ATX-001 is an ionizable lipid, which can be used as a lipid component in lipid nanoparticle (LNP) formulations for mRNA delivery. Its primary function is to enhance the intracellular delivery of nucleic acids by facilitating endosomal escape. While ATX-001 is not a therapeutic molecule per se, its bioactivity is characterized by its physicochemical properties and performance in in vitro and in vivo delivery systems. In published studies, LNPs containing ATX-001 demonstrated efficient mRNA delivery, with ~80–90% transfection efficiency in hepatocytes and other cell types, and robust protein expression following systemic administration in animal models. In mouse models, ATX-001-containing LNPs achieved liver-targeted mRNA expression at doses as low as 0.1 mg/kg, with favorable safety profiles, including minimal liver enzyme elevation and low pro-inflammatory cytokine induction.
MedKoo Cat#: 465713
Name: ATX-001
CAS#: 1777792-33-2
Chemical Formula: C40H74N2O5S
Exact Mass: 694.5318
Molecular Weight: 695.10
Elemental Analysis: C, 69.12; H, 10.73; N, 4.03; O, 11.51; S, 4.61
| Solvent | mg/mL | mM | |
|---|---|---|---|
| Solubility | |||
| DMSO | 250.0 | 359.66 |
The following data is based on the product molecular weight 695.10 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 |
