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Exaluren

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].

Product Specifications

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.

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