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Exaluren disulfate  (Synonyms: ELX-02 disulfate; NB-124 disulfate)

Cat. No.: HY-114231B Purity: ≥98.0%
COA Handling Instructions

Exaluren (ELX-02) disulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.

For research use only. We do not sell to patients.

Exaluren disulfate Chemical Structure

Exaluren disulfate Chemical Structure

CAS No. : 2244622-33-9

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Solution
10 mM * 1 mL in Water USD 1463 In-stock
Solid + Solvent
10 mM * 1 mL
ready for reconstitution
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Solid
1 mg USD 550 In-stock
5 mg USD 980 In-stock
10 mg USD 1500 In-stock
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This product is a controlled substance and not for sale in your territory.

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Based on 1 publication(s) in Google Scholar

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1 Publications Citing Use of MCE Exaluren disulfate

  • Biological Activity

  • Purity & Documentation

  • References

  • Customer Review

Description

Exaluren (ELX-02) disulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations[1].

In Vitro

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

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Cell Cytotoxicity Assay[1]

Cell Line: Wildtype human proximal tubule cells (HK-2)
Concentration: 0, 100 and 400 μg/mL
Incubation Time: 0, 24, 48 and 72 hours
Result: Cytotoxicity assay in wildtype human proximal tubule cells (HK-2) showing no toxic effect of 400 μg/mL at 0, 24, 48 and 72 h.
In Vivo

Exaluren (ELX-02) disulfate (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 Exaluren (ELX-02) disulfate 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). Exaluren (ELX-02) disulfate 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 Exaluren (ELX-02) disulfate accumulates in kidney tissue without overt renal toxicity and that Exaluren (ELX-02) disulfate (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Twenty-nine CtnsY226X/Y226X mice 5-7 month old[1]
Dosage: 10 and 30 mg/kg
Administration: Subcutaneous injection, at dose volume of 5 mL/kg, twice weekly for a period of 28 days (total of 8 doses)
Result: Highest levels were measured in the kidney, followed by spleen and liver, with lower levels in other tissues (lung, heart, cochlea and brain).
Clinical Trial
Molecular Weight

678.68

Appearance

Solid

Formula

C19H42N4O18S2

CAS No.
SMILES

OS(=O)(O)=O.C[C@@H](O)[C@@]([C@@H](O)[C@H](O)[C@H]1N)([H])O[C@]1([H])O[C@H]([C@H](C[C@@H](N)[C@@H]2O)N)[C@]2([H])O[C@@H]3O[C@@]([C@@H](N)C)([H])[C@@H](O)[C@H]3O.OS(=O)(O)=O

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

-20°C, stored under nitrogen

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

Solvent & Solubility
In Vitro: 

H2O : 100 mg/mL (147.34 mM; Need ultrasonic)

DMSO : < 1 mg/mL (insoluble or slightly soluble)

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 1.4734 mL 7.3672 mL 14.7345 mL
5 mM 0.2947 mL 1.4734 mL 2.9469 mL
10 mM 0.1473 mL 0.7367 mL 1.4734 mL
*Please refer to the solubility information to select the appropriate solvent.
In Vivo:
  • 1.

    Add each solvent one by one:  PBS

    Solubility: 100 mg/mL (147.34 mM); Clear solution; Need ultrasonic

*All of the co-solvents are available by MCE.
Purity & Documentation

Purity: ≥98.0%

References
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Exaluren disulfate Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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Exaluren disulfate
Cat. No.:
HY-114231B
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