Title:
COMPOUNDS FOR TARGETING DRUG DELIVERY AND ENHANCING SIRNA ACTIVITY
Document Type and Number:
WIPO Patent Application WO/2012/170952
Kind Code:
A3
Abstract:
Here described are compounds of formula I: wherein R1 and R2 is independently selected from a group consisting of C10 to C18 alkyl, C12 to C18 alkenyl, and oleyl group; wherein R3 and R4 are independently selected from a group consisting of C1 to C6 alkyl, and C2 to C6 alkanol; wherein X is selected from a group consisting of -CH2-, -S-, and -O- or absent; wherein Y is selected from -(CH2)n, -S(CH2)n, -O(CH2)n-, thiophene, -SO2(CH2)n-, and ester, wherein n = 1 -4; wherein a = 1 -4; wherein b=l -4; wherein c=l-4; and wherein Z is a counterion; and compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including one or both of these compounds which are useful for the delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.
Inventors:
NIITSU YOSHIRO (JP)
PAYNE JOSEPH E (US)
HOU ZHENG (US)
GAUDETTE JOHN A (US)
NAGARAJAN SRIDHAR C (US)
KNOPOV VICTOR (US)
WITTE RICHARD P (US)
AHMADIAN MOHAMMAD (US)
PERELMAN LOREN A (US)
TANAKA YASUNOBU (JP)
AKOPIAN VIOLETTA (US)
KARMALI PRIYA (US)
PAYNE JOSEPH E (US)
HOU ZHENG (US)
GAUDETTE JOHN A (US)
NAGARAJAN SRIDHAR C (US)
KNOPOV VICTOR (US)
WITTE RICHARD P (US)
AHMADIAN MOHAMMAD (US)
PERELMAN LOREN A (US)
TANAKA YASUNOBU (JP)
AKOPIAN VIOLETTA (US)
KARMALI PRIYA (US)
Application Number:
PCT/US2012/041753
Publication Date:
April 11, 2013
Filing Date:
June 08, 2012
Export Citation:
Assignee:
NITTO DENKO CORP (JP)
NIITSU YOSHIRO (JP)
PAYNE JOSEPH E (US)
HOU ZHENG (US)
GAUDETTE JOHN A (US)
NAGARAJAN SRIDHAR C (US)
KNOPOV VICTOR (US)
WITTE RICHARD P (US)
AHMADIAN MOHAMMAD (US)
PERELMAN LOREN A (US)
TANAKA YASUNOBU (JP)
AKOPIAN VIOLETTA (US)
KARMALI PRIYA (US)
NIITSU YOSHIRO (JP)
PAYNE JOSEPH E (US)
HOU ZHENG (US)
GAUDETTE JOHN A (US)
NAGARAJAN SRIDHAR C (US)
KNOPOV VICTOR (US)
WITTE RICHARD P (US)
AHMADIAN MOHAMMAD (US)
PERELMAN LOREN A (US)
TANAKA YASUNOBU (JP)
AKOPIAN VIOLETTA (US)
KARMALI PRIYA (US)
International Classes:
C07C237/08; A61K31/16; A61P1/16; C07C237/22; C07C323/60; C07C333/04
Domestic Patent References:
| WO2010061880A1 | 2010-06-03 |
Other References:
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; HORIMOTO, HIKARI ET AL: "Liquid crystal compositions", XP002684897, retrieved from STN Database accession no. 1987:11469
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; HORIMOTO, HIKARI ET AL: "Liquid crystal compositions", XP002684898, retrieved from STN Database accession no. 1987:59428
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; TAGAMI, TATSUAKI ET AL: "Effect of siRNA in PEG-coated siRNA -lipoplex on anti-PEG IgM production", XP002684899, retrieved from STN Database accession no. 2009:776119
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 8 April 2011 (2011-04-08), HADA, MAGDOLNA ET AL: "The first synthesis of PEG-carotenoid conjugates", XP002690323, retrieved from STN Database accession no. 2011:648518
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 2003, BENTLEY, MICHAEL DAVID ET AL: "Water-soluble polymer conjugates of retinoic acid", XP002690324, retrieved from STN Database accession no. 2003:356296
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 1999, SCHREIBER, JORG ET AL: "Disperse multiphase systems linked by covalent bonds into a network", XP002690325, retrieved from STN Database accession no. 1999:12437
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 1998, DIEC, KHIET HIEN ET AL: "Cosmetic or dermatological crosslinked microemulsions", XP002690326, retrieved from STN Database accession no. 1998:239092
