CN114173762A - 用于mRNA递送的合成脂质 - Google Patents
用于mRNA递送的合成脂质 Download PDFInfo
- Publication number
- CN114173762A CN114173762A CN202080054813.2A CN202080054813A CN114173762A CN 114173762 A CN114173762 A CN 114173762A CN 202080054813 A CN202080054813 A CN 202080054813A CN 114173762 A CN114173762 A CN 114173762A
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- mrna
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- C07C323/10—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and singly-bound oxygen atoms bound to the same carbon skeleton
- C07C323/11—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and singly-bound oxygen atoms bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
- C07C323/12—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and singly-bound oxygen atoms bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
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- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C323/00—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
- C07C323/23—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton
- C07C323/24—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
- C07C323/25—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Nanotechnology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
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- Optics & Photonics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Dispersion Chemistry (AREA)
- Dermatology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962857111P | 2019-06-04 | 2019-06-04 | |
| US62/857111 | 2019-06-04 | ||
| PCT/US2020/036085 WO2020247604A1 (en) | 2019-06-04 | 2020-06-04 | Synthetic lipids for mrna delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN114173762A true CN114173762A (zh) | 2022-03-11 |
Family
ID=73652953
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080054813.2A Pending CN114173762A (zh) | 2019-06-04 | 2020-06-04 | 用于mRNA递送的合成脂质 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220168231A1 (https=) |
| EP (1) | EP3979988B1 (https=) |
| JP (1) | JP7691742B2 (https=) |
| KR (1) | KR20220017466A (https=) |
| CN (1) | CN114173762A (https=) |
| AU (1) | AU2020287624A1 (https=) |
| CA (1) | CA3142725A1 (https=) |
| MX (1) | MX2021014911A (https=) |
| WO (1) | WO2020247604A1 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115745788A (zh) * | 2022-11-21 | 2023-03-07 | 国家纳米科学中心 | 一种可电离脂质化合物及其制备方法和应用 |
| WO2024032677A1 (zh) * | 2022-08-11 | 2024-02-15 | 益杰立科(上海)生物科技有限公司 | 一种表观编辑靶点的方法及用途 |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3585895A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Compositions and methods for gene editing |
| 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 |
| AU2022208484A1 (en) * | 2021-01-15 | 2023-08-24 | The Brigham And Women's Hospital, Inc. | Lipid nanoparticles for targeted delivery of mrna |
| CN113402404B (zh) * | 2021-04-30 | 2022-03-11 | 苏州科锐迈德生物医药科技有限公司 | 脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂 |
| CN113209022A (zh) * | 2021-04-30 | 2021-08-06 | 中国科学院化学研究所 | 一种阳离子脂质体与制备方法及其在细菌效应蛋白的递送与抗肿瘤中的应用 |
| 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 |
| WO2023166425A1 (en) * | 2022-03-01 | 2023-09-07 | Crispr Therapeutics Ag | Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions |
| KR20240155955A (ko) * | 2022-03-04 | 2024-10-29 | 베이징 카리우스 바이오 엘티디. | 활성 분자 전달을 위한 분해성 리포솜 및 이의 나노 복합체 |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| CN120916753A (zh) * | 2023-03-29 | 2025-11-07 | 星锐医药(苏州)有限公司 | 编码治疗性多肽的核酸及包含所述核酸的脂质纳米颗粒组合物 |
