Title:
CATIONIC LIPID
Document Type and Number:
WIPO Patent Application WO/2013/065825
Kind Code:
A1
Abstract:
Provided is a cationic lipid expressed in the following formula (I), which facilitates introduction of a nucleic acid into a cell, a composition containing the cationic lipid and the nucleic acid, a method for introducing the nucleic acid into the cell by using the composition containing the cationic lipid and the nucleic acid, and the like (In the formula, R1 and R2 are the same alkenyl or different alkenyls, X1 and X2 are hydrogen atoms or together form a single bond or alkylene, X3 is not present or is alkyl and the like, Y is not present or is an anion, a and b are the same or different between 0 and 3, L3 is a single bond and the like, R3 is alkyl and the like, and L1 and L2 are -O-, -CO-O-, or -O-CO-).
Inventors:
KUBOYAMA TAKESHI (JP)
ERA TOMOHIRO (JP)
NAOI TOMOYUKI (JP)
YAGI NOBUHIRO (JP)
ERA TOMOHIRO (JP)
NAOI TOMOYUKI (JP)
YAGI NOBUHIRO (JP)
Application Number:
PCT/JP2012/078485
Publication Date:
May 10, 2013
Filing Date:
November 02, 2012
Export Citation:
Assignee:
KYOWA HAKKO KIRIN CO LTD (JP)
International Classes:
C07C217/08; A61K31/231; A61K31/24; A61K31/40; A61P29/00; A61P35/00; C07C219/08; C07C237/06; C07C271/20; C07D207/08; C07D207/12; C07D491/056; C12N15/09
Domestic Patent References:
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| WO2000040273A2 | 2000-07-13 | |||
| WO2000030444A1 | 2000-06-02 | |||
| WO2011136369A1 | 2011-11-03 | |||
| WO2011136368A1 | 2011-11-03 | |||
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| WO1997019675A2 | 1997-06-05 | |||
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Other References:
KATHRYN A WHITEHEAD ET AL.: "Synergistic Silencing: Combinations of Lipid-like Materials for Efficacious siRNA Delivery", MOLECULAR THERAPY, vol. 19, no. 9, September 2011 (2011-09-01), pages 1688 - 1694, XP055067340
MUKTHAVARAM RAJESH ET AL.: "Dramatic Influence of the Orientation of Linker between Hydrophilic and Hydrophobic Lipid Moiety in Liposomal Gene Delivery", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 129, no. 37, September 2007 (2007-09-01), pages 11408 - 11420, XP055067341
GOLLAPUDI VENKATA SRILAKSHMIET ET AL.: "Anchor- dependent lipofection with non-glycerol based cytofections containing single 2-hydroxyethyl head groups", BIOCHIMICA ET BIOPHYSICA ACTA, BIOMENBRANES, vol. 1559, no. 2, 2002, pages 87 - 95, XP004341284
I. FLECHSLER ET AL.: "COMPARISON OF ANTISENSE VECTORS AND ANTISENSE OLIGONUCLEOTIDES DELIVERED BY MEANS OF THE NEW CATIONIC LIPIDS UNIFECTIN AND MAXIFECTIN", ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY, vol. 451, 1998, pages 469 - 472, XP009048306
SANTANU BHATTACHARYA ET AL.: "The effects of cholesterol inclusion on the vesicular membranes of cationic lipids", BIOCHIMICA ET BIOPHYSICA ACTA, vol. 1283, no. 1, 1996, pages 21 - 30, XP055067345
IGOR SOLODIN ET AL.: "Synthesis of Amphiphilic Derivatives of N-Methyldiethanolamine. Cationic Lipids 5", SYNLETT, no. 7, July 1996 (1996-07-01), pages 620, XP002035804
IGOR SOLODIN ET AL.: "Synthesis of Novel Cationic Lipids with a Guanidine Group. Cationic Lipids 3", SYNLETT, 1996, pages 617 - 618, XP055151450
T.W. GREENE: "Protective Groups in Organic Synthesis, Third Edition,", 1999, JOHN WILEY & SONS INC.
