8:50 am Chair’s Opening Remarks

  • Robert Kotin Adjunct Professor, University of Massachusetts

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

  • Daozhan Yu President & Chief Executive Officer, AAVnerGene

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

  • Kathy Ivey Senior Director, Gene Therapy Research, Tenaya Therapeutics

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

  • Ye Liu Senior Director, Gene Transfer Technology, R&D, REGENXBIO

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

3:00 pm Chair’s Closing Remarks

  • Robert Kotin Adjunct Professor, University of Massachusetts