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. For oncology applications, the transgene selected is designed to destroy angiogenic blood vessels that feed solid tumors. For other potential applications, such as the treatment of ischemia, a different transgene can be selected that is designed to promote the development of angiogenic blood vessels instead of their destruction.
VBL is developing VB-111 (ofranergene obadenovec), the lead oncology product candidate from our VTS platform technology, for solid tumor indications, with successful proof-of-concept data from Phase 2 clinical trials in rGBM, thyroid cancer and ovarian cancer.
VB-111 is currently being studied in the GLOBE Phase 3 pivotal trial for rGBM, conducted in the US, Canada and Israel under an FDA Special Protocol Assessment (SPA) with top-line data expected in Q2018. VB-111 is also being studied in the OVAL Pivotal Phase 3 Study in Platinum Resistant Ovarian Cancer