ORY-1001 2HCl

ORY-1001 2HCl (CAS No.: 1431326-61-2) is a small-molecule epigenetic modulator, also known as Iadademstat or RG?6016, classified as a selective covalent inhibitor of lysine-specific demethylase 1 (LSD1/KDM1A); LSD1 is a flavin cofactor-dependent amine oxidase responsible for catalyzing the demethylation of K4 and K9 sites on histone H3, thereby affecting chromatin accessibility and gene transcription programs, and engaging regulatory networks related to lineage determination and cell proliferation. In biochemical and cellular systems, this compound shows potent and specific target engagement and inhibition of LSD1, typically inducing accumulation of H3K4 dimethylation and triggering transcriptional reprogramming consistent with a differentiation phenotype at low nanomolar levels; cross-enzyme profiling studies show that it has significant selectivity over related FAD-dependent amine oxidases (such as LSD2, MAO?A and MAO?B), while its effects on several secondary pharmacological targets (including the imidazoline I2 receptor, norepinephrine and dopamine transporters, L-type voltage-gated calcium channels, and spermine oxidase, etc.) mostly appear at submicromolar to micromolar levels, suggesting that the use concentration needs to be precisely controlled in a multi-target context. Its in vitro activity overall spans the nanomolar to micromolar range, with specific effects varying according to the experimental platform, readout indicators, and cellular context; in biomedical research and drug discovery settings, it is commonly used as an epigenetic chemical probe to dissect LSD1-dependent enhancer repression, gene expression circuits, and chromatin regulatory mechanisms, and is widely applied in human cell lines (such as the myeloid model THP?1), primary cell panels, and mouse xenograft models, supporting lead compound discovery and mechanism-of-action studies centered on chromatin regulatory pathways; the treatment concentrations or dose settings used in experiments are generally determined according to the specific experimental design and research objectives.