Two congenitally deaf children can hear for the first time after being treated with gene therapy, according to data presented at a conference Wednesday.
The results are “jaw-droppingly good, just shocking how good. It exceeded the wildest expectations of anybody who started this work,” said Larry Lustig, an otolaryngologist at Columbia University and an investigator on the study.
The new data come from a larger study by Regeneron Pharmaceuticals’ subsidiary Decibel Therapeutics that will treat nearly two dozen children with a specific type of congenital hearing loss with gene therapy. The clinical trial is meant to measure safety and efficacy of DB-OTO, a gene therapy drug Regeneron is developing to treat children who have hearing loss due to changes in the otoferlin gene. Several teams are targeting this rare form of congenital deafness that affects 1% to 8% of deaf people globally. Lustig calls this the “lowest of the low-hanging fruit” for gene therapy deafness treatments.
