PJ Brooks, PhD, on Working to Bring Gene Therapy Development to More Neuromuscular Disorders


The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

“How do we expand some of these successes into additional diseases? [I talked] about some of the projects we’re funding at NIH. The platform vector gene therapies project is focused on how we can use the fact that we use the same AAV serotype as a platform for more than one disease, and how that can potentially streamline the regulatory process... I also was able to talk about the Bespoke Gene Therapy Consortium which is a public-private partnership organized by the Foundation for the NIH.”

While the recent approval of Elevidys (delandistrogene moxeparvovec; Sarepta Therapeutics), the first gene therapy for treating Duchenne muscular dystrophy (DMD) and a muscular dystrophy in general, represents a huge step forward for neuromuscular diseases, there are still thousands of neuromuscular diseases that do not have available disease-modifying therapies.

PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences, National Institutes of Health (NIH), and his colleagues at the NIH are looking to help facilitate gene therapy development for countless of these rare diseases with their platform vector gene therapies project and the Bespoke Gene Therapy Consortium (BGTC) which is aiming to streamline navigation of the regulatory pathway for rare diseases.

Brooks had a chance to discuss these initiatives during a panel discussion he participated in at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida. CGTLive® spoke with Brooks after the discussion to learn more about the projects the NIH is working on to be able to bring more therapies to underserved populations of neuromuscular disease.

Click here to view more coverage of the 2024 MDA Conference.

Hesterlee S, Brooks PJ, Byrne B, Moy J, McNally E. Panel Discussion on NMD in the Age of Therapy. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL.
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