The director of the Center for Biologics Evaluation and Research at the FDA discussed his keynote speech at the 2023 MDA Conference.
“My keynote address really focused on how we can make gene therapy for rare disorders into a reality. And that means trying to address some key things that can get in the way of getting gene therapies to patients. One of them is that manufacturing of these products is still quite expensive and complicated. And we need to try to address that.”
Peter Marks, MD, PhD, the director of the Center for Biologics Evaluation and Research at the FDA, gave the keynote address at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas. He focused on key issues that need to be addressed in order to bring more gene therapies to patients, including manufacturing, clinical trial, commercial viability, and regulatory convergence.
CGTLive spoke with Marks about his keynote address. He discussed the current state of gene therapies, possible future directions of research, and challenges that remain to be addressed. He also touched on data presented at the conference, including positive long-term follow-up data on the gene therapy onasemnogene abeparvovec (Zolgensma; Novartis) for children with spinal muscular atrophy.
Read more coverage of the 2023 MDA Conference here.