RG3039

RG3039 (D‑157495) is a small molecule inhibitor of the scavenger mRNA decapping enzyme DcpS. It was developed as an investigational therapy for spinal muscular atrophy (SMA), a genetic disorder caused by mutations or deletions in the SMN1 gene. By inhibiting DcpS activity and modulating mRNA metabolism—including increasing SMN2 promoter-driven gene expression—RG3039 aims to increase levels of functional survival motor neuron (SMN) protein. Preclinical studies demonstrated that RG3039 increases SMN-dependent reporter activity and stabilizes full-length SMN mRNA in cellular models[3][8][5]. The compound has been studied primarily for SMA but remains investigational.

Other names

2,4-quinazolinediamine, 5-((1-((2,6-dichlorophenyl)methyl)-4-piperidinyl)methoxy)-5-(1-(2,6-dichlorobenzyl)-piperidin-4-yl)methoxyquinazoline-2,4-diamine5-[1-(2,6-Dichlorobenzyl)-Piperidin-4-yl]Methoxyquinazoline-2,4-DiamineRG3039 FREE BASERG-3039 FREE BASERG 3039 FREE BASE