ELX-02 disulfate

ELX-02 disulfate (NB-124 disulfate) is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). ELX-02 disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.

Product information

CAS Number: 2244622-33-9

Molecular Weight: 678.68

Formula: C19H42N4O18S2

Chemical Name: (2R,3S,4R,5R,6S)-5-amino-6-{[(1R,2R,3S,4R,6S)-4,6-diamino-2-{[(2S,3R,4S,5R)-5-[(1S)-1-aminoethyl]-3,4-dihydroxyoxolan-2-yl]oxy}-3-hydroxycyclohexyl]oxy}-2-[(1R)-1-hydroxyethyl]oxane-3,4-diol; bis(sulfuric acid)

Smiles: C[C@H](N)[C@H]1O[C@@H](O[C@H]2[C@H](O[C@H]3O[C@@H]([C@@H](O)[C@H](O)[C@H]3N)[C@@H](C)O)[C@@H](N)C[C@@H](N)[C@@H]2O)[C@H](O)[C@@H]1O.OS(O)(=O)=O.OS(O)(=O)=O

InChiKey: WYGVCSNIMZZOIT-BPQPGTHUSA-N

InChi: InChI=1S/C19H38N4O10.2H2O4S/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;2*1-5(2,3)4/h4-19,24-29H,3,20-23H2,1-2H3;2*(H2,1,2,3,4)/t4-,5+,6+,7-,8+,9-,10+,11-,12-,13+,14+,15+,16+,17+,18+,19-;;/m0../s1

Technical Data

Appearance: Solid Power

Purity: ≥98% (or refer to the Certificate of Analysis)

Solubility: DMSO : < 1 mg/mL (insoluble or slightly soluble). H2O : 100 mg/mL (147.34 mM; Need ultrasonic).

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis

Storage Condition: Dry, dark and -20 ^oC for 1 year or refer to the Certificate of Analysis.

Shelf Life: ≥12 months if stored properly.

Stock Solution Storage: 0 - 4 ^oC for 1 month or refer to the Certificate of Analysis.

Drug Formulation: To be determined

HS Tariff Code: 382200

How to use

In Vitro:

ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.

In Vivo:

ELX-02 (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference. In plasma ELX-02 is rapidly absorbed with a Tmax of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). ELX-02 is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h. In a CtnsY226X nonsense mutant mouse, subcutaneous ELX-02 accumulates in kidney tissue without overt renal toxicity and that ELX-02 (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo.

Products are for research use only. Not for human use.