JPWO2020097520A5 - - Google Patents
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- Publication number
- JPWO2020097520A5 JPWO2020097520A5 JP2021525114A JP2021525114A JPWO2020097520A5 JP WO2020097520 A5 JPWO2020097520 A5 JP WO2020097520A5 JP 2021525114 A JP2021525114 A JP 2021525114A JP 2021525114 A JP2021525114 A JP 2021525114A JP WO2020097520 A5 JPWO2020097520 A5 JP WO2020097520A5
- Authority
- JP
- Japan
- Prior art keywords
- lipid
- nucleic acid
- mol
- particle
- total lipids
- 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
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- 239000002245 particle Substances 0.000 claims 116
- 150000002632 lipids Chemical class 0.000 claims 65
- 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 44
- -1 cationic lipid Chemical class 0.000 claims 43
- 239000000203 mixture Substances 0.000 claims 23
- 235000012000 cholesterol Nutrition 0.000 claims 22
- 108020004707 nucleic acids Proteins 0.000 claims 17
- 102000039446 nucleic acids Human genes 0.000 claims 17
- 150000007523 nucleic acids Chemical class 0.000 claims 17
- 125000002091 cationic group Chemical group 0.000 claims 13
- 150000003904 phospholipids Chemical class 0.000 claims 13
- 125000003342 alkenyl group Chemical group 0.000 claims 10
- 125000001183 hydrocarbyl group Chemical group 0.000 claims 10
- 150000001875 compounds Chemical class 0.000 claims 9
- 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 8
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 claims 7
- 125000000217 alkyl group Chemical group 0.000 claims 7
- 239000004055 small Interfering RNA Substances 0.000 claims 7
- 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims 6
- 239000002202 Polyethylene glycol Substances 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 6
- 229920001223 polyethylene glycol Polymers 0.000 claims 6
- 108020004459 Small interfering RNA Proteins 0.000 claims 5
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 5
- 125000006700 (C1-C6) alkylthio group Chemical group 0.000 claims 4
- 125000005862 (C1-C6)alkanoyl group Chemical group 0.000 claims 4
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims 4
- NQRYJNQNLNOLGT-UHFFFAOYSA-N Piperidine Chemical compound C1CCNCC1 NQRYJNQNLNOLGT-UHFFFAOYSA-N 0.000 claims 4
- 125000004423 acyloxy group Chemical group 0.000 claims 4
- 125000000484 butyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 4
- 125000001559 cyclopropyl group Chemical group [H]C1([H])C([H])([H])C1([H])* 0.000 claims 4
- 125000004356 hydroxy functional group Chemical group O* 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 4
- 125000004191 (C1-C6) alkoxy group Chemical group 0.000 claims 3
- 125000004454 (C1-C6) alkoxycarbonyl group Chemical group 0.000 claims 3
- 125000006755 (C2-C20) alkyl group Chemical group 0.000 claims 3
- 125000006649 (C2-C20) alkynyl group Chemical group 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 3
- 125000000882 C2-C6 alkenyl group Chemical group 0.000 claims 3
- 125000003601 C2-C6 alkynyl group Chemical group 0.000 claims 3
- 125000000304 alkynyl group Chemical group 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000035475 disorder Diseases 0.000 claims 3
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 3
- 229920000642 polymer Polymers 0.000 claims 3
- KILNVBDSWZSGLL-KXQOOQHDSA-N 1,2-dihexadecanoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCC KILNVBDSWZSGLL-KXQOOQHDSA-N 0.000 claims 2
- NOWKCMXCCJGMRR-UHFFFAOYSA-N Aziridine Chemical compound C1CN1 NOWKCMXCCJGMRR-UHFFFAOYSA-N 0.000 claims 2
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 claims 2
- 241000124008 Mammalia Species 0.000 claims 2
- 108700011259 MicroRNAs Proteins 0.000 claims 2
- YNAVUWVOSKDBBP-UHFFFAOYSA-N Morpholine Chemical group C1COCCN1 YNAVUWVOSKDBBP-UHFFFAOYSA-N 0.000 claims 2
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 108020000999 Viral RNA Proteins 0.000 claims 2
- 125000004414 alkyl thio group Chemical group 0.000 claims 2
- HONIICLYMWZJFZ-UHFFFAOYSA-N azetidine Chemical compound C1CNC1 HONIICLYMWZJFZ-UHFFFAOYSA-N 0.000 claims 2
- 125000001246 bromo group Chemical group Br* 0.000 claims 2
- 125000001309 chloro group Chemical group Cl* 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000003937 drug carrier Substances 0.000 claims 2
- 238000001727 in vivo Methods 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 229910052757 nitrogen Inorganic materials 0.000 claims 2
