CN113728101A - 脂质纳米颗粒制剂 - Google Patents

脂质纳米颗粒制剂 Download PDF

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Publication number
CN113728101A
CN113728101A CN201980088382.9A CN201980088382A CN113728101A CN 113728101 A CN113728101 A CN 113728101A CN 201980088382 A CN201980088382 A CN 201980088382A CN 113728101 A CN113728101 A CN 113728101A
Authority
CN
China
Prior art keywords
lipid
nucleic acid
sirna
nucleotides
lipid nanoparticle
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
Application number
CN201980088382.9A
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English (en)
Chinese (zh)
Inventor
林荞梦
艾伦·D·马丁
佩特拉·施赖纳
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Protiva Biotherapeutics Inc
Original Assignee
Protiva Biotherapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Protiva Biotherapeutics Inc filed Critical Protiva Biotherapeutics Inc
Publication of CN113728101A publication Critical patent/CN113728101A/zh
Pending legal-status Critical Current

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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
    • A61K48/0033—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 non-polymeric
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • 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
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D319/00—Heterocyclic compounds containing six-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D319/04—1,3-Dioxanes; Hydrogenated 1,3-dioxanes
    • C07D319/06—1,3-Dioxanes; Hydrogenated 1,3-dioxanes not condensed with other rings

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
CN201980088382.9A 2018-11-09 2019-11-08 脂质纳米颗粒制剂 Pending CN113728101A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862758055P 2018-11-09 2018-11-09
US62/758,055 2018-11-09
PCT/US2019/060595 WO2020097548A1 (en) 2018-11-09 2019-11-08 Lipid nanoparticle formulations

Publications (1)

Publication Number Publication Date
CN113728101A true CN113728101A (zh) 2021-11-30

Family

ID=70612210

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980088382.9A Pending CN113728101A (zh) 2018-11-09 2019-11-08 脂质纳米颗粒制剂

Country Status (7)

Country Link
US (1) US20220001029A1 (https=)
EP (1) EP3877522A4 (https=)
JP (1) JP7506670B2 (https=)
CN (1) CN113728101A (https=)
AU (1) AU2019377131A1 (https=)
CA (1) CA3118559A1 (https=)
WO (1) WO2020097548A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
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CN112877275A (zh) * 2021-02-04 2021-06-01 中国农业科学院兰州兽医研究所 Hdac2基因敲除的bhk-21细胞系及其构建方法和应用
CN112980878A (zh) * 2021-02-04 2021-06-18 中国农业科学院兰州兽医研究所 Hdac8基因敲除的bhk-21细胞系及其构建方法和应用
WO2023165582A1 (zh) * 2022-03-04 2023-09-07 益杰立科(上海)生物科技有限公司 靶向递送系统和方法
CN120037202A (zh) * 2025-01-14 2025-05-27 浙江大学 一种基于连接处含有分支结构的阳离子脂质的脂质纳米颗粒及其应用

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CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
KR20210105889A (ko) 2018-11-09 2021-08-27 아뷰터스 바이오파마 코포레이션 지질 나노입자 제제
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
CN116096886A (zh) 2020-03-11 2023-05-09 欧米茄治疗公司 用于调节叉头框p3(foxp3)基因表达的组合物和方法
EP4132478A1 (en) 2020-04-09 2023-02-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
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
JP2023546175A (ja) 2020-10-14 2023-11-01 ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド 脂質ナノ粒子製造の方法及びそれに由来する組成物
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
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
KR20230053998A (ko) 2021-10-15 2023-04-24 (주)지노믹트리 mRNA 발현을 위한 유전자 구조체
WO2024009316A1 (en) * 2022-07-04 2024-01-11 Bharat Biotech International Limited Cationic lipid based composition, formulation and use for nucleic acid vaccine delivery and preparation thereof
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
EP4722368A1 (en) 2023-05-26 2026-04-08 Samsung Biologics Co., Ltd. Novel nucleic acid construct comprising poly(a) tail having secondary or tertiary structure and uses thereof
KR20250018367A (ko) 2023-07-28 2025-02-05 한국과학기술원 히스톤 코드 리더 도메인을 포함하는 폴리펩티드 및 이의 용도
WO2025144938A1 (en) 2023-12-26 2025-07-03 Emmune, Inc. Systems for nucleic acid transfer
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
WO2025210520A1 (en) * 2024-04-04 2025-10-09 Genevant Sciences Gmbh Lipid nanoparticles for inducing an immunological response
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

