COMPOUNDS AND COMPOSITIONS FOR DELIVERY OF THERAPEUTIC AGENTS

The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel cationic lipid as well as additional lipids such as ionizable lipids, phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNP...

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Bibliographische Detailangaben
Hauptverfasser: LEE, Juneyoung, SEEPERSAUD, Mohindra, BENENATO, Kerry E
Format: Patent
Sprache:eng ; fre
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Zusammenfassung:The disclosure features novel lipids and compositions involving the same. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) include a novel cationic lipid as well as additional lipids such as ionizable lipids, phospholipids, structural lipids, and PEG lipids. Lipid nanoparticles (e.g., empty LNPs or loaded LNPs) further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression. La divulgation concerne de nouveaux lipides et des compositions les comprenant. Les nanoparticules lipidiques (par exemple, les LNP vides ou les LNP chargées) comprennent un nouveau lipide cationique ainsi que d'autres lipides tels que des lipides ionisables, des phospholipides, des lipides structurels et des lipides PEG. Des nanoparticules lipidiques (par exemple, les LNP vides ou les LNP chargées) comprenant en outre des agents thérapeutiques et/ou prophylactiques tels que de l'ARN sont utiles dans l'administration d'agents thérapeutiques et/ou prophylactiques à des cellules ou des organes de mammifère pour, par exemple, réguler l'expression polypeptidique, protéique ou génique.