Breakthrough Gene Therapy: Deaf Baby Girl Hears for First Time

In a groundbreaking milestone, 18-month-old Opal Sandy, born deaf due to a rare genetic defect, experiences the miracle of sound after undergoing a revolutionary gene therapy trial. As the youngest participant globally and the first in the UK, Opal received a single gene therapy injection aimed at rectifying her congenital deafness. Within weeks, she responded to auditory stimuli, and today, boasts functional hearing in her treated ear, eliminating the need for a cochlear implant. This remarkable advancement, part of the CHORD clinical trial sponsored by Regeneron, offers hope for individuals with auditory neuropathy, like Opal, ushering in a potential paradigm shift in treating genetic hearing impairments. Opal’s story, presented at the American Society of Gene and Cell Therapy conference, underscores the transformative power of gene therapies in addressing profound hearing defects.