Joint with: Delivery of Nucleic Acid Therapeutics
Precision Genome Engineering
Jan 22–25, 2024 | Fairmont Banff Springs, Banff, AB, Canada
Scientific Organizers:
Morgan Maeder, Benjamin P. Kleinstiver and Kiran Musunuru
In Person
On Demand
Jan 22–25, 2024 | Fairmont Banff Springs, Banff, AB, Canada
Scientific Organizers:
Morgan Maeder, Benjamin P. Kleinstiver and Kiran Musunuru
Available Formats:
Supported by the Directors' Fund
In Person
On Demand
***Meeting program subject to change.
Available Formats: = In Person = On DemandBooking Function
Fundraising
Merchandise Options
Registration Options
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: Therapeutic Development of Editing Approaches
2:30–4:30 PM
Van Horne B
Lauren Testa, University of Pennsylvania
Base Editing and Prime Editing to Introduce and Correct Pathogenic Variants Causing Pseudoxanthoma Elasticum
Base Editing and Prime Editing to Introduce and Correct Pathogenic Variants Causing Pseudoxanthoma Elasticum
Christiano Alves, MGH
Development of a Customized Base Editor as a Treatment for a Severe Pediatric Vascular Disease
Development of a Customized Base Editor as a Treatment for a Severe Pediatric Vascular Disease
Mandana Arbab, Boston Children's Hospital and Harvard Medical School
Base Editing Rescue of Spinal Muscular Atrophy in Cells and in Mice
Base Editing Rescue of Spinal Muscular Atrophy in Cells and in Mice
Pavel Nash, Univeristy of Cambridge
Innovations in Mitochondrial Gene Therapy: mtZFNs and Beyond
Innovations in Mitochondrial Gene Therapy: mtZFNs and Beyond
Taylor Anglen, Duke University
Epigenetic Control of a Cardiac Gene Regulatory Element Modulates Therapeutically Relevant Phenotypes
Epigenetic Control of a Cardiac Gene Regulatory Element Modulates Therapeutically Relevant Phenotypes
Shawn Liu, Columbia University Medical Center
Edit DNA Methylation in vivo
Edit DNA Methylation in vivo
Coffee Available
4:30–5:00 PM
Van Horne Foyer
Building the Toolbox: New Systems and Technology Development
5:00–7:00 PM
Van Horne B
Benjamin P. Kleinstiver, Massachusetts General Hospital
Engineered CRISPR Enzymes to Improve Genome Editing
Engineered CRISPR Enzymes to Improve Genome Editing
Samuel H. Sternberg, Columbia University
Targeted DNA Integration in Mammalian Cells Using CRISPR-Associated Transposases
Targeted DNA Integration in Mammalian Cells Using CRISPR-Associated Transposases
Tessa M Bertozzi, Whitehead Institute for Biomedical Research
Short Talk: Short Talk: Epigenome Editing as a Tool for Basic Discovery and Therapeutic Intervention
Short Talk: Short Talk: Epigenome Editing as a Tool for Basic Discovery and Therapeutic Intervention
Patrick D. Hsu, Arc Institute | University of California, Berkeley
Short Talk: Short Talk: Discovery of a Unified RNA-guided Mechanism for Programmable Genome Manipulation
Short Talk: Short Talk: Discovery of a Unified RNA-guided Mechanism for Programmable Genome Manipulation
Hiroshi Nishimasu, RCAST, The University of Tokyo
Structure and Engineering of the RNA-Guided Cas7-11-Csx29 Nuclease-Protease Complex
Structure and Engineering of the RNA-Guided Cas7-11-Csx29 Nuclease-Protease Complex
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
Mechanisms of Editing
8:00–11:00 AM
Van Horne B
Jacob E. Corn, ETH Zürich
Repair Mechanisms if Double Strand Breaks
Repair Mechanisms if Double Strand Breaks
Britt Adamson, Princeton University
Highthroughput Profiling of Gene Edited Cells
Highthroughput Profiling of Gene Edited Cells
Alexis C. Komor †, University of California, San Diego
The Mechanisms of Base Editing
The Mechanisms of Base Editing
Ryuei Sato, University of Hawaii at Manoa
Short Talk: Short Talk: Directed Evolution of PhiC31 and Bxb1 Integrases to Integrate Large DNA Sequences in Mammalian Cells with Twin Prime Editing
Short Talk: Short Talk: Directed Evolution of PhiC31 and Bxb1 Integrases to Integrate Large DNA Sequences in Mammalian Cells with Twin Prime Editing
Rachel A Silverstein, Harvard University
Short Talk: Short Talk: Custom PAM-reprogrammed CRISPR-Cas Enzymes via Machine Learning
Short Talk: Short Talk: Custom PAM-reprogrammed CRISPR-Cas Enzymes via Machine Learning
Shengdar Q. Tsai, St. Jude Children's Research Hospital
Short Talk: Short Talk: Population-scale GUIDE-seq-2 Profiling Reveals Human Genetic Variation Frequently Affects Cellular Off-target Genome Editing Activity
Short Talk: Short Talk: Population-scale GUIDE-seq-2 Profiling Reveals Human Genetic Variation Frequently Affects Cellular Off-target Genome Editing Activity
Juliane Weller, Wellcome Sanger Institute
Short Talk: Short Talk: Generating Ultra-long Deletions using Paired Prime Editing
Short Talk: Short Talk: Generating Ultra-long Deletions using Paired Prime Editing
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
New Therapeutic Gene Editing Approaches
5:00–7:00 PM
Van Horne B
Morgan Maeder, Chroma Medicine
Therapeutic Development of Epigenetic Editors
Therapeutic Development of Epigenetic Editors
Andrew Anzalone, Prime Medicine, Inc.
