VBL Therapeutics’ lead oncology product candidate, VB-111 (ofranergene obadenovec), is a targeted anti-cancer gene-based biologic agent that is positioned to potentially treat a wide range of solid tumors. It is conveniently administered as an IV infusion once every 6-8 weeks.
VB-111 is designed to address solid tumors, by two mechanisms: First, by selectively targeting the blood vessels required for tumor growth, and encouraging the programmed cell-death process, or apoptosis and second, as a viral vector, VB-111 recruits immune cells into the tumor. Once immune cells are inside the tumor, they can identify not only VB-111 but also tumor specific proteins. Once familiarized with such tumor epitopes, immune cells can identify and destroy cancer cells throughout the body. Therefore, VB-111 can turn the tumor from being immunologically ‘cold’ to ‘hot’.
VB-111 has been observed to be well-tolerated in >300 cancer patients in an “all comers” Phase 1 trial as well as in three tumor-specific Phase 2 studies, for Ovarian Cancer, recurrent Glioblastoma (rGBM) and Thyroid Cancer. VB-111 is currently being studied in the OVAL Phase 3 study for platinum resistant Ovarian Cancer.
How does it work?
Vascular Targeting System (VTSTM): First-in-Class gene therapy platform technology with pipeline of multiple potential indications
VBL’s innovative, proprietary Vascular Targeting System (VTS™) platform technology enables systemic administration of gene therapy to either destroy or promote angiogenic blood vessels. VTS is both tissue- and condition-specific, allowing for targeted and limited gene expression in endothelial cells, the thin layer of cells that lines the interior surface of blood vessels undergoing angiogenesis.
Our VTS platform technology comprises three components, a viral vector, a promoter and a transgene:
- Viral vector—a modified virus that is used as a delivery vehicle to distribute the promoter and the transgene throughout the body.
- Promoter—our proprietary, genetically modified promoter, called PPE-1-3X, that specifically targets the endothelial cells of angiogenic blood vessels. When present in these cells, the promoter initiates the expression of the transgene.
- Transgene— a genetic sequence designed to yield a specific biologic effect, the expression of which is directed by PPE-1-3X. The particular transgene will vary depending on the therapeutic objectives of the product candidate.
Once the gene therapy has reached the angiogenic blood vessels, the PPE-1-3X promoter activates expression of the transgene to produce a desired protein in the endothelial cells of those vessels.