JP2017535610A5 - - Google Patents
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- JP2017535610A5 JP2017535610A5 JP2017545520A JP2017545520A JP2017535610A5 JP 2017535610 A5 JP2017535610 A5 JP 2017535610A5 JP 2017545520 A JP2017545520 A JP 2017545520A JP 2017545520 A JP2017545520 A JP 2017545520A JP 2017535610 A5 JP2017535610 A5 JP 2017535610A5
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- JP
- Japan
- Prior art keywords
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- 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.)
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- 150000001875 compounds Chemical class 0.000 claims description 80
- 125000000217 alkyl group Chemical group 0.000 claims description 40
- 125000002947 alkylene group Chemical group 0.000 claims description 23
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 18
- 239000005977 Ethylene Substances 0.000 claims description 12
- VGGSQFUCUMXWEO-UHFFFAOYSA-N Ethene Chemical compound C=C VGGSQFUCUMXWEO-UHFFFAOYSA-N 0.000 claims description 11
- 125000003342 alkenyl group Chemical group 0.000 claims description 11
- 125000004805 propylene group Chemical group [H]C([H])([H])C([H])([*:1])C([H])([H])[*:2] 0.000 claims description 10
- 125000001570 methylene group Chemical group [H]C([H])([*:1])[*:2] 0.000 claims description 8
- 150000003839 salts Chemical class 0.000 claims description 8
- 125000004432 carbon atom Chemical group C* 0.000 claims description 7
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims description 6
- -1 alkynyl carbon Chemical compound 0.000 claims description 5
- 125000000304 alkynyl group Chemical group 0.000 claims description 5
- 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 description 5
- 229910052760 oxygen Inorganic materials 0.000 claims description 5
- QQONPFPTGQHPMA-UHFFFAOYSA-N propylene Natural products CC=C QQONPFPTGQHPMA-UHFFFAOYSA-N 0.000 claims description 5
- 229910052717 sulfur Inorganic materials 0.000 claims description 5
- 125000004450 alkenylene group Chemical group 0.000 claims description 4
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 4
- LMBFAGIMSUYTBN-MPZNNTNKSA-N teixobactin Chemical compound C([C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CO)C(=O)N[C@H](CCC(N)=O)C(=O)N[C@H]([C@@H](C)CC)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CO)C(=O)N[C@H]1C(N[C@@H](C)C(=O)N[C@@H](C[C@@H]2NC(=N)NC2)C(=O)N[C@H](C(=O)O[C@H]1C)[C@@H](C)CC)=O)NC)C1=CC=CC=C1 LMBFAGIMSUYTBN-MPZNNTNKSA-N 0.000 claims description 2
- 150000002632 lipids Chemical class 0.000 claims 6
- 239000002245 particle Substances 0.000 claims 5
- 229910052799 carbon Inorganic materials 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims 3
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000012453 solvate Substances 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 description 2
- 201000011510 cancer Diseases 0.000 description 2
- 201000010099 disease Diseases 0.000 description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 2
- 125000000623 heterocyclic group Chemical group 0.000 description 2
- 229910052739 hydrogen Inorganic materials 0.000 description 2
- 239000001257 hydrogen Substances 0.000 description 2
- 125000004435 hydrogen atom Chemical class [H]* 0.000 description 2
- 238000000034 method Methods 0.000 description 2
- 208000024172 Cardiovascular disease Diseases 0.000 description 1
- 206010061218 Inflammation Diseases 0.000 description 1
- 230000005856 abnormality Effects 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 210000000988 bone and bone Anatomy 0.000 description 1
- 210000004556 brain Anatomy 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 230000003176 fibrotic effect Effects 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 208000026278 immune system disease Diseases 0.000 description 1
- 208000027866 inflammatory disease Diseases 0.000 description 1
