- Otonomy (NASDAQ:OTIC) announces preclinical proof-of-concept results for OTO-825 at the ASGCT Annual Meeting. OTO-825 is an AAV-mediated gene therapy developed in collaboration with Applied Genetic Technologies Corporation (NASDAQ:AGTC).
- The results demonstrate that a single administration of OTO-825 rescues hearing loss and cochlear damage in two preclinical models that represent a range of hearing loss severity caused by GJB2 deficiency.
- OTO-825 induces expression of Connexin26, the protein product of the GJB2 gene, in target cochlear cells including support cells of the organ of Corti and spiral limbus, and fibrocytes of the spiral ligament in both rodents and non-human primates.
- Connexin26 expressed by OTO-825 forms functional gap junctions, which are required for proper functioning of cochlear hair cells.
- Two conditional knockout models developed in mice mimic the human setting for GJB2 deficiency by displaying hearing loss and damage to the integrity of cochlear tissue.
- A single intracochlear administration of OTO-825 rescues expression of Connexin26 in both models.
- OTO-825 induces significant improvement in hearing across multiple frequencies and normalizes cochlear morphology in both models.
- AGTC shares up 2.2% premarket trading at $3.75.