“The authors present the results of a clinical trial of DB-OTOF gene therapy, designed to treat congenital deafness caused by otorphelin (OTOF) deficiency. The main finding is that DB-OTOF gene therapy can improve hearing in patients with otorphelin deficiency. Three of the 12 patients in the study achieved normal hearing thresholds, and six out of 12 were able to understand speech without hearing aids. The article presents robust evidence regarding the efficacy and safety of gene therapy in patients with otorphelin deficiency, which is the leading cause of congenital deafness treated by gene therapy.”
Published October 2025; NEJM.org
Authors: Vassili Valayannopoulos, M.D., Ph.D., Manohar Bance, M.B., Ch.B., Daniela S. Carvalho, M.D. John H. Greinwald, Jr., M.D., Steven A. Harvey, M.D., Akira Ishiyama, M.D., Evie C. Landry, M.D., +27
