JP2012526858A5 - - Google Patents
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- Publication number
- JP2012526858A5 JP2012526858A5 JP2012511923A JP2012511923A JP2012526858A5 JP 2012526858 A5 JP2012526858 A5 JP 2012526858A5 JP 2012511923 A JP2012511923 A JP 2012511923A JP 2012511923 A JP2012511923 A JP 2012511923A JP 2012526858 A5 JP2012526858 A5 JP 2012526858A5
- Authority
- JP
- Japan
- Prior art keywords
- carbamate
- polynucleotide
- cholesterol
- peg
- solution according
- Prior art date
- 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.)
- Granted
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- -1 cationic lipid Chemical class 0.000 claims 20
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol group Chemical group [C@@H]1(CC[C@H]2[C@@H]3CC=C4C[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)[C@H](C)CCCC(C)C HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 16
- 239000000203 mixture Substances 0.000 claims 15
- 239000000243 solution Substances 0.000 claims 9
- 229920001223 polyethylene glycol Polymers 0.000 claims 8
- 108091033319 polynucleotide Proteins 0.000 claims 8
- 102000040430 polynucleotide Human genes 0.000 claims 8
- 239000002157 polynucleotide Substances 0.000 claims 8
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims 7
- 235000012000 cholesterol Nutrition 0.000 claims 7
- 229930182558 Sterol Natural products 0.000 claims 6
- 235000003702 sterols Nutrition 0.000 claims 6
- OGQYPPBGSLZBEG-UHFFFAOYSA-N dimethyl(dioctadecyl)azanium Chemical compound CCCCCCCCCCCCCCCCCC[N+](C)(C)CCCCCCCCCCCCCCCCCC OGQYPPBGSLZBEG-UHFFFAOYSA-N 0.000 claims 5
- 150000002632 lipids Chemical class 0.000 claims 4
- 150000003904 phospholipids Chemical class 0.000 claims 4
- 239000002244 precipitate Substances 0.000 claims 4
- 229940063675 spermine Drugs 0.000 claims 4
- 150000003432 sterols Chemical class 0.000 claims 4
- OILXMJHPFNGGTO-UHFFFAOYSA-N (22E)-(24xi)-24-methylcholesta-5,22-dien-3beta-ol Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)C=CC(C)C(C)C)C1(C)CC2 OILXMJHPFNGGTO-UHFFFAOYSA-N 0.000 claims 3
- OQMZNAMGEHIHNN-UHFFFAOYSA-N 7-Dehydrostigmasterol Natural products C1C(O)CCC2(C)C(CCC3(C(C(C)C=CC(CC)C(C)C)CCC33)C)C3=CC=C21 OQMZNAMGEHIHNN-UHFFFAOYSA-N 0.000 claims 3
- JZNWSCPGTDBMEW-UHFFFAOYSA-N Glycerophosphorylethanolamin Natural products NCCOP(O)(=O)OCC(O)CO JZNWSCPGTDBMEW-UHFFFAOYSA-N 0.000 claims 3
- 125000000217 alkyl group Chemical group 0.000 claims 3
- LGJMUZUPVCAVPU-UHFFFAOYSA-N beta-Sitostanol Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(C)CCC(CC)C(C)C)C1(C)CC2 LGJMUZUPVCAVPU-UHFFFAOYSA-N 0.000 claims 3
- 238000009472 formulation Methods 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 239000003960 organic solvent Substances 0.000 claims 3
- 150000008104 phosphatidylethanolamines Chemical class 0.000 claims 3
- 239000007787 solid Substances 0.000 claims 3
