The Future of Cell and Gene Therapy: Navigating Innovation, Regulation, and Manufacturing Challenges

While the CGT space faces unprecedented challenges in 2025, Philip Vanek, chief commercialization officer International Society for Cell & Gene Therapy (ISCT), sees these difficulties as necessary growing pains on the path to revolutionary change. His vision for the CGTs’ evolution centers around three transformative elements that will define the next three to five years—each representing a paradigm shift in how we approach human health.

First, he sees durable and curative medicines through advanced CGTs fundamentally changing treatment paradigms beyond traditional palliative care approaches. “What historically had been seen as intractable illness or chronic illness are now all potential targets for durable and meaningful quality of life driven improvements,” he explains, reflecting ISCT’s mission to drive clinical translation of these breakthrough therapies worldwide.

The second component involves using technology to break down the traditional medical specialty barriers to provide comprehensive to patients. After decades of medical specialization creating isolated silos, Vanek believes the CGT now has the tools to connect these disparate areas. “We’ve got large, rich data sets. We have AI. We have means to unpack the total picture.” He further highlighted that these approaches could enable a more holistic approach to biological outcomes. This holistic approach of using AI to reconnect these barriers is crucial for CGTs manufacturing. These treatments involve intricate manufacturing processes that benefit from understanding the complete patient profile.

The last factor – and perhaps the most important one – is patient access. Given the already high costs and manufacturing complexities associated with personalized CGTs and combined with a chaotic economic environment, this last point is becoming increasingly important. “We can’t leave people behind. We have to find ways to make therapies and interventions much more available to people around the world,” Vanek emphasized, highlighting the critical challenge facing the advanced therapy community.

As industry walks forward into a future riddled with challenges, the question is: is it worth it. The answer is yes.

The recent deployment of Elsa within the FDA has generated both excitement and concern across the CGT industry. Although this system, along with others, has been in development for some time, the announcement did generate a significant amount of buzz within the healthcare sector in early June. However, with further reports that Elsa has limited capabilities and, like CDRH-GPT, is not ready for prime time, the complexity of the CGT space will ultimately be affected. The issues with both Elsa and CDRH-GPT were initially reported by NBC News.

As this adoption evolves, there will understandably be obstacles and challenges to overcome. Vanek identified two major regulatory challenges industry leaders are concerned about in regard to Elsa and to CDRH-GPT.

The first challenge centers on oversight and accountability. “There’s that moralistic center at the heart of this—somebody ultimately is making a potentially life or death decision,” he noted. The risk lies in treating AI-generated reports as “unimpeachable truth” without proper human oversight and interpretation—particularly critical in advanced therapies where individual patient outcomes can vary significantly. This type of cognitive bias from humans is particularly concerning.

His second point touches on legal accountability. With regulatory agencies making life-or-death decisions based on risk assessments built over years of human experience, Vanek worries about the consequences of abdicating that expertise. “We could just hand over everything and say, well, the AI says this, becoming gospel truth, and then suddenly we’re in a world where a bad decision has been made on behalf of mankind by AI, and nobody’s there minding the store.” As we watch the FDA’s adoption of AI into the approval process, both positive and negative consequences may take years to unfold.

Recent layoffs at the FDA and HHS, particularly affecting gene editing experts, have created ripple effects throughout the CGT industry. CGTs are complex. The FDA’s workforce is being cut by 19%, with the vast majority of policy staff eliminated—a particularly challenging development given that the FDA has been seeking to increase staff members to meet the growing workload of CGT reviews in the past few years. Vanek sees this “brain drain” manifesting in two critical areas that directly impact advanced therapy development.

On the regulatory side, the loss of expertise is already causing procedural changes. “We’ve heard that a lot of these reviews are going now to committee review, and usually when you see something going immediately to committee review, it’s because there’s lack of comfort,” he explained. This shift typically slows down the approval process.

These types of bottlenecks contribute negatively to the industry in two ways. The first is that such a delay could essentially make or break a drug developer as funding dries up. While any slowdown in the process could delay critical CGTs reaching patients, multiple approval applications entering committee reviews simultaneously could potentially prevent the drugs from ever going to market.

