JP2012509258A5 - - Google Patents

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Publication number

JP2012509258A5

JP2012509258A5 JP2011536348A JP2011536348A JP2012509258A5 JP 2012509258 A5 JP2012509258 A5 JP 2012509258A5 JP 2011536348 A JP2011536348 A JP 2011536348A JP 2011536348 A JP2011536348 A JP 2011536348A JP 2012509258 A5 JP2012509258 A5 JP 2012509258A5

Authority

JP

Japan

Prior art keywords

independently

substituted

alkyl

peg

cationic lipid

Prior art date

2009-07-31

Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)

Withdrawn

Links

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• 239000002105 nanoparticle Substances 0.000 claims 24
• -1 amino, substituted amino Chemical group 0.000 claims 21
• HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 20
• 239000000203 mixture Substances 0.000 claims 17
• 125000000217 alkyl group Chemical group 0.000 claims 16
• 229910052739 hydrogen Inorganic materials 0.000 claims 12
• 235000012000 cholesterol Nutrition 0.000 claims 10
• 108091034117 Oligonucleotide Proteins 0.000 claims 9
• 229910052799 carbon Inorganic materials 0.000 claims 9
• 150000002632 lipids Chemical class 0.000 claims 9
• 229920001223 polyethylene glycol Polymers 0.000 claims 9
• 229910052760 oxygen Inorganic materials 0.000 claims 8
• 229910052717 sulfur Inorganic materials 0.000 claims 7
• 125000006716 (C1-C6) heteroalkyl group Chemical group 0.000 claims 6
• JZNWSCPGTDBMEW-UHFFFAOYSA-N Glycerophosphorylethanolamin Natural products NCCOP(O)(=O)OCC(O)CO JZNWSCPGTDBMEW-UHFFFAOYSA-N 0.000 claims 6
• 239000001257 hydrogen Substances 0.000 claims 6
• 125000004435 hydrogen atom Chemical class [H]* 0.000 claims 6
• 150000008104 phosphatidylethanolamines Chemical class 0.000 claims 6
• 108020004707 nucleic acids Proteins 0.000 claims 5
• 102000039446 nucleic acids Human genes 0.000 claims 5
• 150000007523 nucleic acids Chemical class 0.000 claims 5
• 229920000362 Polyethylene-block-poly(ethylene glycol) Polymers 0.000 claims 4
• 230000000692 anti-sense effect Effects 0.000 claims 4
• 125000003118 aryl group Chemical group 0.000 claims 4
• 125000002091 cationic group Chemical group 0.000 claims 4
• 229940106189 ceramide Drugs 0.000 claims 4
• 239000008194 pharmaceutical composition Substances 0.000 claims 4
• 125000000547 substituted alkyl group Chemical group 0.000 claims 4
• 125000003107 substituted aryl group Chemical group 0.000 claims 4
• 125000005346 substituted cycloalkyl group Chemical group 0.000 claims 4
• 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims 3
• 125000003710 aryl alkyl group Chemical group 0.000 claims 3
• 230000000799 fusogenic effect Effects 0.000 claims 3
• 125000004405 heteroalkoxy group Chemical group 0.000 claims 3
• 125000000951 phenoxy group Chemical group [H]C1=C([H])C([H])=C(O*)C([H])=C1[H] 0.000 claims 3
• 125000006850 spacer group Chemical group 0.000 claims 3
• 108091023037 Aptamer Proteins 0.000 claims 2
• YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 claims 2
• 108700011259 MicroRNAs Proteins 0.000 claims 2
• CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 claims 2
• 206010028980 Neoplasm Diseases 0.000 claims 2
• 108091093037 Peptide nucleic acid Proteins 0.000 claims 2
• 108020004459 Small interfering RNA Proteins 0.000 claims 2
• 125000003545 alkoxy group Chemical group 0.000 claims 2
• 230000001588 bifunctional effect Effects 0.000 claims 2
• 201000011510 cancer Diseases 0.000 claims 2
• ZVEQCJWYRWKARO-UHFFFAOYSA-N ceramide Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(CO)C(O)C=CCCC=C(C)CCCCCCCCC ZVEQCJWYRWKARO-UHFFFAOYSA-N 0.000 claims 2
• 125000001072 heteroaryl group Chemical group 0.000 claims 2
• 239000002679 microRNA Substances 0.000 claims 2
• VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 claims 2
• 229910052698 phosphorus Inorganic materials 0.000 claims 2
• 239000004055 small Interfering RNA Substances 0.000 claims 2
• 125000004191 (C1-C6) alkoxy group Chemical group 0.000 claims 1
• NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
• FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
• 102100021663 Baculoviral IAP repeat-containing protein 5 Human genes 0.000 claims 1
• 108060000903 Beta-catenin Proteins 0.000 claims 1
• 102000015735 Beta-catenin Human genes 0.000 claims 1
• 108090000994 Catalytic RNA Proteins 0.000 claims 1
• 102000053642 Catalytic RNA Human genes 0.000 claims 1
• 108020004414 DNA Proteins 0.000 claims 1
• 101001046870 Homo sapiens Hypoxia-inducible factor 1-alpha Proteins 0.000 claims 1
• 102100022875 Hypoxia-inducible factor 1-alpha Human genes 0.000 claims 1
• 241000124008 Mammalia Species 0.000 claims 1
• 108091030071 RNAI Proteins 0.000 claims 1
• 108091028664 Ribonucleotide Proteins 0.000 claims 1
• 108091027076 Spiegelmer Proteins 0.000 claims 1
• 108010002687 Survivin Proteins 0.000 claims 1
• 125000003342 alkenyl group Chemical group 0.000 claims 1
• 125000000304 alkynyl group Chemical group 0.000 claims 1
• 239000002246 antineoplastic agent Substances 0.000 claims 1
• 230000005907 cancer growth Effects 0.000 claims 1
• 150000001875 compounds Chemical class 0.000 claims 1
• 125000000753 cycloalkyl group Chemical group 0.000 claims 1
• 229940127089 cytotoxic agent Drugs 0.000 claims 1
• 230000002222 downregulating effect Effects 0.000 claims 1
• 230000014509 gene expression Effects 0.000 claims 1
• 230000009368 gene silencing by RNA Effects 0.000 claims 1
• 125000004404 heteroalkyl group Chemical group 0.000 claims 1
• 125000005842 heteroatom Chemical group 0.000 claims 1
• 210000004185 liver Anatomy 0.000 claims 1
• 230000001394 metastastic effect Effects 0.000 claims 1
• 206010061289 metastatic neoplasm Diseases 0.000 claims 1
• 239000002773 nucleotide Substances 0.000 claims 1
• 125000003729 nucleotide group Chemical group 0.000 claims 1
• WTJKGGKOPKCXLL-RRHRGVEJSA-N phosphatidylcholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCC=CCCCCCCCC WTJKGGKOPKCXLL-RRHRGVEJSA-N 0.000 claims 1
• 230000035755 proliferation Effects 0.000 claims 1
• 239000002336 ribonucleotide Substances 0.000 claims 1
• 125000002652 ribonucleotide group Chemical group 0.000 claims 1
• 108091092562 ribozyme Proteins 0.000 claims 1
• 229920006395 saturated elastomer Polymers 0.000 claims 1
• 125000005017 substituted alkenyl group Chemical group 0.000 claims 1
• 125000004426 substituted alkynyl group Chemical group 0.000 claims 1
• UWYSVYIRGIUDSC-UHFFFAOYSA-N CC(C)CCCC(C)C(CC=C)C(C)(CC1)CC2C1C(C)(CCC(C1)OC(COCCOCCNC(C(CCCCNC3NC3N)NC(N)=N)=O)=O)C1=CC2 Chemical compound CC(C)CCCC(C)C(CC=C)C(C)(CC1)CC2C1C(C)(CCC(C1)OC(COCCOCCNC(C(CCCCNC3NC3N)NC(N)=N)=O)=O)C1=CC2 UWYSVYIRGIUDSC-UHFFFAOYSA-N 0.000 description 1
• IRENVDDCYYVIBT-UHFFFAOYSA-N CC1C(N(C)CCC2)N2CCCC1 Chemical compound CC1C(N(C)CCC2)N2CCCC1 IRENVDDCYYVIBT-UHFFFAOYSA-N 0.000 description 1
• DWXIDAJNMISYJH-UHFFFAOYSA-N CCC(C1OC1N)N=C=N Chemical compound CCC(C1OC1N)N=C=N DWXIDAJNMISYJH-UHFFFAOYSA-N 0.000 description 1
• BWMDMTSNSXYYSP-UHFFFAOYSA-N CCCNC(N)=N Chemical compound CCCNC(N)=N BWMDMTSNSXYYSP-UHFFFAOYSA-N 0.000 description 1
• 0 CC[P@](NCCNC(N)=N)(N=CCC=[O+]CCC(CC(CCC1(C)C2CCC3(C)C(C)C(C)CCCC(C)C)CC1=CCC2C3C=C)=O)=*=CCNC(*)=N Chemical compound CC[P@](NCCNC(N)=N)(N=CCC=[O+]CCC(CC(CCC1(C)C2CCC3(C)C(C)C(C)CCCC(C)C)CC1=CCC2C3C=C)=O)=*=CCNC(*)=N 0.000 description 1
• APKFDBVGPJCXTC-UHFFFAOYSA-N CN1C(CCC2)N2CCC1 Chemical compound CN1C(CCC2)N2CCC1 APKFDBVGPJCXTC-UHFFFAOYSA-N 0.000 description 1