AAVS-SLC26A4

AAVS-SLC26A4 is an adeno-associated virus (AAV) gene therapy developed by Skylark Bio for the treatment of Pendred Syndrome and DFNB4, which are autosomal recessive disorders caused by mutations in the SLC26A4 gene. The therapy utilizes a novel AAV-S capsid to deliver a functional human SLC26A4 coding sequence directly to the inner ear. SLC26A4 encodes the pendrin protein, which is essential for regulating endolymphatic pH and maintaining endocochlear potential. In preclinical studies, AAVS-SLC26A4 demonstrated the ability to restore pendrin function, improve hearing thresholds, and prevent the loss of outer hair cells in Slc26a4 knockout mouse models.