Gene therapy restores sound


United Kingdom

A gene therapy being developed by Regeneron Pharmaceuticals Inc has enabled a young child, suffering from a congenital hearing loss, to overcome deafness. The child, dosed at 11 months of age, is one of two to have responded to the experimental therapy which uses gene replacement to address mutations in the OTOF gene – the cause of the disease. A second child, who received the therapy at the age of four, showed initial improvements after dosing.