A clinical trial sponsored by the biotech company Regeneron has produced 'jaw-droppingly good' early results.
Eighteen-month-old Opal Sandy was born completely deaf due to a rare congenital defect. No matter how loud or close a sound, she did not respond. Today, Opal's able to hear — unaided — after receiving a single dose of a groundbreaking gene therapy as part of an ongoing clinical trial.Opal, whose family lives in Oxfordshire outside London, became the first patient in the UK—and the youngest in the world—to receive an injection of a gene intended to repair the defect that made her deaf.
Gene therapies like the one sponsored by Regeneron have long been anticipated as potential game-changers for people with hearing defects.
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