Shiga, Japan – November 13, 2023 – Takara Bio Inc., an innovative biotechnology company, announced the developed of a novel adeno-associated virus (AAV) vector, SonuAAV™, which exhibits high gene transfer efficiency into inner ear tissues, in collaboration with Dr. Kazusaku Kamiya, Associate Professor at the Department of Otorhinolaryngology in Juntendo University School of Medicine.

 

In recent years, AAV vectors have been widely used in gene therapy for various diseases, and there has been active pursuit of developing gene therapy for ocular and central nervous system diseases in Japan.

 

In the field of otorhinolaryngology, there is ongoing development of gene therapy using AAV vectors to target hereditary hearing loss and other disorders. However, the currently available AAV vectors have insufficient gene transfer efficiency to demonstrate therapeutic efficacy in inner ear organs, which remains a challenge.

 

In this study, Takara Bio Inc. and Dr. Kazusaku Kamiya developed SonuAAV™, a novel AAV vector capable of achieving over 80% gene transfer efficiency to the cochlear organ of mice. The efficiency of SonuAAV™ gene delivery to the inner ear organ was shown to be more than 10 times higher than that of conventional AAV2.

 

Through our CDMO services, we offer manufacturing services for various viral vectors, including AAV vectors. We also provide custom production services for our novel AAV vector, CereAAV™, which enables gene transfer to the brain and retina.

 

We will continue to focus on developing platform technologies for new modalities in gene and cell therapy, including CereAAV™ and other tissue-specific AAV vectors, and make these technologies widely available.