Improved compositions and methods for the delivery of...

C - Chemistry – Metallurgy – 07 – C

Patent

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Details

C07C 219/08 (2006.01) A61K 9/127 (2006.01) A61K 47/18 (2006.01) A61K 47/20 (2006.01) A61K 47/22 (2006.01) A61P 37/04 (2006.01) C07C 215/10 (2006.01) C07C 217/28 (2006.01) C07C 229/12 (2006.01) C07C 271/12 (2006.01) C07C 323/25 (2006.01) C07D 295/088 (2006.01) C07D 317/28 (2006.01) A61K 31/7088 (2006.01) A61K 39/00 (2006.01)

Patent

CA 2709875

The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.

La présente invention concerne des compositions et des procédés pour la délivrance d'agents thérapeutiques à des cellules. En particulier, ceux-ci comprennent de nouveaux lipides et des particules d'acide nucléique-lipide qui permettent une encapsulation efficace des acides nucléiques et une délivrance efficace de l'acide nucléique encapsulé aux cellules in vivo. Les compositions de la présente invention sont hautement puissantes, permettant de cette manière d'inactiver une protéine cible spécifique à des doses relativement basses. En outre, les compositions et les procédés de la présente invention sont moins toxiques et présentent un index thérapeutique supérieur comparativement à des compositions et des procédés précédemment connus dans ce domaine.

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