JPWO2021077066A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021077066A5 JPWO2021077066A5 JP2022523031A JP2022523031A JPWO2021077066A5 JP WO2021077066 A5 JPWO2021077066 A5 JP WO2021077066A5 JP 2022523031 A JP2022523031 A JP 2022523031A JP 2022523031 A JP2022523031 A JP 2022523031A JP WO2021077066 A5 JPWO2021077066 A5 JP WO2021077066A5
- Authority
- JP
- Japan
- Prior art keywords
- lnp
- substituted
- composition
- nucleic acid
- group
- 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
- 239000000427 antigen Substances 0.000 claims 18
- 108091007433 antigens Proteins 0.000 claims 18
- 102000036639 antigens Human genes 0.000 claims 18
- 108020004707 nucleic acids Proteins 0.000 claims 18
- 102000039446 nucleic acids Human genes 0.000 claims 18
- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 150000001875 compounds Chemical class 0.000 claims 15
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims 12
- -1 substituted Chemical class 0.000 claims 10
- 210000001519 tissue Anatomy 0.000 claims 9
- 125000000392 cycloalkenyl group Chemical group 0.000 claims 8
- 239000003814 drug Substances 0.000 claims 8
- 125000001072 heteroaryl group Chemical group 0.000 claims 8
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 8
- 238000000034 method Methods 0.000 claims 8
- 229940124597 therapeutic agent Drugs 0.000 claims 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 7
- 125000000217 alkyl group Chemical group 0.000 claims 6
- 150000002632 lipids Chemical class 0.000 claims 6
- 108020004999 messenger RNA Proteins 0.000 claims 6
- 206010028980 Neoplasm Diseases 0.000 claims 5
- 229910052799 carbon Inorganic materials 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 5
- DHMQDGOQFOQNFH-UHFFFAOYSA-M Aminoacetate Chemical compound NCC([O-])=O DHMQDGOQFOQNFH-UHFFFAOYSA-M 0.000 claims 4
- 125000002252 acyl group Chemical group 0.000 claims 4
- 125000003342 alkenyl group Chemical group 0.000 claims 4
- 125000003545 alkoxy group Chemical group 0.000 claims 4
- 125000000304 alkynyl group Chemical group 0.000 claims 4
- 125000005021 aminoalkenyl group Chemical group 0.000 claims 4
- 125000005014 aminoalkynyl group Chemical group 0.000 claims 4
- 125000005001 aminoaryl group Chemical group 0.000 claims 4
- 125000003118 aryl group Chemical group 0.000 claims 4
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 claims 4
- 150000007942 carboxylates Chemical class 0.000 claims 4
- 125000000753 cycloalkyl group Chemical group 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 4
- 150000002148 esters Chemical class 0.000 claims 4
- 229910052736 halogen Inorganic materials 0.000 claims 4
- 150000002367 halogens Chemical class 0.000 claims 4
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 4
- 125000005020 hydroxyalkenyl group Chemical group 0.000 claims 4
- 125000002768 hydroxyalkyl group Chemical group 0.000 claims 4
- 125000005016 hydroxyalkynyl group Chemical group 0.000 claims 4
- 125000005027 hydroxyaryl group Chemical group 0.000 claims 4
- 229910052757 nitrogen Inorganic materials 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 4
- 125000005017 substituted alkenyl group Chemical group 0.000 claims 4
- 125000000547 substituted alkyl group Chemical group 0.000 claims 4
- 125000004426 substituted alkynyl group Chemical group 0.000 claims 4
- 125000003107 substituted aryl group Chemical group 0.000 claims 4
- 125000005346 substituted cycloalkyl group Chemical group 0.000 claims 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 3