CHEMICAL & PHARMACEUTICAL BULLETIN, vol. 39, 1991, pages 2219
"Jikken Kagaku Kouza , 5th Ed.,", vol. 13, 2005, MARUZEN, article "Synthesis of Organic Compounds I", pages: 374
"Jikken Kagaku Kouza , 5th Ed.,", 2005, MARUZEN, article "Stynthesis of Organic Compounds IV", pages: 1
"jikken Kagaku Kouza , 5th Ed.,", vol. 16, 2005, MARUZEN, article "Synthesis of Organic Compounds IV", pages: 1
"Jikken Kagaku Kouza , 5th Ed.,", vol. 14, 2005, MARUZEN, article "Synthesis of Organic Compounds II", pages: 1
"Jikken Kagaku Kouza , 5th Ed.,", vol. 15, 2005, MARUZEN, article "Synthesis of Organic Compounds III", pages: 1
"Jikken Kagaku Kouza , 5th Ed.,", vol. 15, 2005, MARUZEN, article "Synthesis of Organic Compounds III", pages: 153
"Jikken Kagaku Kouza , 5th Ed.,", vol. 13, 2005, MARUZEN, article "Synthesis of Organic Compounds 1", pages: 374
"Jikken Kagaku Kouza , 5th Ed.,", vol. 16, 2005, MARUZEN, article "Synthesis of Organic Compounds IV", pages: 1
JOURNAL OF AMERICAN CHEMICAL SOCIETY, vol. 103, 1981, pages 4194 - 4199
"Jikken Kagaku Kouza , 5th Ed.,", vol. 17, 2005, MARUZON, article "Synthesis of Organic Compounds V", pages: 186
R.C. LAROCK: "Comprehensive Organic Transformations, 2nd edition,", 1999, VCH VERLAGSGESELLSCHAFT MBH
J. . CHEM. SOC., vol. 124, no. 10, 2002, pages 2092
LIPIDS, vol. 9, no. 11, 1974, pages 913
ACC. CHEM. RES., vol. 32, 1999, pages 624
J. AM. CHEM. SOC., vol. 123, 2001, pages 4653
J. AM. CHEM. SOC., vol. 122, 2000, pages 6900
NUCLEIC ACID RESEARCH, vol. 32, 2004, pages EL75
BANGHAM ET AL., MOL. BIOL., vol. 13, 1965, pages 238 - 252
J. CELL. BIOL., vol. 66, 1975, pages 621 - 634
FEBS LETT., vol. 99, 1979, pages 210 - 214
ARCH. BIOCHEM. BJOPHYS., vol. 212, 1981, pages 186 - 194
PROC. NATL. ACAD SCI. USA, vol. 75, 1978, pages 4194 - 4198
BIOCHIMICA ET BIOPHYSICA ACTA, vol. 1510, 2001, pages 152 - 166
D. D. LASIC; F. MARTIN: "Stealth Liposomes", 1995, CRC PRESS INC., pages: 93 - 102
R. H. MULLER, S. BENITA AND B. BOHM: "Emulsion and Nanosuspensions for the Formulation of Poorly Soluble Drugs", 1998, SCIENTIFIC PUBLISHERS, pages: 267 - 294
See also references of EP 2774912A4
MUKTHAVARAM RAJESH ET AL.: "Dramatic Influence of the Orientation of Linker between Hydrophilic and Hydrophobic Lipid Moiety in Liposomal Gene Delivery", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 129, no. 37, September 2007 (2007-09-01), pages 11408 - 11420, XP055067341
GOLLAPUDI VENKATA SRILAKSHMIET ET AL.: "Anchor- dependent lipofection with non-glycerol based cytofections containing single 2-hydroxyethyl head groups", BIOCHIMICA ET BIOPHYSICA ACTA, BIOMENBRANES, vol. 1559, no. 2, 2002, pages 87 - 95, XP004341284
I. FLECHSLER ET AL.: "COMPARISON OF ANTISENSE VECTORS AND ANTISENSE OLIGONUCLEOTIDES DELIVERED BY MEANS OF THE NEW CATIONIC LIPIDS UNIFECTIN AND MAXIFECTIN", ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY, vol. 451, 1998, pages 469 - 472, XP009048306
SANTANU BHATTACHARYA ET AL.: "The effects of cholesterol inclusion on the vesicular membranes of cationic lipids", BIOCHIMICA ET BIOPHYSICA ACTA, vol. 1283, no. 1, 1996, pages 21 - 30, XP055067345
IGOR SOLODIN ET AL.: "Synthesis of Amphiphilic Derivatives of N-Methyldiethanolamine. Cationic Lipids 5", SYNLETT, no. 7, July 1996 (1996-07-01), pages 620, XP002035804
IGOR SOLODIN ET AL.: "Synthesis of Novel Cationic Lipids with a Guanidine Group. Cationic Lipids 3", SYNLETT, 1996, pages 617 - 618, XP055151450
T.W. GREENE: "Protective Groups in Organic Synthesis, Third Edition,", 1999, JOHN WILEY & SONS INC.
