RGNCY-0062 (2AU-2 r(AUUCU) target)

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2AU-2 is a first-in-class chemical probe that targets RNA AU base pairs in cells.  RNA is known to be an important target for functional chemical probes and lead therapeutics but it has always been difficult to target with small molecules.  2AU-2 is a dimeric small molecule that selectively binds expanded r(AUUCU) repeats in pathogenic RNA that cause spinocerebellar ataxia type 10 (SCA10) in patient derived cells.  2AU-2 (50nM) ameliorates various aspects of SCA10 pathology including improved mitochondrial dysfunction, reduced activation of caspase 3 and reduction of nuclear foci.  2AU-2 is a novel probe for the study of SCA10 RNA toxicity and may define broadly applicable compounds targeting RNA AU base pairs in cells.

Systematic Name:  N-(2-amino-2-oxoethyl)-N-((1-(3-(4-(5-(4-carbamimidoylphenyl)furan-2-yl)benzimidamido)propyl)-1H-1,2,3-triazol-4-yl)methyl)-2-(2-(2-(((1-(3-(4-(5-(4-carbamimidoylphenyl)furan-2-yl)benzimidamido)propyl)-1H-1,2,3-triazol-4-yl)methyl)amino)-N-propylacetamido)-N-propylacetamido)acetamide

SMILES: NC(CN(C(CN(C(CN(C(CNCC1=CN(CCCNC(C2=CC=C(C3=CC=C(C4=CC=C(C(N)

=N)C=C4)O3)C=C2)=N)N=N1)=O)CCC)=O)CCC)=O)CC5=CN(CCCNC(C6=CC=C(C7=CC=

C(C8=CC=C(C(N)=N)C=C8)O7)C=C6)=N)N=N5)=O

Formula:   C62H73N19O6

Mol Wt:  1180.39

PMID:  27248057