Welcome to the 2nd Gene Therapy for Muscular Disorders Summit

Revolutionizing Muscular Gene Therapy Development

Successfully Targeting & Transducing the Muscle Whilst Mitigating Toxicity

Touching on state-of-the-art discovery research to optimally target and transduce muscle cells, through to clinical challenges facing more advanced programs, join leading gene therapy developers focused on AAV and gene editing technologies to:


Overcome immunogenicity challenges and mitigate toxicity with high doses

Optimize their vector and promoter choice and design for more efficient delivery to muscle targets

Discuss the transduction and differentiation of both skeletal and cardiac muscle cells

Define clinically meaningful muscular endpoints to successfully develop and safely deliver the optimal dose to their muscular targets

Understand the patient’s perspective through insights from key patient advocates

Tackle regulatory challenges to demonstrate gene therapy durability

Don’t miss your chance to share knowledge, catch up with friendly faces and make new connections from the likes of Vertex, Genethon, Roche, Novartis and AskBio to help you to develop truly transformative gene therapies targeting the muscle.

View the event guide to see what was discussed last year.

“Speakers were fantastic. Overall a very high quality performance.”
Larry Shoemaker, Associate Professor, University of Florida

“Professional and quality content.”
Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association