CHEMICAL & PHARMACEUTICAL BULLETIN, vol. 39, 1991, pages 2219
"Jikken Kagaku Kouza , 5th Ed.,", vol. 13, 2005, MARUZEN, article "Synthesis of Organic Compounds I", pages: 374
"Jikken Kagaku Kouza , 5th Ed.,", 2005, MARUZEN, article "Stynthesis of Organic Compounds IV", pages: 1
"jikken Kagaku Kouza , 5th Ed.,", vol. 16, 2005, MARUZEN, article "Synthesis of Organic Compounds IV", pages: 1
"Jikken Kagaku Kouza , 5th Ed.,", vol. 14, 2005, MARUZEN, article "Synthesis of Organic Compounds II", pages: 1
"Jikken Kagaku Kouza , 5th Ed.,", vol. 15, 2005, MARUZEN, article "Synthesis of Organic Compounds III", pages: 1
"Jikken Kagaku Kouza , 5th Ed.,", vol. 15, 2005, MARUZEN, article "Synthesis of Organic Compounds III", pages: 153
"Jikken Kagaku Kouza , 5th Ed.,", vol. 13, 2005, MARUZEN, article "Synthesis of Organic Compounds 1", pages: 374
"Jikken Kagaku Kouza , 5th Ed.,", vol. 16, 2005, MARUZEN, article "Synthesis of Organic Compounds IV", pages: 1
JOURNAL OF AMERICAN CHEMICAL SOCIETY, vol. 103, 1981, pages 4194 - 4199
"Jikken Kagaku Kouza , 5th Ed.,", vol. 17, 2005, MARUZON, article "Synthesis of Organic Compounds V", pages: 186
R.C. LAROCK: "Comprehensive Organic Transformations, 2nd edition,", 1999, VCH VERLAGSGESELLSCHAFT MBH
J. . CHEM. SOC., vol. 124, no. 10, 2002, pages 2092
LIPIDS, vol. 9, no. 11, 1974, pages 913
ACC. CHEM. RES., vol. 32, 1999, pages 624
J. AM. CHEM. SOC., vol. 123, 2001, pages 4653
J. AM. CHEM. SOC., vol. 122, 2000, pages 6900
NUCLEIC ACID RESEARCH, vol. 32, 2004, pages EL75
BANGHAM ET AL., MOL. BIOL., vol. 13, 1965, pages 238 - 252
J. CELL. BIOL., vol. 66, 1975, pages 621 - 634
FEBS LETT., vol. 99, 1979, pages 210 - 214
ARCH. BIOCHEM. BJOPHYS., vol. 212, 1981, pages 186 - 194
PROC. NATL. ACAD SCI. USA, vol. 75, 1978, pages 4194 - 4198
BIOCHIMICA ET BIOPHYSICA ACTA, vol. 1510, 2001, pages 152 - 166
D. D. LASIC; F. MARTIN: "Stealth Liposomes", 1995, CRC PRESS INC., pages: 93 - 102
R. H. MULLER, S. BENITA AND B. BOHM: "Emulsion and Nanosuspensions for the Formulation of Poorly Soluble Drugs", 1998, SCIENTIFIC PUBLISHERS, pages: 267 - 294
See also references of EP 2774912A4
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