SITE SPECIFIC DELIVERY OF NUCLEIC ACIDS BY COMBINING TARGETING LIGANDS WITH ENDOSOMOLYTIC COMPONENTS

Japanese Patent JP2015187138

A

PROBLEM TO BE SOLVED: To provide compositions for site-specific delivery of nucleic acids in treatment of cancer, viral disease etc.SOLUTION: There is provided a modular composition containing a nucleic acid, an endosomolytic component, and a targeting ligand, as represented by the structure in the figure as a representative example. [E is the endosomolytic component; L is the targeting ligand; x are each independently 0 or 1; y are each independently an integer from 1 to 6; z are each independently an integer from 0 to 6; and A is a linking moiety having pyrrolidine and phosphate ester structure.]

MUTHIAH MANOHARAN
KALLANTHOTTATHIL G RAJEEV
DAVID BUTLER
MUTHUSAMY JAYARAMAN
NARAYANANNAIR K JAYAPRAKASH
MATSUDA SHIGEO

JP2015112240A

October 29, 2015

June 02, 2015

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ALNYLAM PHARMACEUTICALS INC

A61K31/7088; A61K31/7105; A61K31/7115; A61K31/712; A61K31/7125; A61K31/713; A61K39/395; A61K47/12; A61K47/22; A61K47/24; A61K47/26; A61K47/28; A61K47/32; A61K47/34; A61K47/36; A61K47/42; A61K47/48; A61K48/00; A61P1/04; A61P1/16; A61P3/10; A61P9/00; A61P9/10; A61P11/06; A61P19/02; A61P25/00; A61P25/04; A61P25/14; A61P25/16; A61P25/28; A61P29/00; A61P31/04; A61P31/06; A61P31/08; A61P31/10; A61P31/12; A61P31/14; A61P31/18; A61P31/20; A61P31/22; A61P33/00; A61P33/06; A61P35/00; A61P35/02; A61P37/06; A61P43/00; C12N15/11; C12N15/87

JP2008501319A

2008-01-24

WO2008022309A2

2008-02-21

Yanagita Seiji
Go Sakuma

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