| AU2024279278A1 (en) | 2023-05-31 | 2025-12-18 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025080867A1 (en) * | 2023-10-10 | 2025-04-17 | The Board Of Regents Of The University Of Texas System | Ionizable lipids for improved mrna delivery |
| WO2025182865A1 (ja) * | 2024-03-01 | 2025-09-04 | 国立大学法人東海国立大学機構 | 脂質粒子 |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101835498A (zh) * | 2007-10-05 | 2010-09-15 | 古尔比特公司 | 用于制备与亲水的生物学分布配体偶联的偕二膦酸酯稳定层覆盖的纳米颗粒的新颖的方法 |
| US20110052673A1 (en) * | 2008-01-29 | 2011-03-03 | Arthur Tzianabos | Therapeutic compositions |
| US20160129120A1 (en) * | 2013-05-14 | 2016-05-12 | Tufts University | Nanocomplexes of modified peptides or proteins |
| EP3315125A1 (en) * | 2016-10-31 | 2018-05-02 | Silence Therapeutics (London) Ltd | Lipid nanoparticle formulation |
| WO2018191750A2 (en) * | 2017-04-14 | 2018-10-18 | The Broad Institute Inc. | Novel delivery of large payloads |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101484588B (zh) | 2006-05-11 | 2013-11-06 | 阿尔尼拉姆医药品有限公司 | 抑制pcsk9基因表达的组合物和方法 |
| ES2859923T3 (es) * | 2013-02-28 | 2021-10-04 | Univ Tufts | Compuestos de disulfuro para el suministro de agentes farmacéuticos |
-
2020
- 2020-06-04 JP JP2021571709A patent/JP7691742B2/ja active Active
- 2020-06-04 AU AU2020287624A patent/AU2020287624A1/en not_active Abandoned
- 2020-06-04 KR KR1020227000096A patent/KR20220017466A/ko not_active Ceased
- 2020-06-04 EP EP20817914.3A patent/EP3979988B1/en active Active
- 2020-06-04 MX MX2021014911A patent/MX2021014911A/es unknown
- 2020-06-04 WO PCT/US2020/036085 patent/WO2020247604A1/en not_active Ceased
- 2020-06-04 CA CA3142725A patent/CA3142725A1/en active Pending
- 2020-06-04 CN CN202080054813.2A patent/CN114173762A/zh active Pending
-
2021
- 2021-12-06 US US17/543,173 patent/US20220168231A1/en active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101835498A (zh) * | 2007-10-05 | 2010-09-15 | 古尔比特公司 | 用于制备与亲水的生物学分布配体偶联的偕二膦酸酯稳定层覆盖的纳米颗粒的新颖的方法 |
| US20110052673A1 (en) * | 2008-01-29 | 2011-03-03 | Arthur Tzianabos | Therapeutic compositions |
| US20160129120A1 (en) * | 2013-05-14 | 2016-05-12 | Tufts University | Nanocomplexes of modified peptides or proteins |
| EP3315125A1 (en) * | 2016-10-31 | 2018-05-02 | Silence Therapeutics (London) Ltd | Lipid nanoparticle formulation |
| WO2018191750A2 (en) * | 2017-04-14 | 2018-10-18 | The Broad Institute Inc. | Novel delivery of large payloads |
Non-Patent Citations (2)
| Title |
|---|
| CORY D. SAGO等: "High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing" * |
| MING WANG等: "Combinatorially Designed Lipid-like Nanoparticles for Intracellular Delivery of Cytotoxic Protein for Cancer Therapy" * |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024032677A1 (zh) * | 2022-08-11 | 2024-02-15 | 益杰立科(上海)生物科技有限公司 | 一种表观编辑靶点的方法及用途 |
| CN115745788A (zh) * | 2022-11-21 | 2023-03-07 | 国家纳米科学中心 | 一种可电离脂质化合物及其制备方法和应用 |
| CN115745788B (zh) * | 2022-11-21 | 2025-06-24 | 国家纳米科学中心 | 一种可电离脂质化合物及其制备方法和应用 |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020247604A1 (en) | 2020-12-10 |
| EP3979988A1 (en) | 2022-04-13 |
| AU2020287624A1 (en) | 2022-01-06 |
| US20220168231A1 (en) | 2022-06-02 |
| JP7691742B2 (ja) | 2025-06-12 |
| KR20220017466A (ko) | 2022-02-11 |
| MX2021014911A (es) | 2022-02-21 |
| EP3979988B1 (en) | 2025-02-26 |
| CA3142725A1 (en) | 2020-12-10 |
| EP3979988A4 (en) | 2023-06-21 |
| JP2022535535A (ja) | 2022-08-09 |
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