JP2011529912A5 - - Google Patents
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- Publication number
- JP2011529912A5 JP2011529912A5 JP2011521353A JP2011521353A JP2011529912A5 JP 2011529912 A5 JP2011529912 A5 JP 2011529912A5 JP 2011521353 A JP2011521353 A JP 2011521353A JP 2011521353 A JP2011521353 A JP 2011521353A JP 2011529912 A5 JP2011529912 A5 JP 2011529912A5
- Authority
- JP
- Japan
- Prior art keywords
- peg
- lipid
- cholesterol
- nanoparticle
- oligonucleotide
- 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.)
- Pending
Links
- 239000002105 nanoparticle Substances 0.000 claims 27
- 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 22
- 239000000203 mixture Substances 0.000 claims 15
- 108091034117 Oligonucleotide Proteins 0.000 claims 11
- 235000012000 cholesterol Nutrition 0.000 claims 11
- 150000002632 lipids Chemical class 0.000 claims 10
- -1 cationic lipid Chemical class 0.000 claims 9
- 210000004027 cell Anatomy 0.000 claims 6
- 125000002091 cationic group Chemical group 0.000 claims 5
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- JZNWSCPGTDBMEW-UHFFFAOYSA-N Glycerophosphorylethanolamin Natural products NCCOP(O)(=O)OCC(O)CO JZNWSCPGTDBMEW-UHFFFAOYSA-N 0.000 claims 4
- 206010028980 Neoplasm Diseases 0.000 claims 4
- 230000000692 anti-sense effect Effects 0.000 claims 4
- 201000011510 cancer Diseases 0.000 claims 4
- 150000008104 phosphatidylethanolamines Chemical class 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 claims 3
- 125000000217 alkyl group Chemical group 0.000 claims 3
- 229940106189 ceramide Drugs 0.000 claims 3
- 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 3
- 230000000799 fusogenic effect Effects 0.000 claims 3
- 230000014509 gene expression Effects 0.000 claims 3
- 229910052739 hydrogen Inorganic materials 0.000 claims 3
- 239000001257 hydrogen Substances 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 claims 3
- 108091023037 Aptamer Proteins 0.000 claims 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-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
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 210000005260 human cell Anatomy 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 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
- 102100021569 Apoptosis regulator Bcl-2 Human genes 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
- CPELXLSAUQHCOX-UHFFFAOYSA-M Bromide Chemical compound [Br-] CPELXLSAUQHCOX-UHFFFAOYSA-M 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 101000971171 Homo sapiens Apoptosis regulator Bcl-2 Proteins 0.000 claims 1
- 108700020796 Oncogene Proteins 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 108091028664 Ribonucleotide Proteins 0.000 claims 1
- 108010002687 Survivin Proteins 0.000 claims 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 1
- 239000002246 antineoplastic agent Substances 0.000 claims 1
- 230000004663 cell proliferation Effects 0.000 claims 1
- 229940127089 cytotoxic agent Drugs 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 150000002431 hydrogen Chemical class 0.000 claims 1
- 239000012678 infectious agent Substances 0.000 claims 1
- 230000002401 inhibitory effect Effects 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
- 230000037361 pathway Effects 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
- 230000004647 pro-inflammatory pathway Effects 0.000 claims 1
- 230000006482 proangiogenic pathway Effects 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
