FDA Roundtable Tackles Regulation Reform in Cell and Gene Therapy

Highlights

• At the June 5 FDA-hosted roundtable, HHS Secretary RFK Jr. solicited regulatory rollback ideas, citing President Trump’s “one-in, ten-out” directive.
• Key speakers included FDA Commissioner Dr. Martin Makary, NIH Director Dr. Jay Bhattacharya, and CMS Administrator Dr. Mehmet Oz.
• Attendees voiced concerns over losing global leadership to China and discussed regulatory models to expedite innovation.
• Dr. Carl June suggested adopting China's streamlined two-tier system for clinical trials.
• The case of baby KJ, the first patient to receive a bespoke CRISPR therapy, was hailed as a breakthrough enabled by regulatory flexibility.
• Despite pro-innovation rhetoric, the FDA and NIH face proposed budget cuts of 4% and nearly 40%, respectively, raising concerns about implementation capacity.

Officials Seek to Streamline Innovation in Cell and Gene Therapy

In a high-profile roundtable hosted by the U.S. Food and Drug Administration (FDA) on June 5, federal officials, scientists, and health leaders convened to discuss cutting bureaucratic hurdles in the fast-evolving field of cell and gene therapy. The event was marked by calls to accelerate regulatory processes and eliminate outdated or obstructive rules to boost innovation and maintain U.S. leadership in biomedical research.

During the session, Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. emphasized the Trump administration’s regulatory agenda. “President Trump has given us an executive order that says for every regulation that we put in place, we have to get rid of 10,” he said, inviting attendees to submit a list of regulations they believe should be eliminated.

Urgency in Preserving U.S. Competitiveness

The roundtable discussion repeatedly addressed concerns that the U.S. is at risk of “ceding dominance” in cell and gene therapy to China. Dr. Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, summarized attendees’ sentiments about the growing challenge of global competition and the need for adaptive regulatory models.

Among those joining Prasad and Kennedy were FDA Commissioner Dr. Martin Makary, NIH Director Dr. Jay Bhattacharya, and CMS Administrator Dr. Mehmet Oz. All are recent appointments under President Trump’s second term.

Commissioner Makary stressed the agency’s intent to act: “We’re going to take those messages very seriously,” he stated. “This is not a horse and pony show.”

Learning from China’s Regulatory Approach

Dr. Carl June, director of the Center for Cellular Immunotherapies at the University of Pennsylvania, proposed that the U.S. consider emulating China’s two-tier system. In this model, early human trials require only institutional review board approval, with FDA involvement deferred until larger trials begin.

June remarked: “Why are researchers increasingly taking trials overseas? Simply put, the U.S. process has become too slow, costly and inflexible, while other countries make it easier to innovate.”

KJ Muldoon’s Case as a Paradigm Shift

A compelling example repeatedly cited at the event was the story of infant KJ Muldoon, the first recipient of a custom CRISPR gene therapy tailored to his unique genetic mutations. Dr. Fyodor Urnov of the Innovative Genomics Institute at UC Berkeley, who was part of the team behind KJ’s therapy, heralded the achievement as a turning point.

“The story of baby KJ charts a fundamentally new path to genetic medicines on demand, scalably and affordably,” Urnov stated. He called for greater industry involvement: “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.”

NIH Director Bhattacharya praised the case as proof of what can be accomplished with regulatory flexibility. “KJ I actually got to brag about a little bit, because it was NIH-funded work that actually led to this,” he said.

RFK Jr. Promises Administrative Streamlining

Although initially light on topic-specific remarks, RFK Jr. eventually aligned with the roundtable's theme, emphasizing reduced bureaucratic overhead. “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,” he said.

In closing, he reassured the scientific panel: “We’re going to rely on you to come up with the solutions. 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.”

Budget Cuts Add Complexity to Innovation Push

Despite public promises to promote innovation, financial constraints loom. The FDA is facing a proposed 4% budget cut under President Trump’s 2026 budget. Since the current administration took office, the agency has lost a notable portion of its workforce.

The NIH is expected to be hit harder, with nearly a 40% budget reduction and recent layoffs affecting dozens of employees. These fiscal measures may complicate the administration’s vision of reducing regulatory friction while boosting medical innovation.