Citations (2)

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WO2013126803A1 (en) * 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. Trialkyl cationic lipids and methods of use thereof
US20160151284A1 (en) * 2013-07-23 2016-06-02 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna

Family Cites Families (7)

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EP2621480B1 (en) * 2010-09-30 2018-08-15 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
JP2013545727A (ja) * 2010-10-21 2013-12-26 メルク・シャープ・アンド・ドーム・コーポレーション オリゴヌクレオチド送達用の新規低分子量カチオン性脂質
US9126966B2 (en) * 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
AU2013249548A1 (en) * 2012-04-19 2014-11-06 Sirna Therapeutics, Inc. Novel diester and triester based low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
BR112017006679A2 (pt) 2014-10-02 2017-12-26 Protiva Biotherapeutics Inc moléculas, composição, partícula, composição farmacêutica, métodos para silenciar a expressão de um gene, usos de uma partícula, métodos para melhorar um ou mais sintomas, métodos para tratar uma infecção, usos de uma composição, método para inibir a replicação do vírus da hepatite d
AU2017379901B2 (en) 2016-12-21 2024-02-15 Arbutus Biopharma Corporation Methods for ameliorating infusion reactions
KR20210105889A (ko) * 2018-11-09 2021-08-27 아뷰터스 바이오파마 코포레이션 지질 나노입자 제제

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013126803A1 (en) * 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. Trialkyl cationic lipids and methods of use thereof
CN104321304A (zh) * 2012-02-24 2015-01-28 普洛体维生物治疗公司 三烷基阳离子脂质及其使用方法
US20160151284A1 (en) * 2013-07-23 2016-06-02 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112877275A (zh) * 2021-02-04 2021-06-01 中国农业科学院兰州兽医研究所 Hdac2基因敲除的bhk-21细胞系及其构建方法和应用
CN112980878A (zh) * 2021-02-04 2021-06-18 中国农业科学院兰州兽医研究所 Hdac8基因敲除的bhk-21细胞系及其构建方法和应用
CN112877275B (zh) * 2021-02-04 2023-03-31 中国农业科学院兰州兽医研究所 Hdac2基因敲除的bhk-21细胞系及其构建方法和应用
CN112980878B (zh) * 2021-02-04 2023-03-31 中国农业科学院兰州兽医研究所 Hdac8基因敲除的bhk-21细胞系及其构建方法和应用
WO2023165582A1 (zh) * 2022-03-04 2023-09-07 益杰立科(上海)生物科技有限公司 靶向递送系统和方法
CN120037202A (zh) * 2025-01-14 2025-05-27 浙江大学 一种基于连接处含有分支结构的阳离子脂质的脂质纳米颗粒及其应用
CN120037202B (zh) * 2025-01-14 2025-12-26 浙江大学 一种基于连接处含有分支结构的阳离子脂质的脂质纳米颗粒及其应用

Also Published As

Publication number Publication date
US20220001029A1 (en) 2022-01-06
JP7506670B2 (ja) 2024-06-26
JP2022506955A (ja) 2022-01-17
EP3877522A4 (en) 2022-11-02
AU2019377131A1 (en) 2021-06-03
CA3118559A1 (en) 2020-05-14
EP3877522A1 (en) 2021-09-15
WO2020097548A1 (en) 2020-05-14

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