Gene Therapy Drug Development: A Strategy
to Counter Western Competitors
Still Possible in Time
To create new gene therapy products for incurable diseases, it is crucial to focus on viral vector research and development alongside or even ahead of gene therapy research. Increased development and patenting of new viral vectors will drive further involvement from academia and pharmaceutical companies in gene therapy. Japan has the opportunity to develop unique viral vector tools from a perspective different from that of Western countries. The VVC aims to achieve superior results through innovative approaches before the research gap widens.
The development and approval of gene therapies for intractable diseases in Europe and the U.S. have their roots in extensive basic research. A key focus is the development of viral vectors, which are crucial for gene therapies, and the notable disparity in the number of researchers between Japan and the West. Support for viral vector development is needed for Japan to remain competitive.
The VVC is now developing an inhibitory neuron-specific promoter for treating neurological disorders. Versatile AAV vectors allow research to cover the entire brain and advance potential treatments for Alzheimer's, epilepsy, and other disorders. The VVC is striving to contribute indispensably to Japan neuroscience and is working to expand the potential use of viral vectors in gene therapy.
Method for Targeted Gene Expression Using AAV Vectors
Development of Cell-Type Specific Promoters for AAV Vectors
cmGAD67 Promoter