ELX-02 disulfate (NB-124 disulfate) 是一种首创的的合成的真核核糖体特异性糖苷 (ERSG)，可用于研究无义突变引起的遗传疾病。

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.

Solid

ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells. has not independently confirmed the accuracy of these methods. They are for reference only.Cell Cytotoxicity Assay

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 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.

Room temperature in continental US; may vary elsewhere.

-20°C, stored under nitrogen In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)

[1]. Leubitz A, et al. Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers. Clin Pharmacol Drug Dev. 2019 Jan 16.

In Vitro: H₂O : 100 mg/mL (147.34 mM; Need ultrasonic)DMSO : < 1 mg/mL (insoluble or slightly soluble)配制储备液