Advanced International Journal for Research

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GENE THERAPY TO TREAT DEAFNESS

Author(s) Prof. Jaivin Jaisingh J
Country India
Abstract This article describes the first FDA-approved gene therapy for genetic deafness, Otarmeni (lunsotogene parvec-cwha), developed for pediatric and adult patients with severe-to-profound sensorineural hearing loss caused by biallelic OTOF mutations. The objective was to restore auditory function by addressing the underlying molecular defect responsible for impaired synaptic transmission in inner hair cells. The therapy is delivered as a one-time intracochlear injection using adeno-associated viral vectors that carry a functional OTOF gene, enabling production of otoferlin and reactivation of signal transmission from the cochlea to the auditory nerve.
Clinical data from the pivotal CHORD trial showed substantial hearing improvement after treatment. Approximately 80% of participants achieved hearing thresholds at or below 70 dB within 24 weeks, 42% reached normal or near-normal hearing levels by 48 weeks, and 70% demonstrated confirmed auditory brainstem responses. Benefits appeared durable, with responders maintaining gains through at least 48 weeks and early evidence suggesting continued stability beyond that period. The strongest outcomes were observed in younger patients, particularly those 18 years and under.
Overall, Otarmeni represents a major advance in precision otologic medicine by targeting the genetic cause of deafness rather than bypassing it. These findings suggest that OTOF gene therapy may offer meaningful, durable restoration of natural hearing in appropriately selected patients, while broader application to other genetic forms of hearing loss remains under investigation.
Keywords OTOF gene, deafness, restoration, reactivation, signal transmission
Field Medical / Pharmacy
Published In Volume 7, Issue 3, May-June 2026
Published On 2026-06-06
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E-ISSN 3048-7641
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