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Exaluren

CAT: 0804-HY-114231-01Size: 1 mgDry Ice: NoHazardous: No
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CAT#:0804-HY-114231-01Size:1 mg
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Description
Exaluren (ELX-02) 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].
CAS Number
1375073-93-0
Product Name Alternative
ELX-02; NB-124
UNSPSC
12352005
Target
Others
Type
Reference compound
Related Pathways
Others
Field of Research
Others
Assay Protocol
https://www.medchemexpress.com/exaluren.html
Purity
98.0
Solubility
H2O : 100 mg/mL (ultrasonic)
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
Molecular Formula
C19H38N4O10
Molecular Weight
482.53
References & Citations
[1]Leubitz A, Frydman-Marom A, Sharpe N, van Duzer J, Campbell KCM, Vanhoutte F. 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 Nov;8 (8) :984-994.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Reference compound1
Clinical Information
Phase 2
Citation 01
Genet Med Open. 2025 Nov 17.|Mol Ther. 2023 Apr 5;31 (4) :970-985.|Mol Ther. 2023 Apr 5;31 (4) :970-985.