2024-01-25 01:30:04
An experimental gene therapy developed by a Chinese company has restored hearing in children with congenital deafness, researchers working on a clinical trial reported Wednesday.
Five out of six children with profound deafness were able to regain their hearing and improve their speech skills six months after treatment with Refreshgene Therapeutics’ therapy, according to the report published in The Lancet.
“The results of this study are truly remarkable. We saw the children’s hearing improve dramatically week after week and they also regained their speech,” said Zheng-Yi Chen of Mass Eye and Ear and Harvard Medical School who collaborated on the study, in a statement.
All children suffered from profound deafness caused by mutations in the OTOF (otoferlin) gene. A functioning otoferlin protein is required for the transmission of sound signals from the ear to the brain.
OTOF mutations account for 2 to 8 percent of cases of congenital deafness, according to previous research. One in 1,000 children born in the United States suffers from moderate to profound hearing loss.
In surgical procedures at Fudan University Eye and ENT Hospital, researchers used a harmless virus to deliver a version of the human OTOF gene into patients’ inner ears.
After 26 weeks, five of the six children had regained their hearing, with dramatic improvements in speech perception and the ability to hold conversations, the researchers report.
Most side effects were minor and none had long-term effects, researchers said.
Researchers aren’t sure why the sixth child didn’t respond. One possible explanation is that some of the gene therapy solution leaked from the inner ear during or after surgery.
Refreshgene is also working with OBiO Technology Shanghai 6688238 on gene therapy for the eye disease neovascular age-related macular degeneration.
On Tuesday, another team of researchers announced the restoration of hearing in an 11-year-old who became the first patient to receive Eli Lilly LLY’s OTOF gene therapy.
In October, Regeneron Pharmaceuticals REGN announced positive safety and efficacy results in the first child with profound OTOF-related hearing loss treated with its otoferlin gene therapy.
Current results from these studies will be presented at the Association for Research in Otolaryngology winter meeting in February.
“There has been no effective treatment for deafness since the invention of the cochlear implant 60 years ago,” said Chen. “This is a major milestone that symbolizes a new era in the fight against all types of hearing loss
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