HHS Secretary and Top Health Officials Pledge Support for Cell and Gene Therapies at FDA Roundtable

WASHINGTON D.C. – June 6, 2025 – In a significant move to accelerate medical innovation, U.S. Health and Human Services (HHS) Secretary Robert F. Kennedy Jr. yesterday joined the heads of the Food and Drug Administration (FDA), National Institutes of Health (NIH), and Centers for Medicare & Medicaid Services (CMS) at an FDA-hosted roundtable to formally express unified backing for the advancement of cell and gene therapies.

The June 5 roundtable, led by FDA’s Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad, M.D., heavily focused on speeding up the regulatory process for new cell and gene therapies. Dr. Prasad emphasized, “We want transformational therapies and cures, but we will consider any step forward. We understand progress is not made in a single leap,” indicating the FDA’s willingness to consider surrogate endpoints, real-world data, quality of life, and overall survival for approvals via accelerated pathways. Other key concerns raised by panelists included the risk of the U.S. “ceding dominance” in the field to China, and the potential of genetic engineering for pig organ transplants.

FDA Commissioner Martin Makary, M.D., affirmed the agency’s commitment to acting on the feedback. “We’re going to take those messages very seriously,” he stated, “This is not a horse and pony show.”

A prominent proposal for regulatory streamlining came from Carl June, M.D., Director of the Center for Cellular Immunotherapies at the University of Pennsylvania’s Perelman School of Medicine. Dr. June suggested emulating China’s two-tier regulatory system, where initial “first-in-human” exploratory trials require only institutional review board (IRB) approval, with FDA involvement reserved for larger, multisite trials. He questioned why researchers are increasingly taking trials overseas, asserting the U.S. process has become “too slow, costly and inflexible, while other countries make it easier to innovate.”

A powerful illustration repeatedly cited by attendees and agency heads was the success story of KJ Muldoon, an infant recently treated with the first-ever custom CRISPR gene therapy for his rare genetic mutations. Fyodor Urnov, Ph.D., a scientific director at the Innovative Genomics Institute at the University of California, Berkeley, and a key developer of KJ’s treatment, described it as charting “a fundamentally new path to genetic medicines on demand, scalably and affordably.” Dr. Urnov noted that while the ultimate goal is for the for-profit sector to drive dozens of approved “CRISPR-on-demand” therapies, “such companies are struggling under the old ways of making such therapies.” NIH Director Jay Bhattacharya, Ph.D., highlighted KJ’s story as an example of rapid advancement possible with relaxed regulatory requirements, noting the pioneering therapy was developed in mere months and proudly acknowledging its NIH-funded origins.

Academics, including Harvard’s David Liu, further emphasized the urgent need for a broader national effort in cell and gene therapies. Dr. Liu, a key developer of base and prime editing techniques, projected, “I believe it will be possible by 2030 to treat at least 1,000 rare disease patients.” He underscored the potential for significant cost savings and stressed the necessity of building a “national infrastructure for interventional genetics” to achieve this ambitious goal.

Secretary Kennedy, in his closing remarks, echoed the commitment to regulatory flexibility and fostering innovation. While briefly touching on broader health concerns, he returned to the theme of empowering scientists. “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 stated. He concluded by reassuring the panelists, “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, and do everything that we can to support you.”