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

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

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.

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

ELX-02 disulfate Chemical Structure

ELX-02 disulfate Chemical Structure

CAS No. : 2244622-33-9

Size Price Stock Quantity
10 mM * 1  mL in Water USD 1463 In-stock
Estimated Time of Arrival: December 31
1 mg USD 550 In-stock
Estimated Time of Arrival: December 31
5 mg USD 980 In-stock
Estimated Time of Arrival: December 31
10 mg USD 1500 In-stock
Estimated Time of Arrival: December 31
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Based on 1 publication(s) in Google Scholar

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Description

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].

In Vitro

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

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

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].

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

Formula

C₁₉H₄₂N₄O₁₈S₂

CAS No.

2244622-33-9

SMILES
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.
References

Purity: >98.0%

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Keywords:

ELX-02 disulfateNB-124 disulfateOtherseukaryoticribosomeselectiveglycosideERSGgeneticdiseasesnonsensemutationInhibitorinhibitorinhibit

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ELX-02 disulfate
Cat. No.:
HY-114231B
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