Pre Conference Workshop Day | Tuesday, June 12 2024

8:30 – 10:30 Workshop A

De-Risking the Human Translatability Piece of Next-Generation Vectors

  • Robert Lin CEO, Avista Therapeutics
  • Leszek Lisowski Unit Head Of Translational Vectorology Unit, Children's Medical Research Institute

Synopsis

  • Highlighting different approaches to produce robust, translatable data when transitioning through preclinical studies
  • Better understanding disease mechanisms and anticipated clinical responses to guide vector selection and design
  • Exploring more predictive preclinical models (e.g., human cells, organoids, assembloids) for both vector validation and development
  • Outlining the role of vector bioengineering for fit-for-purpose human tissue types
  • Reducing the number of animal model studies to lower costs, while maintaining good clinical translatability

11:00 – 1:00 Workshop B

Improved AAV Vector Design to Manage Systemic Toxicity & Immunogenicity

Synopsis

  • Determining which vector components trigger liver toxicity and immune responses
  • Capsid and payload optimization strategies to reduce toxicity and immunogenicity
  • Understanding the latest tissue targeting and de-targeting vector design approaches to evade the liver
  • Tissue tropism considerations for choosing a route of administration
  • Levelling up characterisation and tissue tropism validation of novel vectors

2:00 – 4:00 Workshop C

A 101 in Biotech Financial Risk Management

Synopsis

  • How to demonstrate value proposition and attract investment as a biotech with a novel vector platform
  • ‘Build vs Buy’: weighing up whether to develop or license functionalities
  • Identifying healthy business development opportunities
  • Diversifying risk across a portfolio