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

Returning to Boston this April as its first in-person event, the 2nd Gene Therapy for Muscular Disorders Summit 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 will spend 4-days discussing the fields hottest topics from navigating toxicity to state-of-the-art muscular targeting.

View the world-class speaker faculty and topics for discussion here

Your Expert Speakers Include:

Health & Safety

The health, safety and well-being of our community remains our highest priority, therefore, all attendees will be required to complete a health declaration prior to attending the event, as well as comply with the City of Boston’s B Together policy. This policy requires individuals to show you proof of vaccination against COVID-19 in order to gain admittance to the conference venue. For further information around Health and Safety onsite, please visit our FAQs page here or for more information on the B Together Policy, please see here.

* Please note, this is subject to change at any time without prior notice.

New for 2022:


Content: Address the opportunities and challenges facing cardiac muscle targeting


First In-Person Event: Make the most of your opportunity to catch up with familiar faces and make new connections in the muscular disorders field

New Companies: Discover and meet new speakers and companies to improve your understanding of the gene therapy landscape with muscular pipelines

Post-Conference Vector Innovation Day: Take part in the vector day for a more in-depth discussion to optimize vectors targeting the muscle

Who Attended in 2021?

Event Partners

Media Partners

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

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