**FDA Roundtable Discusses Streamlining Cell and Gene Therapy Regulations**

At a recent FDA-hosted roundtable on cell and gene therapy, key leaders and renowned scientists gathered to discuss the current state and future of these revolutionary treatments. The roundtable, held on June 5, focused on ways to expedite the regulatory process for new cell and gene therapies, a recurring theme throughout the discussions.

Robert F. Kennedy Jr. initiated the conversation by asking participants to list regulations they believed were hindering progress. Recognized experts such as Vinay Prasad, M.D., director of the FDA's Center for Biologics Evaluation and Research, voiced concerns about potentially losing dominance in the field to China and discussed leveraging genetic engineering for organ transplants.

The meeting saw the participation of prominent figures including FDA Commissioner Martin Makary, M.D., NIH Director Jay Bhattacharya, Ph.D., and CMS Administrator Mehmet Oz, M.D., all of whom were appointed during President Donald Trump’s second term. Makary assured attendees that their feedback would be taken into serious consideration, stating, "We're going to take those messages very seriously. This is not a horse and pony show."

China's approach to regulation was a significant topic of discussion. Carl June, M.D., from the University of Pennsylvania, suggested the U.S. could benefit from adopting China’s two-tier regulatory system. He critiqued the current U.S. process as "too slow, costly, and inflexible," causing researchers to conduct trials overseas.

The roundtable frequently revisited the inspiring case of KJ Muldoon, an infant who received the first custom CRISPR gene therapy for his rare genetic condition. Fyodor Urnov, Ph.D., from the Innovative Genomics Institute at UC Berkeley, highlighted this event as a potential new direction for scalable and affordable genetic medicines. Urnov stressed the need for regulatory reforms to foster the growth of biotechs capable of providing approved CRISPR-on-demand therapies.

NIH Director Bhattacharya celebrated the success of KJ's treatment as an example of what can be achieved with relaxed regulations. He also noted the significant contributions of NIH-funded work in this breakthrough.

RFK Jr., the Secretary of Health and Human Services, echoed the call for regulatory relaxation and emphasized reallocating budget funds to support innovative medical solutions. He concluded with a commitment to eliminate barriers and back the scientific community in bringing their solutions to market effectively.

This roundtable marks a crucial step towards potentially transformative changes in the regulatory landscape for cell and gene therapies, reflecting a united effort to accelerate medical innovation and provide timely solutions for patients in need.