Gene therapy trial restores hearing

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In a remarkable medical breakthrough, an 18-month-old British girl named Opal Sandy has had her hearing restored through a pioneering gene therapy trial. This significant development marks the first instance worldwide where gene therapy has been successfully used to treat deafness caused by auditory neuropathy, a condition where nerve impulses from the inner ear to the brain are disrupted.

Overview of the Gene Therapy Trial

Opal was treated at Addenbrooke's Hospital in Cambridge, part of the Cambridge University Hospitals NHS Foundation Trust. The gene therapy, developed by the US biotech firm Regeneron, involves delivering a functional copy of the OTOF gene directly to the ear. This gene is crucial for producing otoferlin, a protein that facilitates the communication of cells in the ear with the hearing nerve. The therapy, known as DB-OTO, was administered through a minimally invasive procedure where the gene solution was infused into the inner ear using a catheter. This innovative approach is considered a "one-and-done" therapy, potentially eliminating the need for repeated treatments.

Global Impact and Further Trials

The success of this trial has not only changed Opal's life but also signifies a new era in the treatment of deafness. The trial, named CHORD, is set to expand, with plans to recruit up to 18 children from the UK, Spain, and the US. These participants will be monitored over a five-year period to assess the long-term efficacy and safety of the therapy.

Comparative International Efforts

This breakthrough in the UK is part of a larger global effort to combat genetic forms of deafness. Similar gene therapy trials are being conducted in China and the United States, targeting the same gene but using different technologies and methods of delivery.

Medical and Social Implications

The success of Opal's treatment has been met with enthusiasm from the medical community and advocacy groups. Experts believe that this therapy could eventually lead to treatments for other genetic-related hearing conditions. Additionally, it highlights the importance of genetic testing in diagnosing and treating hearing loss, which is currently covered by the NHS in the UK.


The successful application of gene therapy in restoring hearing to a child with auditory neuropathy is a groundbreaking achievement that could pave the way for treating various genetic auditory conditions. This development not only offers hope to individuals with similar conditions but also represents a significant advancement in genetic medicine and the treatment of sensory impairments.
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