Deaf children enter a world of sound as gene therapy clinical trial shows ‘life-changing’ results

A Chinese-led clinical trial for a gene therapy to cure congenital deafness has restored the hearing of five out of six test patients

The treatment is delivered via a single injection and uses a genetically edited virus as its delivery vector

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A clinical trial of a new gene therapy has shown promising results after it restored the hearing of five children born deaf. Photo: Shutterstock

Published: 8:00pm, 3 Feb 2024Updated: 8:36pm, 4 Feb 2024

A clinical trial of a new gene therapy has delivered startling results for children born with hearing loss.

After just one course of the gene therapy, five out of six children taking part in the trial are gradually returning to a world of sound, according to results published in the peer-reviewed journal The Lancet on January 25.

Involving scientists and clinicians from institutions including the Eye and ENT Hospital of Fudan University in Shanghai and Harvard Medical School, this was the world’s first human clinical trial of gene therapy for congenital deafness with the longest follow-up, it is claimed.

“Bravo!” Cui Xiang, a senior scientist at biotech company Novartis Gene Therapies in the US, posted on social media the day the results were published. “Another life-changing therapy that can be seen to cure congenital deafness with a type of genetic mutation.”

Between October 19, 2022 and June 9, 2023, 425 participants were screened and six children were eventually enrolled in the trial. They had all been diagnosed with autosomal recessive deafness 9, which is caused by mutations in a gene called otoferlin, or OTOF. This had led to the patients’ severe to complete bilateral hearing loss.

Six months after the gene therapy, five of the children showed significantly improved hearing.