ORY-1001(trans) is primarily investigated for its potential in cancer therapy, particularly in the treatment of hematologic malignancies. It functions as a selective inhibitor of lysine-specific demethylase 1 (LSD1), an enzyme involved in the regulation of gene expression. By inhibiting LSD1, ORY-1001(trans) can reactivate silenced tumor suppressor genes and promote differentiation of cancer cells, making it a promising candidate for targeted cancer treatment. Its application is being explored in clinical trials to assess its efficacy and safety in patients with acute myeloid leukemia (AML) and other cancers. Additionally, its role in modulating epigenetic mechanisms offers potential for combination therapies with other anticancer agents.