A British toddler born with a rare form of deafness can hear after receiving a pioneering gene therapy treatment developed by pharmaceutical company Regeneron. The news follows a similar treatment in January by Eli Lilly, allowing 11-year-old Aissam Dam to hear for the first time.
One-and-a-half-year-old Opal Sandy was born with hearing loss caused by a mutated single gene known as otoferlin, affecting roughly 200,000 people globally, or about 2% of all hearing loss cases. Otoferlin is a protein in the inner ear's hair cells that enables sound to be transmitted to the brain. Using a harmless virus, doctors inserted a replacement working gene through the ear canal to restore damage in a procedure lasting 16 minutes. Sandy's hearing in the treated ear reached close to normal capacity, including the ability to hear whispers.
Initial clinical trials will expand the treatment to both ears in higher doses to 18 children. Learn more about how hearing works here.
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