C - Chemistry – Metallurgy – 07 – D
Patent
C - Chemistry, Metallurgy
07
D
C07D 317/28 (2006.01) A61K 9/127 (2006.01) A61K 31/713 (2006.01) A61K 39/00 (2006.01) A61K 47/22 (2006.01) A61P 37/00 (2006.01) C07D 319/06 (2006.01)
Patent
CA 2740000
The present invention provides superior 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 proteins 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.
L'invention concerne des compositions et des procédés de qualité supérieure pour l'administration d'agents thérapeutiques à des cellules. En particulier, ceux-ci comprennent de nouveaux lipides et de nouvelles particules d'acide nucléique-lipide qui fournissent une encapsulation efficace des acides nucléiques, ainsi quune administration efficace de l'acide nucléique encapsulé à des cellules in vivo. Les compositions de la présente invention sont extrêmement puissantes, permettant ainsi d'inactiver les protéines cibles spécifiques à des doses relativement faibles. De plus, les compositions et procédés de la présente invention sont moins toxiques et fournissent un meilleur indice thérapeutique en comparaison des compositions et des procédés précédemment connus dans létat de la technique.
Chen Jianxin
Ciufolini Marco A.
Cullis Pieter R.
Hope Michael J.
Madden Thomas D.
Gowling Lafleur Henderson Llp
Tekmira Pharmaceuticals Corporation
The University Of British Columbia
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