*All times are in EDT
8:50 am Chair’s Opening Remarks
ASSESSING APPROACHES TO DEVELOP MORE TARGETED, LESS IMMUNOGENIC NOVEL CAPSIDS
9:00 am Optimizing AAV Capsids for In Vivo Gene Delivery Using Artificial Intelligence
Synopsis
- Engineered capsids improve the efficiency and tissue specificity of gene delivery relative to natural AAV variants
- Next-gen technologies enable comprehensive mapping of the capsid fitness landscape in NHP models
- Artificial intelligence dramatically accelerates the design of improved capsids for gene delivery
9:30 am Delivering Close-Ended DNA for Next-Generation Non-Viral Gene Therapy
Synopsis
- Utilizing LNPs to deliver gene therapies – POC in mouse disease model, NHP translation
- Understanding specific disease areas/cell types where ceDNA would offer significant advantages over viral approaches
- Solving challenges associated with cell-specific targeting using LNPs
- Non-viral approaches can avoid limitations associated with payload size and immunities of AAVs
10:00 am Standardizing rAAV Vector Production
Synopsis
- Create standardized AAV vector purification reagents and processes
- Optimize rAAV vector purification reagents and processes matched to the full spectrum of rAAV serotypes
- Progression from traditional AAV delivery to non-viral delivery
10:10 am Virtual Speed Networking
10:40 am Morning Break
ADDRESSING IMMUNOGENICITY OF AAV BY MODIFYING VECTOR DESIGN
11:10 am ATHENA: Solving the Puzzle
11:20 am PANEL DISCUSSION: Assessing Capsid Engineering Techniques – Searching for the ‘Perfect’ Capsid
Synopsis
- Discussing different techniques to engineer capsids
- How can we assess which capsids are not only effective but also scalable in manufacturing?
- Will, there ever be a ‘one size fits all’ capsid?
- Highlighting progress in preclinical and clinical trials with optimized AAV capsids
- Can lentiviral capsids be improved in a similar way to AAV – what are the cross-learnings?
12:00 pm Lunch & Networking
INCREASING VECTOR TROPISM TO INCREASE SPECIFICITY & REDUCE TOXICITY
1:30 pm Improving Liver Transduction through AAV Capsid Modifications
Synopsis
- More efficient targeting of specific cells can be achieved through capsid engineering
- Setting up the right model is critical to screen most potent capsids for human application
- Increasing expression in desired targets leads to lower doses required which is key in reducing toxicity in systemic delivery
- Sharing data on novel capsid variants with increased liver tropism
2:00 pm Development of Novel Capsids, Optimized Gene Regulation and Delivery for Safe and Effective AAV-based Cardiac Therapies
Synopsis
- Capsid engineering, microRNA de-targeting, and promoter optimization can all be used to effectively limit transgene expression to specific cell types within the heart
- Careful selection and optimization of the route of administration may facilitate cardiac efficacy while avoiding dose-limiting liver toxicity
- Sf9 manufacturing can enable scalable production of effective AAV therapeutics for prevalent indications such as cardiomyopathy
2:05 pm
A HOLISTIC APPROACH TO GENE THERAPY VECTOR DESIGN & DEVELOPMENT
2:30 pm Discovering the Next Generation AAV Vector Through Capsid Engineering & Expression Cassette Optimization
Synopsis
- Novel methods to build AAV capsid libraries for in vivo screening through rational design and directed evolution
- Tissue specific promoter and enhancer engineering to optimize transgene expression