3-year-old born deaf can hear 2 years after breakthrough gene therapy treatment
a day ago
- #gene therapy
- #medical breakthrough
- #hearing loss
- A 3-year-old girl, Opal Sandy, can hear normally after undergoing a groundbreaking gene therapy treatment for profound hearing loss.
- Opal was the youngest patient (11 months old) to receive the gene therapy injection in her right ear in 2023, targeting mutations in the OTOF gene.
- The 15-minute procedure, performed under general anesthesia, delivered a working copy of the OTOF gene into her cochlea.
- Doctors also placed a cochlear implant in Opal's left ear during the same procedure in the U.K.
- Two years post-treatment, Opal can hear without the cochlear implant, a transformation her parents describe as 'mind blowing.'
- Before the treatment, Opal and her older sister Nora (born with the same condition) could not hear even extremely loud noises.
- The therapy, DB-OTO, is part of a clinical trial involving patients in the U.K., U.S., Spain, and Germany.
- Regeneron reported that 11 of 12 trial patients, including Opal, experienced 'clinically meaningful' hearing improvements within weeks.
- The trial continues to enroll children under 18 for further research.