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

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 focused 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.

2021 attendees included industry leaders from the likes of Sarepta, PTC Therapeutics, Solid Biosciences, Pfizer, AskBio & Novartis and many more.

What Will You Learn?

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How to navigate IP challenges & optimum promoter choice for more efficient delivery

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The next generation capsids being developed for improved muscle selectivity and reduced immunogenic responses

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The biology of different immunogenic responses - trialled strategies to ensure optimal patient safety

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Insights from the latest research in muscular disorder biomarkers

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Clinically meaningful muscular endpoints to successfully and objectively measure muscular disease improvement

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How to tackle regulatory challenges to demonstrate gene therapy durability

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How to overcome clinical challenges to manage patient heterogeneity for patient centric and meaningful trials

Who Attended in 2021?

2021 Event Partner:

Media Partners

Previous Gene Therapy Attendees Have Said

“Inspiring virtual meeting allowing efficient learning about the most recent advances in gene therapy immunogenicity while still having interactions with peers.”
Cornelia Ciorciaro, Director - Safety Science Group Medical, Roche

“It was great to have a conference focused on analytical challenges for testing immune markers and modulating immune responses to gene therapies.”
Michele Stone, Vice President - Translational Development, Kriya Therapeutics

“I learned a lot about the what, who & how of the AAV gene therapy field.”
Robert J Kubiak, Clinical Pharmacologist, AstraZeneca

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