- BQPPJGMMIYJVBR-UHFFFAOYSA-N (10S)-3c-Acetoxy-4.4.10r.13c.14t-pentamethyl-17c-((R)-1.5-dimethyl-hexen-(4)-yl)-(5tH)-Delta8-tetradecahydro-1H-cyclopenta[a]phenanthren Natural products CC12CCC(OC(C)=O)C(C)(C)C1CCC1=C2CCC2(C)C(C(CCC=C(C)C)C)CCC21C BQPPJGMMIYJVBR-UHFFFAOYSA-N 0.000 claims 2
- RQOCXCFLRBRBCS-UHFFFAOYSA-N (22E)-cholesta-5,7,22-trien-3beta-ol Natural products C1C(O)CCC2(C)C(CCC3(C(C(C)C=CCC(C)C)CCC33)C)C3=CC=C21 RQOCXCFLRBRBCS-UHFFFAOYSA-N 0.000 claims 2
- IOWMKBFJCNLRTC-XWXSNNQWSA-N (24S)-24-hydroxycholesterol 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)CC[C@H](O)C(C)C)[C@@]1(C)CC2 IOWMKBFJCNLRTC-XWXSNNQWSA-N 0.000 claims 2
- CHGIKSSZNBCNDW-UHFFFAOYSA-N (3beta,5alpha)-4,4-Dimethylcholesta-8,24-dien-3-ol Natural products CC12CCC(O)C(C)(C)C1CCC1=C2CCC2(C)C(C(CCC=C(C)C)C)CCC21 CHGIKSSZNBCNDW-UHFFFAOYSA-N 0.000 claims 2
- XYTLYKGXLMKYMV-UHFFFAOYSA-N 14alpha-methylzymosterol Natural products CC12CCC(O)CC1CCC1=C2CCC2(C)C(C(CCC=C(C)C)C)CCC21C XYTLYKGXLMKYMV-UHFFFAOYSA-N 0.000 claims 2
- LDGWQMRUWMSZIU-LQDDAWAPSA-M 2,3-bis[(z)-octadec-9-enoxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCCOCC(C[N+](C)(C)C)OCCCCCCCC\C=C/CCCCCCCC LDGWQMRUWMSZIU-LQDDAWAPSA-M 0.000 claims 2
- KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 claims 2
- MVDZEJXAUJRFGW-UHFFFAOYSA-N 24-Isopropyl-cholesterin Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)CCC(C(C)C)C(C)C)C1(C)CC2 MVDZEJXAUJRFGW-UHFFFAOYSA-N 0.000 claims 2
- MVDZEJXAUJRFGW-XKTYQPJFSA-N 24-isopropyl-cholest-5-en-3beta-ol 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)CCC(C(C)C)C(C)C)[C@@]1(C)CC2 MVDZEJXAUJRFGW-XKTYQPJFSA-N 0.000 claims 2
- IOWMKBFJCNLRTC-UHFFFAOYSA-N 24S-hydroxycholesterol Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)CCC(O)C(C)C)C1(C)CC2 IOWMKBFJCNLRTC-UHFFFAOYSA-N 0.000 claims 2
- FPTJELQXIUUCEY-UHFFFAOYSA-N 3beta-Hydroxy-lanostan Natural products C1CC2C(C)(C)C(O)CCC2(C)C2C1C1(C)CCC(C(C)CCCC(C)C)C1(C)CC2 FPTJELQXIUUCEY-UHFFFAOYSA-N 0.000 claims 2
- SGNBVLSWZMBQTH-FGAXOLDCSA-N Campesterol Natural products O[C@@H]1CC=2[C@@](C)([C@@H]3[C@H]([C@H]4[C@@](C)([C@H]([C@H](CC[C@H](C(C)C)C)C)CC4)CC3)CC=2)CC1 SGNBVLSWZMBQTH-FGAXOLDCSA-N 0.000 claims 2
- UCTLRSWJYQTBFZ-UHFFFAOYSA-N Dehydrocholesterol Natural products C1C(O)CCC2(C)C(CCC3(C(C(C)CCCC(C)C)CCC33)C)C3=CC=C21 UCTLRSWJYQTBFZ-UHFFFAOYSA-N 0.000 claims 2
- BDCFUHIWJODVNG-UHFFFAOYSA-N Desmosterol Natural products C1C=C2CC(O)C=CC2(C)C2C1C1CCC(C(C)CCC(CC)C(C)C)C1(C)CC2 BDCFUHIWJODVNG-UHFFFAOYSA-N 0.000 claims 2
- LPFIPZJIWTZLEY-UHFFFAOYSA-N Dinosterol Natural products C1CC2C(C)C(O)CCC2(C)C2C1C1CCC(C(C)C=C(C)C(C)C(C)C)C1(C)CC2 LPFIPZJIWTZLEY-UHFFFAOYSA-N 0.000 claims 2
- DNVPQKQSNYMLRS-NXVQYWJNSA-N Ergosterol Natural products CC(C)[C@@H](C)C=C[C@H](C)[C@H]1CC[C@H]2C3=CC=C4C[C@@H](O)CC[C@]4(C)[C@@H]3CC[C@]12C DNVPQKQSNYMLRS-NXVQYWJNSA-N 0.000 claims 2
- BKLIAINBCQPSOV-UHFFFAOYSA-N Gluanol Natural products CC(C)CC=CC(C)C1CCC2(C)C3=C(CCC12C)C4(C)CCC(O)C(C)(C)C4CC3 BKLIAINBCQPSOV-UHFFFAOYSA-N 0.000 claims 2
- YRPMZHRSQIFCLR-PVNJCSFGSA-N Gorgosterol Chemical compound CC(C)[C@@H](C)[C@@]1(C)C[C@@H]1[C@@H](C)[C@@H]1[C@@]2(C)CC[C@@H]3[C@@]4(C)CC[C@H](O)CC4=CC[C@H]3[C@@H]2CC1 YRPMZHRSQIFCLR-PVNJCSFGSA-N 0.000 claims 2
