Host: The Japanese Society of Toxicology
Since 2010, the number of successful cases in clinical trials and approved products in gene therapy has increased. Gene therapy can be broadly divided into “in vivo gene therapy”, “in vivo virus therapy” and “ex vivo gene therapy”. As of 2020, as in vivo gene therapy, Glybera, Luxturna and Zolgensma using AAV vector, as in vivo virus therapy, Imlygic using oncolytic virus and as ex vivo gene therapy, Kymriah, Yescarta and Tecartus using CAR-T cell therapy have been approved as gene therapy products. With reference to NDA/BLA review reports, the development status is briefly summarized.
