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

Exaluren (ELX-02; NB-124) sulfate is an synthetic eukaryotic ribosome-selective glycoside that induces read-through of nonsense mutations, resulting in normally localized full-length functional proteins. Exaluren sulfate is used for the research of cystic fibrosis caused by nonsense mutations^[1][2].

Exaluren sulfate induces significant premature stop codon read-through with maintained native stop codon fidelity in DMS-114 cells, increasing p53 protein expression and reducing nonsense-mediated mRNA decay of TP53 mRNA^[1].
Exaluren sulfate exhibits consistent nonsense mutation read-through activity and efficacy in human bronchial epithelial cells, Fischer rat thyroid cells, and a dual luciferase cystic fibrosis model^[1].
Exaluren sulfate induces read-through of the CTNS W138X nonsense allele in human cystinosis fibroblasts, increasing cystinosin protein and CTNS mRNA levels while reducing half-cystine levels^[1].

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

Exaluren (30-60 mg/kg; s.c.; daily; 14 days) sulfate achieves superior CFTR functional rescue in a CF mouse model expressing a human CFTR^G542X transgene^[1].
Exaluren (10 mg/kg; s.c.; 7 injections over a 3-week period) sulfate reduces kidney cell cystine levels by 30% in CTNS^Y226X nonsense mutant mice without overt renal toxicity^[1].

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

580.60

C₁₉H₄₀N₄O₁₄S

1375073-94-1

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.[x]

Room temperature in continental US; may vary elsewhere.

Please store the product under the recommended conditions in the Certificate of Analysis.

• [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]