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News in English

Gene therapy restored hearing in children with congenital deafness

Eleven out of twelve children with congenital deafness showed improvements in a study testing Regeneron’s gene therapy for hereditary hearing loss. The U.S. company now plans to apply for approval of the gene therapy.

The conference AAO-HNSF is currently taking place in Indiana, USA. On Sunday results, that were simultaneously published in the New England Journal of Medicine, were presented, attracting significant attention. Regeneron’s treatment for a rare genetic form of deafness has shown promising results.

The treatment, DB-OTO, targets a rare genetic form of deafness caused by mutations in the OTOF gene. Early promising data were presented in February this year. In the newly published study, the children have been followed for six months.

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