- IJGRMHOSHXDMSA-UHFFFAOYSA-N nitrogen Substances N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims 2
- QJGQUHMNIGDVPM-UHFFFAOYSA-N nitrogen group Chemical group [N] QJGQUHMNIGDVPM-UHFFFAOYSA-N 0.000 claims 2
- 125000006274 (C1-C3)alkoxy group Chemical group 0.000 claims 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 1
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 claims 1
- 208000036142 Viral infection Diseases 0.000 claims 1
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims 1
- 150000001412 amines Chemical class 0.000 claims 1
- 150000001450 anions Chemical group 0.000 claims 1
- 125000004432 carbon atom Chemical group C* 0.000 claims 1
- 125000002147 dimethylamino group Chemical group [H]C([H])([H])N(*)C([H])([H])[H] 0.000 claims 1
- 125000003827 glycol group Chemical group 0.000 claims 1
- 125000005843 halogen group Chemical group 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 201000007270 liver cancer Diseases 0.000 claims 1
- 208000019423 liver disease Diseases 0.000 claims 1
- 208000014018 liver neoplasm Diseases 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000002243 precursor Substances 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 239000000758 substrate Substances 0.000 claims 1
- 230000009385 viral infection Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024202026A JP2025019119A (ja) | 2018-11-09 | 2024-11-20 | ケイ素を含有するカチオン性脂質 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862758108P | 2018-11-09 | 2018-11-09 | |
| US62/758,108 | 2018-11-09 | ||
| PCT/US2019/060556 WO2020097520A1 (en) | 2018-11-09 | 2019-11-08 | Cationic lipids containing silicon |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024202026A Division JP2025019119A (ja) | 2018-11-09 | 2024-11-20 | ケイ素を含有するカチオン性脂質 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022501064A JP2022501064A (ja) | 2022-01-06 |
| JP2022501064A5 JP2022501064A5 (https=) | 2022-11-04 |
| JPWO2020097520A5 true JPWO2020097520A5 (https=) | 2022-11-04 |
Family
ID=70612265
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021525114A Pending JP2022501064A (ja) | 2018-11-09 | 2019-11-08 | ケイ素を含有するカチオン性脂質 |
| JP2024202026A Pending JP2025019119A (ja) | 2018-11-09 | 2024-11-20 | ケイ素を含有するカチオン性脂質 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024202026A Pending JP2025019119A (ja) | 2018-11-09 | 2024-11-20 | ケイ素を含有するカチオン性脂質 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US12350383B2 (https=) |
| EP (1) | EP3877001A4 (https=) |
| JP (2) | JP2022501064A (https=) |
| KR (1) | KR20210105888A (https=) |
| CN (1) | CN113286622A (https=) |
| AU (1) | AU2019376006A1 (https=) |
| BR (1) | BR112021008953A2 (https=) |
| CA (1) | CA3119051A1 (https=) |
| EA (1) | EA202191315A1 (https=) |
| IL (1) | IL282957A (https=) |
| MX (1) | MX2021005462A (https=) |
| SG (1) | SG11202104682PA (https=) |
| WO (1) | WO2020097520A1 (https=) |
| ZA (1) | ZA202103229B (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CA3112837A1 (en) * | 2018-10-19 | 2020-04-23 | Translate Bio, Inc. | Pumpless encapsulation of messenger rna |
| AU2020355000A1 (en) | 2019-09-23 | 2022-03-17 | Omega Therapeutics, Inc. | Compositions and methods for modulating apolipoprotein B (APOB) gene expression |
| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| CN116096886A (zh) | 2020-03-11 | 2023-05-09 | 欧米茄治疗公司 | 用于调节叉头框p3(foxp3)基因表达的组合物和方法 |
| AU2021305214A1 (en) * | 2020-07-10 | 2023-02-23 | Genevant Sciences Gmbh | Lipid nanoparticles for delivering therapeutics to lungs |
| CZ310613B6 (cs) | 2020-09-23 | 2026-01-28 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Lipidoidy pro transfekci nukleových kyselin a jejich použití |
| EP4367242A2 (en) | 2021-07-07 | 2024-05-15 | Omega Therapeutics, Inc. | Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression |
| CZ310443B6 (cs) | 2021-07-19 | 2025-06-25 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití |
| WO2024243438A2 (en) | 2023-05-23 | 2024-11-28 | Omega Therapeutics, Inc. | Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression |
| WO2025129128A1 (en) | 2023-12-15 | 2025-06-19 | Vivasor, Inc. | Compositions and methods for non-viral delivery of therapeutic compounds |
| WO2025133951A1 (en) | 2023-12-21 | 2025-06-26 | Genevant Sciences Gmbh | Ionizable lipids suitable for lipid nanoparticles |
| WO2025166323A2 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting lipoprotein (a) expression |
