Pat Furlong, BSN, RN, on Continuing Research With Muscular Dystrophy After Gene Therapy Approval

“PPMD is looking at next generation gene therapies. We certainly are very interested in, as we see these first gene therapies, what's the next generation look like? Are there breakthroughs in manufacturing of the AAV serotypes... So, I think those things are very exciting, we're also thinking about what happens next for these children and young men who've received a gene therapy.”

While patients with Duchenne muscular dystrophy (DMD) enjoyed the breakthrough approval of delandistrogene moxeparvovec (Elevidys) gene therapy in 2023, there remains questions to be answered and more research to be done, especially for people with other types of muscular dystrophies which don’t have available disease modifying therapies. Questions of durable efficacy with gene therapy also remain, as the global, pivotal, phase 3 EMBARK study (Study SRP-9001-301; NCT05096221) that supported Elevidys’ approval failed its primary end point.

CGTLive spoke with Pat Furlong, BSN, RN, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD), to learn more about what the organization thinks are the most pressing concerns and the field and how they are working to address them. Furlong shared the PPMD is looking at next-generation therapies that may overcome challenges with current generation therapies, including improving manufacturing to create more efficient therapies.

With the first approval of gene therapy, PPMD is looking into continuing care for patients after they’ve received gene therapy and investigating the best combinations of therapies for these patients. In terms of other muscular dystrophies, the organization is specifically researching Becker muscular dystrophy and improving cardiac outcomes in those patients. Lastly, Furlong shared her excitement for 2024, PPMD’s 30th anniversary year.

REFERENCE

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