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
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
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?