POST-CONFERENCE VECTOR INNOVATION DAY

8:45 am Welcome & Introduction

9:00 am Machine Learning & the Future of AAV Capsid Design

Synopsis

  • What is Machine Learning and how can it be used to sample the Protein Sequence Space.
  • Types of Neural Network Architecture used in Machine Learning
  • Development of AAV capsid variant training set for Neural Networks
  • Deep Learning Model designed Capsids

9:30 am Optimizing AAV Selectivity to Target Muscle Cells Using Synthetic Promoters

Synopsis

  • Identification of endogenous muscle-selective regulatory elements (enhancers and promoters)
  • Modular design of synthetic promoters utilising muscle-selective regulatory elements
  • Characterization of synthetic promoter activity in vivo

10:00 am Vector Discovery Evaluation: Directed Evolution Vs Rational Design

Synopsis

  • Utilize a rational design approach to modify AAV capsids
  • Goal to increase tropism and expression in muscle
  • Leverage muscle biology and disease understanding to select candidates

10:30 am Coffee Break

11:00 am RoundTable Discussion: Current Challenges & Opportunities

Join a round-table discussion led by a subject matter expert to share your opinion and collectively discuss solutions to existing challenges, specifically associated with targeting the muscle.

A: Cardiac Muscle

B: Vector Discovery

C: Muscular Promoters

D: Non-Viral Vectors

12:00 pm Lunch Break

1:00 pm MyoAAV for Targeted Skeletal Muscle Delivery for DMD & XLMTM

Synopsis

  • A family of muscle-tropic capsids identified by directed evolution in mice and primates
  • MyoAAV transduction is dependent on integrin heterodimers in mouse and human cells
  • MyoAAV administration at low dose results in therapeutic efficacy in disease models
  • Systemically-administered MyoAAV transduces primate muscles highly efficiently

1:30 pm Targeting Cardiac Muscle with AAV: Past, Present & Future

Synopsis

  • AAV9 cardiotropism: pre-clinical and clinical evidence
  • AAV optimization: potential for unintended consequences
  • Enhanced and selective cardiac transduction: potential future strategies

2:00 pm Panel Discussion: Optimizing Muscle Targeting

Synopsis

  • Evaluating the vectors and promoters currently used for muscular disorders
  • Discussing the challenges and opportunities of current vector discovery approaches
  • Which vectors are showing preclinical promise for the muscle? Is non-viral a viable option?

2:30 pm End of Post-Conference Vector Innovation Day