PJ Brooks, PhD, on Developing New Strategies for Novel Clinical Trials


The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed the NIH’s genome editing program and more.

“In the genome editing program we're specifically funding people, not just to do clinical trials and get ready for clinical trials, but to think about new ways to do these clinical trials with genome editing faster and more efficiently, and to go propose and discuss these things with the FDA. And then we're going to disseminate[those findings]. So, I think it's a bit of a different approach to funding research than we've done [in the past]. But I think it's really very timely, because the field is shifting, and it's based on emerging science.”

Thousands of neuromuscular diseases still lack disease-modifying therapies, despite great progress in certain aspects of the field, such as the 2023 approval of Elevidys (delandistrogene moxeparvovec; Sarepta Therapeutics), the first gene therapy for treating Duchenne muscular dystrophy (DMD) and a muscular dystrophy in general.

Sessions at the recent 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, discussed how to facilitate drug development and potentially bring more therapies to underserved populations with neuromuscular disease.

CGTLive® spoke with PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences, National Institutes of Health (NIH), who participated in a panel discussion about this topic. Brooks shared with CGTLive several initiatives and focuses that the NIH is engaged with to try to better drug development for rare diseases, including neuromuscular diseases. One such program is a genome editing program that has a goal of streamlining clinical trials of genome editing products.

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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