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
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WORKSHOP B: Exploring Advances in Next Generation Capsids & Promoters
11.30AM-2.30PM | 8.30AM-11.30AM

Evaluating novel vectors, promoters and micro-dystrophin research for muscular disorders.

  • 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
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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
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