Precision Genome Engineering: From Laboratory to Clinic (J7)
      
      
      
      
      
      January 25-28, 2027
       | Keystone Resort, Keystone, CO, United States
      
      
      
         
          Erik J. Sontheimer and Leonela Amoasii
        
            
      
      
      
          
      
   
            
     
          
    
      
      
        
        
      
        
      
  
      
      
        
                
        
      
      
    
        
      
        
      
  
      
      
        
                
        
      
      
    
        
      
        
      
  
      
      
        
                
        
      
      
    
      
           
      
  
      
        
        
        
          
            | 4:00–8:00 PM | 
            Registration | 
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            | 6:00–8:00 PM | 
            Welcome Mixer | 
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            Tuesday, January 26, 2027
          
           
         
      
        
      
  
      
      
        
                
        
      
      
    
      
           
      
  
        
      
      
        
                
        
      
      
    
      
           
      
  
      
        
        
        
          
            | 8:00–9:00 AM | 
            Welcome and Keynote Address (Joint) | 
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            |   | 
            Eric N. Olson †, University of Texas Southwestern Medical Center Single-AAV base Editing Therapies for Duchenne Muscular Dystrophy | 
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            | 9:00–11:15 AM | 
            Viral Delivery of Genome Editors and Genetic Medicines (Joint) | 
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            |   | 
            Beverly L. Davidson, Children’s Hospital of Philadelphia and University of Pennsylvania AAV Capsid Engineering for Broad CNS Distribution | 
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            Sonia Vallabh †, Broad Institute Development of Medicines to Treat Prion Diseases: A Personal Journey | 
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            Charles Gersbach, Duke University AAV-Delivered Epigenome Editors | 
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            Short Talk(s) Chosen from Abstracts
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            | 9:30–9:50 AM | 
            Coffee Break | 
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            | 11:15–12:15 PM | 
            Panel Discussion: Lessons Learned So Far: From Concept to Clinic | 
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            | 12:15–1:00 PM | 
            Poster Setup | 
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            | 12:15–5:00 PM | 
            On Own for Lunch | 
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            | 1:00–10:00 PM | 
            Poster Viewing | 
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            | 2:30–4:30 PM | 
            Symposia Spotlight 1: Late-breaking research presentations selected from abstract submissions | 
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            Short Talks Chosen from Abstracts
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            | 4:30–5:00 PM | 
            Coffee Available | 
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            | 5:00–7:00 PM | 
            Next-Generation Editing Tools: Continuing Innovation to Push the Limits of Genome Engineering | 
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            |   | 
            Jennifer A. Doudna, HHMI/University of California, Berkeley CRISPR Genome Editing | 
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            |   | 
            Erik J. Sontheimer, University of Massachusetts Medical School Next-Generation Genome Editing Platforms | 
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            |   | 
            Luigi M. Naldini †, San Raffaele Telethon Institute for Gene Therapy Genome Editing in Hematopoietic Stem Cells | 
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            Short Talk(s) Chosen from Abstracts
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            | 7:00–8:00 PM | 
            Social Hour with Dinner | 
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            Wednesday, January 27, 2027
          
           
         
      
        
      
  
      
      
        
                
        
      
      
    
      
           
      
  
        
      
      
        
                
        
      
      
    
      
           
      
  
      
        
        
        
          
            | 8:00–9:00 AM | 
            Keynote Speaker (Joint) | 
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            |   | 
            David R. Liu †, Broad Institute, HHMI, and Harvard University Development and Application of Virus-Like Particles for Genome Editing in vivo | 
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            | 9:00–9:20 AM | 
            Coffee Break | 
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            | 9:00–11:00 AM | 
            In vivo Genome Editing via Non-Viral Delivery (Joint) | 
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            |   | 
            Birgit C Schultes, Intellia Therapeutics Inc. mRNA/LNP based Gene Editing | 
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            |   | 
            Rebecca Ahrens-Nicklas, Children's Hospital of Philadelphia Personalized Therapeutic Genome Editing for Hepatic Inborn Errors of Metabolism | 
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            |   | 
            Leah R Sabin †, Regeneron Pharmaceuticals Novel Delivery Strategies for Genetic Medicines | 
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            |   | 
            Short Talk(s) Chosen from Abstracts
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            | 11:00–1:00 PM | 
            Poster Setup | 
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            | 11:00–5:00 PM | 
            On Own for Lunch | 
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            | 1:00–10:00 PM | 
            Poster Viewing | 
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            | 3:00–4:30 PM | 
            Career Roundtable | 
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            | 4:30–5:00 PM | 
            Coffee Available | 
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            | 5:00–7:00 PM | 
            Editing with Confidence: Evaluating Safety, Specificity, and Genomic Integrity | 
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            |   | 
            Maria Mirotsou, Scribe Therapeutics Advancing In Vivo CRISPR CasX-Based Therapies to Target Key Dyslipidemia Drivers | 
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            |   | 
            Shengdar Q Tsai, St. Jude Children's Research Hospital Off-Target Profiling for Base and Prime Editing | 
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            |   | 
            Marcello Maresca, AstraZeneca Enhancing Prime Editing Platforms for in vivo Applications | 
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            Short Talk(s) Chosen from Abstracts
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            | 7:00–8:00 PM | 
            Social Hour with Dinner | 
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            Thursday, January 28, 2027
          
           
         
      
        
      
  
      
      
        
                
        
      
      
    
      
           
      
  
        
      
      
        
                
        
      
      
    
      
           
      
  
      
        
        
        
          
            | 8:00–11:00 AM | 
            Redefining Editors: From CRISPR Biology to Recombination and Integration | 
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            |   | 
            Sangsu Bae, Seoul National University Precise ADAR Base Editors and Prime Editor Enhancers | 
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            |   | 
            Kathleen Collins, University of California, Berkeley Transgene Insertion Editing with Engineered Retroelements | 
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            |   | 
            Hiroshi Nishimasu, RCAST, The University of Tokyo Engineering Programmable Transposases | 
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            Short Talk(s) Chosen from Abstracts
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            | 9:00–9:20 AM | 
            Coffee Break | 
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            | 11:00–5:00 PM | 
            On Own for Lunch | 
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            | 2:30–4:30 PM | 
            Symposia Spotlight 2: Late-breaking research presentations selected from abstract submissions | 
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            |   | 
            Short Talks Chosen from Abstracts
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            | 4:30–5:00 PM | 
            Coffee Available | 
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            | 5:00–6:45 PM | 
            Machine-Learned Molecules: AI-Driven Design in Genome Engineering & Delivery | 
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            |   | 
            Silvana Konermann †, Stanford University School of Medicine Protein Design and Genome Editor Enhancement | 
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            |   | 
            Peter Cameron †, Profluent Bio Building Customized Editors via Large Language Model-Based Protein Design | 
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            |   | 
            Bruno Emanuel Correia †, École Polytechnique Fédérale de Lausanne BindCraft, Protein Design, and Control over Genome Editing | 
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            Short Talk(s) Chosen from Abstracts
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            | 6:45–7:00 PM | 
            Meeting Wrap-Up: Outcomes and Future Directions (Organizers) | 
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            | 7:00–8:00 PM | 
            Social Hour with Dinner | 
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            | 8:00–11:00 PM | 
            Entertainment | 
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