- 230000003612 virological effect Effects 0.000 claims 1
- 0 CC(*)(*)C(*)(*CCC1)C1(*)N(*)CCC* Chemical compound CC(*)(*)C(*)(*CCC1)C1(*)N(*)CCC* 0.000 description 5
- MZQUWCQHUCBQSW-UHFFFAOYSA-N IN1C(CCC2)N2CCC1 Chemical compound IN1C(CCC2)N2CCC1 MZQUWCQHUCBQSW-UHFFFAOYSA-N 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US8528908P | 2008-07-31 | 2008-07-31 | |
| US61/085,289 | 2008-07-31 | ||
| PCT/US2009/052396 WO2010014895A2 (en) | 2008-07-31 | 2009-07-31 | Nanoparticle compositions for nucleic acids delivery system |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2011529912A JP2011529912A (ja) | 2011-12-15 |
| JP2011529912A5 true JP2011529912A5 (https=) | 2012-07-26 |
Family
ID=41610973
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011521353A Pending JP2011529912A (ja) | 2008-07-31 | 2009-07-31 | 核酸送達システム用のナノ粒子組成物 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20110111044A1 (https=) |
| EP (1) | EP2304026A4 (https=) |
| JP (1) | JP2011529912A (https=) |
| CA (1) | CA2731173A1 (https=) |
| TW (1) | TW201004658A (https=) |
| WO (1) | WO2010014895A2 (https=) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102844435B (zh) * | 2010-02-22 | 2017-05-10 | 库尔纳公司 | 通过抑制吡咯啉‑5‑羧酸还原酶1(pycr1)的天然反义转录物而治疗pycr1相关疾病 |
| WO2011133504A2 (en) * | 2010-04-20 | 2011-10-27 | University Of Florida Research Foundation, Inc. | Nanozymes, methods of making nanozymes, and methods of using nanozymes |
| JP6194248B2 (ja) | 2010-10-28 | 2017-09-06 | パシラ ファーマシューティカルズ インコーポレーテッド | 非ステロイド性抗炎症薬の徐放性処方物 |
| AU2012236099A1 (en) | 2011-03-31 | 2013-10-03 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| US8709466B2 (en) | 2011-03-31 | 2014-04-29 | International Business Machines Corporation | Cationic polymers for antimicrobial applications and delivery of bioactive materials |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| EP2915300A1 (en) * | 2012-11-02 | 2015-09-09 | Telefonaktiebolaget L M Ericsson (Publ) | Network node, user node and methods for channel estimation |
| JP6360074B2 (ja) * | 2013-01-03 | 2018-07-18 | カウンスィル オブ サイエンティフィック アンド インダストリアル リサーチＣｏｕｎｃｉｌ Ｏｆ Ｓｃｉｅｎｔｉｆｉｃ ＆ Ｉｎｄｕｓｔｒｉａｌ Ｒｅｓｅａｒｃｈ | マンノース受容体選択的リシニル化カチオン性両親媒性物質及びその調製方法 |
| EP2971165A4 (en) | 2013-03-15 | 2016-11-23 | Moderna Therapeutics Inc | DISSOLUTION OF DNA FRAGMENTS IN MRNA MANUFACTURING METHODS |
| US10590161B2 (en) | 2013-03-15 | 2020-03-17 | Modernatx, Inc. | Ion exchange purification of mRNA |
| WO2014152031A1 (en) | 2013-03-15 | 2014-09-25 | Moderna Therapeutics, Inc. | Ribonucleic acid purification |
| CA2917348A1 (en) | 2013-07-11 | 2015-01-15 | Moderna Therapeutics, Inc. | Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use |
| TW201521762A (zh) | 2013-08-14 | 2015-06-16 | Univ Florida | 奈米酶、製備奈米酶的方法、以及使用奈米酶的方法 |
| EP3052511A4 (en) | 2013-10-02 | 2017-05-31 | Moderna Therapeutics, Inc. | Polynucleotide molecules and uses thereof |
| WO2015196128A2 (en) | 2014-06-19 | 2015-12-23 | Moderna Therapeutics, Inc. | Alternative nucleic acid molecules and uses thereof |
| US10407683B2 (en) | 2014-07-16 | 2019-09-10 | Modernatx, Inc. | Circular polynucleotides |
| WO2017049286A1 (en) | 2015-09-17 | 2017-03-23 | Moderna Therapeutics, Inc. | Polynucleotides containing a morpholino linker |
| EP3350333B2 (en) | 2015-09-17 | 2025-08-06 | ModernaTX, Inc. | Polynucleotides containing a stabilizing tail region |
| CN107951862B (zh) * | 2016-10-17 | 2021-03-12 | 南京绿叶制药有限公司 | 一种抑制bcl-2的反义寡聚核酸的脂质纳米粒及其制备方法 |
