Eli Lilly says its potential gene therapy ‘restored hearing’ in 11-year-old boy

Eli Lilly has reported that its investigational gene therapy, AK-OTOF, has restored hearing in an 11-year-old boy with genetic hearing loss within 30 days of a single administration. The therapy is being developed to treat sensorineural hearing loss due to mutations in the otoferlin gene (OTOF). The patient, Aissam Dam, was the first participant in a small study run by Eli Lilly.

The key points of this development are:

- The treatment resulted in hearing restoration across sound frequencies within 30 days. In some frequencies, Dam's hearing was within the normal range at day 30.
- The surgical administration procedure and the investigational therapy were well tolerated, with no serious adverse events reported.
- AK-OTOF is a dual adeno-associated viral (AAV) vector-based treatment.
- Lilly plans to present full data from Dam, as well as a second participant in its study, at a medical conference in early February.

While this study is focused on hereditary hearing loss, successes in treating genetic hearing loss may have implications in treating other types of hearing loss. Eli Lilly will present the clinical trial results and reveal data on a second patient at the Association for Research in Otolaryngology 2024 MidWinter Meeting.