STAT+: Skylark Bio kicks off new gene therapy trial for deafness

STAT PlusBiotech Skylar Bio kicks off new gene therapy trial for deafness Skylark is among several biotechs investigating different genetic mutations for future therapies Manage alerts for this article Email this article Share this article By O. Rose BroderickAug. 11, 2026 Disability in Health Care Reporting Fellow O.

Rose Broderick[email protected]Rose covers disability and mental health, and she helps write STAT’s daily Morning Rounds newsletter. Confidential tips can be sent to Rose on Signal at rosebroderick.11. Two years ago, scientists made history with a gene therapy that restored hearing for a small group of children who had been born deaf, allowing some of them to hear for the first time.

Indistinct murmurs turned into audible whispers. The robust gains and lack of safety concerns led the Food and Drug Administration to approve Regeneron’s Otarmeni therapy in April for the small group of people with this rare genetic mutation. “[Otoferlin-related mutations] are more of a Goldilocks.

The ear stays completely intact in animal models, even into geriatric models,” said Joe Burns, former senior vice president of research at Decibel Therapeutics, which was acquired by Regeneron in 2023.Advertisement Scientists are now eyeing other hearing-loss targets, including the holy grail — the GJB2 gene. Startups in the United States, France, and China are racing to concoct a solution, with Skylark Bio emerging from stealth on Tuesday to announce that they have dosed their first patient with a therapy aimed at restoring hearing in a child with a GJB2 mutation. STAT+ Exclusive Story Already have an account?

Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%!

Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, Clinical trials, disability, drug development, Gene therapy, STAT+ Submit a correction requestReprints O.

Rose Broderick Disability in Health Care Reporting Fellow Rose covers disability and mental health, and she helps write STAT’s daily Morning Rounds newsletter. Confidential tips can be sent to Rose on Signal at rosebroderick.11. Newsletter Understand how science, health policy, and medicine shape the world every day Recommended In the Lab August 11, 2026 IRBs have a problem.

Can North Star’s nonprofit model save it? Adam's Take August 11, 2026 STAT Plus: Why is MoonLake Immunotherapeutics scared of releasing data on its drug candidate? Advertisement Biotech August 10, 2026 STAT Plus: In surprise decision, FDA rejects radiopharma therapy expected to rival Novartis treatment The Readout August 10, 2026 STAT Plus: FDA panel to weigh Grail’s Galleri test Biotech August 10, 2026 STAT Plus: Sionna’s experimental cystic fibrosis pill falls flat in mid-stage trial Subscriber Picks