Current status and future perspective of gene therapy products in Japan

Six years have passed since the Pharmaceutical Affairs Law was revised and renamed the Pharmaceuticals, Medical Devices and Other Therapeutic Products Act (PMD Act), which came into force in November 2014. As of January 2021, 10 regenerative medical products have been approved. Research and development of regenerative medical products (cell and gene therapy products) is accelerating, especially for gene therapy products. The development of ex vivo gene therapy products active in Japan and overseas, and commercialization has become a reality through products such as KYMRIAH^® and YESCARTA^®. In addition, the development of in vivo gene therapy products such as the viral vector product, ZOLGENSMA^®, and plasmid vector product, COLLATEGENE^®, has also become a reality. We believe that the development of gene therapy products will become more and more active. This review describes the current status and future perspective of gene therapy products from experiences of the Pharmaceuticals and Medical Devices Agency (PMDA).

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