- BTEISVKTSQLKST-UHFFFAOYSA-N Haliclonasterol Natural products CC(C=CC(C)C(C)(C)C)C1CCC2C3=CC=C4CC(O)CCC4(C)C3CCC12C BTEISVKTSQLKST-UHFFFAOYSA-N 0.000 claims 2
- LOPKHWOTGJIQLC-UHFFFAOYSA-N Lanosterol Natural products CC(CCC=C(C)C)C1CCC2(C)C3=C(CCC12C)C4(C)CCC(C)(O)C(C)(C)C4CC3 LOPKHWOTGJIQLC-UHFFFAOYSA-N 0.000 claims 2
- 108700011259 MicroRNAs Proteins 0.000 claims 2
- CAHGCLMLTWQZNJ-UHFFFAOYSA-N Nerifoliol Natural products CC12CCC(O)C(C)(C)C1CCC1=C2CCC2(C)C(C(CCC=C(C)C)C)CCC21C CAHGCLMLTWQZNJ-UHFFFAOYSA-N 0.000 claims 2
- HIHOWBSBBDRPDW-PTHRTHQKSA-N [(3s,8s,9s,10r,13r,14s,17r)-10,13-dimethyl-17-[(2r)-6-methylheptan-2-yl]-2,3,4,7,8,9,11,12,14,15,16,17-dodecahydro-1h-cyclopenta[a]phenanthren-3-yl] n-[2-(dimethylamino)ethyl]carbamate Chemical compound C1C=C2C[C@@H](OC(=O)NCCN(C)C)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 HIHOWBSBBDRPDW-PTHRTHQKSA-N 0.000 claims 2
- 150000005215 alkyl ethers Chemical class 0.000 claims 2
- 239000000010 aprotic solvent Substances 0.000 claims 2
- 229940076810 beta sitosterol Drugs 0.000 claims 2
- NJKOMDUNNDKEAI-UHFFFAOYSA-N beta-sitosterol Natural products CCC(CCC(C)C1CCC2(C)C3CC=C4CC(O)CCC4C3CCC12C)C(C)C NJKOMDUNNDKEAI-UHFFFAOYSA-N 0.000 claims 2
- SGNBVLSWZMBQTH-PODYLUTMSA-N campesterol 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)CC[C@@H](C)C(C)C)[C@@]1(C)CC2 SGNBVLSWZMBQTH-PODYLUTMSA-N 0.000 claims 2
- 235000000431 campesterol Nutrition 0.000 claims 2
- AVSXSVCZWQODGV-DPAQBDIFSA-N desmosterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@@H](CCC=C(C)C)C)[C@@]1(C)CC2 AVSXSVCZWQODGV-DPAQBDIFSA-N 0.000 claims 2
- QBSJHOGDIUQWTH-UHFFFAOYSA-N dihydrolanosterol Natural products CC(C)CCCC(C)C1CCC2(C)C3=C(CCC12C)C4(C)CCC(C)(O)C(C)(C)C4CC3 QBSJHOGDIUQWTH-UHFFFAOYSA-N 0.000 claims 2
- LPFIPZJIWTZLEY-DAABMGJCSA-N dinosterol Chemical compound C[C@@H]([C@@H]1CC2)[C@@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@H](C)/C=C(\C)[C@H](C)C(C)C)[C@@]2(C)CC1 LPFIPZJIWTZLEY-DAABMGJCSA-N 0.000 claims 2
- DNVPQKQSNYMLRS-SOWFXMKYSA-N ergosterol Chemical compound C1[C@@H](O)CC[C@]2(C)[C@H](CC[C@]3([C@H]([C@H](C)/C=C/[C@@H](C)C(C)C)CC[C@H]33)C)C3=CC=C21 DNVPQKQSNYMLRS-SOWFXMKYSA-N 0.000 claims 2
- 229930182470 glycoside Natural products 0.000 claims 2
- 150000002338 glycosides Chemical class 0.000 claims 2
- YRPMZHRSQIFCLR-UHFFFAOYSA-N gorgosterol Natural products CC(C)C(C)C1(C)CC1C(C)C1C2(C)CCC3C4(C)CCC(O)CC4=CCC3C2CC1 YRPMZHRSQIFCLR-UHFFFAOYSA-N 0.000 claims 2
- AXEYWFGSQDLHDX-UHFFFAOYSA-N hexane-1,3,6-triamine Chemical compound NCCCC(N)CCN AXEYWFGSQDLHDX-UHFFFAOYSA-N 0.000 claims 2
- 230000002452 interceptive effect Effects 0.000 claims 2
- CAHGCLMLTWQZNJ-RGEKOYMOSA-N lanosterol Chemical compound C([C@]12C)C[C@@H](O)C(C)(C)[C@H]1CCC1=C2CC[C@]2(C)[C@H]([C@H](CCC=C(C)C)C)CC[C@@]21C CAHGCLMLTWQZNJ-RGEKOYMOSA-N 0.000 claims 2
