Michael Kelly, PhD, on Gene Therapy’s Continuing Path to Treating DMD


The chief scientific officer of CureDuchenne discussed challenges to tackle in the field.

“We’ve come a long way in the last 10 years that I've been with the organization. A decade ago, there were no clinical trials. Today, we've got a handful of really meaningful therapies that are impacting patients, and I believe the pathway over the next 2 to 3 years is going to be even more exciting. [We have] newer technologies, more muscle-tropic capsids, non-AAV technologies. Right now, it’s about delivery, we've got the tools available in order to go after RNA, DNA and various other things. I think the next few years, we're going to see significant advances that really will impact this disease, and other ones, maybe.”

Challenges and questions with gene therapy for muscular dystrophies are still widespread in the field, despite the landmark approval of delandistrogene moxeparvovec (Elevidys) gene therapy in 2023 for patients with Duchenne muscular dystrophy (DMD).

Elevidys is currently approved for ambulatory patients aged 4 through 5 years, although Sarepta recently announced that the FDA had accepted its efficacy supplement to its biologics license application with priority review, to remove the age and ambulatory restrictions.1 Complicating the motion, the global, pivotal, phase 3 EMBARK study (Study SRP-9001-301; NCT05096221) that supported Elevidys’ approval has failed its primary end point.2

CGTLive spoke with Michael Kelly, PhD, chief scientific officer, CureDuchenne, to learn more about how the treatment field for DMD has evolved over the last decade. He also discussed collaboration with companies in the space, exciting projects in the field, and challenges that need to be tackled.

1. Sarepta Therapeutics announces U.S. FDA acceptance of an efficacy supplement to expand the Elevidysindication. News release. Sarepta Therapeutics, Inc. February 16, 2024. Accessed February 16, 2024.https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-us-fda-acceptance-efficacy?_ga=2.242068545.254033713.1708093077-1826905377.1708093076
2. Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics. October 30, 2023. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0
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