JP2022501064A - ケイ素を含有するカチオン性脂質 - Google Patents

ケイ素を含有するカチオン性脂質 Download PDF

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Publication number
JP2022501064A
JP2022501064A JP2021525114A JP2021525114A JP2022501064A JP 2022501064 A JP2022501064 A JP 2022501064A JP 2021525114 A JP2021525114 A JP 2021525114A JP 2021525114 A JP2021525114 A JP 2021525114A JP 2022501064 A JP2022501064 A JP 2022501064A
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Prior art keywords
lipid
mol
nucleic acid
particles
particle according
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Pending
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JP2021525114A
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Japanese (ja)
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JP2022501064A5 (enExample
Inventor
ジェイ エイブラムス，マイケル
ヘイズ，ジェイムズ
ジェイ ホーランド，リチャード
モン ラム，キェウ
ウッド，マーク
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Arbutus Biopharma Corp
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Tekmira Pharmaceuticals Corp
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Application filed by Tekmira Pharmaceuticals Corp filed Critical Tekmira Pharmaceuticals Corp
Publication of JP2022501064A publication Critical patent/JP2022501064A/ja
Publication of JP2022501064A5 publication Critical patent/JP2022501064A5/ja
Priority to JP2024202026A priority Critical patent/JP2025019119A/ja
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02—Silicon compounds
    • C07F7/08—Compounds having one or more C—Si linkages
    • C07F7/18—Compounds having one or more C—Si linkages as well as one or more C—O—Si linkages
    • C07F7/1804—Compounds having Si-O-C linkages
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02—Silicon compounds
    • C07F7/08—Compounds having one or more C—Si linkages
    • C07F7/18—Compounds having one or more C—Si linkages as well as one or more C—O—Si linkages
    • C07F7/1804—Compounds having Si-O-C linkages
    • C07F7/1872—Preparation; Treatments not provided for in C07F7/20
    • C07F7/1888—Preparation; Treatments not provided for in C07F7/20 by reactions involving the formation of other Si-linkages, e.g. Si-N
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02—Silicon compounds
    • C07F7/08—Compounds having one or more C—Si linkages
    • C07F7/18—Compounds having one or more C—Si linkages as well as one or more C—O—Si linkages
    • C07F7/1804—Compounds having Si-O-C linkages
    • C07F7/1872—Preparation; Treatments not provided for in C07F7/20
    • C07F7/1892—Preparation; Treatments not provided for in C07F7/20 by reactions not provided for in C07F7/1876 - C07F7/1888
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medical Informatics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2021525114A 2018-11-09 2019-11-08 ケイ素を含有するカチオン性脂質 Pending JP2022501064A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024202026A JP2025019119A (ja) 2018-11-09 2024-11-20 ケイ素を含有するカチオン性脂質

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862758108P 2018-11-09 2018-11-09
US62/758,108 2018-11-09
PCT/US2019/060556 WO2020097520A1 (en) 2018-11-09 2019-11-08 Cationic lipids containing silicon

Related Child Applications (1)

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JP2024202026A Division JP2025019119A (ja) 2018-11-09 2024-11-20 ケイ素を含有するカチオン性脂質

Publications (2)

Publication Number Publication Date
JP2022501064A true JP2022501064A (ja) 2022-01-06
JP2022501064A5 JP2022501064A5 (enExample) 2022-11-04

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JP2021525114A Pending JP2022501064A (ja) 2018-11-09 2019-11-08 ケイ素を含有するカチオン性脂質
JP2024202026A Pending JP2025019119A (ja) 2018-11-09 2024-11-20 ケイ素を含有するカチオン性脂質

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JP2024202026A Pending JP2025019119A (ja) 2018-11-09 2024-11-20 ケイ素を含有するカチオン性脂質

