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On January 23, Eli Lilly and Company announced:An 11-year-old boy observed hearing recovery 30 days after receiving the AAV gene therapy AK-OTOF for hereditary hearing loss in October last year, with no adverse events reported.。AK-OTOF-101 1/2 Phase Study Results (Including Initial Data from the Second Subject) to Be Announced on February 3, 2024, at the Winter Meeting of the Association for Otolaryngology Research.Hearing Loss Mediated by the Otoferlin GeneIt is a type of sensorineural hearing loss caused by mutations in the OTOF gene. Mutations or non-functional OTOF prevent auditory signals from being transmitted to the brain, and patients often exhibit severe to profound prelingual nonsyndromic sensorineural deafness. This condition accounts for 8% of congenital hearing loss cases, affecting approximately 20,000 people in the United States and Europe. Currently, there are no approved drug treatments for OTOF-mediated hearing loss or any other form of sensorineural hearing loss.
AK-OTOF was developed by Akouos and later acquired by Eli Lilly and Company for approximately $610 million.AK-OTOF is a gene therapy based on a dual adeno-associated virus (AAV) vector., by delivering the transgene encoding OTOF to the inner hair cells (IHC) of the cochlea to treat patients with OTOF-mediated hearing loss. The purpose of a one-time unilateral intracochlear administration of AK-OTOF is to enable the expression of normal full-length functional otoferlin in the patient's inner hair cells, which may potentially restore the patient’s auditory function.In April 2021, AK-OTOF was granted Orphan Drug and Rare Pediatric Disease designations by the FDA.The positive results of the AK-OTOF study highlight the therapeutic significance of gene drugs for Otoferlin gene-mediated hearing loss.
References:
https://investor.lilly.com/news-releases/news-release-details/positive-phase-12-clinical-trial-data-investigational-gene
