Exaluren (disulfate)
Exaluren (ELX-02) disulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG) . Exaluren disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations[1].
Product Specifications
CAS Number
2244622-33-9
Product Name Alternative
ELX-02 (disulfate) ; NB-124 (disulfate)
UNSPSC
12352005
Target
Others
Type
Reference compound
Related Pathways
Others
Applications
Metabolism-protein/nucleotide metabolism
Field of Research
Metabolic Disease
Assay Protocol
https://www.medchemexpress.com/exaluren-disulfate.html
Concentration
10mM
Purity
98.0
Solubility
H2O : 100 mg/mL (ultrasonic)
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
Molecular Formula
C19H42N4O18S2
Molecular Weight
678.68
References & Citations
[1]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.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, stored under nitrogen)
Scientific Category
Reference compound1
Clinical Information
Phase 2
Citation 01
Mol Ther. 2023 Apr 5;31 (4) :970-985.|Genet Med Open. 2025 Nov 17.|Mol Ther. 2023 Apr 5;31 (4) :970-985.
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