Cell and Gene Therapy Industry Faces Uncertainty as FDA Champion Peter Marks Departs

Peter Marks, a key figure in the advancement of cell and gene therapies in the United States, has resigned from his position as head of the Food and Drug Administration's Center for Biologics Evaluation and Research (CBER). His departure, citing differences with Health and Human Services Secretary Robert F. Kennedy Jr., leaves the rapidly evolving field without its most prominent regulatory advocate.

Marks' Legacy and Impact on Cell and Gene Therapy Development

During his tenure at the FDA since 2016, Marks oversaw the approval of numerous groundbreaking treatments, including the first gene therapy, the first cellular treatment for cancer, and the first CRISPR gene editing medicine. His leadership was instrumental in creating flexible regulatory pathways that accelerated the development and approval of these complex therapies.

Stephan Grupp, head of Children's Hospital of Philadelphia's cell therapy and transplant section, called Marks "an absolute scientific and regulatory giant at the agency," emphasizing his role in keeping the field both safe and progressive. Katherine High, a prominent gene therapy researcher, praised Marks' vision and commitment to building a rigorous regulatory infrastructure for novel therapeutics.

Under Marks' guidance, the FDA approved 43 cell or gene therapies, according to the Alliance for Regenerative Medicine. His advocacy for using unorthodox approval pathways, particularly for rare diseases, was aimed at encouraging investment in these cutting-edge treatments.

Industry Concerns and Market Reactions

Marks' resignation has sent ripples through the pharmaceutical industry, particularly affecting companies focused on cell and gene therapies. The departure adds regulatory uncertainty to an already challenging landscape for these innovative but complex treatments.

Several analysts have expressed concerns about the potential impact on ongoing drug development programs:

• Leerink Partners analyst David Risinger noted that cell and gene therapy companies are "particularly exposed to FDA disruption risks."
• RBC Capital Markets analyst Brian Abrahams suggested there might be a "stricter path to market" for new Duchenne muscular dystrophy therapies and "less leniency" towards cell therapies for autoimmune diseases.
• Mani Foroohar of Leerink highlighted the additional risk created by uncertainties regarding Marks' successor and potential staff turnover at the agency.

The news has negatively affected stock prices of several gene therapy firms, reflecting investor concerns about the regulatory landscape's stability.

Future Outlook and Industry Response

Despite the uncertainty, some industry watchers remain cautiously optimistic. Baird analyst Jack Allen noted that Marks' dispute with Kennedy appeared to focus on vaccines rather than novel treatments like gene therapies. The team at William Blair pointed out that other FDA officials, such as Nicole Verdun, director of the Office of Therapeutic Products, have also been advocates for accelerated approvals.

However, experts in the field express worry about the FDA's ability to maintain its current approach to cell and gene therapy regulation. The agency already faces staffing deficits, which could be exacerbated by recent government-mandated job cuts.

Industry leaders are calling for swift action to address the leadership vacuum. Nicole Paulk, CEO of gene therapy startup Siren Biotechnology, urged industry leaders and investors to lobby for Marks' reinstatement. Pat Furlong, head of Parent Project Muscular Dystrophy, emphasized the need for "strong, experienced leadership" to continue progress in the field.

As the cell and gene therapy sector navigates this period of regulatory uncertainty, the industry's ability to adapt and collaborate with the FDA will be crucial in maintaining the momentum of innovation and bringing these potentially transformative treatments to patients.