Chemistry Newsletter Fall 2017 | Page 19

ix Based on UNC Chemistry identify RNA motifs in therapeutically compelling RNAs that have cave-like clefts and crevices. Small molecules that bind in these clefts may modulate RNA function and the underlying biology. If successful, Ribometrix could make finding drugs that target RNA straightforward in the sense that targeting RNA will be no harder than tar- geting proteins, the cellular targets of most drugs. Weeks notes, “targeting proteins is still a big challenge, of course, but Ribometrix technologies are easily adapted to high-throughput screening of many RNAs and small molecules, so there is also a tremendous opportunity here.” D rug discovery is expensive and so Weeks and War- ner spent roughly two years working to convince funding agencies and outside investors that they had a special angle on drug discovery, especially on poten- tially high-reward but high-risk drugs that target RNA. Ribometrix has since received multiple grants from the National Institutes of Health and, critically, private equi- ty funding, together totaling roughly nine million dollars. “Although Katie and I keep getting congratulated on all this fund raising, I do not really see this as success at all,” emphasizes Weeks. “Drug discovery is an incredibly diffi- cult and no-nonsense endeavor, which suits our person- alities. We will not consider this completely successful until we have created new chemical matter that makes people’s lives better.” R ibometrix has moved to lab space in a renovated for- mer tobacco-processing factory in Durham and has recruited leadership and scientific teams.The group is focusing on Huntington’s disease and on several tar- gets that were previously thought to be “undrugable” but probably can be drugged with the novel RNA technolo- gies that underlie the Ribometrix approach to drug dis- covery. “We now have a good shot at proving our ideas,” concludes Weeks. “Ultimately, if you want to make the largest possible impact with your university-created technology and ideas, a commercial company is often the best and most direct way to impact human lives. Lots of challenges ahead, but we are giving this our most ag- gressive efforts.” CHEM.UNC.EDU | CHEMISTRY AT THE UNIVERSITY OF NORTH CAROLINA | 19