2244622-33-9
Chemical Structure
Exaluren disulfate
Synonym(s): ELX-02 disulfate; NB-124 disulfate
- CAS No.: 2244622-33-9
- Formula:C19H42N4O18S2
- Molecular Weight:678.68
IUPAC Name: (2R,3S,4R,5R,6S)-5-amino-6-(((1R,2R,3S,4R,6S)-4,6-diamino-2-(((2S,3R,4S,5R)-5-((S)-1-aminoethyl)-3,4-dihydroxytetrahydrofuran-2-yl)oxy)-3-hydroxycyclohexyl)oxy)-2-((R)-1-hydroxyethyl)tetrahydro-2H-pyran-3,4-diol bis(sulfate)
InChIKey: WYGVCSNIMZZOIT-BPQPGTHUSA-N
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
Biological Activity: Exaluren (ELX-02; NB-124) disulfate is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren disulfate is used for the research of cystic fibrosis caused by nonsense mutations[1][2].
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Exaluren disulfate | ≥98.0% | Exaluren (ELX-02; NB-124) disulfate is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren disulfate is used for the research of cystic fibrosis caused by nonsense mutations. | ||||||||||||||||||||
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References
- [1]. Kerem E, et al. ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease. Expert Opin Investig Drugs. 2020;29(12):1347-1354. [Content Brief]
- [2]. 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. [Content Brief]
