The U.S. Food and Drug Administration (FDA) has approved the first-ever gene therapy for inherited deafness.
The therapy, called Otarmeni, is approved to treat a form of hearing loss caused by mutations in the OTOF gene, which codes for a protein called otoferlin. Cells in the inner ear need otoferlin to translate vibrations into signals that can be interpreted by the brain. When people carry two defective copies of the OTOF gene — one from each parent — this line of communication between the inner ear and brain is cut, resulting in severe-to-profound hearing loss.
