Story perspectives

Breakthrough Gene Therapy Restores Hearing in Children

10/19/2025

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Summary

Story summary

DB-OTO, a Regeneron gene therapy, has shown promise to restore hearing in children with OTOF mutations.
The therapy uses a single injection to deliver a functional OTOF copy into the cochlea.
In a clinical trial, 11 of 12 children improved hearing within weeks.
Three-year-old Opal Sandy, the youngest recipient, can hear normally without a cochlear implant.
The advance could redefine hereditary hearing loss treatment and spur early genetic-testing policies.

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Story summary

DB-OTO, a Regeneron gene therapy, has shown promise to restore hearing in children with OTOF mutations.
The therapy uses a single injection to deliver a functional OTOF copy into the cochlea.
In a clinical trial, 11 of 12 children improved hearing within weeks.
Three-year-old Opal Sandy, the youngest recipient, can hear normally without a cochlear implant.
The advance could redefine hereditary hearing loss treatment and spur early genetic-testing policies.