Gene therapy cures genetic hearing loss

Child with hearing aids

In the first case of its kind, gene therapy has restored hearing in a patient in the United States.

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 Phase I/II study participant.

AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF).

Akouos, a wholly owned subsidiary of Eli Lilly, will present the results during the Late Breaking Presidential Symposium at the 2024 Association for Research in Otolaryngology (ARO) MidWinter Meeting.

The first participant to receive AK-OTOF in the study, an 11-year-old at the time of AK-OTOF administration with profound hearing loss from birth, experienced restored hearing within 30 days of AK-OTOF administration.

In this individual, hearing was restored across all tested frequencies, achieving thresholds of 65 to 20dBHL, and within the normal hearing range at some frequencies at the Day 30 visit. Both the surgical administration procedure and the investigational therapy were well tolerated, and no serious adverse events were reported.

“Gene therapy for hearing loss is something physicians and scientists around the world have been working toward for over 20 years,” said Professor John Germiller, Director of Clinical Research in the Division of Otolaryngology at Children’s Hospital of Philadelphia, and a principal investigator of the AK-OTOF-101 Clinical Trial. “These initial results show that it may restore hearing better than many thought possible.”

“Children with OTOF-mediated hearing loss are often born with profound hearing loss, yet only a small fraction have undergone genetic testing to receive a definitive diagnosis,” added Dr Oliver Haag, Head of Otolaryngology at Sant Joan de Deu Hospital in Barcelona. “The AK-OTOF-101 Clinical Trial and AK-OTOF-NHS-002 Natural History Study demonstrate the power of international collaboration in the development of new medicines for rare genetic conditions.”

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