OTO-825

OTO-825 is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of congenital hearing loss caused by mutations in the GJB2 gene. The GJB2 gene encodes Connexin 26, a protein essential for the formation of gap junctions in the inner ear that maintain potassium homeostasis; deficiency in this protein is the most common cause of genetic hearing loss. OTO-825 was developed to deliver a functional copy of the GJB2 gene to the cochlea via a single local administration to restore hearing function. The program was initially a strategic collaboration between Otonomy and Applied Genetic Technologies Corporation (AGTC). Following Otonomy's financial difficulties and subsequent bankruptcy in 2023, the asset was sold to Decibel Therapeutics (which was later acquired by Regeneron).