TargetMol

ELX-02

Product Code:
 
TAR-T31616
Product Group:
 
Inhibitors and Activators
Supplier:
 
TargetMol
Regulatory Status:
 
RUO
Shipping:
 
cool pack
Storage:
 
-20℃
1 / 1
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TAR-T31616-5mg5mgEnquire
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Further Information

Bioactivity:
ELX-02, also known as NB-124, is a eukaryotic ribosomal selective glycoside (ERSG) designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to restore activity.
CAS:
1375073-95-2
Formula:
C19H38N4O10
Molecular Weight:
482.531
Purity:
0.98
SMILES:
C[C@@H](N)[C@H]1O[C@@H](O[C@@H]2[C@@H](O)[C@H](N)C[C@H](N)[C@H]2O[C@H]2O[C@H]([C@@H](C)O)[C@@H](O)[C@H](O)[C@H]2N)[C@H](O)[C@@H]1O

References

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 Jan 16. doi: 10.1002/cpdd.647. [Epub ahead of print] PubMed PMID: 30650260.