- 230000004054 inflammatory process Effects 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 208000017169 kidney disease Diseases 0.000 description 1
- 210000004185 liver Anatomy 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 150000007523 nucleic acids Chemical class 0.000 description 1
- 102000039446 nucleic acids Human genes 0.000 description 1
- 108020004707 nucleic acids Proteins 0.000 description 1
- 210000000952 spleen Anatomy 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US14/546,105 | 2014-11-18 | ||
| US14/546,105 US9593077B2 (en) | 2013-11-18 | 2014-11-18 | Ionizable cationic lipid for RNA delivery |
| US14/707,876 | 2015-05-08 | ||
| US14/707,876 US9365610B2 (en) | 2013-11-18 | 2015-05-08 | Asymmetric ionizable cationic lipid for RNA delivery |
| US14/707,796 US9567296B2 (en) | 2013-11-18 | 2015-05-08 | Ionizable cationic lipid for RNA delivery |
| US14/707,796 | 2015-05-08 | ||
| PCT/US2015/030218 WO2016081029A1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017535610A JP2017535610A (ja) | 2017-11-30 |
| JP2017535610A5 true JP2017535610A5 (https=) | 2018-09-27 |
| JP6637988B2 JP6637988B2 (ja) | 2020-01-29 |
Family
ID=56014367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017545520A Active JP6637988B2 (ja) | 2014-11-18 | 2015-05-11 | Rna送達のためのイオン化可能カチオン性脂質 |
Country Status (20)
| Country | Link |
|---|---|
| EP (1) | EP3221293B1 (https=) |
| JP (1) | JP6637988B2 (https=) |
| KR (1) | KR102380363B1 (https=) |
| CN (1) | CN107207428A (https=) |
| AU (1) | AU2015350561B2 (https=) |
| CA (1) | CA2968060C (https=) |
| DK (1) | DK3221293T3 (https=) |
| ES (1) | ES2946110T3 (https=) |
| FI (1) | FI3221293T3 (https=) |
| HK (1) | HK1244479A1 (https=) |
| HR (1) | HRP20230342T1 (https=) |
| HU (1) | HUE062130T2 (https=) |
| IL (1) | IL252291B (https=) |
| LT (1) | LT3221293T (https=) |
| NZ (1) | NZ732777A (https=) |
| PL (1) | PL3221293T3 (https=) |
| PT (1) | PT3221293T (https=) |
| RS (1) | RS64078B1 (https=) |
| SI (1) | SI3221293T1 (https=) |
| WO (1) | WO2016081029A1 (https=) |
Families Citing this family (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9593077B2 (en) | 2013-11-18 | 2017-03-14 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US12460231B2 (en) | 2014-04-02 | 2025-11-04 | Editas Medicine, Inc. | Crispr/CAS-related methods and compositions for treating primary open angle glaucoma |
| CN110582301A (zh) | 2016-12-14 | 2019-12-17 | 利甘达尔股份有限公司 | 用于核酸和蛋白质有效负载递送的方法和组合物 |
| WO2018119163A1 (en) * | 2016-12-21 | 2018-06-28 | Payne Joseph E | Ionizable cationic lipid for rna delivery |
| US10526284B2 (en) | 2016-12-21 | 2020-01-07 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| US10383952B2 (en) * | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| WO2018183901A1 (en) * | 2017-03-30 | 2018-10-04 | The Government Of The United States Of America As Represented By The Secretary Of The Army | Nucleic acid vaccine composition comprising a lipid formulation, and method of increasing the potency of nucleic acid vaccines |
| ES3039647T3 (en) * | 2017-05-31 | 2025-10-23 | Arcturus Therapeutics Inc | Synthesis and structure of high potency rna therapeutics |
| US12083224B2 (en) | 2018-03-30 | 2024-09-10 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
| MX2020011166A (es) | 2018-04-25 | 2022-10-19 | Ethris Gmbh | Agentes crioprotectores para formulaciones en forma de particulas. |
| KR20190127277A (ko) * | 2018-05-04 | 2019-11-13 | 주식회사 삼양바이오팜 | mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법 |
| PL3805198T3 (pl) * | 2018-06-08 | 2024-02-12 | Fujifilm Corporation | Związek, jego sól i cząstki lipidowe |
| JP7445657B2 (ja) | 2018-12-06 | 2024-03-07 | アークトゥラス・セラピューティクス・インコーポレイテッド | オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法 |
| EP3942050A4 (en) * | 2019-03-19 | 2023-02-22 | Arcturus Therapeutics, Inc. | PROCEDURE FOR THE PRODUCTION OF LIPID-COATED RNA NANOPARTICLES |
