Session Details

The recent advances in clinical gene and cell therapy highlight that real-world clinical data need to be incorporated in future gene and cell therapies. The emerging potential differences between AAV-mediated gene delivery for different transgenes in the same tissue (liver, Hemophilia A and B) is worth reviewing and underlines the need for more nuanced liver gene therapy programs in the future. The potential toxicity associated with high-dose systemic AAV delivery also warrants a timely discussion and should help improve the safety and efficacy of upcoming AAV gene therapies. The use of cell therapy as a clinical treatment for metabolic diseases such as diabetes represents an exciting development in the field and will be of high interest to the ASGCT community.