Strike the Optimal Balance Between High Dose & Mitigating Toxicity to Maintain Successful Expression in Muscle

With a focus on AAV and gene editing technologies and insights from Pfizer, Solid Biosciences, Audentes and Vertex, this niche 3-day agenda has been specifically designed to help industry experts to:

  • Optimize your promoter choice and navigate IP challenges for more efficient delivery to muscular targets
  • Discover the next generation capsids being developed for improved muscle selectivity and reduced immunogenic responses
  • Understand the biology of different immunogenic responses and hear feedback from trialled strategies to ensure optimal patient safety
  • Hear the latest research in muscular disorder biomarkers
  • Define clinically meaningful muscular endpoints to successfully and objectively measure muscular disease improvement
  • Tackle regulatory challenges to demonstrate gene therapy durability
  • Overcome clinical challenges to manage patient heterogeneity for patient centric and meaningful trials

Join your peers to share, learn and discuss at the first industry-focused Gene Therapy for Muscular Disorders. Overcome your immunogenicity challenges and mitigate toxicity with high doses targeting the muscle to successfully demonstrate durability with your gene therapy product.

View the event guide for the full list of hot topics and the expert industry speaker faculty.