Gene Editing Helped One Baby—Could It Help Thousands?

image via New Scientist

November 4, 2025, 1:00 PM

• •A clinical trial using base editing will be launched to treat children with genetic mutations.
• •The therapy was initially developed and used to successfully treat baby KJ Muldoon.
• •The FDA is accepting safety data from KJ's treatment to expedite the trial process.
• •Researchers aim to reduce the time needed to produce a therapy from six months to three or four.
• •The team is publishing correspondence with the FDA to serve as a model for other researchers.

Researchers are launching a clinical trial using a gene-editing technique called base editing, following the successful treatment of a baby boy with a rare genetic disorder. The therapy, developed in record time, made precise changes to the baby's DNA, allowing him to produce a crucial liver enzyme and improve his condition. The trial aims to treat multiple children with similar genetic mutations, with the hope of accelerating the process and making this technology accessible to more patients. This marks a significant step towards personalized gene-editing therapies, potentially revolutionizing the treatment of rare diseases.

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