Regeneron, which licenses technology developed by UF researcher Sanford Boye and team, is advancing gene therapy research that could restore hearing in people with inherited deafness.
The US Food and Drug Administration approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a clinical trial who were born without hearing.
The condition it treats is rare, affecting up to about 50 babies born each year in the US with mutations in a gene known as OTOF. But the therapy’s effect can be profound: In a trial of 20 kids, 16 had improvements in hearing about five months after treatment. Five of 12 who were followed for at least 11 months had their hearing essentially restored to normal.
Read more about FDA Approves First Gene Therapy for Inherited Deafness, Shown To Restore Hearing for Children With Rare Condition.
