JP2011529912A5 - - Google Patents
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- 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 |
|---|---|---|---|---|
| RU2608496C2 (ru) * | 2010-02-22 | 2017-01-18 | Курна, Инк. | Лечение заболеваний, связанных с пирролин-5 карбоксилатредуктазой 1(pycr1), путем ингибирования природного антисмыслового транскрипта к pycr1 |
| EP2561072A4 (en) | 2010-04-20 | 2016-04-06 | Univ Florida | NANOZYME, METHOD FOR THE PRODUCTION OF NANOCYMENTS AND METHOD FOR THE USE OF NANOCYMENTS |
| EP2632553B1 (en) | 2010-10-28 | 2020-01-01 | Pacira Pharmaceuticals, Inc. | A sustained release formulation of a non-steroidal anti-inflammatory drug |
| EP2691101A2 (en) | 2011-03-31 | 2014-02-05 | 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 |
| US9374747B2 (en) * | 2012-11-02 | 2016-06-21 | Telefonaktiebolaget Lm 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 |
| WO2014152031A1 (en) | 2013-03-15 | 2014-09-25 | Moderna Therapeutics, Inc. | Ribonucleic acid purification |
| WO2014144767A1 (en) | 2013-03-15 | 2014-09-18 | Moderna Therapeutics, Inc. | Ion exchange purification of mrna |
| WO2014152030A1 (en) | 2013-03-15 | 2014-09-25 | Moderna Therapeutics, Inc. | Removal of dna fragments in mrna production process |
| SMT202100691T1 (it) * | 2013-07-11 | 2022-01-10 | Modernatx Inc | Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso |
| CN104519875A (zh) | 2013-08-14 | 2015-04-15 | 佛罗里达大学研究基金会公司 | 纳米酶、制备纳米酶的方法、以及使用纳米酶的方法 |
| WO2015051169A2 (en) | 2013-10-02 | 2015-04-09 | 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 |
| PL3350333T3 (pl) | 2015-09-17 | 2022-03-07 | Modernatx, Inc. | Polinukleotydy zawierające region stabilizujący ogon |
| WO2017049286A1 (en) | 2015-09-17 | 2017-03-23 | Moderna Therapeutics, Inc. | Polynucleotides containing a morpholino linker |
| 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 |
| EP3662913A4 (en) * | 2017-08-04 | 2021-06-30 | Kyowa Kirin Co., Ltd. | Nucleic-acid-containing lipid nanoparticles |
| EP3740241A1 (en) * | 2018-01-18 | 2020-11-25 | eTheRNA Immunotherapies NV | Lipid nanoparticles |
| CN113166783B (zh) | 2018-10-09 | 2024-10-11 | 不列颠哥伦比亚大学 | 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法 |
| 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 |
| CN117642155A (zh) * | 2021-06-01 | 2024-03-01 | 内诺维什疗法股份有限公司 | 使用脂质纳米颗粒的dna载体递送 |
| 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 |
| AU2023238223A1 (en) | 2022-03-23 | 2024-10-10 | Nanovation Therapeutics Inc. | High sterol-containing lipid nanoparticles |
| CN119654166A (zh) * | 2022-04-01 | 2025-03-18 | 内诺维什疗法股份有限公司 | mRNA递送方法及其组合物 |
| WO2024085149A1 (ja) * | 2022-10-18 | 2024-04-25 | 第一三共株式会社 | テイラー反応装置及びカプセル粒子の製造方法 |
| CN121568686A (zh) * | 2023-07-24 | 2026-02-24 | 北京生命科学研究所 | 用于皮肤疾病局部治疗的脂质纳米颗粒组合物 |
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 |
| CA2256900A1 (en) * | 1996-05-29 | 1997-12-04 | Xiao-Ying Meng | Cationic polymer/lipid nucleic acid delivery vehicles |
| CA2289702C (en) * | 1997-05-14 | 2008-02-19 | Inex Pharmaceuticals Corp. | 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) |
| WO2006007712A1 (en) * | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| JP5042863B2 (ja) * | 2005-02-14 | 2012-10-03 | サーナ・セラピューティクス・インコーポレイテッド | 生物学的に活性な分子をデリバリーするための脂質ナノ粒子系組成物および方法 |
| US7404969B2 (en) * | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| US7919466B2 (en) * | 2006-02-01 | 2011-04-05 | The Regents Of The University Of California | Lymphatic zip codes in tumors and pre-malignant lesions |
| US20070293449A1 (en) * | 2006-06-20 | 2007-12-20 | Nastech Pharmaceutical Company Inc. | Compositions and methods for delivery of double-stranded rna |
| EP2076257A4 (en) * | 2006-09-15 | 2014-04-16 | Belrose Pharma Inc | POLYMER CONJUGATES WITH POSITIVELY LOADED PARTS |
-
2009
- 2009-07-31 TW TW098125813A patent/TW201004658A/zh unknown
- 2009-07-31 US US13/003,816 patent/US20110111044A1/en not_active Abandoned
- 2009-07-31 JP JP2011521353A patent/JP2011529912A/ja active Pending
- 2009-07-31 CA CA2731173A patent/CA2731173A1/en not_active Abandoned
- 2009-07-31 WO PCT/US2009/052396 patent/WO2010014895A2/en not_active Ceased
- 2009-07-31 EP EP09803646.0A patent/EP2304026A4/en not_active Withdrawn
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