***Meeting program subject to change.
Available Formats: = In Person
= On Demand
Welcome and Keynote Address (Joint)
8:00–9:00 AM
Eric N. Olson †, University of Texas Southwestern Medical Center
Single-AAV base Editing Therapies for Duchenne Muscular Dystrophy
Viral Delivery of Genome Editors and Genetic Medicines (Joint)
9:00–11:15 AM
Panel Discussion: Lessons Learned So Far: From Concept to Clinic
9:00–11:15 AM
Beverly L. Davidson, Children’s Hospital of Philadelphia and University of Pennsylvania
AAV Capsid Engineering for Broad CNS Distribution
Sonia Vallabh †, Broad Institute
Development of Medicines to Treat Prion Diseases: A Personal Journey
Charles Gersbach †, Duke University
AAV-Delivered Epigenome Editors
Short Talk(s) Chosen from Abstracts
Symposia Spotlight 1: Late-breaking research presentations selected from abstract submissions
2:30–4:30 PM
Short Talks Chosen from Abstracts
Next-Generation Editing Tools: Continuing Innovation to Push the Limits of Genome Engineering
5:00–7:00 PM
Jennifer A. Doudna †, HHMI/University of California, Berkeley
CRISPR Genome Editing
Erik J. Sontheimer, University of Massachusetts Medical School
Next-Generation Genome Editing Platforms
Luigi M. Naldini †, San Raffaele Telethon Institute for Gene Therapy
Genome Editing in Hematopoietic Stem Cells
Short Talk(s) Chosen from Abstracts
David R. Liu †, Broad Institute, HHMI, and Harvard University
Development and Application of Virus-Like Particles for Genome Editing in vivo
In vivo Genome Editing via Non-Viral Delivery (Joint)
9:00–11:00 AM
Birgit C. Schultes †, IntelliaTherapeutics Inc.
Clinical Results from Liver Genome Editing in vivo
Rebecca Ahrens-Nicklas †, Children's Hospital of Philadelphia
Personalized Therapeutic Genome Editing for Hepatic Inborn Errors of Metabolism
Leah R. Sabin †, Regeneron Pharmaceuticals
Novel Delivery Strategies for Genetic Medicines
Short Talk(s) Chosen from Abstracts
Editing with Confidence: Evaluating Safety, Specificity, and Genomic Integrity
5:00–7:00 PM
Maria Mirotsou, Scribe Therapeutics
Compact, Self-Inactivating CasX Editing in vivo
Shengdar Q. Tsai †, St. Jude Children's Research Hospital
Off-Target Profiling for Base and Prime Editing
Marcello Maresca †, AstraZeneca
Enhancing Prime Editing Platforms for in vivo Applications
Short Talk(s) Chosen from Abstracts
Redefining Editors: From CRISPR Biology to Recombination and Integration
8:00–11:00 AM
Feng Zhang †, Broad Institute of MIT and Harvard University
Non-Canonical CRISPR Systems for Genome Editing
Sangsu Bae †, Seoul National University
Precise ADAR Base Editors and Prime Editor Enhancers
Kathleen Collins †, University of California, Berkeley
Transgene Insertion Editing with Engineered Retroelements
Hiroshi Nishimasu †, RCAST, The University of Tokyo
Engineering Programmable Transposases
Short Talk(s) Chosen from Abstracts
Symposia Spotlight 2: Late-breaking research presentations selected from abstract submissions
2:30–4:30 PM
Short Talks Chosen from Abstracts
Machine-Learned Molecules: AI-Driven Design in Genome Engineering & Delivery
5:00–6:45 PM
Silvana Konermann †, Stanford University School of Medicine
Protein Design and Genome Editor Enhancement
Peter Cameron †, Profluent Bio
Building Customized Editors via Large Language Model-Based Protein Design
Bruno Emanuel Correia †, École Polytechnique Fédérale de Lausanne
BindCraft, Protein Design, and Control over Genome Editing
Short Talk(s) Chosen from Abstracts
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
6:45–7:00 PM