TG693 is an orally available, selective, ATP-competitive CLK1 inhibitor with IC50 of 112.6 nM.
TG693 also potently inhibits haspin activity and weakly inhibits DYRK kinases in a panel of 313 recombinant kinases.
TG693 promotes the skipping of the endogenous mutated exon 31 in DMD patient-derived cells and increases the production of the functional exon 31-skipped dystrophin protein.
TG693 inhibits the phosphorylation of CLK1 substrate serine/arginine-rich proteins and modulates pre-mRNA splicing in the skeletal muscle in mice.
