A 3-year-old girl in the U.K., born completely deaf, can now hear without assistance two years after undergoing a groundbreaking gene therapy procedure.
Opal Sandy made history in 2023 when, at just 11 months old, she became the youngest patient to receive a gene therapy injection targeting the OTOF gene, which causes inherited deafness. During a 15-minute procedure, doctors delivered a working copy of the gene to her right cochlea and placed a cochlear implant in her left ear.
Today, Opal can hear normally even when her cochlear implant is turned off. Her mother, Jo Sandy, described the change as “mind blowing,” noting that before the treatment, neither Opal nor her older sister could detect even the loudest noises.
The therapy, known as DB-OTO, is part of an ongoing clinical trial involving patients in the U.K., U.S., Spain, and Germany. Regeneron, the pharmaceutical company leading the trial, recently announced that 11 of 12 children showed significant hearing improvements within weeks of the procedure.
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