| EP3541815A4 (en) | 2016-11-18 | 2020-07-15 | Pacira Pharmaceuticals, Inc. | ZINC MELXICAM COMPLEX MICROPARTICLE MULTIVESICULAR LIPOSOME FORMULATIONS AND METHODS OF MAKING THE SAME |
| WO2019027055A1 (ja) * | 2017-08-04 | 2019-02-07 | 協和発酵キリン株式会社 | 核酸含有脂質ナノ粒子 |
| CA3088485A1 (en) * | 2018-01-18 | 2019-07-25 | Etherna Immunotherapies Nv | Lipid nanoparticles |
| HRP20240586T1 (hr) | 2018-10-09 | 2024-07-19 | The University Of British Columbia | Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima |
| CN109549934B (zh) * | 2018-12-25 | 2021-03-09 | 上海交通大学 | 一种多糖基脂质纳米粒子的制备方法 |
| KR20240027630A (ko) * | 2021-06-01 | 2024-03-04 | 나노베이션 테라퓨틱스 인크. | 지질 나노입자를 사용한 dna 벡터 전달 |
| TWI851090B (zh) * | 2022-03-17 | 2024-08-01 | 國立成功大學 | 雙股寡去氧核苷酸、包含該雙股寡去氧核苷酸的組合物及其用途 |
| ES2994098T3 (en) | 2022-03-21 | 2025-01-17 | R G C C Holdings Ag | Liposomal compositions |
| WO2023178425A1 (en) | 2022-03-23 | 2023-09-28 | Nanovation Therapeutics Inc. | High sterol-containing lipid nanoparticles |
| WO2023184038A1 (en) * | 2022-04-01 | 2023-10-05 | Nanovation Therapeutics Inc. | Mrna delivery method and composition thereof |
| EP4606372A1 (en) * | 2022-10-18 | 2025-08-27 | Tipton Corp. | Taylor reactor and capsule particle manufacturing method |
| 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 (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5837533A (en) * | 1994-09-28 | 1998-11-17 | American Home Products Corporation | Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent |
| WO1997045069A1 (en) * | 1996-05-29 | 1997-12-04 | Cell Genesys, Inc. | Cationic polymer/lipid nucleic acid delivery vehicles |
| AU733310C (en) * | 1997-05-14 | 2001-11-29 | University Of British Columbia, The | High efficiency encapsulation of charged therapeutic agents in lipid vesicles |
| US6794499B2 (en) * | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US6841537B1 (en) * | 1998-04-22 | 2005-01-11 | Protiva Biotherapeutics Inc. | Combination therapy using nucleic acids and conventional drugs |
| US20070042983A1 (en) * | 2001-05-18 | 2007-02-22 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA) |
| US20080207542A1 (en) * | 2002-03-26 | 2008-08-28 | Sirna Therapeutics, Inc. | RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA) |
| US20060051405A1 (en) * | 2004-07-19 | 2006-03-09 | Protiva Biotherapeutics, Inc. | Compositions for the delivery of therapeutic agents and uses thereof |
| US7404969B2 (en) * | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| WO2007086881A2 (en) * | 2005-02-14 | 2007-08-02 | Sirna Therapeutics, Inc. | Cationic lipids and formulated molecular compositions containing them |
| EP1996607A4 (en) * | 2006-02-01 | 2009-06-24 | Burnham Inst Medical Research | LYMPH ADDRESS CODES FOR TUMORS AND PREMALARY LESIONS |
| US20070293449A1 (en) * | 2006-06-20 | 2007-12-20 | Nastech Pharmaceutical Company Inc. | Compositions and methods for delivery of double-stranded rna |
| WO2008034123A2 (en) * | 2006-09-15 | 2008-03-20 | Enzon Pharmaceuticals, Inc. | Polymeric conjugates containing positively-charged moieties |
-
2009
- 2009-07-31 TW TW098125813A patent/TW201004658A/zh unknown
- 2009-07-31 WO PCT/US2009/052396 patent/WO2010014895A2/en not_active Ceased
- 2009-07-31 CA CA2731173A patent/CA2731173A1/en not_active Abandoned
- 2009-07-31 US US13/003,816 patent/US20110111044A1/en not_active Abandoned
- 2009-07-31 EP EP09803646.0A patent/EP2304026A4/en not_active Withdrawn
- 2009-07-31 JP JP2011521353A patent/JP2011529912A/ja active Pending
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