Gene Therapy Restores Hearing in Child

Eli Lilly has made a significant breakthrough with its experimental gene therapy, AK-OTOF, which has reportedly restored the hearing of a child within a month of treatment. The 11-year-old patient, who had been deaf since birth due to a genetic disorder, began to regain his hearing after receiving the therapy, reaching levels within the normal range for certain frequencies by day 30.

The therapy was deemed safe and well-tolerated, with no serious side effects observed. The success of this trial is particularly notable as it marks a major step forward in the field of gene therapy for hearing loss, an area that has seen extensive research for over two decades.

The treatment involves a surgical procedure to deliver the gene therapy, which is designed to provide a functional version of the otoferlin gene to the hair cells in the inner ear. This gene mutation is responsible for a form of genetic hearing loss that affects approximately 200,000 individuals globally, for whom there are currently no approved treatments.

AK-OTOF is a product of a dual adeno-associated viral vector and was originally developed by Akouos, a precision medicine company acquired by Eli Lilly for nearly $500 million in 2022. The therapy has received both Orphan Drug and Rare Pediatric Disease designations from the FDA, highlighting its potential to address unmet medical needs.

The Phase I/II trial, AK-OTOF-101, is evaluating the safety, tolerability, and effectiveness of the therapy at various doses. It aims to enroll 14 patients, aged between two and 17 years, across three locations. The Children’s Hospital of Philadelphia is one of the sites participating in the trial.

Eli Lilly's progress puts the company in competition with Regeneron, which is also working on an otoferlin gene therapy known as DB-OTO. Regeneron has reported early positive findings from its own trial involving an infant patient.

This advancement in gene therapy could potentially pave the way for treatments targeting over 150 other genes associated with childhood hearing loss, offering hope to many families affected by this condition. The success of AK-OTOF and similar therapies underscores the ongoing commitment to innovation in medical science, aiming to improve and transform the lives of those living with genetic disorders.