Ben Samelson-Jones, MD, PhD, on Conservative Management Following Treatment With DMD Gene Therapy Elevidys

“I think the big picture take-home is that AAV has a set of evolving and unique toxicities, and the clinical care of AAV recipients requires collaboration and developing special specialty expertise. I think that's really important to safely administer these therapies. I think this case shows that it can be done, but it requires forethought and planning and some institutional support to do that as safely as possible.”

As one of the prominent FDA approved gene therapy products based on the use of an adeno-associated virus (AAV) vector, Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a gene therapy intended to treat Duchenne muscular dystrophy (DMD), has attracted some amount of attention lately regarding potential safety concerns associated with AAV vectors. As such, careful monitoring of patients after treatment, and rapid intervention in the case of any warning signs, is of great importance.

At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA, Ben Samelson-Jones, MD, PhD, an assistant professor of pediatric hematology at Perelman School of Medicine, University of Pennsylvania, and the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia (CHOP), presented a late-breaking case study regarding a patient treated with Elevidys at CHOP. The study, entitled “Conservative management of acute myocarditis and thrombotic microangiopathy after Elevidys (delandistrogene moxeparvovec)” covered the case of a patient who experienced adverse events following treatment with the gene therapy that were able to be effectively addressed because of early monitoring and intervention efforts.

In a conversation with CGTLive® at the conference, Samelson-Jones spoke about the case and gave his view on the main takeaways from it for the clinical community. He shared his view that the case shows that AAV can be administered safely provided that careful and conservative monitoring is implemented. Samelson-Jones also noted that the clinical model is still evolving and the community will need to continue working out best practices going forward.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.

REFERENCES
1. Sarepta Therapeutics provides update on Elevidys. News release. Sarepta Therapeutics, Inc. April 4, 2025. Accessed April 9, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-update-elevidys?_ga=2.105497091.1449420572.1744214373-2026491411.1742335673
2. Samelson-Jones B. Conservative management of acute myocarditis and thrombotic microangiopathy after Elevidys (delandistrogene moxeparvovec). Presented at: ASGCT 28th Annual Meeting, May 13 to 17, 2024, New Orleans, LA. Abstract #2000