CB096 binds to the 5′CGG/3′GGC internal loop structure of r(G4C2)exp RNA and interferes with its gain-of-function mechanism, thereby regulating nucleolar focusing, RNA splicing defects, RNA metabolism, nucleocytoplasmic transport dysfunction, and toxic dipeptide repeats (DPRs) generated by RNA translation. CB096 inhibits RAN translation in HEK293T cells with an IC50 of 20 μM.

Physicochemical Properties

Molecular Formula	C14H11N3O4
Molecular Weight	285.25
CAS #	108883-90-5
SMILES	COC1=C(C=CC(=C1)C2=NC3=C(C=C(C=C3)[N+](=O)[O-])N2)O
Storage	Powder-20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month
Shipping Condition	Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

Biological Activity

References

[1]. Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD. ACS Chem Biol. 2020 Dec 18;15(12):3112-3123.

Solubility Data

Preparing Stock Solutions		1 mg	5 mg	10 mg
	1 mM	3.5057 mL	17.5285 mL	35.0570 mL
	5 mM	0.7011 mL	3.5057 mL	7.0114 mL
	10 mM	0.3506 mL	1.7528 mL	3.5057 mL

*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.