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ELX-02 disulfate

CAS No. : 2244622-33-9

产品活性：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.

研究领域：Others

作用靶点：Others

In Vitro: ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.

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.
In 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.
In 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.

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