The departure of Peter Marks from the Food and Drug Administration marks the end of an era for cell and gene therapy development in the United States. Marks, who served as director of the FDA’s Center for Biologics Evaluation and Research (CBER) since 2016, resigned on Friday citing disagreements with Health and Human Services Secretary Robert F. Kennedy Jr.
During his tenure, Marks played a pivotal role in advancing numerous groundbreaking treatments, including the approval of the first gene therapy, initial cellular therapy for cancer, and pioneering CRISPR gene editing medicine. His leadership style emphasized flexibility in the review process and the creation of expedited development pathways that, while sometimes controversial, helped bring dozens of innovative therapies to market.
The impact of Marks’ departure is particularly significant for the cell and gene therapy sector, where his advocacy has been instrumental in establishing regulatory frameworks and facilitating drug
development. Leading figures in the field have expressed concern about the void his resignation creates. Dr. Stephan Grupp of Children’s Hospital of Philadelphia described Marks as “an absolute scientific and regulatory giant,” whose departure represents “a huge loss to the field.”
Under Marks’ leadership, CBER approved 43 cell or gene therapies since 2017, demonstrating his commitment to advancing these complex treatments. His approach included building specialized expertise within the agency and modernizing regulatory frameworks to handle the increasing number of applications in this field.
Marks was particularly noted for his support of accelerated approval pathways for rare disease treatments, believing this approach would encourage pharmaceutical companies to invest in these areas. His decision-making sometimes generated controversy, as exemplified by the 2024 approval of Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys, where he overruled other FDA officials to support the treatment’s authorization.
The timing of his exit creates additional uncertainty for an industry already facing significant challenges. Many cell and gene therapy companies are struggling to secure funding, and the stock prices of several firms declined following news of his resignation. Industry analysts have suggested that future regulatory reviews might become more stringent, particularly for Duchenne therapies and cell therapies targeting autoimmune conditions.
Despite these concerns, some observers remain optimistic about the field’s future. Incoming FDA commissioner Marty Makary has indicated support for flexible approval pathways in cases of rare,
life-threatening diseases. Additionally, other FDA leaders, including Nicole Verdun, director of the Office of Therapeutic Products, have demonstrated support for accelerated approvals.
However, the cell and gene therapy sector faces additional challenges beyond Marks’ departure. The Trump administration’s recent
announcement of widespread job cuts at federal agencies adds to existing staffing concerns at the FDA. Industry experts worry that these factors could slow the progress of innovative treatments reaching patients.
In his resignation letter, Marks emphasized the need for continued rapid development of treatments for genetic diseases, highlighting the transformative potential of these therapies. His legacy includes not just the approvals achieved during his tenure, but also the regulatory infrastructure and scientific expertise he helped establish within the FDA.
The search for Marks’ successor comes at a critical juncture for the cell and gene therapy field, with industry leaders emphasizing the importance of maintaining strong, experienced leadership to continue the progress made under his direction. As one expert noted, finding someone to match his combination of scientific knowledge, regulatory expertise, and patient-focused approach will be challenging.
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