Lillys Investigational Gene Therapy Restores Hearing in First Treated Patient

Lilly's investigational gene therapy, AK-OTOF, has successfully restored hearing in the first treated patient, an 11-year-old boy with genetic hearing loss. Within 30 days of a single administration of AK-OTOF, the patient experienced hearing restoration across sound frequencies. The therapy was well-tolerated, and no serious adverse events were reported.

AK-OTOF is a gene therapy developed to treat sensorineural hearing loss due to mutations in the otoferlin gene (OTOF). The therapy delivers copies of the otoferlin gene into the ear via an engineered virus, resulting in the production of the needed protein. Otoferlin-mediated hearing loss is estimated to affect around 200,000 people worldwide.

The patient's hearing restoration was observed within the normal range at some sound frequencies. Lilly plans to present full data from this patient and a second participant in the study at a medical conference in early February.

This early-stage success puts Lilly in direct competition with Regeneron, which is also developing an otoferlin gene therapy called DB-OTO. In October 2023, Regeneron reported improvements in auditory response for the first patient treated, an infant younger than two years of age, through week six.

While more data is needed to confirm the results of this study, gene therapies for restoring hearing are a significant area of focus. Hearing restoration is a major breakthrough, as currently, there are no approved pharmacologic treatments designed to restore hearing.