Small Molecules Against RNA

The start-up Ribometrix in North Carolina is scouring the three-dimensional, 3D, or tertiary, structures of RNA to find potential targets for small-molecule drugs. “If you’re going after tertiary structures, it looks like any other kind of drug discovery,” says Kevin Weeks, a biochemist at the University of North Carolina in Chapel Hill and founder of Ribometrix. “Just like with protein folding, you can find appropriate clefts and crevices withinRNA to target.” The company is searching for microRNAs and mRNAs associated with cancer, fibrosis, Huntington’s disease and multiple sclerosis. According to Weeks, the company has identified ten targets. “What gets me excited about these small molecules is if you draw structures, they look like normal drugs,” Weeks says. “They don’t look like anything special.” He adds that the company hopes to have multiple clinical leads by 2019….