Precision Genome Engineering (Q8)
January 22-25, 2024
| Fairmont Banff Springs, Banff, AB, Canada
Morgan Maeder, Benjamin P. Kleinstiver and Kiran Musunuru
Scholarship Deadline: Nov. 2, 2023 | Abstract Deadline: Nov. 2, 2023 | Early Registration Deadline: Nov. 21, 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: Therapeutic Development of Editing Approaches |
Van Horne B |
| |
Lauren Testa, University of Pennsylvania
Base Editing and Prime Editing to Introduce and Correct Pathogenic Variants Causing Pseudoxanthoma Elasticum |
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| |
Christiano Alves, MGH
Development of a Customized Base Editor as a Treatment for a Severe Pediatric Vascular Disease |
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Mandana Arbab, Boston Children's Hospital and Harvard Medical School
Base Editing Rescue of Spinal Muscular Atrophy in Cells and in Mice |
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Pavel Nash, Univeristy of Cambridge
Innovations in Mitochondrial Gene Therapy: mtZFNs and Beyond |
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| |
Taylor Anglen, Duke University
Epigenetic Control of a Cardiac Gene Regulatory Element Modulates Therapeutically Relevant Phenotypes |
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| |
Shawn Liu, Columbia University Medical Center
Edit DNA Methylation in vivo |
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| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–7:00 PM |
Building the Toolbox: New Systems and Technology Development |
Van Horne B |
| |
Benjamin P. Kleinstiver, Massachusetts General Hospital
Engineered CRISPR Enzymes to Improve Genome Editing |
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| |
Samuel H. Sternberg, Columbia University
Targeted DNA Integration in Mammalian Cells Using CRISPR-Associated Transposases |
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Tessa M Bertozzi, Whitehead Institute for Biomedical Research
Short Talk: Short Talk: Epigenome Editing as a Tool for Basic Discovery and Therapeutic Intervention |
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| |
Patrick D. Hsu, Arc Institute | University of California, Berkeley
Short Talk: Short Talk: Discovery of a Unified RNA-guided Mechanism for Programmable Genome Manipulation |
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| |
Hiroshi Nishimasu, RCAST, The University of Tokyo
Structure and Engineering of the RNA-Guided Cas7-11-Csx29 Nuclease-Protease Complex |
|
| 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 |
Mechanisms of Editing |
Van Horne B |
| |
Jacob E. Corn, ETH Zürich
Repair Mechanisms if Double Strand Breaks |
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Britt Adamson, Princeton University
Highthroughput Profiling of Gene Edited Cells |
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| |
Alexis C. Komor †, University of California, San Diego
The Mechanisms of Base Editing |
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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 |
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Rachel A Silverstein, Harvard University
Short Talk: Short Talk: Custom PAM-reprogrammed CRISPR-Cas Enzymes via Machine Learning |
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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 |
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| |
Juliane Weller, Wellcome Sanger Institute
Short Talk: Short Talk: Generating Ultra-long Deletions using Paired Prime Editing |
|
| 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 |
New Therapeutic Gene Editing Approaches |
Van Horne B |
| |
Morgan Maeder, Chroma Medicine
Therapeutic Development of Epigenetic Editors |
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Andrew Anzalone, Prime Medicine, Inc.
Therapeutic Development of Prime Editing |
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Blythe Duke Sather, Tune Therapeutics
Harnessing the Power of Epi Editing for Clinical Benefit |
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Dominique Brooks, University of Pennsylvania
Short Talk: Short Talk: In Vivo Correction of Human Phenylketonuria Variants Via Prime Editing and Base Editing |
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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 |
|
| 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: Technology Development |
Van Horne B |
| |
Nora Enright, Stanford University
Characterization and Design of Large Serine Recombinases Using Domain Swaps |
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Chad Moon, Arc Institute
Combinatorial Domain Screening for the Discovery of Stage-specific Epigenetic Interactions |
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Tanner Wiegand, Columbia University
Emergence of RNA-guided Transcription Factors via Domestication of Transposon-encoded Nucleases |
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| |
Kyle Trudeau, Sana Biotechnology
Retargetable Fusogens Enable Delivery of Gene Editing Payloads with High Cell-specificity via Direct Coupling of Receptor Binding to Fusion |
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Addison Wright, Scribe Therapeutics
Engineering CasX to Create a Gene Editor with Potent Activity in Non-human Primates |
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Jonas Koeppel, Wellcome Sanger Institute
Randomizing the Human Genome by Engineering Recombination between Repeat Elements |
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Shannon M Miller, Scripps Research
Engineering SpCas9 Towards Compatibility with Fully Chemically Modified sgRNA |
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| |
Olivier Belli, ETH Zurich
High-Throughput Precision Genome Editing Screens to Study Variants of Uncertain Significance in Their Genomic Context |
|
| 4:30–5:00 PM |
Coffee Available |
Van Horne Foyer |
| 5:00–6:45 PM |
Gene Editing in the Clinic |
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 |
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| |
Waseem Qasim, University in London
CRISPR and Base Edited Therapeutic T Cells |
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Janice Chen, Mammoth Biosciences, Inc.
Developing in vivo Therapeutics with Ultracompact CRISPR Systems |
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| |
Giulia Schiroli, Tessera Therapeutics
Short Talk: Short Talk: In Vivo HSC Gene Editing for Correction of Sickle Cell Mutation using RNA Gene Writers |
|
| 6:45–7:00 PM |
Meeting Wrap-Up: Outcomes and Future Directions (Organizers) |
Van Horne B |
| 7:00–8:00 PM |
Social Hour with Lite Bites |
President's Hall |
| 7:30–10:00 PM |
Poster Session 3 |
Van Horne C |
