AAV.Anc80L65.SLC26A4

AAV.Anc80L65.SLC26A4 is an experimental gene therapy candidate designed for the treatment of DFNB4, a form of hereditary hearing loss caused by mutations in the SLC26A4 gene. The therapy utilizes a synthetic adeno-associated virus (AAV) vector, Anc80L65, which is engineered to efficiently transduce key inner ear epithelial tissues, including the endolymphatic sac and the spiral prominence of the cochlear lateral wall. By delivering a functional copy of the SLC26A4 gene, the treatment aims to restore the expression of pendrin, a protein essential for maintaining ionic balance in the inner ear. Preclinical studies in mouse models have demonstrated that postnatal administration can improve auditory brainstem response (ABR) thresholds, preserve hair cells, and reduce endolymphatic sac enlargement, suggesting a potential therapeutic window for hearing restoration in humans.

Other names

AAV-Anc80L65-SLC26A4AAV-Anc-80L65-SLC26A4AAV-Anc 80L65-SLC26A4