| WO2020246581A1 (ja) | 2019-06-07 | 2020-12-10 | 富士フイルム株式会社 | 脂質組成物 |
| US12544437B2 (en) | 2019-10-04 | 2026-02-10 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant AAV |
| EP4059505B1 (en) | 2019-11-15 | 2024-02-21 | FUJIFILM Corporation | Lipid composition |
| JP7832114B2 (ja) | 2020-03-03 | 2026-03-17 | アークトゥラス・セラピューティクス・インコーポレイテッド | オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法 |
| AU2021244559A1 (en) | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| MX2022011805A (es) | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| US20230227826A1 (en) * | 2020-06-18 | 2023-07-20 | Arcturus Therapeutics, Inc. | Una oligomers for the treatment of polyglutamine diseases |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| WO2022056413A1 (en) | 2020-09-13 | 2022-03-17 | Arcturus Therapeutics, Inc. | Lipid nanoparticles encapsulation of large rna |
| MX2023009345A (es) * | 2021-02-10 | 2023-10-24 | Oncorus Inc | Compuestos, composiciones y metodos de uso de estos. |
| AU2022260111A1 (en) | 2021-04-20 | 2023-11-30 | Anjarium Biosciences Ag | Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof |
| CN117881786A (zh) | 2021-04-27 | 2024-04-12 | 世代生物公司 | 表达治疗性抗体的非病毒dna载体及其用途 |
| EP4329884A1 (en) | 2021-04-27 | 2024-03-06 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| MX2024002726A (es) | 2021-09-03 | 2024-03-20 | CureVac SE | Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos. |
| WO2023073228A1 (en) | 2021-10-29 | 2023-05-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| EP4430024A1 (en) | 2021-11-08 | 2024-09-18 | Orna Therapeutics, Inc. | Lipid nanoparticle compositions for delivering circular polynucleotides |
| JP2025504404A (ja) | 2022-01-14 | 2025-02-12 | アンジャリウム バイオサイエンシズ エージー | Viii因子をコードするdna分子の組成物、それを作製する方法、及びその使用方法 |
| US20250099614A1 (en) | 2022-01-28 | 2025-03-27 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
| CN114213295B (zh) * | 2022-02-22 | 2022-05-20 | 中国科学院基础医学与肿瘤研究所(筹) | 一种阳离子化合物、制备方法及其复合物和用途 |
| WO2023177655A1 (en) | 2022-03-14 | 2023-09-21 | Generation Bio Co. | Heterologous prime boost vaccine compositions and methods of use |
| WO2023227608A1 (en) | 2022-05-25 | 2023-11-30 | Glaxosmithkline Biologicals Sa | Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide |
| EP4536197A1 (en) | 2022-06-07 | 2025-04-16 | Generation Bio Co. | Lipid nanoparticle compositions and uses thereof |
| WO2024040222A1 (en) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
| KR20250110209A (ko) | 2022-09-23 | 2025-07-18 | 엔크로마 바이오, 인크. | Hbv 유전자 발현의 에피제네틱 조절을 위한 조성물 및 방법 |
| WO2024089638A1 (en) | 2022-10-28 | 2024-05-02 | Glaxosmithkline Biologicals Sa | Nucleic acid based vaccine |
| CN120390656A (zh) | 2022-11-08 | 2025-07-29 | 欧纳医疗公司 | 环状rna组合物 |
| WO2024102762A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Lipids and lipid nanoparticle compositions for delivering polynucleotides |
| TW202425959A (zh) | 2022-11-08 | 2024-07-01 | 美商歐納醫療公司 | 遞送多核苷酸的脂質及奈米顆粒組合物 |
| JP2025540076A (ja) | 2022-12-01 | 2025-12-11 | ジェネレーション バイオ カンパニー | 核酸、イオン化可能な脂質、ステロール、脂質アンカーポリマー、及びヘルパー脂質を含む脂質ナノ粒子、それらの使用 |
| KR20250131271A (ko) | 2022-12-01 | 2025-09-02 | 제너레이션 바이오 컴퍼니 | 신규한 폴리글리세롤-접합된 지질 및 이를 포함하는 지질 나노입자 조성물 |
| KR20250114116A (ko) | 2022-12-01 | 2025-07-28 | 제너레이션 바이오 컴퍼니 | 세포 표적화를 위한 스텔스 지질 나노입자 조성물 |
| WO2024119103A1 (en) | 2022-12-01 | 2024-06-06 | Generation Bio Co. | Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers |
| KR20250153298A (ko) | 2023-03-08 | 2025-10-24 | 큐어백 에스이 | 핵산 전달을 위한 신규의 지질 나노입자 제형 |
| WO2024205657A2 (en) | 2023-03-29 | 2024-10-03 | Orna Therapeutics, Inc. | Lipids and lipid nanoparticle compositions for delivering polynucleotides |
| WO2024233308A2 (en) | 2023-05-05 | 2024-11-14 | Orna Therapeutics, Inc. | Circular rna compositions and methods |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| IL324634A (en) | 2023-05-15 | 2026-01-01 | Nchroma Bio Inc | Compositions and methods for epigenetic regulation of hbv gene expression |
