Access to life-saving cell and gene therapies depends not only on scientific breakthroughs but also on the systems that bring those breakthroughs to patients. Regulations, infrastructure, and collaboration across borders determine whether a therapy moves from the lab to the bedside, or remains out of reach
This urgent reality set the stage for the November 2025 Bridging the Gap webinar, “From Discovery to Delivery: Building Smarter Regulatory Pathways for Cell and Gene Therapy,” featuring Dr. Bambi Jo Grilley, Professor of Pediatrics and Director of Clinical Research and Early Product Development at the Center for Cell and Gene Therapy at Baylor College of Medicine. Dr. Grilley also serves as Chief Regulatory Officer at the International Society for Cell and Gene Therapy (ISCT).
She joined moderator Dr. Patrick Hanley, Chief and Director of the Cellular Therapy Program at Children’s National Hospital, along with George Eastwood, Executive Director of the Emily Whitehead Foundation, and Olga Bukatova from Azenta Life Sciences, to explore how the regulatory ecosystem must evolve to keep pace with rapid scientific progress.
A Career Shaped by Collaboration and Innovation
Dr. Grilley began her career as a pharmacist at MD Anderson Cancer Center, where her experience with early investigational drugs led her to the world of clinical research and regulation. “I became convinced that the only way forward in treating cancer was to move into new areas and really do new things,” she reflected.
That mindset carried her to Baylor, where she helped secure dozens of Investigational New Drug (IND) applications and supported over 130 clinical trials in cell and gene therapy.
Her early work with pioneers such as Dr. Malcolm Brenner, Dr. Helen Heslop, and Dr. Cliona Rooney helped shape what would become the modern era of immunotherapy. “What makes our field so fun,” Dr. Grilley said, “is that it’s very science-based, but also very collaborative. You can actually come to meetings, talk to people, and see things changing while we’re moving ahead.”
Dr. Hanley agreed, noting, “It’s a small field, but it’s special. We all support each other’s trials because ultimately, we’re trying to help each other’s patients.”
A Turning Point in Gene Therapy
Nearly every transformative field endures a defining crisis that tests its purpose and integrity. For gene therapy, that moment came in 1999, when 18-year-old Jesse Gelsinger experienced a fatal outcome during a clinical trial for a rare metabolic disorder—an event that paused much of the field’s momentum and prompted sweeping regulatory reform. The tragedy underscored the need for rigorous, transparent reporting of all adverse events, no matter how minor, to ensure patient safety and scientific accountability.
Dr. Grilley reflected on that pivotal time, recalling the heightened scrutiny and regulatory engagement that followed. Despite these challenges, the community persevered. “A lot of centers didn’t make it through,” she noted, “but those that did helped establish the foundation for the approvals and advancements we see today.”
The resulting regulatory framework for CGT evolved to emphasize stronger oversight of vector-delivery systems, better manufacturing practices (cGMP), improvements in informed consent, and closer monitoring by the regulatory bodies. Rather than stopping innovation, the hard-won lessons from early failures strengthened the field—laying the groundwork for safer, more credible therapies today.
Global Regulation: Progress, Cost, and Equity
As Chief Regulatory Officer of ISCT, Dr. Grilley has a front-row view of how global differences shape access. She noted that Europe’s regulatory environment is “very, very highly regulated—and getting stricter all the time,” while the United States, she said, “is in a kind of stasis,” with no major changes for better or worse. Meanwhile, Asia—especially Japan and India—is emerging as a powerful hub for development.
As a result, developers increasingly weigh whether to begin trials in regions like the U.S. or EU—or to start elsewhere to reach patients faster. India’s example particularly stood out:
“They’re charging one-tenth the cost for some CAR T-cell products,” Dr. Grilley said. “It raises important questions—what are they doing differently, and can that model be adapted globally?”.
She noted that without mechanisms for shared regulatory review, promising data generated in one region often cannot be efficiently leveraged in another.
She also cautioned about the rise of unregulated programs in countries lacking formal oversight. “As automated manufacturing systems become more common, ensuring consistent oversight will be increasingly important—especially in regions without strong regulatory controls,” Dr. Grilley cautioned. “These therapies aren’t like making a pill; they’re complex biologic products made from and for individual patients, where every step matters.”
Dr. Grilley warned that overly rigid systems risk pushing innovation into regions with fewer safeguards, underscoring the need to balance safety with practical pathways to patient access. Rather than calling for uniform global rules, she advocated for greater regulatory collaboration, where agencies build on each other’s reviews instead of starting from scratch.
To foster collaboration and global standards, ISCT hosts a Global Regulatory Summit, inviting regulators from countries with developing systems to learn and build their own frameworks. The goal, she explained, is not regulatory dependence but work-sharing—accelerating access while preserving local authority.