- 229940058690 lanosterol Drugs 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- XVUQPECVOGMPRU-ZPPAUJSGSA-N n,n-dimethyl-1,2-bis[(9z,12z)-octadeca-9,12-dienoxy]propan-1-amine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCCOC(C)C(N(C)C)OCCCCCCCC\C=C/C\C=C/CCCCC XVUQPECVOGMPRU-ZPPAUJSGSA-N 0.000 claims 2
- 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 2
- 150000003905 phosphatidylinositols Chemical class 0.000 claims 2
- 239000013612 plasmid Substances 0.000 claims 2
- KZJWDPNRJALLNS-VJSFXXLFSA-N sitosterol 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)CC[C@@H](CC)C(C)C)[C@@]1(C)CC2 KZJWDPNRJALLNS-VJSFXXLFSA-N 0.000 claims 2
- 229950005143 sitosterol Drugs 0.000 claims 2
- 239000008247 solid mixture Substances 0.000 claims 2
- 239000002904 solvent Substances 0.000 claims 2
- 229940063673 spermidine Drugs 0.000 claims 2
- QYSXJUFSXHHAJI-YRZJJWOYSA-N vitamin D3 Chemical compound C1(/[C@@H]2CC[C@@H]([C@]2(CCC1)C)[C@H](C)CCCC(C)C)=C\C=C1\C[C@@H](O)CCC1=C QYSXJUFSXHHAJI-YRZJJWOYSA-N 0.000 claims 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- ARKJJRYSZYFUNJ-UHFFFAOYSA-N 2,3-dihydroxypropyl carbamate Chemical compound NC(=O)OCC(O)CO ARKJJRYSZYFUNJ-UHFFFAOYSA-N 0.000 claims 1
- 108091023037 Aptamer Proteins 0.000 claims 1
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 claims 1
- DTQVDTLACAAQTR-UHFFFAOYSA-M Trifluoroacetate Chemical compound [O-]C(=O)C(F)(F)F DTQVDTLACAAQTR-UHFFFAOYSA-M 0.000 claims 1
- HZYXFRGVBOPPNZ-UHFFFAOYSA-N UNPD88870 Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)=CCC(CC)C(C)C)C1(C)CC2 HZYXFRGVBOPPNZ-UHFFFAOYSA-N 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000013011 aqueous formulation Substances 0.000 claims 1
- 239000007864 aqueous solution Substances 0.000 claims 1
- 125000004432 carbon atom Chemical group C* 0.000 claims 1
- 229940106189 ceramide Drugs 0.000 claims 1
- 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 1
- 150000001875 compounds Chemical class 0.000 claims 1
- UMGXUWVIJIQANV-UHFFFAOYSA-M didecyl(dimethyl)azanium;bromide Chemical compound [Br-].CCCCCCCCCC[N+](C)(C)CCCCCCCCCC UMGXUWVIJIQANV-UHFFFAOYSA-M 0.000 claims 1
- PSLWZOIUBRXAQW-UHFFFAOYSA-M dimethyl(dioctadecyl)azanium;bromide Chemical compound [Br-].CCCCCCCCCCCCCCCCCC[N+](C)(C)CCCCCCCCCCCCCCCCCC PSLWZOIUBRXAQW-UHFFFAOYSA-M 0.000 claims 1
- UAKOZKUVZRMOFN-JDVCJPALSA-M dimethyl-bis[(z)-octadec-9-enyl]azanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC[N+](C)(C)CCCCCCCC\C=C/CCCCCCCC UAKOZKUVZRMOFN-JDVCJPALSA-M 0.000 claims 1
- FRKBLBQTSTUKOV-UHFFFAOYSA-N diphosphatidyl glycerol Natural products OP(O)(=O)OCC(OP(O)(O)=O)COP(O)(O)=O FRKBLBQTSTUKOV-UHFFFAOYSA-N 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000000178 monomer Substances 0.000 claims 1
- OZBZDYGIYDRTBV-RSLAUBRISA-N n,n-dimethyl-1,2-bis[(9z,12z,15z)-octadeca-9,12,15-trienoxy]propan-1-amine Chemical compound CC\C=C/C\C=C/C\C=C/CCCCCCCCOC(C)C(N(C)C)OCCCCCCCC\C=C/C\C=C/C\C=C/CC OZBZDYGIYDRTBV-RSLAUBRISA-N 0.000 claims 1
- 230000007935 neutral effect Effects 0.000 claims 1
