Exaluren (ELX-02) disulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside ( ERSG ). Exaluren disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.
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Product Description
ELX-02 disulfate (NB-124 disulfate) is an investigational, advanced synthetic eukaryotic ribosome selective glycoside ( ERSG ). ELX-02 disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.
In Vitro
ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells. MCE has not independently confirmed the accuracy of these methods. They are for reference only. Cell Cytotoxicity AssayCell Line: Wildtype human proximal tubule cells (HK-2) Concentration: 0, 100 and 400 μg/mL Incubation Time: 0, 24, 48 and 72 hours Result: Cytotoxicity assay in wildtype human proximal tubule cells (HK-2) showing no toxic effect of 400 μg/mL at 0, 24, 48 and 72 h.
In Vivo
ELX-02 (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference . ?\nIn plasma ELX-02 is rapidly absorbed with a T max of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). ELX-02 is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T 1/2 ) of 0.5 h . ?\nIn a Ctns Y226X nonsense mutant mouse, subcutaneous ELX-02 accumulates in kidney tissue without overt renal toxicity and that ELX-02 (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo . MCE has not independently confirmed the accuracy of these methods. They are for reference only. Animal Model: Twenty-nine Ctns Y226X/Y226X mice 5-7 month old Dosage: 10 and 30 mg/kg Administration: Subcutaneous injection, at dose volume of 5 mL/kg, twice weekly for a period of 28 days (total of 8 doses) Result: Highest levels were measured in the kidney, followed by spleen and liver, with lower levels in other tissues (lung, heart, cochlea and brain).
This compound belongs to the class of organic compounds known as 4,5-disubstituted 2-deoxystreptamines. These are 2-deoxystreptamine aminoglycosides that a glycosidically linked to a pyranose of furanose unit at the C4- and C5-positions.
External Descriptors
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1. Djoumbou Feunang Y, Eisner R, Knox C, Chepelev L, Hastings J, Owen G, Fahy E, Steinbeck C, Subramanian S, Bolton E, Greiner R, and Wishart DS. ClassyFire: Automated Chemical Classification With A Comprehensive, Computable Taxonomy. Journal of Cheminformatics, 2016, 8:61.
