16-Minute Gene Therapy Restores Hearing To British Baby, A World First

An 18-month-old British child has regained her hearing through a pioneering gene therapy, marking a significant stride in the treatment of deafness.

What Happened: Opal Sandy, who was born with a rare genetic condition that left her completely deaf, has now been able to hear without the need for external devices, according to Cambridge University Hospitals.

The therapy, administered at Addenbrooke’s in Cambridge, is the first of its kind and has sparked hope for the treatment of deafness.

The gene therapy, known as DB-OTO, was part of the CHORD trial that commenced in May last year. The trial aimed to assess the effectiveness and safety of the therapy in cases with OTOF mutations.

Opal received a dose of the therapy via the AAV1 virus, which was injected into her cochlea under general anesthesia. The virus delivered a copy of the gene material to cells, effectively restoring her hearing.

“These results are spectacular and better than I expected. Gene therapy has been the future in otology and audiology for many years and I'm so excited that it is now finally here. This is hopefully the start of a new era for gene therapies for the inner ear and many types of hearing loss,” said Professor Manohar Bance, an ear surgeon at Cambridge University Hospitals NHS Foundation Trust and chief investigator of the trial.

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Opal’s parents, Jo and James Sandy, expressed their excitement at their daughter’s newfound ability to hear even the faintest sounds. They also hope that this breakthrough will benefit other children and their families.

Why It Matters: This pioneering gene therapy represents a significant breakthrough in the treatment of deafness. The therapy’s success in restoring Opal’s hearing has the potential to revolutionize the treatment of auditory neuropathy, a rare genetic condition that affects approximately 1 to 2 babies in every 10,000 born, as per the report by Interesting Engineering.

Tesla CEO Elon Musk recently lauded the potential of genetic engineering technology in treating COVID-19, highlighting its potential as “digital medicine.” This stance contrasts with Republican skepticism towards COVID-19 vaccines, many of which utilize mRNA technology. Musk’s endorsement underscores the transformative potential of mRNA in healthcare and its expected growth in the coming years.

Meanwhile a Pfizer gene therapy trial resulted in the death of a young patient. These developments underscore the need for continued research and regulation in the field of gene therapy.

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