- 125000003368 amide group Chemical group 0.000 claims 3
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Natural products 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 3
- 208000035475 disorder Diseases 0.000 claims 3
- 206010022000 influenza Diseases 0.000 claims 3
- 150000003904 phospholipids Chemical class 0.000 claims 3
- 229920000642 polymer Polymers 0.000 claims 3
- 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 2
- LVNGJLRDBYCPGB-UHFFFAOYSA-N 1,2-distearoylphosphatidylethanolamine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OCC(COP([O-])(=O)OCC[NH3+])OC(=O)CCCCCCCCCCCCCCCCC LVNGJLRDBYCPGB-UHFFFAOYSA-N 0.000 claims 2
- 108020004414 DNA Proteins 0.000 claims 2
- 102000053602 DNA Human genes 0.000 claims 2
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 2
- 108060001084 Luciferase Proteins 0.000 claims 2
- 239000005089 Luciferase Substances 0.000 claims 2
- 208000012902 Nervous system disease Diseases 0.000 claims 2
- 208000025966 Neurological disease Diseases 0.000 claims 2
- 239000002202 Polyethylene glycol Substances 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 210000001744 T-lymphocyte Anatomy 0.000 claims 2
- 239000002671 adjuvant Substances 0.000 claims 2
- 125000004103 aminoalkyl group Chemical group 0.000 claims 2
- 230000000692 anti-sense effect Effects 0.000 claims 2
- 230000001580 bacterial effect Effects 0.000 claims 2
- 235000012000 cholesterol Nutrition 0.000 claims 2
- 150000001841 cholesterols Chemical class 0.000 claims 2
- 239000002299 complementary DNA Substances 0.000 claims 2
- 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 2
- 230000002538 fungal effect Effects 0.000 claims 2
- 208000016361 genetic disease Diseases 0.000 claims 2
- 108091070501 miRNA Proteins 0.000 claims 2
- 239000002679 microRNA Substances 0.000 claims 2
- 229910052760 oxygen Inorganic materials 0.000 claims 2
- 230000003071 parasitic effect Effects 0.000 claims 2
- 229920001223 polyethylene glycol Polymers 0.000 claims 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 2
- 230000001105 regulatory effect Effects 0.000 claims 2
- 229910052717 sulfur Inorganic materials 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 229960005486 vaccine Drugs 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 claims 1
- 208000023275 Autoimmune disease Diseases 0.000 claims 1
- 208000035143 Bacterial infection Diseases 0.000 claims 1
- 208000024172 Cardiovascular disease Diseases 0.000 claims 1
- 206010017533 Fungal infection Diseases 0.000 claims 1
- 208000031888 Mycoses Diseases 0.000 claims 1
- 208000030852 Parasitic disease Diseases 0.000 claims 1
- 208000036142 Viral infection Diseases 0.000 claims 1
- 150000001408 amides Chemical class 0.000 claims 1
- 206010003246 arthritis Diseases 0.000 claims 1
- 210000003719 b-lymphocyte Anatomy 0.000 claims 1
- 208000022362 bacterial infectious disease Diseases 0.000 claims 1
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 210000005013 brain tissue Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 210000001072 colon Anatomy 0.000 claims 1
- 210000002889 endothelial cell Anatomy 0.000 claims 1
- 210000002919 epithelial cell Anatomy 0.000 claims 1
- 210000003608 fece Anatomy 0.000 claims 1
- 230000008175 fetal development Effects 0.000 claims 1
- 230000001605 fetal effect Effects 0.000 claims 1
- 238000001476 gene delivery Methods 0.000 claims 1
- 208000019622 heart disease Diseases 0.000 claims 1