Therapeutic Development of Prime Editing
Therapeutic Development of Prime Editing
Blythe Duke Sather, Tune Therapeutics
Harnessing the Power of Epi Editing for Clinical Benefit
Harnessing the Power of Epi Editing for Clinical Benefit
Dominique Brooks, University of Pennsylvania
Short Talk: Short Talk: In Vivo Correction of Human Phenylketonuria Variants Via Prime Editing and Base Editing
Short Talk: Short Talk: In Vivo Correction of Human Phenylketonuria Variants Via Prime Editing and Base Editing
Gabriele Casirati, Boston Children's Hospital/Dana Farber Cancer Institute
Short Talk: Short Talk: Epitope Edited Hematopoietic Stem Cells to Enable Immune-Based In Vivo Selection Of Genome-Engineered Cells
Short Talk: Short Talk: Epitope Edited Hematopoietic Stem Cells to Enable Immune-Based In Vivo Selection Of Genome-Engineered Cells
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: Technology Development
2:30–4:30 PM
Van Horne B
Nora Enright, Stanford University
Characterization and Design of Large Serine Recombinases Using Domain Swaps
Characterization and Design of Large Serine Recombinases Using Domain Swaps
Chad Moon, Arc Institute
Combinatorial Domain Screening for the Discovery of Stage-specific Epigenetic Interactions
Combinatorial Domain Screening for the Discovery of Stage-specific Epigenetic Interactions
Tanner Wiegand, Columbia University
Emergence of RNA-guided Transcription Factors via Domestication of Transposon-encoded Nucleases
Emergence of RNA-guided Transcription Factors via Domestication of Transposon-encoded Nucleases
Kyle Trudeau, Sana Biotechnology
Retargetable Fusogens Enable Delivery of Gene Editing Payloads with High Cell-specificity via Direct Coupling of Receptor Binding to Fusion
Retargetable Fusogens Enable Delivery of Gene Editing Payloads with High Cell-specificity via Direct Coupling of Receptor Binding to Fusion
Addison Wright, Scribe Therapeutics
Engineering CasX to Create a Gene Editor with Potent Activity in Non-human Primates
Engineering CasX to Create a Gene Editor with Potent Activity in Non-human Primates
Jonas Koeppel, Wellcome Sanger Institute
Randomizing the Human Genome by Engineering Recombination between Repeat Elements
Randomizing the Human Genome by Engineering Recombination between Repeat Elements
Shannon M Miller, Scripps Research
Engineering SpCas9 Towards Compatibility with Fully Chemically Modified sgRNA
Engineering SpCas9 Towards Compatibility with Fully Chemically Modified sgRNA
Olivier Belli, ETH Zurich
High-Throughput Precision Genome Editing Screens to Study Variants of Uncertain Significance in Their Genomic Context
High-Throughput Precision Genome Editing Screens to Study Variants of Uncertain Significance in Their Genomic Context
Coffee Available
4:30–5:00 PM
Van Horne Foyer
Gene Editing in the Clinic
5:00–6:45 PM
Van Horne B
Kiran Musunuru, University of Pennsylvania
Therapeutic Gene Editing for Cardiovascular and Metabolic Diseases: From the Leading Cause of Death to N-of-1 Disorders
Therapeutic Gene Editing for Cardiovascular and Metabolic Diseases: From the Leading Cause of Death to N-of-1 Disorders
Waseem Qasim, University in London
CRISPR and Base Edited Therapeutic T Cells
CRISPR and Base Edited Therapeutic T Cells
Janice Chen, Mammoth Biosciences, Inc.
Developing in vivo Therapeutics with Ultracompact CRISPR Systems
Developing in vivo Therapeutics with Ultracompact CRISPR Systems
Giulia Schiroli, Tessera Therapeutics
Short Talk: Short Talk: In Vivo HSC Gene Editing for Correction of Sickle Cell Mutation using RNA Gene Writers
Short Talk: Short Talk: In Vivo HSC Gene Editing for Correction of Sickle Cell Mutation using RNA Gene Writers
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
6:45–7:00 PM
Van Horne B
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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