Drug intelligence / Profile preview

SonuAAV

Development stage
Preclinical
Lead developer
Takara Bio
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Otic, Intracochlear, Local Injection
01

Overview

SonuAAV is a novel adeno-associated virus (AAV) vector developed by Takara Bio in collaboration with Dr. Kazusaku Kamiya at Juntendo University School of Medicine. It is designed for highly efficient gene transfer to inner ear tissues, achieving over 80% gene transfer efficiency to the cochlear organ in mice—more than ten times higher than conventional AAV2 vectors. The primary application of SonuAAV is as a platform for gene therapy targeting hereditary hearing loss and other otorhinolaryngologic diseases. In preclinical studies, local injection of a GJB2-expressing version (SonuAAV-CX26) into the mouse inner ear improved hearing in Gjb2-deficient mice six weeks after administration[1][3][5]. The mechanism involves delivery and expression of therapeutic genes such as GJB2 (gap junction protein beta 2), which plays a critical role in auditory function.

Brand names
SonuAAV
02

Targets

GJB2 (Gap junction protein beta-2)

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