Host: The Japanese Society of Toxicology
Name : The 49th Annual Meeting of the Japanese Society of Toxicology
Date : June 30, 2022 - July 02, 2022
More than 30 years have passed since the world's first gene therapy for ADA (Adenosine Deaminase Deficiency) deficiency was implemented in the United States, and currently more than 3000 gene therapy clinical trials are being conducted. Recently, gene therapy drugs for cancers and genetic diseases have been approved as regenerative medicine products in Japan. On the other hand, there have been reports of serious adverse events in the development of these gene therapy drugs, including deaths caused by in vivo administration of adenovirus vectors and the development of leukemia caused by ex vivo gene therapy using retrovirus vectors. It is still fresh in our minds that adverse events including fatalities occurred last year in gene therapy trials for X-linked myotubular myopathy and Duchenne muscular dystrophy using adeno-associated viral vectors (AAV). The type and frequency of various toxicities, including such severe toxicities, vary depending on the viral vector used, its dosage, administration method, target cells, and other factors. We are developing gene therapy for hypophosphatasia using AAV and are investigating high titer, low volume, localized administration to increase safety and minimize toxicity as much as possible. In this symposium, we will focus on in vivo gene therapies and will discuss their efficacy, safety, and future challenges considering the gene therapy drugs for hypophosphatasia currently under development in our laboratory.
