The Food and Drug Administration’s cell and gene therapy landscape faces significant upheaval following the departure of Peter Marks, who resigned Friday citing disagreements with Health and Human Services Secretary Robert F. Kennedy Jr. As the director of the Center for Biologics Evaluation and Research (CBER) since 2016, Marks played an instrumental role in advancing revolutionary treatments, including the first gene therapy approval, pioneering cancer cell therapies, and groundbreaking CRISPR-based medicines.
During his tenure, Marks championed flexible regulatory approaches that facilitated faster development pathways for these innovative treatments. His leadership saw the approval of over 40 cell and gene therapies, establishing a regulatory framework that balanced safety with the urgency of bringing treatments to patients with serious diseases.
Medical experts and industry leaders have expressed concern about the void left by Marks’ exit. Dr. Stephan Grupp from Children’s Hospital of Philadelphia described him as “an absolute scientific and regulatory giant,” while renowned gene therapy researcher Katherine High praised his vision and commitment to building robust regulatory infrastructure for novel therapeutics.
The impact of Marks’ departure extends beyond leadership changes. The cell and gene therapy sector, already facing challenges in attracting investment, now confronts additional regulatory uncertainty. Stock prices of several gene therapy companies declined following the announcement, reflecting market concerns about potential shifts in regulatory approach.
Under Marks’ guidance, CBER developed specialized expertise and expanded its staff to handle the increasing complexity of cell and gene therapy applications. His advocacy for accelerated approval pathways, particularly for rare disease treatments, helped encourage pharmaceutical companies to invest in these innovative therapies.
A notable example of Marks’ impact was the 2024 approval of Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys. Despite mixed clinical trial results, Marks supported the therapy’s approval, arguing that even modest benefits could be meaningful for patients with severe conditions. While patient advocacy groups celebrated this decision, some critics questioned whether it represented an inappropriate lowering of approval standards.
The resignation comes at a particularly challenging time for the biomedical field, coinciding with recently announced government job cuts. Industry analysts express varying views on the future regulatory landscape. While some, like Baird analyst Jack Allen, note that Marks’ disputes with Kennedy appeared focused on vaccine-related issues rather than gene therapies, others worry about potential impacts on development timelines and approval processes.
William Blair analysts point to positive signs, including the continued presence of Nicole Verdun, director of the Office of Therapeutic Products, who shares Marks’ support for accelerated approvals. Additionally, incoming FDA commissioner Marty Makary has indicated openness to flexible approaches for rare, life-threatening conditions.
However, concerns persist about maintaining the momentum in cell and gene therapy development. Nicole Paulk, CEO of Siren Biotechnology, emphasized Marks’ role as the field’s “biggest champion,” particularly in advancing treatments for rare pediatric diseases. Pat Furlong, leader of Parent Project Muscular Dystrophy, stressed the importance of maintaining strong, experienced leadership to continue progress.
In his resignation letter, Marks urged the FDA to maintain its commitment to expediting treatment development for genetic diseases, emphasizing the transformative potential of these therapies. As the industry adapts to this transition, the challenge lies in preserving the regulatory framework and development-friendly environment that Marks helped establish while ensuring continued advancement of these innovative treatments.
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