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.

AVROBIO Safety System Overview


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).