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Some deaf children are hearing again because of a new gene therapy

A medical field that almost died is quietly fixing one disease at a time.

In a lab room, a toddler, deaf from birth, sits while a tone plays. There’s no reaction. His face does not change.

Six weeks later, after a single injection of an experimental gene therapy, the same toddler is back in the same room. The tone plays. The toddler’s head turns toward the sound. And somewhere just off screen, the child’s grandfather says his name. The boy turns and looks. He can hear.

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“When the parents realized their child had a response to sound they cried,” says Dr. Yilai Shu of the Eye & ENT Hospital of Fudan University, who co-led the trial, in a video that showed the results. “The whole family cried.” The video cuts to another child, thirteen weeks post-treatment, dancing to music.

This is what gene therapy can do in 2026. The clip comes from the international clinical trial of an OTOF gene therapy run by Mass Eye and Ear and China’s Fudan University that provided the underlying science behind a drug the Food and Drug Administration (FDA) approved last week.

On April 23, the FDA granted accelerated approval to Otarmeni, a gene therapy from the pharma company Regeneron for severe-to-profound hearing loss caused by mutations in a gene called OTOF. In a pivotal trial, 80 percent of treated patients gained measurable hearing, and 42 percent reached the level needed to pick up whispers. Two and a half years after treatment, 90 percent of patients in the underlying multi-center trial were still hearing.

The one huge obstacle standing in the way of progress on gene-editing medicine

It’s a drug that certainly feels like a miracle to those in the trials, taking patients from silence to sound. But what can feel almost as miraculous is how far the broader field of gene therapies like Otarmeni — which deliver a working copy of a broken gene directly into a patient’s cells — have come.

In 1999, the nascent field of gene therapy all but........

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