- 210000002865 immune cell Anatomy 0.000 claims 1
- 230000000968 intestinal effect Effects 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 210000005067 joint tissue Anatomy 0.000 claims 1
- 210000002429 large intestine Anatomy 0.000 claims 1
- 230000003902 lesion Effects 0.000 claims 1
- 210000005228 liver tissue Anatomy 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 230000032630 lymph circulation Effects 0.000 claims 1
- 210000001165 lymph node Anatomy 0.000 claims 1
- 210000002540 macrophage Anatomy 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 229910052698 phosphorus Inorganic materials 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
- 210000000813 small intestine Anatomy 0.000 claims 1
- 210000000952 spleen Anatomy 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 210000004881 tumor cell Anatomy 0.000 claims 1
- 230000009385 viral infection Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025185622A JP2026012402A (ja) | 2019-10-18 | 2025-11-04 | 薬物送達のための脂質及び脂質ナノ粒子製剤 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962923258P | 2019-10-18 | 2019-10-18 | |
| US62/923,258 | 2019-10-18 | ||
| PCT/US2020/056252 WO2021077066A1 (en) | 2019-10-18 | 2020-10-19 | Lipid and lipid nanoparticle formulation for drug delivery |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025185622A Division JP2026012402A (ja) | 2019-10-18 | 2025-11-04 | 薬物送達のための脂質及び脂質ナノ粒子製剤 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022552009A JP2022552009A (ja) | 2022-12-14 |
| JP2022552009A5 JP2022552009A5 (https=) | 2023-10-23 |
| JPWO2021077066A5 true JPWO2021077066A5 (https=) | 2023-10-23 |
Family
ID=75538682
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523031A Pending JP2022552009A (ja) | 2019-10-18 | 2020-10-19 | 薬物送達のための脂質及び脂質ナノ粒子製剤 |
| JP2025185622A Pending JP2026012402A (ja) | 2019-10-18 | 2025-11-04 | 薬物送達のための脂質及び脂質ナノ粒子製剤 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025185622A Pending JP2026012402A (ja) | 2019-10-18 | 2025-11-04 | 薬物送達のための脂質及び脂質ナノ粒子製剤 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220396556A1 (https=) |
| EP (1) | EP4045020A4 (https=) |
| JP (2) | JP2022552009A (https=) |
| KR (1) | KR20220084365A (https=) |
| CN (1) | CN114828836A (https=) |
| AU (1) | AU2020368556A1 (https=) |
| CA (1) | CA3155074A1 (https=) |
| WO (1) | WO2021077066A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL303195A (en) | 2020-11-25 | 2023-07-01 | Akagera Medicines Inc | Lipid nanoparticles for delivery of nucleic acids and related methods of use |
| CN113785975B (zh) * | 2021-09-09 | 2023-08-29 | 天津医科大学 | 精胺、亚精胺脂质体在抗氧化、抗衰老中的应用 |
| AR128959A1 (es) * | 2022-04-04 | 2024-06-26 | Spark Therapeutics Inc | Mejora inmunológica del tratamiento del cáncer |
| US20230322689A1 (en) * | 2022-04-08 | 2023-10-12 | SunVax mRNA Therapeutics Inc. | Ionizable lipid compounds and lipid nanoparticle compositions |
| EP4531819A2 (en) | 2022-05-25 | 2025-04-09 | Akagera Medicines, Inc. | Lipid nanoparticles for delivery of nucleic acids and methods of use thereof |
| WO2024061354A1 (zh) * | 2022-09-23 | 2024-03-28 | 北京键凯科技股份有限公司 | 一种抑制top1基因表达的干扰rna及其应用 |
| AU2024231082A1 (en) * | 2023-03-09 | 2025-10-16 | Maxirna (Shanghai) Pharmaceutical Co., Ltd. | Ionizable lipid molecule, preparation method therefor and use thereof |
| CN116082184B (zh) * | 2023-04-12 | 2023-06-30 | 山东大学 | 基于环己二胺的可电离脂质、脂质纳米颗粒及其制备方法与应用 |
| WO2025038494A1 (en) | 2023-08-11 | 2025-02-20 | Tune Therapeutics, Inc. | Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation |
| WO2025059112A1 (en) * | 2023-09-11 | 2025-03-20 | The Trustees Of The University Of Pennsylvania | Compositions and methods for t cell targeted extrahepatic delivery of therapeutic agents |
| 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 |
| WO2025209467A1 (en) * | 2024-04-01 | 2025-10-09 | Longuide Biopharma Corporation | Lipids and lipid nanoparticle formulations |
| WO2026003582A2 (en) | 2024-06-27 | 2026-01-02 | Axelyf ehf. | Lipids and lipid nanoparticles |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| ES2646630T3 (es) * | 2008-11-07 | 2017-12-14 | Massachusetts Institute Of Technology | Lipidoides aminoalcohólicos y usos de los mismos |
| US7635707B1 (en) * | 2008-11-10 | 2009-12-22 | Intermune, Inc. | Pirfenidone treatment for patients with atypical liver function |
| WO2015011633A1 (en) * | 2013-07-23 | 2015-01-29 | Protiva Biotherapeutics, Inc. | Compositions and methods for delivering messenger rna |
| WO2018081638A1 (en) * | 2016-10-27 | 2018-05-03 | The Trustees Of The University Of Pennsylvania | Nucleoside-modified rna for inducing an adaptive immune response |
-
2020
- 2020-10-19 AU AU2020368556A patent/AU2020368556A1/en active Pending
- 2020-10-19 WO PCT/US2020/056252 patent/WO2021077066A1/en not_active Ceased
- 2020-10-19 CA CA3155074A patent/CA3155074A1/en active Pending
- 2020-10-19 KR KR1020227016630A patent/KR20220084365A/ko not_active Ceased
- 2020-10-19 US US17/769,858 patent/US20220396556A1/en active Pending
- 2020-10-19 JP JP2022523031A patent/JP2022552009A/ja active Pending
- 2020-10-19 CN CN202080088019.XA patent/CN114828836A/zh active Pending
- 2020-10-19 EP EP20877084.2A patent/EP4045020A4/en active Pending
-
2025
- 2025-11-04 JP JP2025185622A patent/JP2026012402A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2021077067A5 (https=) | ||
| JP2025175052A5 (https=) | ||
| JP2026012402A5 (https=) | ||
| JP7641308B2 (ja) | Ｒｎａ送達のためのイオン化可能なカチオン性脂質 | |
| JPWO2021077066A5 (https=) | ||
| US10626393B2 (en) | Delivering CRISPR therapeutics with lipid nanoparticles | |
| US20180245074A1 (en) | Treating hepatitis b virus infection using crispr | |
| JPWO2020051223A5 (https=) | ||
| JP2012509258A (ja) | 核酸送達系のための分岐カチオン性脂質 | |
| JP2012509366A (ja) | 核酸送達系のための放出可能ポリマー脂質 | |
| JP2011126892A (ja) | 脂質に封入された干渉ｒｎａ | |
| FI4426270T3 (fi) | Lipidi-nanopartikkelit oligonukleotidien kuljetusta varten | |
| JP2024105237A (ja) | 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法 | |
| CA3198599A1 (en) | Tissue-specific nucleic acid delivery by 1,2-dioleoyl-3-trimethylammonium-propane (dotap) lipid nanoparticles | |
| EP4667004A1 (en) | Lipid composition targeting antigen-presenting cells and use thereof | |
| Lee et al. | Tetraiodothyroacetic acid-tagged liposomes for enhanced delivery of anticancer drug to tumor tissue via integrin receptor | |
| JP2025541727A (ja) | 新規のポリグリセロールコンジュゲート脂質及びそれを含む脂質ナノ粒子組成物 | |
| WO2018232330A1 (en) | Therapeutic compositions and methods for treating hepatitis b | |
| CN120835876A (zh) | 包括具有支化结构的电离脂质的脂质纳米粒制剂及其用途 | |
| RU2476204C2 (ru) | Носитель лекарственного средства | |
| US10722599B2 (en) | Lipid assemblies and uses thereof and some pH and electrostatic modulating lipids to be used in said assemblies | |
| WO2023085440A1 (ja) | 難治性ウイルス感染症の治療剤 | |
| JP2005513073A (ja) | ポリヌクレオチドを細胞へトランスフェクションするための組成物の製造のためのリゾ脂質の使用 | |
| KR20220026197A (ko) | 세포 투과성 핵산 복합체를 유효성분으로 함유하는 류마티스 관절염의 예방 또는 치료용 조성물 | |
| JP2002533354A (ja) | 安定なポジプレックスの懸濁液の製造法 |