JPWO2022235935A5 - - Google Patents
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- Publication number
- JPWO2022235935A5 JPWO2022235935A5 JP2023567891A JP2023567891A JPWO2022235935A5 JP WO2022235935 A5 JPWO2022235935 A5 JP WO2022235935A5 JP 2023567891 A JP2023567891 A JP 2023567891A JP 2023567891 A JP2023567891 A JP 2023567891A JP WO2022235935 A5 JPWO2022235935 A5 JP WO2022235935A5
- Authority
- JP
- Japan
- Prior art keywords
- compound
- lipid
- lipid composition
- pharma
- acceptable salt
- 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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- 150000002632 lipids Chemical class 0.000 claims description 97
- 150000001875 compounds Chemical class 0.000 claims description 77
- 239000002105 nanoparticle Substances 0.000 claims description 55
- 239000000203 mixture Substances 0.000 claims description 31
- 108020004707 nucleic acids Proteins 0.000 claims description 19
- 102000039446 nucleic acids Human genes 0.000 claims description 19
- 150000007523 nucleic acids Chemical class 0.000 claims description 19
- 150000003839 salts Chemical class 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 102000004169 proteins and genes Human genes 0.000 claims description 13
- 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 description 12
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 11
- 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 description 9
- GVNVAWHJIKLAGL-UHFFFAOYSA-N 2-(cyclohexen-1-yl)cyclohexan-1-one Chemical compound O=C1CCCCC1C1=CCCCC1 GVNVAWHJIKLAGL-UHFFFAOYSA-N 0.000 claims description 9
- 101150065749 Churc1 gene Proteins 0.000 claims description 9
- 108090000790 Enzymes Proteins 0.000 claims description 9
- 102000004190 Enzymes Human genes 0.000 claims description 9
- 102100038239 Protein Churchill Human genes 0.000 claims description 9
- 125000002947 alkylene group Chemical group 0.000 claims description 9
- 239000008194 pharmaceutical composition Substances 0.000 claims description 9
- 230000001225 therapeutic effect Effects 0.000 claims description 9
- 239000000427 antigen Substances 0.000 claims description 7
- 102000036639 antigens Human genes 0.000 claims description 7
- 108091007433 antigens Proteins 0.000 claims description 7
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 6
- 235000012000 cholesterol Nutrition 0.000 claims description 6
- 238000010362 genome editing Methods 0.000 claims description 6
- 108020004999 messenger RNA Proteins 0.000 claims description 6
- 238000000034 method Methods 0.000 claims description 6
- CITHEXJVPOWHKC-UUWRZZSWSA-N 1,2-di-O-myristoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCC CITHEXJVPOWHKC-UUWRZZSWSA-N 0.000 claims description 4
- 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 description 4
- 239000002202 Polyethylene glycol Substances 0.000 claims description 4
- 125000000217 alkyl group Chemical group 0.000 claims description 4
- 229960003724 dimyristoylphosphatidylcholine Drugs 0.000 claims description 4
- 229960005160 dimyristoylphosphatidylglycerol Drugs 0.000 claims description 4
- MWRBNPKJOOWZPW-CLFAGFIQSA-N dioleoyl phosphatidylethanolamine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC(COP(O)(=O)OCCN)OC(=O)CCCCCCC\C=C/CCCCCCCC MWRBNPKJOOWZPW-CLFAGFIQSA-N 0.000 claims description 4
- BPHQZTVXXXJVHI-AJQTZOPKSA-N ditetradecanoyl phosphatidylglycerol Chemical compound CCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@@H](O)CO)OC(=O)CCCCCCCCCCCCC BPHQZTVXXXJVHI-AJQTZOPKSA-N 0.000 claims description 4
- 239000002245 particle Substances 0.000 claims description 4
- 229920001223 polyethylene glycol Polymers 0.000 claims description 4
