| Size | Price | Stock | Qty |
|---|---|---|---|
| 1mg |
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| 5mg | |||
| 100mg | |||
| Other Sizes |
| Targets |
Eukaryotic ribosome. ELX-02 disulfate is a synthetic eukaryotic ribosomal selective glycoside (ERSG) that binds to the ribosome and promotes readthrough of nonsense mutations. By inducing ribosomal readthrough, it allows translation to continue past premature stop codons, restoring production of full-length proteins in genetic diseases caused by nonsense mutations.
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| ln Vitro |
In human cells, exaluren (ELX-02) disulfate (100–400 μg/mL) may detect nonsense mutations without causing harm [1].
ELX-02 disulfate promotes ribosomal readthrough of nonsense mutations in vitro. The compound's activity is measured by its ability to restore protein expression in cell lines carrying nonsense mutations in target genes. It shows potent readthrough activity with minimal effects on normal stop codon recognition. The compound is being developed for genetic diseases caused by nonsense mutations. |
| ln Vivo |
Exaluren (ELX-02) disulfate (10 and 30 mg/kg; subcutaneous dose; every two doses for a total of 8 doses) accumulates in tissues in a dose-dependent manner with no gender differences [1]. In India, Exaluren (ELX-02) disulfate is available as a single (single subcutaneous injection of 10 mg/kg with a dosage volume of 5 mL/kg) and repeat formulations (twice weekly with 10 mg/kg/dose , lasts for 21 days; 7 times in total) for quicker absorption. Exaluren (ELX-02) disulfate is rapidly removed from dormancy in a biphasic manner with a terminal half-life (T1/2) of 0.5 hours [1]. In CtnsY226 null Accumulation of cystine [1]
In vivo, ELX-02 disulfate has been studied in animal models of genetic diseases caused by nonsense mutations. The compound demonstrates restoration of functional protein expression and improvement of disease phenotypes. ELX-02 disulfate is currently in development as a therapy for genetic diseases caused by nonsense mutations. |
| Enzyme Assay |
Nonsense mutation readthrough assays are performed in cell lines containing a reporter gene (e.g., luciferase or GFP) with a premature stop codon. Cells are treated with serial dilutions of test compound, and readthrough is quantified by measuring reporter activity. IC₅0 or EC₅0 values are calculated from dose-response curves. Protein restoration is confirmed by Western blotting.
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| Cell Assay |
Cytotoxicity assay[1]
Cell Types: Wild-type human proximal tubule cells (HK-2) Tested Concentrations: 0, 100 and 400 μg/mL Incubation Duration: 0, 24, 48 and 72 hrs (hours) Experimental Results: Wild-type human proximal tubule Cytotoxicity assay of cells (HK-2) demonstrated 400 μg/mL at 0, 24, 48 and 72 hrs (hours). Cells carrying nonsense mutations in the gene of interest (e.g., CFTR for cystic fibrosis, DMD for Duchenne muscular dystrophy) are treated with ELX-02 disulfate at various concentrations. Functional protein expression is assessed by Western blotting, ELISA, or functional assays. Cell viability and off-target effects are also evaluated. |
| Animal Protocol |
Animal/Disease Models: 29 CtnsY226X/Y226X mice, 5-7 months old [1]
Doses: 10 and 30 mg/kg Route of Administration: subcutaneous injection, dose volume 5 mL/kg, weekly Two times for 28 days. (8 doses total) Experimental Results: The highest levels were measured in the kidneys, followed by the spleen and liver, with lower levels in other tissues (lungs, heart, cochlea and brain). Animal models of nonsense mutation diseases (e.g., CFTR-mutant mice, DMD mice) are administered ELX-02 disulfate via subcutaneous injection or oral gavage. Protein restoration is assessed in target tissues by Western blotting or immunohistochemistry. Functional improvement is evaluated by disease-specific endpoints (e.g., chloride transport for CF, muscle function for DMD). |
| ADME/Pharmacokinetics |
ELX-02 disulfate has a molecular weight of 678.68 g/mol and formula C1₉H42N4O1₈S2. As a glycoside, it is water-soluble and formulated for parenteral administration. PK parameters are being characterized in preclinical and clinical studies. The disulfate salt form provides favorable solubility and stability properties.
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| Toxicity/Toxicokinetics |
ELX-02 disulfate is in clinical development and toxicity data are being generated through ongoing clinical trials. Preclinical toxicology studies would have been conducted to support clinical development. As a ribosomal targeting agent, potential toxicities may include effects on normal protein synthesis and off-target readthrough.
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| References | |
| Additional Infomation |
ELX-02 disulfate (Exaluren) is a clinical-stage investigational drug for genetic diseases caused by nonsense mutations. It is not FDA-approved. The compound represents a novel therapeutic approach for diseases such as cystic fibrosis, Duchenne muscular dystrophy, and other nonsense mutation disorders. It is a first-in-class ERSG and is being developed by Eloxx Pharmaceuticals.
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| Molecular Formula |
C19H40N4O14S
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|---|---|
| Molecular Weight |
580.604305267334
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| Exact Mass |
678.193
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| CAS # |
2244622-33-9
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| Related CAS # |
Exaluren;1375073-93-0;Exaluren sulfate;1375073-94-1
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| PubChem CID |
138454758
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| Appearance |
White to light yellow solid powder
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| Hydrogen Bond Donor Count |
14
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| Hydrogen Bond Acceptor Count |
22
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| Rotatable Bond Count |
6
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| Heavy Atom Count |
43
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| Complexity |
733
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| Defined Atom Stereocenter Count |
16
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| SMILES |
S(=O)(=O)(O)O.O([C@@H]1[C@@H]([C@H]([C@@H]([C@@H]([C@@H](C)O)O1)O)O)N)[C@@H]1[C@H](C[C@H]([C@@H]([C@H]1O[C@H]1[C@@H]([C@@H]([C@@H]([C@H](C)N)O1)O)O)O)N)N
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| InChi Key |
WYGVCSNIMZZOIT-BPQPGTHUSA-N
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| InChi Code |
InChI=1S/C19H38N4O10.2H2O4S/c1-4(20)14-12(28)13(29)19(30-14)33-17-9(25)6(21)3-7(22)16(17)32-18-8(23)10(26)11(27)15(31-18)5(2)24;2*1-5(2,3)4/h4-19,24-29H,3,20-23H2,1-2H3;2*(H2,1,2,3,4)/t4-,5+,6+,7-,8+,9-,10+,11-,12-,13+,14+,15+,16+,17+,18+,19-;;/m0../s1
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| Chemical Name |
(2R,3S,4R,5R,6S)-5-amino-6-[(1R,2R,3S,4R,6S)-4,6-diamino-2-[(2S,3R,4S,5R)-5-[(1S)-1-aminoethyl]-3,4-dihydroxyoxolan-2-yl]oxy-3-hydroxycyclohexyl]oxy-2-[(1R)-1-hydroxyethyl]oxane-3,4-diol;sulfuric acid
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
H2O : ~100 mg/mL (~147.34 mM)
DMSO :< 1 mg/mL |
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| Solubility (In Vivo) |
Solubility in Formulation 1: 100 mg/mL (147.34 mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication.
(Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 1.7224 mL | 8.6118 mL | 17.2236 mL | |
| 5 mM | 0.3445 mL | 1.7224 mL | 3.4447 mL | |
| 10 mM | 0.1722 mL | 0.8612 mL | 1.7224 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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