Akouos, Inc. is a subsidiary of Eli Lilly and Company that focuses on the development of gene therapies for the treatment of hearing loss and deafness. Their new investigational dual-AAV gene therapy, AK-OTOF, has been in Phase 1/2 clinical trials and targets the otoferlin (OTOF) gene. The OTOF gene makes an essential protein, also called otoferlin, that is required for normal hearing. Mutations or changes in the OTOF gene affect proper sound transmission from inner hair cells to the auditory nerves, and children with OTOF mutations are often born with profound hearing loss and require cochlear implants to access sound.
During the February 2024 Association for Research in Otolaryngology MidWinter Meeting, Akouos announced positive initial results from their AK-OTOF clinical trials. Their first patient, an 11-year-old with profound hearing loss from birth, experienced partially restored hearing within 30 days of receiving one dose of the AK-OTOF gene therapy. This patient’s hearing was restored across all frequencies, with some frequencies reaching normal hearing range, and no side effects were reported. Additional patients continue to be enrolled in these trials, and new trial results are to be expected at a later date.
What this means for Usher syndrome: If this investigational dual-AAV gene therapy continues to deliver positive results through its clinical trials, it may become an FDA-approved therapy, providing patients with OTOF-mediated hearing loss a promising path to restored hearing without the need for cochlear implants. Additionally, the potential success of this dual-AAV gene therapy approach will encourage researchers to study this method to address other genetic mutations that are responsible for Usher syndrome.
