ATX-012 is an ionizable cationic lipid for the assembly of mRNA -delivering lipid nanoparticles (LNPs). ATX-012 blends with helper lipids, cholesterol and PEG-lipid conjugates to form mRNA -containing nanocarriers, which support efficient cellular internalization and the generation of functional proteins including CFTR. ATX-012 constitutes the vital building block of LNPs applied in cystic fibrosis-related biological studies [1].
In Vivo, ATX-012 (intratracheal administration)-formulated LNPs deliver mRNA to mouse lung epithelial cells and drive protein expression throughout large and small airways[1]. ATX-012 (1 mg/kg; intranasal administration; once daily for two days)-based LNPs loaded with hCFTR mRNA restore functional chloride channel activity in CFTR knockout mice[1].
In Vitro, ATX-012 can form LNPs featuring high mRNA encapsulation efficiency (> 95%) and a small particle size ranging from 70 to 90 nm, and such LNPs achieve efficient mRNA delivery into lung epithelial cells in vitro and in vivo[1].
References:
[1]. Carlos G, et al. Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis. US20230159449A1. United States. 2022-11-03.
In Vivo, ATX-012 (intratracheal administration)-formulated LNPs deliver mRNA to mouse lung epithelial cells and drive protein expression throughout large and small airways[1]. ATX-012 (1 mg/kg; intranasal administration; once daily for two days)-based LNPs loaded with hCFTR mRNA restore functional chloride channel activity in CFTR knockout mice[1].
In Vitro, ATX-012 can form LNPs featuring high mRNA encapsulation efficiency (> 95%) and a small particle size ranging from 70 to 90 nm, and such LNPs achieve efficient mRNA delivery into lung epithelial cells in vitro and in vivo[1].
References:
[1]. Carlos G, et al. Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis. US20230159449A1. United States. 2022-11-03.