New Gene Therapy Helps Doctors Bring Hearing to Children Born Deaf

Cincinnati Children’s Hospital in Ohio has announced a remarkable advancement in the field of hearing restoration, showcasing a groundbreaking gene therapy that is enabling profoundly deaf children to hear for the first time. The therapy, named Otarmeni, is being implemented in a clinical trial involving two young siblings from Uzbekistan, both of whom suffer from a rare genetic variant linked to their hearing loss.

Three-year-old Abdu and his one-year-old sister, Sumaya, were brought to the hospital after their parents sought out otolaryngologist Dr. John Greinwald Jr., an expert in the molecular genetics of deafness. The family, having researched Dr. Greinwald online, approached him with the hope that he could help their children regain their hearing. “I was at one of our outlying clinics when they arrived, and they asked if I could assist them,” Dr. Greinwald recalled.

Both children were diagnosed with severe deafness attributed to a biallelic variant in the OTOF gene, which is integral for hearing. This specific genetic mutation results in a form of congenital deafness that is characterised by an inability to hear sounds as loud as a lawnmower just a few feet away. Traditional treatment options for this condition have primarily included cochlear implants, which serve to bypass the damaged parts of the inner ear.

However, Dr. Greinwald and his team are pioneering the Otarmeni treatment, which is a gene therapy designed for those suffering from severe-to-profound sensorineural hearing loss caused by OTOF gene mutations. Abdu and Sumaya qualified for the trial due to their specific genetic profile, their functioning outer hair cells, and the fact that they had not received cochlear implants.

In April 2025, Abdu underwent the innovative treatment, which involved a surgical procedure resembling that of cochlear implantation. The process entails accessing the cochlea through the bone and administering the gene therapy directly. According to the hospital, the results were astounding; within four weeks of the treatment, Abdu began to detect sounds and has since progressed to hearing within the normal range, also developing his speech skills.

Following Abdu’s positive response to the therapy, the research team decided to provide Sumaya with the same treatment. Remarkably, both siblings have experienced minimal complications, and Dr. Greinwald expressed his amazement at their swift progress. “It is something that we would never have imagined in our careers, that children would be able to hear without requiring devices,” he stated.

The outcomes of the clinical trial are promising. Published findings in The New England Journal of Medicine reported that nine out of the 12 initial participants could hear sounds as quiet as 70 decibels or below within six months of receiving the therapy—an achievement significant enough that these individuals would traditionally not be suitable candidates for cochlear implants.

As researchers continue to follow this and emerging cases, Dr. Greinwald and his team believe that the results of the study could herald major advancements in gene therapy for individuals with hearing impairments. He commended the collective effort that brought this treatment to fruition, stating, “It has taken a village. Hundreds of researchers and clinical staff have contributed to this work.”

The extraordinary nature of the Otarmeni therapy and the first-hand accounts of the siblings underscore the potential for gene therapy to transform the lives of children with profound hearing loss. This pioneering work not only showcases the emerging possibilities of genetic medicine but also highlights the ongoing efforts of medical professionals to innovate and improve the quality of life for their patients.