ORY-1001 (RG-6016) 2HCl is primarily investigated for its potential in treating various cancers, particularly those involving hematological malignancies. It functions as a selective inhibitor of lysine-specific demethylase 1 (LSD1), an enzyme that plays a critical role in regulating gene expression and is often overactive in cancer cells. By inhibiting LSD1, this compound can reactivate silenced tumor suppressor genes and disrupt the growth and survival of cancer cells. It has shown promise in preclinical and early clinical trials for acute myeloid leukemia (AML) and small cell lung cancer (SCLC), where it may enhance the efficacy of existing therapies or serve as a standalone treatment. Additionally, its ability to modulate epigenetic mechanisms makes it a candidate for combination therapies targeting drug-resistant cancers.