Interview 1

Long Years of Basic Research Are Coming to Fruition

　Professor Hirokazu Hirai , who focuses on developing gene introduction techniques to understand brain functions, has played an important role in the national project of “Brain Mapping by Integrated Neurotechnologies for Disease Studies” by the Japan Agency for Medical Research and Development. His work involves using adeno-associated virus (AAV) vectors to activate specific cell groups in the primate brain. In 2018, he received government funding to establish Japan’s first “Virus Vector Core” based on his achievements, enhancing virus vector research in Japan.

　Professor Hirai observes the rapid advancements in gene therapy research in Europe and the U.S. with mixed feelings, as these regions have long-standing involvement in basic research that has led to treatment approvals for incurable diseases like spinal muscular atrophy and hemophilia.

　Gene therapy is becoming increasingly popular among researchers and companies in Europe and the U.S., supported by decades of foundational research, particularly in viral technologies. The significant difference in membership numbers between the gene and cell therapy societies in the U.S. and Japan, highlights the disparity in their research environments.

　Noteworthy gene therapy drugs like Zolgensma have demonstrated considerable success in treating severe diseases like spinal muscular atrophy with just a single infusion, although costs remain high. Viral vectors serve as essential tools in gene therapy to deliver genes to target cells and have diverse applications, from fundamental research to vaccine development.

　Over the years, various viruses have been adapted for use as vectors, with AAVs becoming particularly prominent since 2010 due to their versatility. The development and patenting of advanced viral vectors are vital to therapeutic drug development, as they lay the groundwork for effective gene therapy.

　There is a noticeable gap in viral vector research in Japan compared to the U.S.; however, initiatives like those at Gunma University are working to advance the field domestically by creating and providing state-of-the-art viral vectors.

　The Viral Vector Core (VVC), established at Gunma University Initiative for Advanced Research in 2019, focuses on developing advanced viral vectors for various life science fields and is Japan’s first specialized research institution in this area. The VVC is led by Professor Hirai, whose research group has over 20 years of experience in gene therapy research. It also serves as the most prominent "Virus Vector Core" in Japan, producing AAV vectors for other institutions while developing new vectors. One of the university's strengths is a marmoset research facility that supports the evaluation and screening of AAV vectors.

　Since 2018, the VVC has distributed approximately 900 virus vectors to various institutions nationwide, significantly contributing to brain research in Japan and supporting a wide range of applications. This initiative is crucial to advancing Japanese neuroscience.

　Numerous papers using AAV vectors from Gunma University have been published in top journals, with Professor Hirai authoring many. Developing unique virus vectors and securing patents is crucial for competing internationally in gene therapy, a mission the VVC has undertaken.