Regeneron Pharmaceuticals announced that its experimental gene therapy showed improved hearing in 10 of 11 children aged 10 months to 16 years who were born with hearing loss caused by genetic mutations.
Promising results for children with hereditary deafness
The company explained that the first child who received the gene therapy, who was 10 months old at the start of the study, showed hearing improvement to normal levels across most of the frequencies important for speech.
The updated data were presented at the 48th Annual Meeting of the Association for Research in Otolaryngology, adding to the growing evidence of the effectiveness of these treatments in children with profound deafness resulting from mutations in the OTOFERLIN (OTOF) gene.
Other treatments for hereditary hearing loss
These efforts are not limited to Regeneron. Other companies, such as Chinese company Refreshgene and Eli Lilly, have developed similar gene therapies that have shown success in restoring hearing in children during early clinical trials.
Jonathan Whitton, head of Genetic Medicines at Regeneron, described this condition as “extremely rare,” noting that fewer than 50 babies are born each year with this genetic mutation.
How does gene therapy work?
Otoferlin is a protein found in the inner ear hair cells, which vibrate to transmit sound signals to the brain. Whitton explained that the treatment works by sending instructions encoded in DNA to these cells, helping them produce the otoferlin protein necessary for hearing.
Gene Therapy Results
Of the 11 children evaluated after receiving the treatment, 10 showed significant hearing improvements at various decibel levels.
Additionally, 24-week evaluations showed that 3 of the 5 patients had achieved “near-normal” or “normal” hearing levels. The first child to receive the treatment, 48 weeks after the dose, was able to recognize words like “mama,” “biscuit,” and “plane” at a normal conversational level without the need for visual cues.
Last year, Regeneron announced that the first child reached normal hearing levels in just 24 weeks, while another showed significant improvement after six weeks of treatment.
The Future of Gene Therapies for Hearing Loss
These findings represent an important step toward developing effective gene therapies for hereditary hearing loss, offering hope for children with this rare condition and potentially opening the door to more similar treatments in the future.
