RG3039 是一种具有口服活性、能够透过血脑屏障的DcpS抑制剂，其IC50=0.069 nM。

RG3039 (PF-06687859) is a potent DcpS inhibitor. DcpS is a therapeutic target for spinal muscular atrophy. Spinal muscular atrophy (SMA) is caused by deletion or mutation of both copies of the SMN1 gene which produces an essential protein known as SMN. RG3039 improves motor function in SMA mice. RG3039 also showed activity to improve survival, function and motor unit pathologies in two SMA mouse models.

1005504-62-0

C21H23Cl2N5O

432.35

PF-06687859;Rg3039

H2O:< 0.1 mg/mL (insoluble)
DMSO:6 mg/mL (13.88 mM),Need ultrasonic and warming
Powder: -20°C for 3 years
In solvent: -80°C for 2 years