Over 90% of congenital deafness cases have been cured by gene therapy developed by Regeneron Pharmaceuticals.
Recently, Regeneron revealed that gene therapy had improved the hearing of 10 out of 11 children with congenital hearing loss, ranging in age from 10 months to 16 years.
The findings were presented at the 48th Annual MidWinter Meeting of the Association for Research in Otolaryngology.
Mutations in the otoferlin (OTOF) gene caused hearing impairments in all of the study’s children from birth. Inner hair cells express a protein called otoferlin, which transforms sound waves into neural signals and sends them to the brain.
Regeneron’s innovative gene therapy instructs the DNA in inner hair cells to produce the otoferlin protein.
According to Regeneron, three of the five children who underwent hearing tests over the 24 weeks experienced hearing improvements to normal or nearly normal levels, and ten of the eleven children who underwent at least one hearing test following gene therapy experienced improvements in hearing across a range of decibel levels.
