Our founding discovery, the H19 gene, is a controlling element for key malignant cell processes that plays a role in carcinogenesis, tumor progression, drug resistance and metastasis in a spectrum of human cancers. H19 is normally silent in most adult somatic tissues, but it is expressed at high levels in a wide variety of malignancies, including in bladder cancer.
Inodiftagene vixteplasmid (formerly BC-819), our lead product candidate, is a first-in-class and first-of-its kind gene therapy. Inodiftagene is a targeted recombinant DNA construct that employs H19 gene regulatory sequences in order to drive the expression of an encoded lethal toxin specifically in malignant cells.
Clinical studies of inodiftagene are currently focused on the treatment of early stage bladder cancer.