Safety System

Whilst the lentiviral gene therapy technology has a highly impressive safety record, the aggressive attack on disease requires equally careful patient safety considerations. For our immuno-oncology therapy we have built-in a powerful safety system, which can be triggered to eliminate the genetically-modified cells by administering the well studied drug AZT to the patient. The AZT is converted only inside the the genetically-modified cell into a chemical which kills the cells.

Publications

Sato T, Neschadim A, Nakagawa R, Yanagisawa T, Medin JA. Evaluation of Bystander Cell Killing Effects in Suicide Gene Therapy of Cancer: Engineered Thymidylate Kinase (TMPK)/AZT Enzyme-Prodrug Axis.
Methods in Molecular Biology 1317:55-67 (2015).

Sato T, Neschadim A, Lavie A, Yanagisawa T, Medin JA. The engineered thymidylate kinase (TMPK)/AZT enzyme-prodrug axis offers efficient bystander cell killing for suicide gene therapy of cancer.
PLoS ONE 8(10):e78711 (2013).

Sato T, Neschadim A, Konrad M, Fowler DH, Lavie A, Medin JA. Engineered human tmpk/AZT as a novel enzyme/prodrug axis for suicide gene therapy.
Molecular Therapy: J. American Society Gene Therapy. 15(5):962-70 (2007).