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Single-Dose OTOF Gene Therapy Restores Inherited Hearing in 90%

4/24/2026

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Summary

Story summary

Single-dose OTOF gene therapy cured inherited deafness in most trial participants.
Chinese researchers treated 42 patients at eight sites, restoring hearing in about 90% of treated ears.
The therapy delivers a functional OTOF gene via a single inner-ear injection using a hollowed-out virus.
Participants from infants to adults showed hearing improvement within weeks, with younger children and bilateral treatment improving the most.

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Story summary

Single-dose OTOF gene therapy cured inherited deafness in most trial participants.
Chinese researchers treated 42 patients at eight sites, restoring hearing in about 90% of treated ears.
The therapy delivers a functional OTOF gene via a single inner-ear injection using a hollowed-out virus.
Participants from infants to adults showed hearing improvement within weeks, with younger children and bilateral treatment improving the most.