Pre-Conference Delivery Focus Day

Tuesday 28th November 2023

10:00 am Chair’s Opening Remarks

10:15 am Peptide Mediated Efficient Delivery of CRISPR Enzymes

  • Ross Wilson Adjunct Assistant Professor, Wilson Lab, University of California at Berkeley


  • Outlining a mixed peptide-CRISPR cargo method for efficient delivering
  • Maximizing both delivery efficiency and manufacturing scalability
  • Defining the ongoing research efforts needed to expand therapeutic capabilities 

10:45 am Efficient & precise in vivo CRISPR Delivery With Engineered Virus-Like Particles (eVLPs)


  • Understanding the basic platform and engineering efforts deployed to overcome delivery limitations
  • Assessing on- and off- target editing levels in comparison with AAV
  • Exploring in vivo therapeutic efficiency in the liver and retina

11:15 am Morning Refreshment Break & Networking

11:30 am Extracellular Vesicles (EVs): An Emerging Promising Delivery Vehicle


  • A focused look at the delivery mechanism of EVs
  • Outlining preclinical results to-date from EV delivery studies
  • Addressing where progress needs to be made to potentially improve loading capacity, safety, stability, and targeting profiles

12:00 pm Enabling Tissue-Specific in vivo Targeting With Lipid Nanoparticles


  • Outlining the key challenges facing tissue-specific CRISPR delivery
  • Examining the impacts of LNP composition on tissue targeting
  • Understanding the role of surface ligand conjugations for non-hepatic targeting and hepatic detargeting

12:30 pm Lunch & Networking

1:30 pm Next-Generation Engineering of AAV Vectors

  • Jing Liao Director - Vector Development & Operations, Alexion Pharmaceuticals


  • Presenting a novel AAV platform: where have improvements been made to traditional AAV vectors?
  • What role can novel AAV vectors play in precise genome engineering tool delivery?
  • Overcoming immunogenicity concerns

2:00 pm Digital Evolution of Gene Delivery Vehicles


  • Current state-of-play of viral gene delivery vehicles
  • Opportunities to improve vector discovery by digital simulation
  • Deep dive into specific examples of artificial intelligence-supported capsid evolution

2:30 pm Chair’s Closing Remarks