Drugs

FDA to Unveil Faster Pathway for Gene-Editing Therapies

FDA is preparing to introduce a new, accelerated pathway for approving custom gene-editing therapies, aimed at treating patients with rare diseases. The announcement, highlighted in an interview with CBER director Vinay Prasad, signals a major shift in the agency’s regulatory approach.

Prasad told Bloomberg News that advances in technologies like CRISPR have made it possible to develop one-time, patient-specific treatments. “Regulation has to evolve as fast as science evolves,” he said. “The agency is going to be extremely flexible and work very fast with the scientists who want to bring these therapies to kids who need it.”

The FDA’s new approach will allow combined trials across patients with related genetic disorders, rather than requiring a separate trial for every mutation or condition. According to researchers Rebecca Ahrens-Nicklas and Kiran Musunuru, who are designing CRISPR trials at the Children’s Hospital of Philadelphia and University of Pennsylvania, this framework enables a single gene-editing platform to be adjusted for multiple patients with different mutations.

“It allows for the amazing ability to tackle multiple genetic diseases at once,” Ahrens-Nicklas told Bloomberg. Ahrens-Nicklas and Musunuru are now seeking FDA approval to start clinical trials for phenylketonuria and other urea cycle disorders, diseases affecting a small number of newborns and often requiring costly interventions like liver transplants.

Prasad plans to publish a paper in early November outlining the agency’s new pathway. He predicted it will spark investment in treatments for conditions affecting only a handful of patients, describing the effect as turning “the spigot on” for funding and research.

The new framework builds on real-world success: in 2025, 10-month-old KJ Muldoon became the first person to have his genes custom-edited to cure an inherited disease. While the case drew international attention, many families with rare diseases still face limited access to these therapies.

FDA’s shift could dramatically change the landscape for ultra-rare diseases, enabling rapid development of personalized therapies while reducing cost and time barriers that previously limited innovation.

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome