VL-422 

VL-422 is an ionizable cationic lipid. VL-422 delivers CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA to enable in vitro and in vivo gene editing. LNPs containing VL-422 loaded with Cas9 mRNA and sgRNA targeting the ANGPTL3 gene induce the deletion of premature stop codons within the ANGPTL3 gene in the liver of cynomolgus monkeys. Loss-of-function of ANGPTL3 leads to decreased levels of LDL, HDL and cholesterol in plasma. The VL-422 delivery system can be used for the research of gene editing strategies targeting lipid metabolism diseases^[1].

Ionizable cationic lipids are essentially "pH-responsive molecular switches". They exist in a neutral state in the blood to ensure delivery safety; after entering cells, they can be activated in an acidic environment to achieve membrane disruption and nucleic acid release; after completing the delivery task, they can also be rapidly degraded, while simultaneously addressing the four core challenges in nucleic acid drug delivery, namely nucleic acid stability, delivery efficiency, tissue targeting, and biosafety^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

1052.59

C₆₂H₁₁₇NO₁₁

2765519-47-7

Liquid

Colorless to light yellow

O=C(OCC(COC(CCCCCCC(OC(CCCCCCCC)CCCCCCCC)=O)=O)COC(CCCCCCC(OC(CCCCCCCC)CCCCCCCC)=O)=O)OCCCN(CC)CC

Room temperature in continental US; may vary elsewhere.

Solution, -20°C, 2 years

• [1]. Kallanthottathil G. Rajeev, et al. Lipid formulations for gene editing. WO2022060871A1.

  [2]. Atmuri NDP, et al. Design of cationic ionizable lipids for the delivery of therapeutic nucleic acids. Mol Ther Methods Clin Dev. 2025 Sep 2;33(4):101585.  [Content Brief]