With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. Despite the successes, there are still withstanding challenges for gene therapy developers targeting the muscle when it comes to optimizing delivery and navigating toxicity to develop truly safe and transformative therapeutics for muscular disorders patients.
The first industry-dedicated Gene Therapy for Muscular Disorders Summit will focus on overcoming the translational and clinical challenges of developing genetic therapies targeting muscular disorders, including Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy (LGMD), Pompe Disease and X-Linked Myotubular Myopathy.
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.
To know more about Gene Therapy for Muscular Disorders please click here.