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。
货号:ajcx38844
CAS:2244622-33-9
分子式:C19H42N4O18S2
分子量：678.68
溶解度:H2O : 100 mg/mL (147.34 mM; Need ultrasonic)|DMSO :< 1 mg/mL (insoluble or slightly soluble)
纯度：98%
存储：Store at -20°C
库存：现货

Background:

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[1].

ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells[1].

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[1].In plasma ELX-02 is rapidly absorbed with a Tmax 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 (T1/2) of 0.5 h[1]. In a CtnsY226X 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[1].

[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.