“Japan has taken a mentoring role with other Asian countries without strong regulatory processes,” she said. “We need more of that worldwide: more harmonization, more work-sharing.”.
Beyond the Lab: Building the Workforce for the Future
Workforce development is another key priority for the future of cell and gene therapy. Dr. Grilley noted that the field draws talent from many disciplines—including pharmacy, nursing, and manufacturing—and emphasized that expanding science-based training programs will help equip the next generation with the expertise needed to advance complex biologic therapies.
Dr. Hanley added that companies consistently report gaps in regulatory and GMP expertise. “The people who embrace those fields really excel,” he said. “We need to make regulatory work something people are excited to do.”
Bukatova agreed, noting that this mindset reflects the field’s broader spirit of innovation. “It’s not about sitting all day reading PDFs,” she said. “It’s about finding real-world solutions so patients can receive the therapy they need.”
Keeping Patients at the Center: Why Proximity Matters
One theme consistently reinforced throughout the discussion was the importance of staying close to the patient, a defining characteristic of cell and gene therapy that sets it apart from many other therapeutic modalities. In academic medical centers, researchers, regulators, and clinicians are often just steps away from the patients they serve, creating a sense of urgency that shapes decision-making at every stage of development.
Dr. Grilley emphasized that this proximity fundamentally changes how innovation happens. “Our field is different,” she explained. “We’re not working on abstract problems, we’re working for patients who can’t wait.” That reality drives a mindset focused on finding safe, compliant ways to move forward, rather than defaulting to “no.”
Eastwood echoed this perspective from the patient advocacy side, noting that families navigating rare and life-threatening diseases often share remarkably similar journeys, regardless of diagnosis. “Approval is not the finish line,” he said. “Delivery at scale is the real challenge, and that’s where regulation, infrastructure, and coordination must evolve.”
Together, the panel underscored that keeping patients visible throughout development is not just an ethical imperative, it is a practical one. Patient proximity accelerates learning, informs smarter regulatory decisions, and ensures that innovation remains grounded in real-world need.
Looking Ahead: Regulation as a Catalyst, Not a Constraint
When asked where she sees the field by 2030, Dr. Grilley was both pragmatic and optimistic. “We’re moving toward helping smaller and smaller patient populations: ultra-rare and even N-of-one diseases,” she said. “That means more collaboration, more harmonization, and hopefully a global view of what makes a therapy approvable.”
For Eastwood and the Emily Whitehead Foundation, this future is about ensuring no patient is left behind. “Eight years post-approval, we’re still not delivering at scale,” he said. “Regulation, cost, and infrastructure are the next bottlenecks to solve.”
Dr. Grilley closed with a hopeful reminder:
“These products aren’t small molecules—they’re made differently, for patients who can’t wait. The regulations should reflect that.”
Key Takeaways
- Global collaboration is critical for harmonizing regulatory frameworks and accelerating access to therapies.
- Cost and infrastructure, not science alone, are now the main barriers to delivery.
- Emerging regions, such as India and Japan, are redefining what efficient, ethical regulation can look like.
- Regulatory affairs is a creative, patient-centered discipline—an essential bridge from innovation to access.
- Academic and industry partnerships must remain rooted in patient proximity and real-world urgency.
As therapies grow more personalized and timelines compress, the panel made clear that regulation must evolve in parallel with science. Smarter regulatory pathways, designed for complex biologics, adaptive manufacturing, and ultra-rare populations, will determine whether innovation reaches patients in time. When aligned early, regulatory strategy becomes not a bottleneck, but a force multiplier for safe, scalable access.
The conversation underscored that advancing cell and gene therapy requires more than scientific innovation; it demands a united global effort to align regulation, technology, and access for every patient in need.
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About the Guest
Bambi Jo Grilley, RPh, RAC, CIP, CCRC, CCRP
Professor, Department of Pediatrics; Director, Clinical Research & Early Product Development, Center for Cell and Gene Therapy, Baylor College of Medicine; Chief Regulatory Officer, International Society for Cell & Gene Therapy
For more than three decades, Dr. Bambi Jo Grilley has been a driving force in the translation of cell and gene therapies from academic discovery to clinical application. A Professor of Pediatrics and Director of Clinical Research and Early Product Development at Baylor College of Medicine’s Center for Cell and Gene Therapy, she has guided over 130 clinical trials and 69 research INDs, shaping national standards for clinical research quality and regulatory oversight.
As Chief Regulatory Officer for the International Society for Cell & Gene Therapy (ISCT), Dr. Grilley champions global collaboration, equitable access, and regulatory innovation—recently representing ISCT in high-level FDA and international summits. Through her consultancy, QB Regulatory Consulting, she supports emerging biotech companies in developing sound regulatory strategies. A passionate advocate for patient access, Dr. Grilley continues to bridge the gap between innovation and regulation to ensure life-saving therapies reach those who need them most.