BG00001

BG00001 is an experimental gene therapy consisting of a replication-deficient adenoviral vector (Ad5) carrying the human interferon-beta (IFN-β) transgene. Developed by researchers at the Abramson Cancer Center at Penn Medicine with vector supply from Biogen Idec, it was designed for the treatment of malignant pleural mesothelioma and malignant pleural effusions. The therapy is administered intrapleurally via an indwelling catheter, where the vector transfects mesothelial and tumor cells to produce high local concentrations of IFN-β. This local expression aims to trigger direct tumor cell death and stimulate a systemic antitumor immune response. Clinical development reached Phase I dose-escalation trials, which demonstrated successful gene transfer and some clinical activity, but the program was discontinued after the vector supply was withdrawn.

Other names

recombinant adenovirus-hIFN-betaAd.IFN-beta gene transfer