SonuAAV-GJB2

SonuAAV-GJB2 is an adeno-associated virus (AAV) gene therapy being co-developed by Takara Bio and Gap Junction Therapeutics for the treatment of hereditary hearing loss caused by mutations in the GJB2 gene (encoding Connexin 26). The therapy utilizes Takara Bio's proprietary SonuAAV™ vector, which is specifically engineered for high tropism and transduction efficiency in inner ear tissues, particularly cochlear supporting cells. By delivering a functional copy of the GJB2 gene, the therapy aims to restore gap junction intercellular communication and potassium homeostasis in the cochlea, which are essential for auditory function. Preclinical studies in mouse models have demonstrated significant restoration of hearing following local injection. Under a 2024 agreement, Gap Junction Therapeutics is responsible for clinical development, while Takara Bio handles manufacturing and process development, retaining exclusive manufacturing rights post-approval.

Other names

AAV-GJB2 gene therapyAAV-GJB-2 gene therapyAAV-GJB 2 gene therapyGJB2 gene therapy (Takara Bio)SonuAAV hereditary hearing loss program