- 229920001184 polypeptide Polymers 0.000 claims description 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 3
- 238000010354 CRISPR gene editing Methods 0.000 claims description 3
- 108091026890 Coding region Proteins 0.000 claims description 3
- 108020004414 DNA Proteins 0.000 claims description 3
- 108020004459 Small interfering RNA Proteins 0.000 claims description 3
- 238000010459 TALEN Methods 0.000 claims description 3
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims description 3
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 3
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 3
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 3
- 239000003814 drug Substances 0.000 claims description 3
- 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 description 3
- 239000013612 plasmid Substances 0.000 claims description 3
- 201000010099 disease Diseases 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 230000009851 immunogenic response Effects 0.000 claims description 2
- 238000001727 in vivo Methods 0.000 claims description 2
- 239000002479 lipoplex Substances 0.000 claims description 2
- 239000002502 liposome Substances 0.000 claims description 2
- 229910052760 oxygen Inorganic materials 0.000 claims description 2
- 229910052717 sulfur Inorganic materials 0.000 claims description 2
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 claims 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 1
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerine Chemical compound OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 9
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 3
- 229930006000 Sucrose Natural products 0.000 description 3
- 239000003446 ligand Substances 0.000 description 3
- 239000005720 sucrose Substances 0.000 description 3
- JKMHFZQWWAIEOD-UHFFFAOYSA-N 2-[4-(2-hydroxyethyl)piperazin-1-yl]ethanesulfonic acid Chemical compound OCC[NH+]1CCN(CCS([O-])(=O)=O)CC1 JKMHFZQWWAIEOD-UHFFFAOYSA-N 0.000 description 2
- 239000007995 HEPES buffer Substances 0.000 description 2
- 108091034117 Oligonucleotide Proteins 0.000 description 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 2
- 239000002577 cryoprotective agent Substances 0.000 description 2
- QQONPFPTGQHPMA-UHFFFAOYSA-N propylene Natural products CC=C QQONPFPTGQHPMA-UHFFFAOYSA-N 0.000 description 2
- 125000004805 propylene group Chemical group [H]C([H])([H])C([H])([*:1])C([H])([H])[*:2] 0.000 description 2
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 239000011780 sodium chloride Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163185303P | 2021-05-06 | 2021-05-06 | |
| US63/185,303 | 2021-05-06 | ||
| PCT/US2022/027874 WO2022235935A2 (en) | 2021-05-06 | 2022-05-05 | Ionizable cationic lipids for rna delivery |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024518375A JP2024518375A (ja) | 2024-05-01 |
| JPWO2022235935A5 true JPWO2022235935A5 (https=) | 2025-05-13 |
| JP2024518375A5 JP2024518375A5 (https=) | 2025-05-13 |
Family
ID=83932495
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023567891A Pending JP2024518375A (ja) | 2021-05-06 | 2022-05-05 | Ｒｎａ送達のためのイオン化可能なカチオン性脂質 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220389422A1 (https=) |
| EP (1) | EP4352038A4 (https=) |
| JP (1) | JP2024518375A (https=) |
| KR (1) | KR20240008872A (https=) |
| CN (2) | CN121517316A (https=) |
| AU (1) | AU2022268975A1 (https=) |
| CA (1) | CA3219192A1 (https=) |
| TW (1) | TW202313557A (https=) |
| WO (1) | WO2022235935A2 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| IL303195A (en) | 2020-11-25 | 2023-07-01 | Akagera Medicines Inc | Lipid nanoparticles for delivery of nucleic acids and related methods of use |
| EP4619378A2 (en) * | 2022-11-16 | 2025-09-24 | Seawolf Therapeutics, Inc. | Ionizable lipids and lipid nanoparticle compositions for the delivery of nucleic acids |
| EP4683905A1 (en) * | 2023-03-22 | 2026-01-28 | Nanovation Therapeutics Inc. | Ionizable anionic lipids |
| IL324383A (en) * | 2023-05-10 | 2026-01-01 | Arcturus Therapeutics Inc | Ionizable cationic lipids for rna delivery |
| US12311033B2 (en) | 2023-05-31 | 2025-05-27 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| CN117482066B (zh) * | 2023-11-02 | 2024-08-13 | 深圳市易瑞生物技术股份有限公司 | 脂质组合物和用于脂质组合物的化合物 |
| WO2025117732A1 (en) * | 2023-11-29 | 2025-06-05 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| WO2025129201A1 (en) | 2023-12-15 | 2025-06-19 | Capstan Therapeutics, Inc. | Humanized anti-cd8 antibodies and uses thereof |
| WO2025134071A1 (en) * | 2023-12-22 | 2025-06-26 | Sanofi | Malic and glutaric acid based ionizable lipids |
| CN117820149B (zh) * | 2023-12-29 | 2025-01-07 | 北京剂泰医药科技有限公司 | 可电离脂质化合物 |
| WO2025140421A1 (zh) * | 2023-12-29 | 2025-07-03 | 北京剂泰医药科技有限公司 | 可电离脂质化合物及其在递送核酸疫苗中的用途 |
| 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 |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
| CN121021427A (zh) * | 2024-07-30 | 2025-11-28 | 广州市恒诺康医药科技有限公司 | 可离子化脂质及其用途 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW201019969A (en) * | 2008-11-17 | 2010-06-01 | Enzon Pharmaceuticals Inc | Branched cationic lipids for nucleic acids delivery system |
| US9579338B2 (en) * | 2011-11-04 | 2017-02-28 | Nitto Denko Corporation | Method of producing lipid nanoparticles for drug delivery |
| CA2853685C (en) * | 2011-11-04 | 2019-09-03 | Nitto Denko Corporation | Single use system for sterilely producing lipid-nucleic acid particles |
| EP2788316B1 (en) * | 2011-12-07 | 2019-04-24 | Alnylam Pharmaceuticals, Inc. | Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents |
| EP2788006B1 (en) * | 2011-12-07 | 2026-01-07 | Alnylam Pharmaceuticals, Inc. | Biodegradable lipids for the delivery of active agents |
| US9687448B2 (en) * | 2012-12-07 | 2017-06-27 | Alnylam Pharmaceuticals, Inc. | Nucleic acid lipid particle formulations |
| WO2015011633A1 (en) * | 2013-07-23 | 2015-01-29 | Protiva Biotherapeutics, Inc. | Compositions and methods for delivering messenger rna |
| US10383952B2 (en) * | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| JP7436990B2 (ja) * | 2018-08-10 | 2024-02-22 | 国立大学法人京都大学 | カチオン性脂質を用いた心筋細胞へのトランスフェクション方法 |
| MY202837A (en) * | 2019-01-11 | 2024-05-24 | Acuitas Therapeutics Inc | Lipids for lipid nanoparticle delivery of active agents |
| EP3942050A4 (en) * | 2019-03-19 | 2023-02-22 | Arcturus Therapeutics, Inc. | PROCEDURE FOR THE PRODUCTION OF LIPID-COATED RNA NANOPARTICLES |
| US20230097090A1 (en) * | 2019-08-14 | 2023-03-30 | Acuitas Therapeutics, Inc. | Improved lipid nanoparticles for delivery of nucleic acids |
-
2022
- 2022-05-05 KR KR1020237041979A patent/KR20240008872A/ko active Pending
- 2022-05-05 AU AU2022268975A patent/AU2022268975A1/en active Pending
- 2022-05-05 TW TW111116949A patent/TW202313557A/zh unknown
- 2022-05-05 EP EP22799597.4A patent/EP4352038A4/en active Pending
- 2022-05-05 US US17/737,690 patent/US20220389422A1/en active Pending
- 2022-05-05 CA CA3219192A patent/CA3219192A1/en active Pending
- 2022-05-05 WO PCT/US2022/027874 patent/WO2022235935A2/en not_active Ceased
- 2022-05-05 CN CN202511412284.XA patent/CN121517316A/zh active Pending
- 2022-05-05 CN CN202280043621.0A patent/CN117715886B/zh active Active
- 2022-05-05 JP JP2023567891A patent/JP2024518375A/ja active Pending
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