Our founding discovery, the H19 gene, is a controlling element for central malignant cell processes that plays a key role in carcinogenesis, tumor progression, drug resistance and metastasis in a spectrum of human cancers. H19 is normally silent in adult somatic tissues. But it is expressed at high levels in a wide variety of malignancies, including in bladder cancer.
BC-819, our current focus, is a first-in-class and first-of-its-kind gene therapy that harnesses the H19 genetic elements that control its cancer-specific expression into a targeted, recombinant DNA construct that encodes a lethal toxin specifically in malignant cells.
Clinical studies on BC-819 are currently focused on the treatment of early stage, superficial bladder cancer.