Lipid encapsulated interfering rna

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/88 (2006.01) A61K 9/127 (2006.01) A61K 31/7105 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2569664

The present invention is directed to lipid-based formulations for delivering, e.g., introducing, nucleic acid-lipid particles to a cell, and assays for optimizing the delivery efficiency of such lipid-based formulations. The nucleic acid-lipid particles comprise an interference RNA molecule, a cationic lipid with alkyl side chains from about 10 to about 20 carbon atoms having more than a single site of unsaturation, a non~cationic lipid and a conjugated lipid that inhibits aggregation of the particle such as a polyethyleneglycol (PEG)-lipid conjugate or a polyamide (ATTA)-conjugate).

La présente invention concerne des formulations à base de lipides utiles pour apporter et par exemple pour introduire dans une cellule, des particules de lipide-acide nucléique et des dosages permettant d'optimiser l'efficacité de l'apport de telles formulations à base de lipides. Les particules de lipide-acide nucléique comprennent une molécule d'ARN interférant, un lipide cationique comportant des chaînes latérales alkyle constituées d'environ 10 à environ 20 atomes de carbone et présentant plus d'un seul site d'insaturation, un lipide non cationique et un lipide conjugué qui inhibe l'agrégation de la particule comme un conjugué de polyéthylèneglycol (PEG)-lipide ou un conjugué de polyamide (ATTA).

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