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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- Japan
- Prior art keywords
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- 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 |
| US14/707,796 | 2015-05-08 | ||
| US14/707,876 US9365610B2 (en) | 2013-11-18 | 2015-05-08 | Asymmetric ionizable cationic lipid for RNA delivery |
| US14/707,876 | 2015-05-08 | ||
| US14/707,796 US9567296B2 (en) | 2013-11-18 | 2015-05-08 | Ionizable cationic lipid for RNA delivery |
| 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 (enExample) | 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 (enExample) |
| JP (1) | JP6637988B2 (enExample) |
| KR (1) | KR102380363B1 (enExample) |
| CN (1) | CN107207428A (enExample) |
| AU (1) | AU2015350561B2 (enExample) |
| CA (1) | CA2968060C (enExample) |
| DK (1) | DK3221293T3 (enExample) |
| ES (1) | ES2946110T3 (enExample) |
| FI (1) | FI3221293T3 (enExample) |
| HK (1) | HK1244479A1 (enExample) |
| HR (1) | HRP20230342T1 (enExample) |
| HU (1) | HUE062130T2 (enExample) |
| IL (1) | IL252291B (enExample) |
| LT (1) | LT3221293T (enExample) |
| NZ (1) | NZ732777A (enExample) |
| PL (1) | PL3221293T3 (enExample) |
| PT (1) | PT3221293T (enExample) |
| RS (1) | RS64078B1 (enExample) |
| SI (1) | SI3221293T1 (enExample) |
| WO (1) | WO2016081029A1 (enExample) |
Families Citing this family (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6486955B2 (ja) | 2013-11-18 | 2019-03-20 | アークトゥルス セラピューティクス, インコーポレイテッド | Rna送達のためのイオン化可能なカチオン性脂質 |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| JP7317706B2 (ja) | 2016-12-14 | 2023-07-31 | リガンダル インコーポレイテッド | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
| 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 |
| EP3600396A4 (en) * | 2017-03-30 | 2021-01-13 | The Government of the United States of America as represented by the Secretary of the Army | VACCINE COMPOSITION OF NUCLEIC ACID INCLUDING A LIPIDIC FORMULATION, AND PROCESS FOR INCREASING THE EFFECTIVENESS 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 |
| AU2019258678B2 (en) | 2018-04-25 | 2024-08-22 | Ethris Gmbh | Lipid-based formulations for the delivery of RNA |
| KR20190127277A (ko) * | 2018-05-04 | 2019-11-13 | 주식회사 삼양바이오팜 | mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법 |
| CN116172975B (zh) * | 2018-06-08 | 2025-03-28 | 富士胶片株式会社 | 脂质颗粒、包含该脂质颗粒的核酸药物以及核酸递送用组合物 |
| US11685906B2 (en) | 2018-12-06 | 2023-06-27 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| US11737979B2 (en) | 2019-03-19 | 2023-08-29 | Arcturus Therapeutics, Inc. | Method of making lipid-encapsulated RNA nanoparticles |
| CN113924128B (zh) | 2019-06-07 | 2024-09-06 | 富士胶片株式会社 | 脂质组合物 |
| EP4038194A1 (en) | 2019-10-04 | 2022-08-10 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| KR20250057122A (ko) | 2019-11-15 | 2025-04-28 | 후지필름 가부시키가이샤 | 지질 조성물 |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| CA3172591A1 (en) | 2020-03-24 | 2021-09-30 | Douglas Anthony KERR | Non-viral dna vectors and uses thereof for expressing gaucher therapeutics |
| KR20230003477A (ko) | 2020-03-24 | 2023-01-06 | 제너레이션 바이오 컴퍼니 | 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도 |
| US20230227826A1 (en) * | 2020-06-18 | 2023-07-20 | Arcturus Therapeutics, Inc. | Una oligomers for the treatment of polyglutamine diseases |
| US20230272432A1 (en) | 2020-07-27 | 2023-08-31 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| MX2023002928A (es) | 2020-09-13 | 2023-06-12 | Arcturus Therapeutics Inc | Encapsulacion de arn grande en nanoparticulas lipidicas. |
| IL305064A (en) | 2021-02-10 | 2023-10-01 | Oncorus Inc | Compounds, preparations and methods of using them |
| KR20240012370A (ko) | 2021-04-20 | 2024-01-29 | 안자리움 바이오사이언시스 아게 | 아밀로-알파-1, 6-글루코시다제, 4-알파-글루카노트랜스퍼라제를 인코딩하는 dna 분자의 조성물, 이를 제조하는 방법 및 이를 사용하는 방법 |
| KR20240011714A (ko) | 2021-04-27 | 2024-01-26 | 제너레이션 바이오 컴퍼니 | 치료용 항체를 발현하는 비바이러스성 dna 벡터 및 이의 용도 |
| US20240216535A1 (en) | 2021-04-27 | 2024-07-04 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| JP2024538489A (ja) | 2021-09-03 | 2024-10-23 | キュアバック エスイー | 核酸を送達するための新規な脂質ナノ粒子 |
| US20250027108A1 (en) | 2021-10-29 | 2025-01-23 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| MX2024005521A (es) | 2021-11-08 | 2024-05-21 | Orna Therapeutics Inc | Composiciones de nanoparticulas de lipidos para la administracion de polinucleotidos circulares. |
