WARNING: This product is for research use only, not for human or veterinary use.
Hodoodo CAT#: H578000
CAS#: 1375073-96-3 (sulfate)
Description: Exaluren, also known as NB-124 and ELX 02 is a a synthetic aminoglycoside and a translational read-through inducer. ELX-02 suppresses translation termination at PTCs (i.e., readthrough) by promoting the insertion of an amino acid at the PTC and restoring expression of full-length CFTR protein. After treatment with ELX-02 and CFTR correctors, CFTR function rescued from the G550X allele in FRTs reached 20–40% of the wild-type level. Exaluren is being developed as a therapy for genetic diseases caused by nonsense mutations.
Hodoodo Cat#: H578000
Name: Exaluren sulfate
CAS#: 1375073-96-3 (sulfate)
Chemical Formula: C19H40N4O14S
Exact Mass: 580.23
Molecular Weight: 580.600
Elemental Analysis: C, 39.31; H, 6.94; N, 9.65; O, 38.58; S, 5.52
Related CAS #: 1375073-93-0 (free base) 1375073-96-3 (sulfate)
Synonym: NB-124; NB 124; NB124; NB-124; ELX 02; ELX-02; ELX 02; Exaluren sulfate;
IUPAC/Chemical Name: (2R,3S,4R,5R,6S)-5-amino-6-(((1R,2R,3S,4R,6S)-4,6-diamino-2-(((2S,3R,4S,5R)-5-((S)-1-aminoethyl)-3,4-dihydroxytetrahydrofuran-2-yl)oxy)-3-hydroxycyclohexyl)oxy)-2-((R)-1-hydroxyethyl)tetrahydro-2H-pyran-3,4-diol sulfate
InChi Key: BOIAUNVCOPGNOT-STOTURBNSA-N
InChi Code: 1S/C19H38N4O10.H2O4S/c1-4(20)14-12(28)13(29)19(30-14)33-17-9(25)6(21)3-7(22)16(17)32-18-8(23)10(26)11(27)15(31-18)5(2)24;1-5(2,3)4/h4-19,24-29H,3,20-23H2,1-2H3;(H2,1,2,3,4)/t4-,5+,6+,7-,8+,9-,10+,11-,12-,13+,14+,15+,16+,17+,18+,19-;/m0./s1
SMILES Code: 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]3O[C@H]([C@@H](C)O)[C@@H](O)[C@H](O)[C@H]3N)[C@H](O)[C@@H]1O.OS(=O)(=O)O
Appearance: Solid powder
Purity: >98% (or refer to the Certificate of Analysis)
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility: To be determined
Shelf Life: >2 years if stored properly
Drug Formulation: To be determined
Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code: 2934.99.9001
More Info:
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The following data is based on the product molecular weight 580.60 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
| Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
|---|---|---|---|
| 1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
| 5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
| 10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
| 50 mM | 0.02 mL | 0.12 mL | 0.23 mL |
| Formulation protocol: | |
| In vitro protocol: | |
| In vivo protocol: |
1. Bidou L, Bugaud O, Belakhov V, Baasov T, Namy O. Characterization of new-generation aminoglycoside promoting premature termination codon readthrough in cancer cells. RNA Biol. 2017 Mar 4;14(3):378-388. doi: 10.1080/15476286.2017.1285480. Epub 2017 Feb 1. PMID: 28145797; PMCID: PMC5367250.
2. Friesen WJ, Johnson B, Sierra J, Zhuo J, Vazirani P, Xue X, Tomizawa Y, Baiazitov R, Morrill C, Ren H, Babu S, Moon YC, Branstrom A, Mollin A, Hedrick J, Sheedy J, Elfring G, Weetall M, Colacino JM, Welch EM, Peltz SW. The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential. PLoS One. 2018 Oct 25;13(10):e0206158. doi: 10.1371/journal.pone.0206158. PMID: 30359426; PMCID: PMC6201930.
3. Xue X, Mutyam V, Tang L, Biswas S, Du M, Jackson LA, Dai Y, Belakhov V, Shalev M, Chen F, Schacht J, J Bridges R, Baasov T, Hong J, Bedwell DM, Rowe SM. Synthetic aminoglycosides efficiently suppress cystic fibrosis transmembrane conductance regulator nonsense mutations and are enhanced by ivacaftor. Am J Respir Cell Mol Biol. 2014 Apr;50(4):805-16. doi: 10.1165/rcmb.2013-0282OC. PMID: 24251786; PMCID: PMC4068923.
