OTOV-201

OTOV-201 is an investigational adeno-associated virus (AAV) gene therapy being developed by Suzhou Otovia Therapeutics for the treatment of hereditary non-syndromic hearing loss (NSHL) caused by mutations in the GJB2 gene. The GJB2 gene encodes connexin 26, a critical gap junction protein responsible for maintaining potassium homeostasis in the inner ear, which is essential for the survival and function of sensory hair cells. OTOV-201 works through AAV-mediated gene replacement to deliver a functional copy of the GJB2 gene to the cochlea, aiming to restore functional connexin 26 expression and improve or restore hearing. As GJB2 mutations are among the most prevalent causes of hereditary deafness globally, OTOV-201 represents a significant expansion of the company's pipeline into common forms of genetic hearing loss. The therapy is currently in the IND-enabling stage of development.