OTO-825 / Otonomy, Beacon Therapeutics - LARVOL DELTA

[VIRTUAL] AAV-Mediated GJB2 Gene Therapy Rescues Hearing Loss and Cochlear Damage in a Mouse Model of Congenital Hearing Loss Caused by Conditional Connexin26 Knockout (ASGCT 2021) - "Sub-cellular localization of the CX26 protein in rescued animals was normal and apparent in support cells throughout the cochlea, and these animals showed increased numbers of surviving hair cells. These preclinical results with AAV-mediated GJB2 gene therapy support the use of OTO-825 as a clinical candidate to treat congenital hearing loss caused by GJB2 deficiency."

Preclinical • Gene Therapies • Immunology • Otorhinolaryngology