At the Hagiwara Laboratory, Department of Drug Discovery Medicine, we have established a completely novel approach to drug discovery, transcriptome-based drug discovery, founded on our fundamental research in RNA regulation. Through this approach, we are advancing the research and development of candidate therapeutics for diseases that have long been difficult to treat with conventional pharmacotherapy, including hereditary disorders, malignant tumors, neurodegenerative diseases, and chronic pain. Several of these compounds are already being co-developed with university spin-off ventures and have progressed to the clinical trial stage.