Advances in Gene Therapy for Neuromuscular Diseases: Kevin Flanigan, MD

WATCH TIME: 4 minutes

“Despite the success we've had so far like with spinal muscular atrophy, there's still a lot of challenges. The full safety and efficacy profile for the newest gene therapies for Duchenne muscular dystrophy is not yet clear. We do anticipate that there will be continued issues of safety that we have to address in the long term.”

In May 2019, FDA approved onasemnogene abeparvovec-xioi (Zolgensma; AveXis) for the treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age with mutations in the SMN1 gene.¹ The treatment became the first and only gene therapy FDA-approved for SMA, including presymptomatic patients and was based on data from the phase 3 STR1VE trial (NCT03306277) as well as the phase 1 START trial (NCT03421977), which included patients with SMA type 1 who experienced symptoms at less than 6 months.

Recently, at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Kevin Flanigan, MD, gave a talk on the safety issues and concerns of gene therapies in neuromuscular diseases in a session on updates in gene therapy. The session reviewed the novel approaches of gene therapies for SMA and Duchenne muscular dystrophy (DMD) and compared them with other disorders with multiple available and emerging therapy options. Additionally, the session discussed pressing questions for the future of gene therapy such as consideration for dosing antibody-positive patients, redosing strategies, and prenatal gene therapy.²

At the conference, Flanigan, director of the center for gene therapy at Nationwide Children's Hospital, sat down with NeurologyLive® in an interview to talk about updates in gene therapies for neuromuscular diseases such as SMA and DMD. In addition, he discussed some of the most recent advancements in care for patients as well as challenges including earlier diagnosis and prevention.

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REFERENCES
1. AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA). News Release. Novartis. Published May 24, 2019. Accessed March 30, 2023. https://www.novartis.com/news/media-releases/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma
2. Flanigan K. Safety Issues: Gene Therapy Administration and Management. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Updates on Gene Therapy in NMD: Current & Emerging Therapies session.