At the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference on Monday, a keynote address was delivered by an FDA official, outlining the agency’s efforts to promote the progress of gene therapies for rare disorders.
In an interview with Neurology Live, Peter Marks, who serves as the director of the FDA’s Center for Biologics Evaluation and Research (CBER), summarized his speech and emphasized the MDA’s efforts.
His comments came only three days after the FDA announced its intent to gather an advisory committee meeting concerning Sarepta Therapeutics’ developmental gene therapy for Duchenne muscular dystrophy (DMD) before the deadline of May 29.
During the interview, Marks explained that the FDA is focusing on three main areas to facilitate the delivery of gene therapies to patients, which are manufacturing, clinical trials, and commercial feasibility.
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