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ELX-02 sulfate

产品编号 T19307 CAS 1375073-94-1

别名: NB-124 sulfate

ELX-02 sulfate, also known as NB-124 sulfate, is a novel compound currently under development as a therapeutic agent for genetic diseases triggered by nonsense mutations. This investigational compound, an advanced synthetic eukaryotic ribosome selective glycoside (ERSG), shows promising potential for targeted treatment.

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ELX-02 sulfate, CAS 1375073-94-1

规格	价格/CNY		货期	数量
25 mg	￥ 10,600		待询

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其他形式的 ELX-02 sulfate:

ELX-02 disulfate
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ELX-02
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生物活性

化学信息

存储 & 溶解度

参考文献

产品描述	ELX-02 sulfate, also known as NB-124 sulfate, is a novel compound currently under development as a therapeutic agent for genetic diseases triggered by nonsense mutations. This investigational compound, an advanced synthetic eukaryotic ribosome selective glycoside (ERSG), shows promising potential for targeted treatment.
体外活性	ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.
体内活性	ELX-02 is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h. ?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.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 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).

别名	NB-124 sulfate
分子量	580.6
分子式	C19H40N4O14S
CAS No.	1375073-94-1

存储

Powder: -20°C for 3 years | In solvent: -80°C for 1 year

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参考文献

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.

剂量换算

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体内实验配液计算器

请在以下方框中输入您的动物实验信息后点击计算，可以得到母液配置方法和体内配方的制备方法：比如您的给药剂量是10 mg/kg，每只动物体重20 g，给药体积100 μL，一共给药动物10 只，您使用的配方为5% DMSO+30% PEG300+5% Tween 80+60% ddH2O。那么您的工作液浓度为2 mg/mL。

母液配置方法：2 mg 药物溶于 50 μL DMSO (母液浓度为 40 mg/mL)，如您需要配置的浓度超过该产品的溶解度，请先与我们联系。

体内配方的制备方法：取 50 μL DMSO 主液，加入 300 μL PEG300，混匀澄清，再加 50 μL Tween 80，混匀澄清，再加 600 μL ddH2O, 混匀澄清。

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第二步：请输入动物体内配方组成，不同的产品配方组成不同，如有配方需求，可先联系我们提供正确的体内配方。

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