Pre-Conference Workshop Day
Tuesday, May 25 2021
WORKSHOP A: Gene Therapy Immunogenicity: The Biology & Strategies
8AM-11AM | 5AM-8AM
As you seek to learn more about the gene therapy field, join this workshop to:
- Get up to speed with immunogenicity challenges specific to gene therapies targeting muscular disorders
- Improve your understanding of the different types of immunogenic responses to vector, transgene and complement activation.
- Discuss the strategies available and being explored to help mitigate immunogenic reactions both preclinical and in clinical trials

WORKSHOP B: Exploring Advances in Next Generation Capsids & Promoters
11.30AM-2.30PM | 8.30AM-11.30AM
Moving beyond oncology applications, discussing the latest preclinical research to optimize the patient conditioning process for ex vivo gene therapies.
- Hear the latest advances in AAV vectors and biodistribution to the muscle
- Discuss the suitability of novel non-viral vectors including nanoparticles and exosomes
- Understand the benefits and disadvantages of novel vectors

WORKSHOP C: Discovering the Latest Biology Research for Muscular Disorders
3PM-6PM | 8.30AM-11.30AM
- Learn about the latest muscle biology research and discuss how this may inform future drug development
- Debate the effect of satellite cells and understand the consequences of cell division and muscle regeneration
- Discuss fast twitch vs slow twitch fibres
- Explore smooth, cardiac and skeletal muscle differences to address gene therapy targeting
