on Biological Function
Daniel J. Siegwart, University of Texas Southwestern Medical Center
Selective Organ Targeting (SORT) Lipid Nanoparticles (LNPs) for Tissue-Specific Gene Editing
Selective Organ Targeting (SORT) Lipid Nanoparticles (LNPs) for Tissue-Specific Gene Editing
Christopher Baehr, UC Berkeley
Short Talk: Short Talk: Engineered Neuron Targeting CRISPR RNPs for Improved Genome Editing in the Brain
Short Talk: Short Talk: Engineered Neuron Targeting CRISPR RNPs for Improved Genome Editing in the Brain
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
Short Talk: Short Talk: Development of Immune Modulating Ionizable Lipids and Their Lipid Nanoparticle Formulation for mRNA Vaccines and Therapeutics
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
Workshop 2
2:30–4:30 PM
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
Lipid Nanoparticle-Mediated RNA Delivery to Hematopoietic and T-Cells in Humanized Mice and Non-Human Primates
Won Jong Kim, POSTECH
Polymer Nanoparticles for mRNA Delivery
Polymer Nanoparticles for mRNA Delivery
Emma Kang, UBC
mRNA-LNP Engineering of Donor Platelets is Compatible with Current Blood Banking Practices
mRNA-LNP Engineering of Donor Platelets is Compatible with Current Blood Banking Practices
Will Dowell, The University of Vermont
Hallmarks of mRNA/LNP Immunogenicity for the Rational Design of Vaccines and Therapeutics
Hallmarks of mRNA/LNP Immunogenicity for the Rational Design of Vaccines and Therapeutics
Blake Lash, MIT
Engineering Domesticated Retroelements for Therapeutic mRNA Delivery
Engineering Domesticated Retroelements for Therapeutic mRNA Delivery
Tetiana Korzun, OHSU
Probing Reactogenicity Mechanisms for Safer mRNA LNP Formulations
Probing Reactogenicity Mechanisms for Safer mRNA LNP Formulations
Coffee Available
4:30–5:00 PM
Van Horne Foyer
Applications of Nucleic Acid Delivery
5:00–6:45 PM
Van Horne A
Niren Murthy, University of California, Berkeley
Acid degradable lipid nanoparticles efficiently deliver mRNA to multiple organs in vivo
Acid degradable lipid nanoparticles efficiently deliver mRNA to multiple organs in vivo
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
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
Delivery of mRNA Vaccines
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
6:45–7:00 PM
Van Horne A
Social Hour with Lite Bites
7:00–8:00 PM
President's Hall
Poster Session 3
7:30–10:00 PM
Van Horne C
Friday, January 26, 2024
Departure
7:00–11:59 PM
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