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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.”
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