Turning Gene Therapy Into Reality: Peter Marks, MD, PhD

WATCH TIME: 3 minutes

"Another is how to move them [gene therapies] through clinical trials in a relatively facile manner, making use of what we have at our disposal at the agency, which is things like the use of accelerated approval."

The 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference kicked off Monday with the Opening and Keynote Address given by Donald S. Wood, PhD, president and chief executive officer, MDA, and Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research (CBER), FDA. CBER is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies.

Marks, who’s resume includes several years in the pharmaceutical industry on the clinical development of hematology and oncology products, focused his speech on the phenomenon behind gene therapies, and the steps needed to turn them into reality. Gene therapies represent a potentially revolutionary approach to treating neuromuscular disorders; however, executing on these approaches has been difficult to date. The healthcare community saw its first major breakthrough with gene therapies in 2019, when the FDA approved onasemnogene abeparvovec-xioi (Zolgensma; Novartis) to treat children with spinal muscular atrophy, a rare, fatal neuromuscular condition.

In an interview with CGTLive^®, a sister publication to NeurologyLive^®, Marks provided an overview of his talk, and the reasons for why he chose this topic. He spoke on the steps needed to continue the advancement of gene therapies, and why speaking to the MDA community can have major impacts.

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