rg-3039

RG3039, also known as PF-06687859, is the first small molecule developed specifically for treatment of Spinal muscular atrophy (SMA). This drug is used in SMA patients that have reached early stage clinical trials. It was shown that RG3039 improved survival, function and motor unit pathologies in SMA mouse models. It is known, that RG3039 is a potent inhibitor of the mRNA decapping scavenger enzyme (DcpS). DcpS is a nuclear shuttling protein that binds and hydrolyzes the m(7)GpppN mRNA cap structure and a modulator of RNA metabolism. Therefore, DcpS represents an intrigueing therapeutic target for modulating gene expression by a small molecule. The exact therapeutic mechanism remains unknown. — NCATS