| Company Name: |
ChemeGen(Shanghai) Biotechnology Co.,Ltd.
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| Tel: |
18818260767 |
| Email: |
sales@chemegen.com |
| Products Intro: |
Product Name:ELX-02 disulfate
CAS:2244622-33-9
Purity:98% Package:10 mg;50 mg;100 mg;500 mg;1 g;5 g;10 g
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| | ELX-02 (disulfate) Basic information |
| | ELX-02 (disulfate) Chemical Properties |
| storage temp. | Store at -20°C, stored under nitrogen | | solubility | |DMSO : < 1 mg/mL (insoluble or slightly soluble) | | Water Solubility | Water : 100 mg/mL (147.34 mM; Need ultrasonic) |
| | ELX-02 (disulfate) Usage And Synthesis |
| Biological Activity | 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]. | | References | [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. |
| | ELX-02 (disulfate) Preparation Products And Raw materials |
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