Problem

Mitochondria is a promising target for cancer treatment. However, current strategies to specifically and effectively destroy cancer mitochondria in vivo are limited.

Solution

• mLumiOpto is a mitochondrial-targeted luminoptogenetics gene therapy that includes the co-expression of a blue light-gated cationic channelrhodopsin (CoChR) in the inner mitochondrial membrane of cancer cells and of an emission-spectrum matched nanoluciferase (NLuc) in the cytosol of the same cells. Induction of intracellular NLuc bioluminescence activates CoChR, triggering mitochondrial depolarization and cancer cell death
• The mLumiOpto genes are selectively delivered to cancer cells in vivo by a cancer-targeted monoclonal antibody-tagged exosome-associated adeno-associated virus (mAb-Exo-AAV) carrying a cancer-specific promoter
• mLumiOpto reduces tumor burden in glioblastoma (GBM) and triple-negative breast cancer (TNBC) xenograft mouse models and induced an anti-tumor immune response, increasing infiltration of dendritic cells and CD8+ T cells in the tumor microenvironment (TME)
• mLumiOpto induces citotoxicity in cancer cells primarily through mitochondrial-mediated intrinsic apoptosis and DNA damage

Applications

• Cancer therapy for glioblastoma, non-small cell lung cancer, triple-negative breast cancer and other metastatic tumors

Competitive Advantages

• High specificity: The technology targets cancer cells specifically, thus minimizing off-target effects and systemic toxicity
• Great versatility: This system can be adapted to various cancer types by changing the targeting mAb on the exosome-associated AAV
• Reduced drug resistance: The technology would be less prone to resistance compared to conventional therapies by directly targeting inner mitochondrial membrane

Seeking Opportunities for Licensing and collaboration