Eli Lilly gene therapy restores hearing in first dosed patient

Jan. 24, 2024

According to Eli Lilly, early phase data from its investigational gene therapy demonstrated pharmacologic hearing restoration within 30 days of administration in the first trial participant.

Akouos, a Lilly subsidiary, announced positive initial clinical results from the phase 1/2 AK-OTOF-101 study testing its dual adeno-associated viral vector-based gene therapy, AK-OTOF, in OTOF-mediated hearing loss. At day 30, the participant, an 11-year-old boy with profound hearing loss from birth, had hearing restored across all tested frequencies and within the normal hearing range at some frequencies.

AK-OTOF is designed to restore auditory function by gene transfer and durable expression of normal, functional otoferlin protein to the inner hair cells of the cochlea. In the trial, eligible participants received a single, unilateral intracochlear administration of AK-OTOF, with hearing restoration assessed by behavioral audiometry and auditory brainstem response.

Eli Lilly plans to present the trial results, including initial data from a second participant, at the 2024 Association for Research in Otolaryngology MidWinter Meeting on February 3.