| WO2025213138A1 (en) | 2024-04-05 | 2025-10-09 | Editas Medicine, Inc. | Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma |
| WO2026022656A2 (en) | 2024-07-22 | 2026-01-29 | Genevant Sciences Gmbh | Lipid particle formulations with permanently charged cationic lipids |
| WO2026022677A1 (en) | 2024-07-22 | 2026-01-29 | Genevant Sciences Gmbh | Permanently charged cationic lipids |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4618689A (en) | 1985-07-18 | 1986-10-21 | General Electric Company | Novel aminofunctional silicone compositions |
| JPH08166679A (ja) * | 1994-12-13 | 1996-06-25 | Fuji Xerox Co Ltd | 電子写真感光体およびのその製造方法 |
| DE10058620A1 (de) * | 2000-11-25 | 2002-05-29 | Degussa | Verfahren zur Herstellung von Aminoalkylsilanen |
| US7901708B2 (en) | 2002-06-28 | 2011-03-08 | Protiva Biotherapeutics, Inc. | Liposomal apparatus and manufacturing methods |
| ES2559828T3 (es) | 2003-07-16 | 2016-02-16 | Protiva Biotherapeutics Inc. | ARN de interferencia encapsulado en lípidos |
| WO2006113679A2 (en) * | 2005-04-15 | 2006-10-26 | Board Of Regents, The University Of Texas System | Delivery of sirna by neutral lipid compositions |
| US20070292688A1 (en) * | 2005-08-18 | 2007-12-20 | Eastman Kodak Company | Silylamine modified nanoparticulate carriers |
| ITMI20061766A1 (it) * | 2006-09-15 | 2008-03-16 | N P T S R L | Prepolimeri silano-terminati e relativi formulati adesivo-sigillanti |
| AU2009238175C1 (en) | 2008-04-15 | 2023-11-30 | Arbutus Biopharma Corporation | Novel lipid formulations for nucleic acid delivery |
| CN104119242B (zh) | 2008-10-09 | 2017-07-07 | 泰米拉制药公司 | 改善的氨基脂质和递送核酸的方法 |
| MY156621A (en) * | 2009-08-21 | 2016-03-15 | China Petroleum & Chemical | A catalyst component for ethylene polymerization, preparation thereof and a catalyst comprising the catalyst component |
| WO2012000104A1 (en) | 2010-06-30 | 2012-01-05 | Protiva Biotherapeutics, Inc. | Non-liposomal systems for nucleic acid delivery |
| US9028603B2 (en) * | 2011-06-09 | 2015-05-12 | The Research Foundation Of State University Of New York | Anti-fouling coating compositions and methods for preventing the fouling of surfaces |
| US10517822B2 (en) * | 2013-11-06 | 2019-12-31 | The University Of Chicago | Nanoscale carriers for the delivery or co-delivery of chemotherapeutics, nucleic acids and photosensitizers |
| CN108350455A (zh) * | 2015-07-29 | 2018-07-31 | 阿布特斯生物制药公司 | 用于使b型肝炎病毒基因表达沉默的组合物和方法 |
| CN106834355B (zh) * | 2015-12-07 | 2021-02-23 | 北京大学 | 一种阳离子复合脂质纳米盘及其制备方法和用途 |
| EP3269785A1 (de) * | 2016-07-13 | 2018-01-17 | Nitrochemie Aschau GmbH | Zusammensetzung für silikonkautschukmassen |
| AU2017379901B2 (en) * | 2016-12-21 | 2024-02-15 | Arbutus Biopharma Corporation | Methods for ameliorating infusion reactions |
| WO2019051257A2 (en) | 2017-09-11 | 2019-03-14 | Arbutus Biopharma Corporation | METHODS OF TREATING HEPATITIS B TYPE INFECTIONS |
| AU2020262437A1 (en) | 2019-04-26 | 2021-12-23 | Genevant Sciences Gmbh | Lipid nanoparticles |
| AU2021305214A1 (en) * | 2020-07-10 | 2023-02-23 | Genevant Sciences Gmbh | Lipid nanoparticles for delivering therapeutics to lungs |
| AU2021403156A1 (en) * | 2020-12-18 | 2023-07-13 | Genevant Sciences Gmbh | Peg lipids and lipid nanoparticles |
-
2019
- 2019-11-08 MX MX2021005462A patent/MX2021005462A/es unknown
- 2019-11-08 KR KR1020217017640A patent/KR20210105888A/ko not_active Ceased
- 2019-11-08 AU AU2019376006A patent/AU2019376006A1/en active Pending
- 2019-11-08 JP JP2021525114A patent/JP2022501064A/ja active Pending
- 2019-11-08 EA EA202191315A patent/EA202191315A1/ru unknown
- 2019-11-08 US US17/291,873 patent/US12350383B2/en active Active
- 2019-11-08 CA CA3119051A patent/CA3119051A1/en active Pending
- 2019-11-08 SG SG11202104682PA patent/SG11202104682PA/en unknown
- 2019-11-08 WO PCT/US2019/060556 patent/WO2020097520A1/en not_active Ceased
- 2019-11-08 EP EP19881045.9A patent/EP3877001A4/en active Pending
- 2019-11-08 BR BR112021008953-5A patent/BR112021008953A2/pt unknown
- 2019-11-08 CN CN201980088071.2A patent/CN113286622A/zh active Pending
-
2021
- 2021-05-05 IL IL282957A patent/IL282957A/en unknown
- 2021-05-12 ZA ZA2021/03229A patent/ZA202103229B/en unknown
-
2024
- 2024-11-20 JP JP2024202026A patent/JP2025019119A/ja active Pending
-
2025
- 2025-05-27 US US19/219,802 patent/US20250281422A1/en active Pending
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