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

The 2nd Gene Therapy for Muscular Disorders Summit took place in April this year, and is the field’s only industry-focused event specifically designed to help gene therapy developers successfully target the muscle.

With a disease-agnostic agenda, but highlighting novel research in Duchenne’s (DMD), Limb-Girdle (LGMD), Pompe Disease and X-Linked Myotubular Myopathy (XLMTM), the expert industry speakers from the likes of Solid Biosciences, Sarepta Therapeutics, Astellas Gene Therapies and Pfizer spent 4-days discussing the fields hottest topics from navigating toxicity to state-of-the-art muscular targeting.

View the 2022 speaker faculty and agenda here.

Expert Speakers Included:

Event Partners Included:

Media Partners Included:

Hear What Our Audience Say:

“Fantastic opportunity to learn from the experts!”
Karin Lucas, Associate Director, Sarepta Therapeutics

“The conference overall expanded my knowledge in gene therapy targeting muscle diseases but also offered additional learning in the areas of immunology and redosing strategies.”
Paul Fitzpatrick, Senior Director - Bioprocess Technology, Zogenix Inc

“Being new to the space this was a great opportunity to learn about the relevant areas for developing gene therapies in muscle.”
Lori Getts, Vice President - Preclinical & Process Development, Code Biotherapeutics

Other Events In The Gene Therapy Series: