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Acoramidis is a kinetic stabilizer of transthyretin (TTR).1 It selectively binds to wild-type TTR and TTRV122I with negative cooperativity (Kd1s = 4.8 and 6.2 nM, respectively; Kd2s = 314 and 139 nM, respectively) over COX-1, COX-2, and the thyroid hormone nuclear receptor (IC50s = >10 µM for all). Acoramidis inhibits pH-induced amyloidogenesis of wild-type TTR and TTRV122I, indicating tetramer stabilization, when used at stoichiometric ratios of 1:1 and 1:0.5 with the number of TTR binding sites. It stabilizes TTRV122I in serum isolated from patients with familial amyloid cardiomyopathy when used at a concentration of 10 µM.
WARNING This product is not for human or veterinary use.
1. AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-