STAT+: Skylar Bio kicks off new gene therapy trial for deafness
Biotech startups in the U.S., France, and China are racing to bring new gene therapies for deafness by targeting different genetic mutations.
Two years ago, scientists achieved a breakthrough with gene therapy, restoring hearing for a small group of children born deaf, allowing them to hear for the first time. Their progress and safety made the Food and Drug Administration approve Regeneron’s Otarmeni therapy in April for individuals with a rare genetic mutation. Joe Burns, a former senior vice president of research at Decibel Therapeutics, which Regeneron acquired in 2023, noted that these gene mutations are "more of a Goldilocks."
The ear remains intact in animal models, even in geriatric models. Now, researchers are focusing on other hearing loss targets, including the GJB2 gene. On Tuesday, Skylark Bio emerged from stealth to announce that they have initiated treatment with their first patient for a therapy aimed at restoring hearing in a child with a GJB2 mutation.
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