Gene therapy helps five out of six deaf children regain their hearing

A Chinese biotechnology company’s experimental gene therapy has demonstrated encouraging outcomes in reinstating hearing abilities among children born with congenital deafness, as per the findings disclosed in The Lancet by investigators participating in a clinical examination. The treatment, devised by Refreshgene Therapeutics, resulted in noteworthy enhancements in auditory perception and speech recognition in five out of six children grappling with profound deafness after six months of intervention.

Under the leadership of Zheng-Yi Chen from Mass Eye and Ear and Harvard Medical School, the study underscored the considerable advancements observed in the auditory capacities and linguistic progression of the children throughout the trial. Each participant bore profound deafness owing to mutations in the OTOF gene, pivotal for transmitting auditory signals from the ear to the brain.

Previous investigations suggest that OTOF mutations contribute substantially to congenital deafness cases, affecting an estimated 2% to 8% of individuals afflicted by the condition. Moderate to profound hearing loss is approximately one in every 1,000 births in the United States.

Throughout the trial, investigators at the Eye & ENT Hospital of Fudan University employed a benign virus to transport an adapted variant of the human OTOF gene into the inner portion of the ears of the patients through surgical procedures. Following 26 weeks, five of the six children displayed restored hearing capabilities, coupled with significant improvements in speech comprehension and conversational skills.

Most of the reported side effects of the gene therapy were minor and fleeting, with no enduring consequences observed. Nevertheless, the researchers indicated that one child failed to respond to the treatment, potentially due to the leakage of the gene therapy solution from the inner ear during or post-surgery.

Refreshgene is collaborating with OBiO Technology Shanghai to develop a gene therapy targeting neovascular age-related macular degeneration, broadening their focus beyond auditory-related conditions. Furthermore, distinct research teams have reported successful restoration of hearing in patients subjected to gene therapies by Eli Lilly and Regeneron Pharmaceuticals, both directed toward the OTOF gene.

The upcoming Association for Research in Otolaryngology MidWinter Meeting in February will unveil updated findings from these trials, signifying substantial strides in the pursuit of efficacious treatments for diverse forms of hearing impairment.

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