Eli Lilly (NYSE: LLY) has announced preliminary results from a Phase I/II study conducted by its subsidiary Akouos for gene therapy AK-OTOF, which is being investigated for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF). The press release reported that the first participant in the study regained hearing across all tested frequencies within 30 days of treatment, following a history of profound hearing loss since birth. Both the intracochlear delivery device and the gene therapy candidate were well tolerated by patients.
AK-OTOF is a dual adeno-associated viral (AAV) vector-based therapy designed to restore the expression of normal, functional otoferlin protein to the inner hair cells of the cochlea. This restoration has the potential to return high acuity physiologic hearing to patients with this specific genetic mutation.- Flcube.com
