JP2022552009A - 薬物送達のための脂質及び脂質ナノ粒子製剤 - Google Patents

薬物送達のための脂質及び脂質ナノ粒子製剤 Download PDF

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JP2022552009A
JP2022552009A JP2022523031A JP2022523031A JP2022552009A JP 2022552009 A JP2022552009 A JP 2022552009A JP 2022523031 A JP2022523031 A JP 2022523031A JP 2022523031 A JP2022523031 A JP 2022523031A JP 2022552009 A JP2022552009 A JP 2022552009A
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lnp
substituted
mol
nucleic acid
antigens
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JP2022552009A5 (https=
JPWO2021077066A5 (https=
Inventor
ミッチェル マイケル
ビリンズリー マーガレット
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ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
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Publication of JP2022552009A publication Critical patent/JP2022552009A/ja
Publication of JP2022552009A5 publication Critical patent/JP2022552009A5/ja
Publication of JPWO2021077066A5 publication Critical patent/JPWO2021077066A5/ja
Priority to JP2025185622A priority Critical patent/JP2026012402A/ja
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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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/12—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/125—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/13—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/12—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/135—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms separated by carbocyclic rings or by carbon chains interrupted by carbocyclic rings
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Medicinal Chemistry (AREA)
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  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
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  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2022523031A 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 Pending JP2022552009A (ja)

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

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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

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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

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JP2022552009A true JP2022552009A (ja) 2022-12-14
JP2022552009A5 JP2022552009A5 (https=) 2023-10-23
JPWO2021077066A5 JPWO2021077066A5 (https=) 2023-10-23

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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

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Country Status (8)

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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)

* Cited by examiner, † Cited by third party
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

Citations (1)

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JP2012508253A (ja) * 2008-11-10 2012-04-05 インターミューン， インコーポレイテッド 異型肝機能をもつ患者に対するピルフェニドン治療

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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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012508253A (ja) * 2008-11-10 2012-04-05 インターミューン， インコーポレイテッド 異型肝機能をもつ患者に対するピルフェニドン治療

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CANCER LETTERS, vol. 458, JPN6024038516, 2019, pages 102 - 112, ISSN: 0005630745 *
J.CHEM.PHAEM.SCI., vol. 27(9), JPN6024038515, 2018, pages 608 - 616, ISSN: 0005630744 *
NANO LETTERS, vol. 17(3), JPN6024038517, 2017, pages 1326 - 1335, ISSN: 0005630746 *
NAT REV DRUG DISCOV, vol. 18(3), JPN6024038518, 2019, pages 175 - 196, ISSN: 0005630747 *

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Publication number Publication date
JP2026012402A (ja) 2026-01-23
AU2020368556A1 (en) 2022-05-19
WO2021077066A1 (en) 2021-04-22
CN114828836A (zh) 2022-07-29
CA3155074A1 (en) 2021-04-22
EP4045020A4 (en) 2024-02-21
US20220396556A1 (en) 2022-12-15
KR20220084365A (ko) 2022-06-21
EP4045020A1 (en) 2022-08-24

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