ORY1001 是一种具有口服活性的选择性赖氨酸特异性去甲基化酶 LSD1/KDM1A 抑制剂，对相关的 FAD 依赖性氨氧化酶具有高选择性，IC50<20 nM。

ORY-1001 (RG-6016) is an orally active and selective lysine-specific demethylase LSD1/KDM1A inhibitor, with high selectivity against related FAD dependent aminoxidases with IC50 of<20 nM.

In THP-1 (MLL-AF9) cells, ORY-1001 results in a time/dose dependent me2H3K4 accumulation at KDM1A target genes and concomitant induction of differentiation markers. ORY-1001 also induces apoptosis in THP-1 and inhibits proliferation and colony formation of MV(4;11) (MLL-AF4) cells. [1]

In rodent MV(4;11) xenografts, ORY-1001 (<0.020 mg/kg, p.o.) leads to significantly reduced tumor growth. [1]

1431326-61-2

C15H22N2·2HCl

303.27

ORY-1001 (RG-6016) 2HCl;ORY-1001;RG-6016;ORY 1001

DMSO:5 mg/mL (16.48 mM)
Ethanol:1 mg/mL(3.3 mM)
H2O:56 mg/mL (184.7 mM)
Powder: -20°C for 3 years
In solvent: -80°C for 2 years