OF-02

OF-02 is a liver-targeted ionizable cationic lipid that has been used in combination with other lipids in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA, prime editor guide RNA (pegRNA), and nicking guide RNA (ngRNA) in vitro and in vivo.¹ LNPs containing OF-02 localize to the liver after intravenous administration in mice. Intravenous administration of LNPs containing OF-02 and encapsulating human erythropoietin (EPO) mRNA increases serum EPO levels in mice. OF-02-containing LNPs encapsulating prime editing cargo optimized to correct the phenylalanine hydroxylase (PAH) variant associated with phenylketonuria (PKU), c.1222C>T, induce efficient prime editing and reduce serum levels of phenylalanine in R408W mice, which harbor the c.1222C>T variant.² LNPs containing OF-02 and encapsulating prime editing cargo optimized to install a +1 TTAC frameshift insertion at Pcsk9 have a higher prime editing efficiency than those containing DLin-MC3-DMA (Item No. 34364) or SM-102 (Item No. 33474).

WARNING This product is not for human or veterinary use.

1. Fenton, O.S., Kauffman, K.J., McClellan, R.L., et al. Bioinspired alkenyl amino alcohol ionizable lipid materials for highly potent in vivo mRNA delivery. Adv. Mater. 28(15), 2939-2943 (2016).

2. Jiang, A.Y., Cristian, A., Brooks, D.L., et al. Efficient prime editing in vivo and in vitro using lipid nanoparticles. Nat. Nanotechnol. (2026).