JP2024526202A - ナノ粒子を含有する核酸 - Google Patents

ナノ粒子を含有する核酸 Download PDF

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Publication number
JP2024526202A
JP2024526202A JP2023579371A JP2023579371A JP2024526202A JP 2024526202 A JP2024526202 A JP 2024526202A JP 2023579371 A JP2023579371 A JP 2023579371A JP 2023579371 A JP2023579371 A JP 2023579371A JP 2024526202 A JP2024526202 A JP 2024526202A
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JP
Japan
Prior art keywords
nanoparticles
apolipoprotein
nucleic acid
nanoparticle
lipid
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Pending
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JP2023579371A
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English (en)
Japanese (ja)
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JP2024526202A5 (enExample
Inventor
デア メール，ロイ ヴァン
ジェイ．エム． マルダー，ウィレム
クルザ，エヴェリナ
ホフストラート，スティン
アンベルゲン，トム
コルネリス ズウォルスマン，ロビー
マリー ヤンセン，ヘンリクス
ミシェル フランセン，ピーター
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バイオ－トリップ ベーフェー
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Publication of JP2024526202A publication Critical patent/JP2024526202A/ja
Publication of JP2024526202A5 publication Critical patent/JP2024526202A5/ja
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
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/08—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
    • 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
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • 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
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • 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
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Transplantation (AREA)
  • Dermatology (AREA)
  • Dispersion Chemistry (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2023579371A 2021-06-22 2022-06-22 ナノ粒子を含有する核酸 Pending JP2024526202A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21180786 2021-06-22
EP21180786.2 2021-06-22
PCT/EP2022/067073 WO2022268913A1 (en) 2021-06-22 2022-06-22 Nucleic acid containing nanoparticles

Publications (2)

Publication Number Publication Date
JP2024526202A true JP2024526202A (ja) 2024-07-17
JP2024526202A5 JP2024526202A5 (enExample) 2025-06-27

Family

ID=76553579

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023579371A Pending JP2024526202A (ja) 2021-06-22 2022-06-22 ナノ粒子を含有する核酸

Country Status (10)

Country Link
US (1) US20240216291A1 (enExample)
EP (1) EP4358936A1 (enExample)
JP (1) JP2024526202A (enExample)
KR (1) KR20240025611A (enExample)
CN (1) CN117545467A (enExample)
AU (1) AU2022296780A1 (enExample)
CA (1) CA3222851A1 (enExample)
IL (1) IL309578A (enExample)
MX (1) MX2023014901A (enExample)
WO (1) WO2022268913A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
KR20250029793A (ko) 2022-05-24 2025-03-05 바이오-트립 비.브이. 지질 나노입자를 위한 표적화 바디를 갖는 변형된 아포리포단백질
EP4688163A1 (en) 2023-03-29 2026-02-11 Bio-TRIP B.V. Apolipoprotein lipid nanoparticle
WO2025190968A1 (en) 2024-03-11 2025-09-18 Bio-Trip B.V. Apolipoprotein lipid nanoparticle
WO2026029597A1 (ko) * 2024-07-31 2026-02-05 주식회사 메디치바이오 신규한 스테롤 지질 또는 이의 유도체를 함유하는 지질 나노입자 조성물
GB202412885D0 (en) * 2024-09-03 2024-10-16 Imperial College Innovations Ltd Three component lipid nanoparticles
CN120607455B (zh) * 2025-08-06 2025-10-21 中国科学院长春应用化学研究所 可电离脂质、其立体异构体或药学上可接受的盐、制备方法和应用
CN121059567A (zh) * 2025-11-07 2025-12-05 中国医学科学院输血研究所 一种脂质纳米颗粒及其应用、药物递送载体

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4995423B2 (ja) * 2002-12-03 2012-08-08 ブランシェット・ロックフェラー・ニューロサイエンスィズ・インスティテュート 物質を血液−脳関門を渡って輸送するための人工低密度リポタンパク質キャリア
EP2217221B1 (en) * 2007-10-17 2018-06-27 Korea Advanced Institute of Science and Technology Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US8268796B2 (en) * 2008-06-27 2012-09-18 Children's Hospital & Research Center At Oakland Lipophilic nucleic acid delivery vehicle and methods of use thereof
EP3243504A1 (en) * 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
US10525152B2 (en) * 2009-10-09 2020-01-07 Signablok, Inc. Methods and compositions for targeted imaging
EP3713548A4 (en) 2017-11-21 2021-06-23 Icahn School of Medicine at Mount Sinai Promoting trained immunity with therapeutic nanobiologic compositions
GB201800370D0 (en) * 2018-01-10 2018-02-21 Ucl Business Plc Anionic nanocomplexes for nucleic acid delivery
EP3947646A1 (en) * 2019-04-05 2022-02-09 Precision BioSciences, Inc. Methods of preparing populations of genetically-modified immune cells
WO2020219833A1 (en) * 2019-04-26 2020-10-29 Northwestern University High density lipoprotein nanoparticles and rna templated lipoprotein particles for ocular therapy

Also Published As

Publication number Publication date
EP4358936A1 (en) 2024-05-01
MX2023014901A (es) 2024-04-29
US20240216291A1 (en) 2024-07-04
IL309578A (en) 2024-02-01
KR20240025611A (ko) 2024-02-27
AU2022296780A9 (en) 2023-12-14
CA3222851A1 (en) 2022-12-29
WO2022268913A1 (en) 2022-12-29
CN117545467A (zh) 2024-02-09
AU2022296780A1 (en) 2023-12-07

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