Agenda
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
Synopsis
- 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)
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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
Synopsis
- 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