1375073-93-0
Chemical Structure
Exaluren
Synonym(s): ELX-02; NB-124
- CAS. Nr.: 1375073-93-0
- Formula:C19H38N4O10
- Molecular Weight:482.53
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
InChIKey: KJBRSTPUILEBDR-DBMIJKFDSA-N
SMILES: 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
Biological Activity: Exaluren (ELX-02; NB-124) is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren is used for the research of cystic fibrosis caused by nonsense mutations[1][2].
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Exaluren | 98.0% | Exaluren (ELX-02; NB-124) is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren is used for the research of cystic fibrosis caused by nonsense mutations. | ||||||||||||||||||||
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- [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;8(8):984-994. [Content Brief]
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