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000003880 polar aprotic solvent Substances 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- HCXVJBMSMIARIN-PHZDYDNGSA-N stigmasterol 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)/C=C/[C@@H](CC)C(C)C)[C@@]1(C)CC2 HCXVJBMSMIARIN-PHZDYDNGSA-N 0.000 claims 1
- 229940032091 stigmasterol Drugs 0.000 claims 1
- 235000016831 stigmasterol Nutrition 0.000 claims 1
- BFDNMXAIBMJLBB-UHFFFAOYSA-N stigmasterol Natural products CCC(C=CC(C)C1CCCC2C3CC=C4CC(O)CCC4(C)C3CCC12C)C(C)C BFDNMXAIBMJLBB-UHFFFAOYSA-N 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US17896209P | 2009-05-16 | 2009-05-16 | |
| US61/178,962 | 2009-05-16 | ||
| PCT/US2010/035049 WO2010135207A1 (en) | 2009-05-16 | 2010-05-16 | Compositions comprising cationic amphiphiles and colipids for delivering therapeutics molecules |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012526858A JP2012526858A (ja) | 2012-11-01 |
| JP2012526858A5 true JP2012526858A5 (enExample) | 2013-07-04 |
| JP5675784B2 JP5675784B2 (ja) | 2015-02-25 |
Family
ID=43126456
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012511923A Active JP5675784B2 (ja) | 2009-05-16 | 2010-05-16 | 治療用分子を送達するためのカチオン性両親媒性物質と共脂質とを含む組成物 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US9080186B2 (enExample) |
| EP (1) | EP2430168B1 (enExample) |
| JP (1) | JP5675784B2 (enExample) |
| CN (1) | CN102985548B (enExample) |
| AU (1) | AU2010249881B2 (enExample) |
| CA (1) | CA2762095A1 (enExample) |
| WO (1) | WO2010135207A1 (enExample) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010008582A2 (en) | 2008-07-18 | 2010-01-21 | Rxi Pharmaceuticals Corporation | Phagocytic cell drug delivery system |
| CN108165548B (zh) | 2008-09-22 | 2022-10-14 | 菲奥医药公司 | 减小大小的自递送RNAi化合物 |
| WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| IN2012DN00570A (enExample) | 2009-07-14 | 2015-06-12 | Polypid Ltd | |
| AU2011208374B2 (en) | 2010-01-19 | 2016-09-08 | Polypid Ltd. | Sustained-release nucleic acid matrix compositions |
| FR2998899B1 (fr) * | 2012-11-30 | 2015-07-17 | Commissariat Energie Atomique | Methode de criblage a haut-debit pour l'identification de biomarqueurs, cibles therapeutiques ou d'agents therapeutiques |
| US10253315B2 (en) | 2012-08-30 | 2019-04-09 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Method for transfection of nucleic acids into eukaryotic cells in 3D scaffold |
| FR2994849B1 (fr) * | 2012-08-30 | 2015-01-02 | Commissariat Energie Atomique | Formulation pour la delivrance de sequences nucleotidiques susceptibles de moduler des mecanismes endogenes d'arn interferents |
| EP2719765A1 (de) * | 2012-10-11 | 2014-04-16 | Secutech International Pte. Ltd. | Ex vivo-Verfahren zur Einbringung von Nukleinsäuren in Zellen |
| JP6400479B2 (ja) * | 2012-10-29 | 2018-10-03 | 株式会社カルディオ | 肺疾患特異的治療剤 |
| EP3027222A1 (en) * | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Sphingolipid-polyalkylamine-oligonucleotide compounds |
| WO2015184326A1 (en) * | 2014-05-30 | 2015-12-03 | Cerulean Pharma Inc. | Conjugates, particles, compositions, and related methods |
