Cell and Gene Therapy Sector Faces Pivotal Moment Amid Leadership Changes and Global Challenges

The cell and gene therapy (CGT) sector is entering a critical period following significant leadership changes at the FDA and mounting global challenges. Recent developments have brought both uncertainty and cautious optimism to an industry already grappling with manufacturing hurdles and commercial obstacles.

FDA Leadership Transition Sparks Debate on Regulatory Approach

The departure of Peter Marks as director of the FDA's Center for Biologics Evaluation and Research (CBER) has raised questions about the future direction of CGT regulation. Marks, widely regarded as a champion for cell and gene therapies, particularly in rare diseases, leaves behind a legacy of advocacy for flexible trial designs and accelerated approval pathways.

New FDA Commissioner Marty Makary appears to be continuing some of Marks' initiatives. In a recent interview, Makary proposed a conditional approval pathway based on "plausible scientific mechanism" for incurable diseases affecting small populations. This approach aligns with Marks' previous suggestions for non-randomized, single-arm trials in rare disease gene therapy development.

Industry experts express mixed reactions to the leadership change. Jonathan Wofford, chief commercial officer at Title21 Health Solutions, noted, "There is no doubt about the impact that Dr. Marks has had on the CGT space. He has been instrumental in shaping a regulatory framework that I believe has accelerated not only the advancement of clinical pipeline in the United States and therapy approvals, but also the advancement of the entire CGT sector."

However, some see potential benefits in fresh perspectives. Ignacio Núñez, chief operations officer at CDMO CellReady, commented, "After seven years, maybe it's not bad to have a fresh view of the whole thing. . . . It doesn't need to be negative. It could also be positive."

Global Economic Pressures Threaten to Reshape CGT Landscape

The CGT sector faces significant headwinds from global economic pressures, particularly the implementation of tariffs. The industry's reliance on a global supply chain for critical components such as viral vectors, plasmids, and reagents makes it particularly vulnerable to trade disruptions.

Audrey Greenberg, founder of the Center for Breakthrough Medicines, warned of the potential consequences: "If tariffs hit these key components from China or the EU, we're going to see increased cost of goods, tighter margins, more volatility and production timelines. And that's bad news for patients and companies."

The impact of tariffs could be especially pronounced for CGT products, which are already among the most expensive medicines on the market. Recent approvals like Orchard Therapeutics' Lenmeldy for metachromatic leukodystrophy, priced at $4.25 million per treatment, exemplify the high costs associated with these innovative therapies.

Some companies are already adapting their strategies in response to these challenges. Sekar Kathiresan, CEO of Verve Therapeutics, noted that his company maintains manufacturers in both the U.S. and Europe to navigate the evolving trade landscape effectively.

While tariffs pose significant challenges, they may also drive positive changes in the industry. Núñez suggested that increased tariffs could incentivize more U.S.-based manufacturing, potentially leading to faster and more cost-effective supply chains.