CB-096

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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1. Ursu, A., Wang, K.W., Bush, J.A., et al. Structural features of small molecules targeting the RNA repeat expansion that causes genetically defined ALS/FTD. ACS Chem. Biol. 15(12), 3112-3123 (2020).

2. Navarrete-Vázquez, G., Hidalgo-Figueroa, S., Torres-Piedra, M., et al. Synthesis, vasorelaxant activity and antihypertensive effect of benzo[d]imidazole derivatives. Bioorg. Med. Chem. 18(11), 3985-3991 (2010).