| CN116270543B (zh) * | 2023-05-19 | 2023-09-26 | 清华大学 | 脂质纳米颗粒在制备通过雾吸或鼻滴给药递送核酸的药物中的用途 |
| US12311033B2 (en) | 2023-05-31 | 2025-05-27 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025049690A1 (en) | 2023-08-29 | 2025-03-06 | Orna Therapeutics, Inc. | Circular polyethylene glycol lipids |
| WO2025052180A2 (en) | 2023-09-07 | 2025-03-13 | Axelyf ehf. | Lipids and lipid nanoparticles |
| WO2025080939A1 (en) | 2023-10-13 | 2025-04-17 | Ultragenyx Pharmaceutical, Inc. | Compositions and methods for treating conditions associated with cartilage oligomeric matrix protein (comp) mutations |
| WO2025101501A1 (en) | 2023-11-07 | 2025-05-15 | Orna Therapeutics, Inc. | Circular rna compositions |
| WO2025128853A2 (en) | 2023-12-13 | 2025-06-19 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with ube3a overexpression |
| WO2025129201A1 (en) | 2023-12-15 | 2025-06-19 | Capstan Therapeutics, Inc. | Humanized anti-cd8 antibodies and uses thereof |
| WO2025166323A2 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting lipoprotein (a) expression |
| WO2025189064A1 (en) | 2024-03-08 | 2025-09-12 | Genzyme Corporation | Lipid nanoparticles |
| 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 |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
| WO2026030375A2 (en) | 2024-07-30 | 2026-02-05 | Genzyme Corporation | Lipid nanoparticles and methods of manufacture and use thereof |
| WO2026064512A1 (en) | 2024-09-18 | 2026-03-26 | Generation Bio Co. | Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4511069A (en) | 1981-06-04 | 1985-04-16 | The Pharmasol Corporation | Dispensing system |
| US4778810A (en) | 1987-01-08 | 1988-10-18 | Nastech Pharmaceutical Co., Inc. | Nasal delivery of caffeine |
| JP3257675B2 (ja) | 1990-10-12 | 2002-02-18 | マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー.ファウ. | 修飾リボザイム |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| US5849902A (en) | 1996-09-26 | 1998-12-15 | Oligos Etc. Inc. | Three component chimeric antisense oligonucleotides |
| JP5902616B2 (ja) * | 2010-04-28 | 2016-04-13 | 協和発酵キリン株式会社 | カチオン性脂質 |
| CN107043334B (zh) * | 2011-06-08 | 2020-06-16 | 日东电工株式会社 | 用于定向药物输送和增强siRNA活性的化合物 |
| US20140308304A1 (en) * | 2011-12-07 | 2014-10-16 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| SI2858974T1 (sl) * | 2012-06-08 | 2019-01-31 | Nitto Denko Corporation | Lipidi za formulacije za dostavo terapevtskega sredstva |
| US9593077B2 (en) * | 2013-11-18 | 2017-03-14 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
-
2015
- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active Active
- 2015-05-11 HK HK18103903.0A patent/HK1244479A1/zh unknown
- 2015-05-11 NZ NZ732777A patent/NZ732777A/en unknown
- 2015-05-11 WO PCT/US2015/030218 patent/WO2016081029A1/en not_active Ceased
- 2015-05-11 AU AU2015350561A patent/AU2015350561B2/en active Active
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
- 2015-05-11 HR HRP20230342TT patent/HRP20230342T1/hr unknown
- 2015-05-11 HU HUE15724466A patent/HUE062130T2/hu unknown
- 2015-05-11 CN CN201580073670.9A patent/CN107207428A/zh active Pending
- 2015-05-11 RS RS20230231A patent/RS64078B1/sr unknown
- 2015-05-11 EP EP15724466.6A patent/EP3221293B1/en active Active
- 2015-05-11 PT PT157244666T patent/PT3221293T/pt unknown
- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
- 2015-05-11 DK DK15724466.6T patent/DK3221293T3/da active
- 2015-05-11 ES ES15724466T patent/ES2946110T3/es active Active
- 2015-05-11 CA CA2968060A patent/CA2968060C/en active Active
- 2015-05-11 JP JP2017545520A patent/JP6637988B2/ja active Active
- 2015-05-11 LT LTEPPCT/US2015/030218T patent/LT3221293T/lt unknown
- 2015-05-11 FI FIEP15724466.6T patent/FI3221293T3/fi active
-
2017
- 2017-05-15 IL IL252291A patent/IL252291B/en unknown
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