Akouos

Gene Therapy for Hearing Disorders

AK-CLRN1 Clin0 Usher syndrome type 3A; AK-antiVEGF Clin0 vestibular schwannoma; GJB2 RD GJB2-mediated hearing loss; 2 undisclosed programs RD hearing loss

Akouos is developing AAV-based gene therapies to treat hearing loss that results from mutations in genes for inner ear function. Akouos uses specialized vectors and surgical procedures to target the inner ear. Akouos has exclusive rights to AAV vectors that were designed specifically for diseases of the inner ear, hearing, and balance. For example, the company uses a vector called Anc8OL65 that transduces sensory cells and cochlear cells with high efficiency. Akouos delivers their gene therapies via minimally invasive surgery along the sensory epithelium, where target cells of the inner ear are located. The therapies work by various mechanisms, including delivery of a healthy gene in loss-of-function mutations, delivery of RNAi, and delivery of a protein that alters a disease pathway.