Entrepreneurial Support Program(Explore Course)
- Principal Investigator
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Tokyo University of Agriculture and Technology
Masayki Tera
- Adopted Theme
Development of pharmaceuticals based on small-molecule drug discovery targeting gene structures
- Subject of Research
- Development of pharmaceuticals based on small-molecule drug discovery targeting gene structures
- Overview
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Our research is focused on developing a platform technology for small molecule drug discovery that targets guanine quadruplex (G4) structures found in disease-causing RNAs. The G4 forming sites in RNAs are predicted by the AI-based G4 prediction system (step 1 in the figure). From the predicted G4 structures, the most suitable ones are selected as drug targets (Step 2). Small-molecule compounds that specifically bind to these targets are identified (Step 3). These small molecules are expected to bind to the G4 structures and inhibit the synthesis of disease-related proteins.
Using this platform technology, we are currently developing our first drug pipeline: therapeutic for leukemia. To date, we have identified an optimal G4 structure on a leukemia-causing gene as a small molecule target (Step 1 completed) and acquired multiple hits that bind to this structure (Step 2 in progress).
- Business Models（when applying）
Our goal is to establish collaborations with multiple pharmaceutical companies to co-develop drug pipelines using our enabling technology. To demonstrate the value of this technology, we will develop our first pipeline to clinical proof of concept (PoC) and then license it to pharmaceutical companies.
- Activity Planning（when applying）
- Strengthen the platform technology to improve the efficiency of hit compound discovery
- Expand the chemical structures of hit compounds and acquire lead compounds
- Evaluate the efficacy and safety of lead compounds
- Develop global business strategies and explore partnership opportunities for the foundational technology
- Strengthen the required intellectual property portfolio