What we learned from the FDA’s cell and gene therapy roundtable
As part of new efforts to improve FDA processes and enhance communication with stakeholders, they hosted a roundtable on cell and gene therapy on June 5th, 2025. Various academics, physicians, patient advocates, and industry experts were present. With major recent advancements in cell and gene therapy, this initiative signaled that the FDA is keen on building the momentum and spearheading the next wave of therapeutics. In this blog, we provide an overview of the main themes and key takeaways from the 3-hour discussion.
The need for innovative trial design
Vinay Prasad, CMO and CSO at the FDA and Director of the Center for Biologics Evaluation and Research (CBER), highlighted in his opening remarks the FDA’s understanding that randomized controlled trials (RCTs) may not always be the appropriate model for certain treatments or disease areas. Accordingly, the FDA is willing to accept innovative trial design, including the use of real-world data (RWD).
One example he mentioned was target trial emulation, which allows researchers to use RWD to retrospectively structure an RCT. By designing a study to mimic a series of randomized comparisons using large-scale RWD, it becomes possible to generate evidence while bypassing cost, patient burden, and limitations of small patient cohorts.
Interestingly, a recent study showed the success of this approach. Researchers used an US electronic health record (EHR) database to investigate whether the use of semaglutide (to treat obesity and type 2 diabetes) could reduce the risk of Alzheimer’s disease. With an overall cohort of over 1 million adults, the different emulated trials showed that semaglutide was associated with a 40-70% lower risk of developing Alzheimer’s disease.
Various panelists echoed the importance of flexible trial design, particularly in rare diseases. Furthermore, in the increasingly common case of n-of-1 therapies, even flexible trial design models are not suitable. Rather, experts mentioned that case studies may be needed to stand in for fully developed trials in these examples. Regulatory processes should be streamlined for these types of therapies, particularly considering the low feasibility of running trials at such high costs for one patient.
If you’re interested in learning more about trial design strategies in precision medicine, read our Designing smarter trials: Precision medicine strategies for a new era of clinical research whitepaper here.
Addressing market failures in pediatric rare disease
One of the more emotionally charged topics during the roundtable was the lack of options for children with rare diseases, particularly in oncology. Experts emphasized that the challenge in this space is not scientific failure, but market failure. Many promising gene therapies have been developed in academic labs but never make the leap to commercialization due to poor financial incentives and regulatory bottlenecks.
Despite a growing pipeline of pediatric gene therapies, very few have made it to patients. Panelists described how chemotherapy remains the frontline treatment for many children, even though safer and more effective cell therapies exist. This is largely due to the limited commercial viability of ultra-rare therapies that leads to almost negligible industry interest. This gap between academic innovation and market access leads to what some call “valley of death,” where potentially life-saving therapies are eliminated before reaching patients.
Panelists also highlighted promising strategies to overcome these challenges, namely platform designation that reduces regulatory barriers and financial incentivization in these therapeutic areas. The FDA recently applied platform designations to allow specific technologies to be reused across multiple therapeutic indications without undergoing full re-evaluation each time. Expanding this pathway could reduce costs and timelines significantly.
Transparent regulatory processes and enhanced data sharing
To realize the full potential of bespoke gene therapies, several participants underscored the need for greater infrastructure to share data and outcomes. Creating platforms that allow gene therapy developers to build on each other’s work can help reduce redundancy, speed up regulatory decision-making, and improve patient access. This kind of open data ecosystem would be especially valuable in rare disease, where individual patient experiences can be scientifically and clinically meaningful.
Another key point raised was the need for greater transparency when the FDA rejects or delays a therapy. Feedback on rejections is often unclear, making it difficult for sponsors to respond adequately or resubmit successfully. Several attendees argued that consistent, structured communication around regulatory decisions could help foster more innovation by reducing uncertainty, streamlining timelines, and making expectations clearer for both biotech startups and academic labs. This would also increase confidence for investors who express concerns over the risk of regulatory bottlenecks, which is particularly valuable considering the current difficulties in biotech funding.
Keeping the US competitive in a global race
Participants also expressed concerns about the U.S. falling behind on the global stage of clinical development. A growing number of US-developed therapies are being trialed in China first due to faster and more flexible regulatory pathways. Panelists and FDA officials alike warned that if this trend continues, it could erode America’s leadership in the field, reduce investor confidence, and stifle domestic biotech innovation.
Suggestions to combat this included allowing tiered IRB approvals for early-phase trials in specific therapeutic areas and creating clearer pathways for exploratory trials. In China’s two-tier model, early-stage studies move quickly through local approvals and only require full-scale review once they have shown promise. This approach could offer a more agile model that supports both innovation and safety evaluation.
Overall, this roundtable highlighted the FDA’s growing willingness to evolve. From embracing RWD and innovative trial designs, to considering new regulatory structures for platform-based and bespoke gene therapies, the agency appears poised to play a more proactive and collaborative role in the future of therapeutics.
Nonetheless, change will require more than policy. If the ideas discussed at this roundtable are supported by intentional changes and investment in infrastructure, the next decade in cell and gene therapy could be defined by a new era of accessible, inclusive, and fast development of life-saving treatments.