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 1998, SCHREIBER, JOERG ET AL: "Crosslinked structures with double lipid membranes or based on peptides", XP002690327, retrieved from STN Database accession no. 1998:208404
MOON ET AL: "Regulation of adipocyte differentiation by PEGylated all-trans retinoic acid: reduced cytotoxicity and attenuated lipid accumulation", JOURNAL OF NUTRITIONAL BIOCHEMISTRY, BUTTERWORTH PUBLISHERS, STONEHAM, GB, vol. 18, no. 5, 19 April 2007 (2007-04-19), pages 322 - 331, XP022035106, ISSN: 0955-2863, DOI: 10.1016/J.JNUTBIO.2006.06.004
LI JING ET AL: "Synthesis of polyethylene glycol (PEG) derivatives and PEGylated-peptide biopolymer conjugates", BIOMACROMOLECULES, AMERICAN CHEMICAL SOCIETY; US, vol. 4, no. 4, 17 May 2003 (2003-05-17), pages 1055 - 1067, XP002328259, ISSN: 1525-7797, DOI: 10.1021/BM034069L
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; NIITSU, YOSHIRO ET AL: "Retinoid-liposomes for enhancing modulation of human hsp47 expression for treatment of fibrosis", XP002690328, retrieved from STN Database accession no. 2012:1819002
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; HORIMOTO, HIKARI ET AL: "Liquid crystal compositions", XP002684898, retrieved from STN Database accession no. 1987:59428
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; TAGAMI, TATSUAKI ET AL: "Effect of siRNA in PEG-coated siRNA -lipoplex on anti-PEG IgM production", XP002684899, retrieved from STN Database accession no. 2009:776119
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 8 April 2011 (2011-04-08), HADA, MAGDOLNA ET AL: "The first synthesis of PEG-carotenoid conjugates", XP002690323, retrieved from STN Database accession no. 2011:648518
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 2003, BENTLEY, MICHAEL DAVID ET AL: "Water-soluble polymer conjugates of retinoic acid", XP002690324, retrieved from STN Database accession no. 2003:356296
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 1999, SCHREIBER, JORG ET AL: "Disperse multiphase systems linked by covalent bonds into a network", XP002690325, retrieved from STN Database accession no. 1999:12437
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 1998, DIEC, KHIET HIEN ET AL: "Cosmetic or dermatological crosslinked microemulsions", XP002690326, retrieved from STN Database accession no. 1998:239092
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; 1998, SCHREIBER, JOERG ET AL: "Crosslinked structures with double lipid membranes or based on peptides", XP002690327, retrieved from STN Database accession no. 1998:208404
MOON ET AL: "Regulation of adipocyte differentiation by PEGylated all-trans retinoic acid: reduced cytotoxicity and attenuated lipid accumulation", JOURNAL OF NUTRITIONAL BIOCHEMISTRY, BUTTERWORTH PUBLISHERS, STONEHAM, GB, vol. 18, no. 5, 19 April 2007 (2007-04-19), pages 322 - 331, XP022035106, ISSN: 0955-2863, DOI: 10.1016/J.JNUTBIO.2006.06.004
LI JING ET AL: "Synthesis of polyethylene glycol (PEG) derivatives and PEGylated-peptide biopolymer conjugates", BIOMACROMOLECULES, AMERICAN CHEMICAL SOCIETY; US, vol. 4, no. 4, 17 May 2003 (2003-05-17), pages 1055 - 1067, XP002328259, ISSN: 1525-7797, DOI: 10.1021/BM034069L
DATABASE CA [online] CHEMICAL ABSTRACTS SERVICE, COLUMBUS, OHIO, US; NIITSU, YOSHIRO ET AL: "Retinoid-liposomes for enhancing modulation of human hsp47 expression for treatment of fibrosis", XP002690328, retrieved from STN Database accession no. 2012:1819002
Attorney, Agent or Firm:
KNUDSEN, Peter, J. (2929 Arch StreetCira Centre, 12th Floo, Philadelphia PA, US)
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