| US20150368648A1 (en) | 2014-06-20 | 2015-12-24 | Yale University | Compositions and methods of inhibiting gene expression in a lung |
| KR101671864B1 (ko) * | 2014-08-29 | 2016-11-04 | 세종대학교 산학협력단 | 요오드화 오일을 함유하는 양이온성 에멀젼 및 바이러스 벡터의 유전자 전달 증진을 위한 이의 용도 |
| CN104922141B (zh) * | 2015-05-28 | 2016-05-25 | 厦门成坤生物技术有限公司 | 一种用于治疗乙型病毒性肝炎的siRNA组合物 |
| CN105079021A (zh) * | 2015-08-03 | 2015-11-25 | 厦门成坤生物技术有限公司 | 包含m10c1化合物与活性成分的组合物及其制备方法 |
| DK3350157T3 (da) | 2015-09-17 | 2022-02-14 | Modernatx Inc | Forbindelser og sammensætninger til intracellulær afgivelse af terapeutiske midler |
| AU2016377681B2 (en) | 2015-12-22 | 2021-05-13 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
| WO2017151733A1 (en) * | 2016-03-01 | 2017-09-08 | Molecular Transfer, Inc. | Plant virus movement proteins and methods of using the same |
| US11583504B2 (en) | 2016-11-08 | 2023-02-21 | Modernatx, Inc. | Stabilized formulations of lipid nanoparticles |
| AU2018234692B2 (en) | 2017-03-15 | 2022-06-02 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| CA3055653A1 (en) * | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Lipid nanoparticle formulation |
| WO2018232120A1 (en) | 2017-06-14 | 2018-12-20 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
| CN107802508A (zh) * | 2017-11-01 | 2018-03-16 | 武汉百思凯瑞纳米科技有限公司 | 一种阳离子脂质体纳米组合物及其制备方法和应用 |
| KR102056144B1 (ko) | 2018-03-08 | 2019-12-16 | 주식회사 파마리서치프로덕트 | Dna 단편 혼합물이 고농도로 함유된 유동성을 갖는 액제 조성물 및 이의 제조방법 |
| US12263248B2 (en) | 2018-09-19 | 2025-04-01 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| WO2020061317A1 (en) * | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | Sterol purification |
| WO2020061332A1 (en) * | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | Sterol analogs and uses thereof |
| CA3154618A1 (en) | 2019-09-19 | 2021-03-25 | Modernatx, Inc. | Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents |
| CN111904934B (zh) * | 2020-09-21 | 2022-02-25 | 中国医学科学院医药生物技术研究所 | miRNA185抑制剂的脂质体及其制法 |
| CN120775847A (zh) | 2021-10-20 | 2025-10-14 | 厦门甘宝利生物医药有限公司 | Agt抑制剂及其用途 |
| EP4495130A4 (en) * | 2022-03-15 | 2025-12-17 | Fujifilm Corp | CONNECTION AND LIPIDE COMPOSITION |
| WO2023229023A1 (ja) * | 2022-05-25 | 2023-11-30 | 学校法人 名城大学 | 粉末剤及びその製造方法 |
| CN116814621B (zh) | 2022-08-05 | 2025-09-09 | 厦门甘宝利生物医药有限公司 | 一种抑制apoc3基因表达的rna抑制剂及其应用 |
| CN115197431B (zh) * | 2022-09-15 | 2022-11-29 | 清华大学 | 脂质偶联完全可降解水溶性聚合物的合成及应用 |
| CN115851723B (zh) | 2022-10-24 | 2023-10-03 | 厦门甘宝利生物医药有限公司 | 一种抑制lpa基因表达的rna抑制剂及其应用 |
| WO2025021083A1 (en) * | 2023-07-24 | 2025-01-30 | National Institute Of Biological Sciences, Beijing | Lipid nanoparticle compositions for localized treatment of skin diseases |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE3272873D1 (en) | 1981-06-04 | 1986-10-02 | Pharmasol Corp | Pressurized container with dispensing pump |
| US5208036A (en) | 1985-01-07 | 1993-05-04 | Syntex (U.S.A.) Inc. | N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4778810A (en) | 1987-01-08 | 1988-10-18 | Nastech Pharmaceutical Co., Inc. | Nasal delivery of caffeine |
