Cayman Cb-096; 100 Mg; 108883-90-5; ≥98%

CB-096 is an inhibitor of repeat-associated non-ATG (RAN) translation.¹ It selectively binds to the hexanucleotide GGGGCC repeat expansion (r(G₄C₂)^exp) hairpin r(G₄C₂)₈ (EC₅₀ = 19 µM) over the anti-sense hairpin r(G₂C₄)₈ and base-paired construct r(GGCC)₁₀. CB-096 (25 µM) inhibits RAN translation and the formation of nuclear stress granules in HEK293T cells expressing r(G₄C₂)₆₆ in an in vitro model of C9orf72 amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) (c9ALS/FTD). It also induces relaxation of rat thoracic aortic rings pre-contracted with norepinephrine (EC₅₀ = 1.81 µM) and decreases systolic arterial pressure in conscious spontaneously hypertensive rats when administered at a dose of 100 mg/kg.²

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