Approximately 1.7 per 1000 babies born in the US are born with congenital deafness. A majority of these cases are due to genetic deficiencies. Children born with hearing loss are typically treated with hearing aids or cochlear implants, or communicate via American Sign Language (ASL) as part of the Deaf community.

Recent research is showing the possibility of another treatment option through the use of gene therapy for hearing loss caused from specific genetic deficiencies.

Variants of the OTOF gene which encodes otoferlin is responsible for 1-3% of cases of congenital deafness. Children with this genetic hearing loss are typically profoundly deaf, and utilize cochlear implants for treatment of the hearing loss.

Children with cochlear implants often receive great benefit from their devices, however, music appreciation and speech perception in background noise may still prove to be challenging.

While studies are showing progress in treating hearing loss with the use of gene therapy, this only applies to a percentage of the population with hearing loss at this time. As children with OTOF variants have intact inner ear cellular structures, they present differently than a majority of adults with acquired hearing loss who have damage to the inner ear cellular structures.

It is important that patients do not delay treatment through the use of assistive devices such as hearing aids and cochlear implants in anticipation of gene therapy becoming a readily available option.

Have You Heard The News?

A 2026 research study investigated the efficacy of gene therapy for children with OTOF variant genetic deafness. This study treated 12 children under the age of 18 with DB-OTO gene therapy and evaluated hearing following treatment.

The children received an intra-cochlear infusion and were re-evaluated 24 weeks after treatment. The researchers found that 3 out of the 12 children had normal hearing following treatment, and 6 out of 12 children could hear soft speech without the use of assistive devices.

Source: Valayannopoulos, V., Bance, M., Carvalho, D. S., Greinwald Jr, J. H., Harvey, S. A., Ishiyama, A., ... & Whitton, J. P. (2026). DB-OTO gene therapy for inherited deafness. New England Journal of Medicine, 394(11), 1074-1083.

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That's A Fact!

Congenital deafness affects 1.7 per 1000 babies born in the US.

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