792371 | LP-01
(9Z,12Z)-3-((4,4-bis(octyloxy)butanoyl)oxy)-2-((((3-(diethylamino)propoxy)carbonyl)oxy)methyl)propyloctadeca-9,12-dienoate
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LP-01
(9Z,12Z)-3-((4,4-bis(octyloxy)butanoyl)oxy)-2-((((3-(diethylamino)propoxy)carbonyl)oxy)methyl)propyloctadeca-9,12-dienoate
LP-01 is a synthetic ionizable lipid with a pKa value of 6.1 that is predominantly used in the formation of LNPs for mRNA delivery as well as in CRISPR-Cas9 gene therapies. LNPs using LP-01 are shown to result in accumulation primarily in the liver and to a much lesser extent in the spleen and kidneys.
In CRISPR/Cas9 mediated genome editing, LP01 has been used in mice to enable significant editing of the mouse transthyretin (Ttr) gene in the liver. This LNP formulation resulted in a reduction in serum proteins that persisted for nearly 12 months while also showing a high level of biodegradability.1,2
References:
1. Gautam, M., Jozic, A., Su, G.LN. et al. Lipid nanoparticles with PEG-variant surface modifications mediate genome editing in the mouse retina. Nat Commun 14, 6468 (2023). https://doi.org/10.1038/s41467...
2. Jonathan D. Finn, Amy Rhoden Smith, Mihir C. Patel, Lucinda Shaw, Madeleine R. Youniss, Jane van Heteren, Tanner Dirstine, Corey Ciullo, Reynald Lescarbeau, Jessica Seitzer, Ruchi R. Shah, Aalok Shah, Dandan Ling, Jacqueline Growe, Melissa Pink, Ellen Rohde, Kristy M. Wood, William E. Salomon, William F. Harrington, Christian Dombrowski, Walter R. Strapps, Yong Chang, David V. Morrissey, A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing, Cell Reports, Volume 22, Issue 9, 2018, Pages 2227-2235, ISSN 2211-1247, https://doi.org/10.1016/j.celr.... (https://www.sciencedirect.com/...)
CAS Registry Number is a Registered Trademark of the American Chemical Society
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