POST-CONFERENCE VECTOR INNOVATION DAY

8:30 am Welcome & Introduction

9:00 am Comparison of Different AAV Vectors: Xenograft & Other Models

Synopsis

  • Talk details to follow

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 Non-Viral Expression of Full-Length Dystrophin for Treatment of Duchenne Muscular Dystrophy

  • Stan Froehner Co-Founder & Chief Executive Officer, Myosana Therapeutics

Synopsis

  • Using a non-viral gene delivery platform targeted to skeletal and cardiac muscle, we can express full-length dystrophin in dystrophic animals
  • Since the Myosana platform is non-viral, it allows for multiple doses which increases the level of full-length dystrophin expression
  • Myosana’s platform is applicable to many genetic diseases of skeletal and cardiac muscle

2:00 pm Optimizing Vectors for Cardiac Muscle Targeting

  • Jose Trevejo Chief Development Officer & Senior Vice President of AAV, Rocket Pharmaceuticals

Synopsis

  • Talk details to follow

2:30 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?

3:30 pm End of Post-Conference Vector Innovation Day