AAV-Sia6e

AAV-Sia6e is an engineered adeno-associated virus (AAV) vector derived from AAV6, specifically optimized for gene delivery to the inner ear. Developed through capsid shuffling by researchers at Juntendo University, Gap Junction Therapeutics, and Takara Bio, the vector is designed to target cochlear gap junction-forming cells. Its primary application is the treatment of hereditary hearing loss caused by mutations in the GJB2 gene, which encodes the Connexin 26 (CX26) protein. Preclinical studies have demonstrated that AAV-Sia6e can efficiently deliver gene replacement or genome editing payloads to restore gap junction function and improve hearing in adult mouse models and non-human primates. The vector is part of a translational platform aimed at providing postnatal and adult-targeted interventions for genetic deafness.