Until now, many forms of severe inherited hearing loss have been managed with assistive devices such as hearing aids and cochlear implants. Today, a first-of-its-kind gene therapy may offer new hope for patients with a rare mutation in the OTOF gene, which plays an essential role in transmitting sound signals from the inner ear to the brain. Dr. Ksenia Aaron, a leading otolaryngologist at Rambam Health Care Campus (Rambam), offers her perspective on this breakthrough in auditory medicine.
Gene therapy is a medical approach that treats disease by delivering, correcting, or modifying genetic material within a patient’s cells. In what is being hailed as a breakthrough in auditory medicine, the U.S. Food and Drug Administration has recently approved a new therapy, Otarmeni™, developed by the U.S.-based biotechnology company Regeneron, to treat a rare form of severe inherited hearing loss caused by mutations in the OTOF gene. The OTOF gene plays a critical role in transmitting sound signals from the inner ear to the brain.
“This specific therapy aims to restore hearing at the biological level,”
“This specific therapy aims to restore hearing at the biological level,” says Dr. Ksenia Aaron, an attending physician in Rambam’s Department of Otolaryngology–Head & Neck Surgery, “enabling a more natural perception of sound without relying on electro-mechanical stimulation of the auditory nerve.”
Instead of relying on external devices like hearing aids or cochlear implants, Otarmeni delivers a functional copy of the OTOF gene with the goal of restoring the natural transmission of sound signals from the inner ear to the brain. Currently intended for a very small group of patients with this specific genetic mutation, long-term outcomes are still being studied.
“Cochlear implants can significantly improve hearing and developmental outcomes in children with severe hearing loss, particularly in speech and language acquisition,” explains Dr. Aaron. “However, they have limitations—especially in sound fidelity—as patients may struggle with background noise, fine frequency discrimination, and music appreciation.
Gene therapy typically uses viral vectors to deliver the therapeutic gene into target cells
At the same time, Aaron cautions that gene therapy does not guarantee normal hearing in every patient. Some patients may still require additional amplification, such as a hearing aid, to achieve optimal function.
She emphasises that the research is still in its early stages. “Only about two years have passed since the first gene therapy infusions were administered, so long-term outcomes over five to ten years remain unknown. While the results are encouraging, they are not uniform.” She points out, “Some patients who initially benefited experienced high-frequency hearing loss, likely related to the procedure rather than the therapy itself.”
Availability in countries such as Israel will depend on the progress of ongoing clinical trials
Dr. Aaron also mentions that gene therapy typically uses viral vectors to deliver the therapeutic gene into target cells. “The body may mount an immune response to these vectors, which could complicate repeat treatments,” she explains. “Drawing on experience from gene therapies used in ophthalmology,” she adds, “improvements may not always be permanent.”
The therapy is currently approved for use only in the United States, with access limited to a small number of specialised centres. Its future availability in countries such as Israel will depend on the progress of ongoing clinical trials, the necessary medical infrastructure, the prevalence of relevant genetic mutations within the population, and regulatory approval outside the United States.
Based on a Hebrew article that first appeared on ynet
