Targeted Delivery of Nucleic Acid Medicines (J8)
      
      
      
      
      
      January 25-28, 2027
       | Keystone Resort, Keystone, CO, United States
      
      
      
         
          C. Frank Bennett, Beverly L. Davidson and Daniel G. Anderson
        
            
      
      
      
          
      
   
            
     
          
    
      
      
        
        
      
        
      
  
      
      
        
                
        
      
      
    
        
      
        
      
  
      
      
        
                
        
      
      
    
        
      
        
      
  
      
      
        
                
        
      
      
    
      
           
      
  
      
        
        
        
          
            | 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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            | 11:15–1:00 PM | 
            Poster Setup | 
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            | 11:15–5:00 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 | 
            Tissue Targeting of LNPs | 
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            |   | 
            Daniel G. Anderson, Massachusetts Institute of Technology LNP Delivery of Nucleic Acid Medicines | 
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            |   | 
            Jessica A. Couch †,  ReCode Therapeutics LNP Delivery with the CNS | 
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            |   | 
            Kathryn Whitehead †, Carnegie Mellon University Optimizing LNPs Delivery Vehicles | 
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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 Address (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 | 
            Ligand Mediated Delivery of Nucleic Acid Medicines | 
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            |   | 
            Deborah Law †, Xaira Therapeutics Machine Learning Design of Ligands for Targeted Delivery of Nucleic Acid Medicines | 
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            |   | 
            Vadim Dudkin, Souffle Therapeutics Novel Ligands for Cell Selective Gene Silencing | 
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            |   | 
            C. Frank Bennett, Ionis Pharmaceuticals, Inc. Targeted Delivery of Oligonucleotide Drugs | 
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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 | 
            Translation of Genetic Medicines to the Clinic | 
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            |   | 
            Joseph B. Bolen †, Orna Therapeutics Tissue Selective Delivery of Circular RNAs | 
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            |   | 
            Christian Mueller, Sanofi Genome Editing in vivo via Non-Viral Delivery Platforms | 
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            |   | 
            Dirk Grimm, University of Heidelberg AAV and Bocavirus Vector Engineering | 
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            |   | 
            Nicole K. Paulk, University of California, San Francisco AAV Delivery to Cancer Cells | 
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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 | 
             | 
          
        
        
        
         
      
      
        
                
        
      
      
    
      
           
      
  
      
        
      
  
      
      
        
        
        
        
          
            |   | 
            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 | 
            New Delivery Technologies | 
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            |   | 
            Dong-Jiunn Jeffery Truong †, Helmholtz Munich VLP Deliver of cas Enzymes | 
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            |   | 
            Akin Akinc, Aera Therapeutics Target LNPs for In Vivo T Cell Engineering | 
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            |   | 
            Bowen Li, University of Toronto AI-Driven Development of Nonviral Delivery Tools for Nucleic Acid Medicines | 
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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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