| EP4463475A2 (en) | 2022-01-14 | 2024-11-20 | Anjarium Biosciences AG | Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof |
| WO2023144330A1 (en) | 2022-01-28 | 2023-08-03 | 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 |
| JP2025509607A (ja) | 2022-03-14 | 2025-04-11 | ジェネレーション バイオ カンパニー | 異種プライムブーストワクチン組成物及び使用の方法 |
| KR20250028547A (ko) | 2022-05-25 | 2025-02-28 | 큐어백 에스이 | 대장균 FimH 항원성 폴리펩타이드를 인코딩하는 핵산 기반 백신 |
| CA3258303A1 (en) | 2022-06-07 | 2023-12-14 | Generation Bio Co | Compositions of lipid nanoparticles and their uses |
| WO2024040222A1 (en) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
| EP4590822A1 (en) | 2022-09-23 | 2025-07-30 | nChroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| US12186389B2 (en) | 2022-10-28 | 2025-01-07 | Glaxosmithkline Biologicals Sa | Nucleic acid base vaccine against emerging SARS-CoV-2 variants |
| WO2024102762A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Lipids and lipid nanoparticle compositions for delivering polynucleotides |
| WO2024102730A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Lipids and nanoparticle compositions for delivering polynucleotides |
| CN120390656A (zh) | 2022-11-08 | 2025-07-29 | 欧纳医疗公司 | 环状rna组合物 |
| WO2024119039A2 (en) | 2022-12-01 | 2024-06-06 | Generation Bio Co. | Stealth lipid nanoparticles and uses thereof |
| WO2024119103A1 (en) | 2022-12-01 | 2024-06-06 | Generation Bio Co. | Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers |
| EP4626401A1 (en) | 2022-12-01 | 2025-10-08 | Generation Bio Co. | Stealth lipid nanoparticle compositions for cell targeting |
| KR20250131271A (ko) | 2022-12-01 | 2025-09-02 | 제너레이션 바이오 컴퍼니 | 신규한 폴리글리세롤-접합된 지질 및 이를 포함하는 지질 나노입자 조성물 |
| 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 |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | 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 |
| US20250332281A2 (en) | 2023-12-15 | 2025-10-30 | Capstan Therapeutics, Inc. | Humanized anti-cd8 antibodies and uses thereof |
| WO2025166325A1 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | MODIFIED GUIDE RNAs |
| 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 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS58500845A (ja) | 1981-06-04 | 1983-05-26 | デラウェア・ケミカルス・コーポレーション | ポンプ |
| US4778810A (en) | 1987-01-08 | 1988-10-18 | Nastech Pharmaceutical Co., Inc. | Nasal delivery of caffeine |
| US5672695A (en) | 1990-10-12 | 1997-09-30 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Modified ribozymes |
| 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 |
| EP2567951B1 (en) * | 2010-04-28 | 2018-09-19 | Kyowa Hakko Kirin Co., Ltd. | Cationic lipid |
| CA2837101C (en) * | 2011-06-08 | 2020-12-15 | Priya Karmali | Compounds for targeting drug delivery and enhancing sirna activity |
| US20140308304A1 (en) * | 2011-12-07 | 2014-10-16 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| WO2013185116A1 (en) * | 2012-06-08 | 2013-12-12 | Payne Joseph E | Lipids for therapeutic agent delivery formulations |
| JP6486955B2 (ja) * | 2013-11-18 | 2019-03-20 | アークトゥルス セラピューティクス, インコーポレイテッド | Rna送達のためのイオン化可能なカチオン性脂質 |
-
2015
- 2015-05-11 JP JP2017545520A patent/JP6637988B2/ja active Active
- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active Active
- 2015-05-11 CN CN201580073670.9A patent/CN107207428A/zh active Pending
- 2015-05-11 NZ NZ732777A patent/NZ732777A/en unknown
- 2015-05-11 FI FIEP15724466.6T patent/FI3221293T3/fi active
- 2015-05-11 AU AU2015350561A patent/AU2015350561B2/en active Active
- 2015-05-11 HR HRP20230342TT patent/HRP20230342T1/hr unknown
- 2015-05-11 LT LTEPPCT/US2015/030218T patent/LT3221293T/lt unknown
- 2015-05-11 CA CA2968060A patent/CA2968060C/en active Active
- 2015-05-11 RS RS20230231A patent/RS64078B1/sr unknown
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
- 2015-05-11 DK DK15724466.6T patent/DK3221293T3/da active
- 2015-05-11 PT PT157244666T patent/PT3221293T/pt unknown
- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
- 2015-05-11 WO PCT/US2015/030218 patent/WO2016081029A1/en not_active Ceased
- 2015-05-11 HK HK18103903.0A patent/HK1244479A1/zh unknown
- 2015-05-11 HU HUE15724466A patent/HUE062130T2/hu unknown
- 2015-05-11 ES ES15724466T patent/ES2946110T3/es active Active
- 2015-05-11 EP EP15724466.6A patent/EP3221293B1/en active Active
-
2017
- 2017-05-15 IL IL252291A patent/IL252291B/en unknown
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