The innovation impact may prove even more significant for the advanced therapies space. Vanek highlighted how the FDA’s transformation over the past decade, particularly under Scott Gottlieb’s leadership, fostered collaborative relationships with the biotech and pharmaceutical industries. These partnerships enabled early-stage discussions that accelerated advanced therapy development. “Those interactions and those discussions were extremely meaningful to progress the advanced therapies space,” Vanek noted, worrying that the loss of institutional knowledge will create larger gaps between therapeutic breakthroughs and the manufacturing tools needed to make them accessible.

The intersection of manufacturing reshoring, Most Favored Nation (MFN) policy, and tariff uncertainties has created what Vanek described as a “small dumpster fire” of complexity for the CGT industry. While acknowledging the appeal of bringing manufacturing back to the United States, he cautioned against oversimplifying the unique challenges facing advanced therapies. There is also an apparent mismatch of development times. While there is a push for rapid reshoring, the reality is that building new manufacturing plants which meet U.S. regulatory standards typically takes between five to 10 years.

“The uncertainty is always going to cause a ripple effect in the industry,” Vanek observed, noting that companies are slowing investments and hiring while waiting to understand the full implications of policy changes. The tariff situation, in particular, has been “a disaster for investors” due to its unpredictable rollout and communication.

Vanek takes a nuanced view of the MFN policy, which aims to alleviate high U.S. drug prices, by pointing out that drug pricing is not a single actor issue in a complex ecosystem. “You can’t point to one thing. It’s the confluence of all these different actors all causing the problem,” he stated, advocating for comprehensive system reform rather than targeting individual players—particularly important for CGTs where development costs and manufacturing complexity create unique pricing pressures.

The manufacturing challenge extends far beyond simply moving production facilities, especially for cell and gene therapies. Vanek emphasized, “Supply chains for advanced therapies are global by necessity, involving animal-derived, chemical-derived, and plant-derived raw materials that may not be readily available domestically.” This sentiment is shared throughout the industry as expressed by Richard Pollack, president and CEO of American Hospital Association in a letter to the White House, nearly 30% of raw ingredients for major drugs come from China alone. For CGTs, the specialized nature of materials—viral vectors, cell culture media, cytokines—makes this dependency even more acute.

For advanced therapies specifically, manufacturing complexity has always been a primary consideration—unlike traditional pharmaceuticals where manufacturing is often an afterthought. However, the broader regulatory mandate requires multiple suppliers and backup systems to ensure product availability. “If you break the supply chain, you go against some of the regulatory mandates requiring secondary suppliers and having backup suppliers so that a drug is always available,” Vanek explained. This is particularly challenging for regenerative medicines that are sometimes produced for just a handful of patients annually, making redundant supply chains economically unfeasible.

The economic reality of domestic manufacturing presents challenges unique to CGTs. Noting the ultimate goal is patient access, domestic manufacturing, while important, may “require significant automation and scale to compete with global alternatives, potentially changing the fundamental nature of how these products are made,” Vanek stated. This is a particular challenge for autologous therapies that require patient-specific manufacturing. Domestic manufacturing, even without considering ongoing issues such as tariffs, may not be cost-effective enough for the United States healthcare system.

Despite the challenges, Vanek remains optimistic about the CGT space’s trajectory, particularly for chronic disease management. With over 4,000 candidates in the pipeline and 76 therapies already approved globally, the momentum is undeniable. However, Vanek emphasized, “Success will depend on maintaining the collaborative relationships between regulators and industry that have driven recent innovations in advanced therapies.”

The current regulatory and manufacturing challenges, while significant, may ultimately forge a stronger, more resilient industry. Vanek believes that the path to success is to balance innovation with safety, efficiency with access, and global collaboration with domestic capabilities.

While the decisions made in the coming months will test the industry’s resilience, they also present an unprecedented opportunity to build a more sustainable, accessible future for these life-saving therapies. The journey through today’s challenges is the crucible in which the next generation of curative medicines will be forged, ultimately bringing hope to millions of patients who need them most worldwide.

Special thanks to Tozaro for facilitating this interview. This article was written in partnership with Tozaro.