At a June 5 cell and gene therapyroundtablehosted by the FDA, Robert F. Kennedy Jr. asked the assembled scientists and advocates to provide a list of regulations they’d like to see gone.
“President Trump has given us anexecutive orderthat says for every regulation that we put in place, we have to get rid of 10,” the secretary of the Department of Health and Human Services (HHS) said. “So we would solicit, right now, a list from all of you of any regulations you think we ought to be getting rid of.”
Speeding up the regulatory process for new cell and gene therapies was one of the roundtable’s recurring themes.
Other major points from the attendees, as summarized by Vinay Prasad, M.D., director of the FDA’s Center for Biologics Evaluation and Research, included the risk of “ceding dominance” in the field to China and using genetic engineering to edit pig organs for transplants.
Joining Kennedy and Prasad were FDA Commissioner Martin Makary, M.D.; National Institutes of Health (NIH) Director Jay Bhattacharya, Ph.D.; and Centers for Medicare & Medicaid Services Administrator Mehmet Oz, M.D. All leaders have been nominated and appointed under President Donald Trump's second term.
Makary emphasized that the FDA is determined to act based on the panelists’ input.
“We're going to take those messages very seriously,” he said. “This is not a horse and pony show.”
Though China was mostly spoken about as an adversary, one attendee proposed looking to the country as a model. Carl June, M.D., director of the Center for Cellular Immunotherapies at the University of Pennsylvania’s Perelman School of Medicine, proposed copying China’s two-tier regulatory system, where first-in-human exploratory trials require only the approval of institutional review boards. The FDA would then only become involved when the therapy is ready for larger, multisite trials.
“Why are researchers increasingly taking trials overseas?” June asked. “Simply put, the U.S. process has become too slow, costly and inflexible, while other countries make it easier to innovate.”
A story that attendees and agency heads alike continually returned to was that of KJ Muldoon, the infant who wasrecently announcedto have received the first-ever custom CRISPR gene therapy designed specifically for the genetic mutations causing his rare disease.
“The story of baby KJ charts a fundamentally new path to genetic medicines on demand, scalably and affordably,” said Fyodor Urnov, Ph.D., a scientific director of the Innovative Genomics Institute at the University of California, Berkeley, who helped develop KJ’s treatment. “Our ultimate goal is to have the for-profit sector step in in a major way, and for there to be dozens of flourishing biotechs providing approved CRISPR-on-demand therapies.”
“But at the present time, as many speakers have mentioned, such companies are struggling under the old ways of making such therapies," Urnov said.
Bhattacharya extolled KJ’s story as an example of what can be achieved when regulatory requirements are relaxed, as the pioneering therapy was made in just a few months. He also took the chance to highlight the NIH’s role in the groundbreaking achievement.
“KJ I actually got to brag about a little bit, because it was NIH-funded work that actually led to this,” Bhattacharya said.
When it was RFK Jr.’s turn to speak, the HHS secretary at first had little to say about cell and gene therapy. He began by highlighting his friendship with the “renegades” seated next to him, and touched on familiar bugbears like declining sperm counts in young men and the sharp rise in chronic disease cases since his uncle, John F. Kennedy, was president.
When he did get to the topic of cell and gene therapy, he followed a similar line as the other agency heads, promising relaxed regulations and fully realized scientific potential.
“There's a lot of people who criticize us, they say we cut the budget of the agency,” RFK Jr. said. “We didn't cut any of the budget of the agency. We have that money, and we are taking away from the administrative costs and redundancies, and we're going to reapply it to spawn and foster and fortify innovation and unleash it.”
“We're going to rely on you to come up with the solutions,” RFK Jr. told the panelists in conclusion. “And we're going to do everything in our power to sweep away the barriers from you getting those solutions to market and getting them funded, and do everything that we can to support you.”
Under Trump’s proposed 2026 budget, the FDA isfacing a 4% budget cut. The agency hasshed a significant number of staffsince Trump and RFK Jr. came into power.
At the NIH, the agency isfacing a nearly 40% budget cutand recentlylaid off scores of employees.
(20min delay)