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Two companies have launched clinical trials to see if they can restore hearing to children with a rare type of genetic deafness.
Akouos and Decibel Therapeutics, both based in Boston, are testing experimental therapies in children with severe hearing loss due to variations in a gene called OTOF. A third firm, Sensorion, in France, is gearing up to begin a similar trial in Europe. The companies are all aiming to deliver functioning copies of this gene to the inner ear. The approach is known as gene therapy. It's designed to be given once and have lasting effects.
Genetic causes account for 50 to 60 percent of hearing loss in babies, and up to 8 percent of those cases are due to mutations in the OTOF gene. About 20,000 people across the United States, United Kingdom, Germany, France, Spain, and Italy are thought to be affected. People with alterations in this gene lack a key protein needed for hearing called otoferlin.
“We want to restore physiological hearing on all frequencies so these babies can hear early on, they...
