Risk assessment of AAV transmission to the next generation-in-situ hybridization using gonadal tissues of monkeys administered in-house AAV-

Abstract

In AAV gene therapy, a genomic integration into non-target cells, especially germline cells should be minimized. ICH indicate the necessity to evaluate the distribution, persistence and localization in gonadal tissues. However, details have not been specified, although EMA guidelines indicate the in-situ hybridization (ISH) as a method.

Therefore, we examined the usefulness of ISH with reproductive tissues of AAV treated cyno. monkeys. We will show the results, suggesting low germline risk in male, and report the possibility of semi-quantitative evaluation by algorithm analysis using AI.