BC-819 is a first-in-class, first-of-its-kind gene therapy. Its fundamental technology is a targeted recombinant DNA-based gene construct for bladder cancer, engineered to express a lethal toxin specifically in malignant cells.
The plasmid BC-819 is a 4.5 kb recombinant DNA molecule containing 832 bp of H19 regulatory sequences driving the expression of diphtheria toxin A chain only in malignant cells.
Diphtheria toxin A is an extraordinarily potent payload: one molecule may kill a cell, binding NAD and EF2 and inhibiting protein translation. In BC-819, the lack of the diphtheria toxin B chain prevents transfer of the toxin molecule between cells. The mechanism of action of diphtheria toxin is very well understood. Unlike most cancer therapies, there is no need to validate a new target or pathway.