CN114828836A - 脂质和用于药物递送的脂质纳米颗粒配方 - Google Patents

脂质和用于药物递送的脂质纳米颗粒配方 Download PDF

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CN114828836A
CN114828836A CN202080088019.XA CN202080088019A CN114828836A CN 114828836 A CN114828836 A CN 114828836A CN 202080088019 A CN202080088019 A CN 202080088019A CN 114828836 A CN114828836 A CN 114828836A
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lnp
substituted
nucleic acid
mol
cells
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Chinese (zh)
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迈克尔·米切尔
玛格丽特·比林斯利
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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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    • A61K39/00—Medicinal preparations containing antigens or antibodies
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0011—Cancer antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
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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)
  • Veterinary Medicine (AREA)
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  • Molecular Biology (AREA)
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  • 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)
CN202080088019.XA 2019-10-18 2020-10-19 脂质和用于药物递送的脂质纳米颗粒配方 Pending CN114828836A (zh)

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

Publications (1)

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CN114828836A true CN114828836A (zh) 2022-07-29

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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=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117482066A (zh) * 2023-11-02 2024-02-02 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
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
WO2025209467A1 (en) * 2024-04-01 2025-10-09 Longuide Biopharma Corporation Lipids and lipid nanoparticle formulations

Families Citing this family (10)

* 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
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
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Citations (4)

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WO2010053572A2 (en) * 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US20110256175A1 (en) * 2008-10-09 2011-10-20 The University Of British Columbia Amino lipids and methods for the delivery of nucleic acids
CN105555757A (zh) * 2013-07-23 2016-05-04 普洛体维生物治疗公司 用于递送信使rna的组合物和方法
US20190274968A1 (en) * 2016-10-27 2019-09-12 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response

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US7635707B1 (en) * 2008-11-10 2009-12-22 Intermune, Inc. Pirfenidone treatment for patients with atypical liver function

Patent Citations (5)

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US20110256175A1 (en) * 2008-10-09 2011-10-20 The University Of British Columbia Amino lipids and methods for the delivery of nucleic acids
CN102245590A (zh) * 2008-10-09 2011-11-16 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
WO2010053572A2 (en) * 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
CN105555757A (zh) * 2013-07-23 2016-05-04 普洛体维生物治疗公司 用于递送信使rna的组合物和方法
US20190274968A1 (en) * 2016-10-27 2019-09-12 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response

Non-Patent Citations (2)

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Title
ALVIN J. MUKALELA;等: "Nanoparticles for Nucleic Acid Delivery: Applications in Cancer Immunotherapy", CANCER LETT., vol. 458, 31 August 2019 (2019-08-31), pages 1 - 14 *
李燕;等: "基因治疗药物输递系统的研究现状及发展趋势", 生物化学与生物物理进展, vol. 40, no. 10, 4 December 2013 (2013-12-04), pages 998 - 1007 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN117482066A (zh) * 2023-11-02 2024-02-02 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

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JP2026012402A (ja) 2026-01-23
JP2022552009A (ja) 2022-12-14
AU2020368556A1 (en) 2022-05-19
WO2021077066A1 (en) 2021-04-22
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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