H19 Program

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).



Phase II Trial of BC-819 Intravesical Gene Therapy in Combination with BCG in Patients with Non-Muscle Invasive Bladder Cancer. Halachmi et al. ASCO GU Symposium 2018. [Download Poster]

DNA Based Therapy with Diptheria Toxin-A BC-819: A Phase 2b Marker Lesion Trial in Patients with Intermediate Risk Nonmuscle Invasive Bladder Cancer. J Urol. 2014 Jun;191(6):1697-702. 

Phase I/II Marker Lesion Study of Intravesical BC-819 DNA Plasmid in H19 Over Expressing Superficial Bladder Cancer Refractory to Bacillus Calmette-Guerin. J Urol. 2008 Dec;180(6):2379-83.

Regulatory Sequences of the H19 Gene in DNA Based therapy of Bladder Cancer. Gene Ther Mol Biol. 2004; Vol 8, 181-192.