Barry J Byrne, MD, PhD, on MDA Care Centers and the Future of Gene Therapy in Neuromuscular Disease

This is the second part of an interview with Barry J Byrne, MD, PhD. For the first part, click here.

“One of our own efforts [for the future] is to ensure that a level of expression can be sustained over the lifespan, meaning the potential for redosing AAV vectors. This would be relevant to individuals who either received first generation products or may receive a treatment at a very early age.”

CGTLive® recently spoke with Barry J Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida, about what attendees can expect at the upcoming 2025 MDA Clinical & Scientific Conference, which will be held from March 16 to 19, in Dallas, Texas. As part of the discussion, CGTLive asked Byrne about the MDA Care Centers, which the organization has established throughout the United States, and the future of the field of gene therapy for neuromuscular disease in general.

Byrne discussed the role and impact of MDA Care Centers, emphasizing their multidisciplinary approach to patient care. With over 150 sites across the US, these centers provide comprehensive support for people with neuromuscular diseases, including amyotrophic lateral sclerosis (ALS). The model ensures coordinated care, access to essential resources like durable equipment and testing, and availability of emerging therapies. Notably, in 2025, MDA will host a Care Center Directors meeting before the annual conference to share best practices, foster collaboration, and seek to enhance long-term patient outcomes.

With regard to the future of the field, Byrne highlighted recent advancements in gene therapy, particularly in the treatment of Duchenne muscular dystrophy (DMD). Notable progress includes improvements in capsid targeting and adeno-associated virus (AAV) vector manufacturing, which will lower costs and expand treatment accessibility. A major focus in DMD is on developing a larger dystrophin protein that more closely mimics the full-length, functional version found in unaffected individuals. Byrne also discussed MDA’s own efforts to innovate in the gene therapy space.

Byrne concluded by acknowledging the 75th anniversary of MDA. He expressed pride in MDA’s role in improving patient outcomes and contributing to the discovery of new therapies, and expressed hope that the coming years will bring continued progress.

Click here to register for the upcoming 2025 MDA Conference.