| US5279833A (en) | 1990-04-04 | 1994-01-18 | Yale University | Liposomal transfection of nucleic acids into animal cells |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| DE69123979T2 (de) | 1990-10-12 | 1997-04-30 | Max Planck Gesellschaft | Abgeänderte ribozyme |
| US5283185A (en) | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| DE69527206T2 (de) | 1994-09-30 | 2003-02-27 | Inex Pharmaceuticals Corp., Vancouver | Mittel zum einbringen polyanionischer materialien in zellen |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| US5767099A (en) | 1994-12-09 | 1998-06-16 | Genzyme Corporation | Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules |
| US5948767A (en) | 1994-12-09 | 1999-09-07 | Genzyme Corporation | Cationic amphiphile/DNA complexes |
| US6331524B1 (en) * | 1994-12-09 | 2001-12-18 | Genzyme Corporation | Organ-specific targeting of cationic amphiphile / DNA complexes for gene therapy |
| US20020022264A1 (en) * | 1995-05-26 | 2002-02-21 | Sullivan Sean M. | Delivery vehicles comprising stable lipid/nucleic acid complexes |
| US7422902B1 (en) * | 1995-06-07 | 2008-09-09 | The University Of British Columbia | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
| US5849902A (en) | 1996-09-26 | 1998-12-15 | Oligos Etc. Inc. | Three component chimeric antisense oligonucleotides |
| US6358523B1 (en) | 1996-12-06 | 2002-03-19 | The Regents Of The University Of California | Macromolecule-lipid complexes and methods for making and regulating |
| JP2002524473A (ja) | 1998-09-09 | 2002-08-06 | ジエンザイム コーポレイション | プラスミドベクターのメチル化 |
| WO2002087541A1 (en) * | 2001-04-30 | 2002-11-07 | Protiva Biotherapeutics Inc. | Lipid-based formulations for gene transfer |
| SG190613A1 (en) * | 2003-07-16 | 2013-06-28 | Protiva Biotherapeutics Inc | Lipid encapsulated interfering rna |
| EP1664316B1 (en) * | 2003-09-15 | 2012-08-29 | Protiva Biotherapeutics Inc. | Polyethyleneglycol-modified lipid compounds and uses thereof |
| JP4764426B2 (ja) | 2004-06-07 | 2011-09-07 | プロチバ バイオセラピューティクス インコーポレイティッド | カチオン性脂質および使用方法 |
| US8598333B2 (en) * | 2006-05-26 | 2013-12-03 | Alnylam Pharmaceuticals, Inc. | SiRNA silencing of genes expressed in cancer |
| US20080299177A1 (en) * | 2007-06-06 | 2008-12-04 | Biovaluation & Analysis, Inc. | Supramolecular Complexes for Use in Acoustically Mediated Intracellular Drug Delivery in vivo |
| WO2011084721A2 (en) | 2009-12-21 | 2011-07-14 | Agave Pharma Inc | Cationic molecule and method of use |
-
2010
- 2010-05-16 EP EP10778182.5A patent/EP2430168B1/en active Active
- 2010-05-16 JP JP2012511923A patent/JP5675784B2/ja active Active
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- 2010-05-16 WO PCT/US2010/035049 patent/WO2010135207A1/en not_active Ceased
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- 2010-05-16 CN CN201080031986.9A patent/CN102985548B/zh active Active
- 2010-05-16 CA CA2762095A patent/CA2762095A1/en not_active Abandoned
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