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 or expressed at low levels in normal tissue, and is expressed at high levels in a wide variety of malignancies, including in bladder cancer. Illustrated below is a section of bladder cancer demonstrating specific localization of H19 to tumor cells.
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.
Pivotal clinical studies of inodiftagene are focused on the treatment of non-muscle invasive bladder cancer (NMIBC).