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US (2) US12350383B2 (enExample)
EP (1) EP3877001A4 (enExample)
JP (2) JP2022501064A (enExample)
KR (1) KR20210105888A (enExample)
CN (1) CN113286622A (enExample)
AU (1) AU2019376006B2 (enExample)
BR (1) BR112021008953A2 (enExample)
EA (1) EA202191315A1 (enExample)
IL (1) IL282957A (enExample)
MX (1) MX2021005462A (enExample)
SG (1) SG11202104682PA (enExample)
WO (1) WO2020097520A1 (enExample)
ZA (1) ZA202103229B (enExample)

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US12678515B2 (en) 2015-12-30 2026-07-14 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
DK3864163T3 (da) 2018-10-09 2024-05-06 Univ British Columbia Sammensætninger og systemer omfattende transfektionskompetente vesikler fri for organiske opløsningsmidler og rengøringsmidler og fremgangsmåder forbundet dermed
CN112888451A (zh) * 2018-10-19 2021-06-01 川斯勒佰尔公司 信使rna的无泵包封
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 Sepia Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
WO2021183720A1 (en) 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
MX2023000511A (es) * 2020-07-10 2023-02-13 Genevant Sciences Gmbh Nanoparticulas lipidicas para administrar agentes terapeuticos en los pulmones.
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
WO2023283359A2 (en) 2021-07-07 2023-01-12 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
EP4716741A2 (en) 2023-05-23 2026-04-01 Flagship Labs 114, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
AU2025217182A1 (en) 2024-02-02 2026-08-06 Editas Medicine, Inc. MODIFIED GUIDE RNAs
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
WO2026022677A1 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Permanently charged cationic lipids
WO2026022656A2 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Lipid particle formulations with permanently charged cationic lipids
CN121622610A (zh) * 2024-09-05 2026-03-10 苏州慧疗生物医药科技有限公司 向细胞内递送遗传物质的三组分脂质纳米颗粒及组合物
WO2026102260A1 (en) 2024-11-08 2026-05-15 Editas Medicine, Inc. Crispr-related methods and compositions targeting angiopoietin like 3 (angptl3)

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JP2007528863A (ja) * 2003-07-16 2007-10-18 プロチバ バイオセラピューティクス インコーポレイティッド 脂質に封入された干渉ｒｎａ
JP2009504742A (ja) * 2005-08-18 2009-02-05 ケアストリーム ヘルス，インコーポレイティド シリルアミンで改質されたナノ粒子状キャリヤ
JP2012505250A (ja) * 2008-10-09 2012-03-01 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
US20120312192A1 (en) * 2011-06-09 2012-12-13 Michael Ray Detty Anti-Fouling Coating Compositions and Methods For Preventing the Fouling of Surfaces
CN106834355A (zh) * 2015-12-07 2017-06-13 北京大学 一种阳离子复合脂质纳米盘及其制备方法和用途
WO2018011360A1 (de) * 2016-07-13 2018-01-18 Nitrochemie Aschau Gmbh Zusammensetzung für silikonkautschukmassen
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JP2002173494A (ja) * 2000-11-25 2002-06-21 Degussa Ag アミノアルキルシランの製造方法
JP2007528863A (ja) * 2003-07-16 2007-10-18 プロチバ バイオセラピューティクス インコーポレイティッド 脂質に封入された干渉ｒｎａ
JP2009504742A (ja) * 2005-08-18 2009-02-05 ケアストリーム ヘルス，インコーポレイティド シリルアミンで改質されたナノ粒子状キャリヤ
JP2012505250A (ja) * 2008-10-09 2012-03-01 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
US20120312192A1 (en) * 2011-06-09 2012-12-13 Michael Ray Detty Anti-Fouling Coating Compositions and Methods For Preventing the Fouling of Surfaces
US20180208932A1 (en) * 2015-07-29 2018-07-26 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
CN106834355A (zh) * 2015-12-07 2017-06-13 北京大学 一种阳离子复合脂质纳米盘及其制备方法和用途
WO2018011360A1 (de) * 2016-07-13 2018-01-18 Nitrochemie Aschau Gmbh Zusammensetzung für silikonkautschukmassen

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CN113286622A (zh) 2021-08-20
EP3877001A1 (en) 2021-09-15
BR112021008953A2 (pt) 2021-08-10
KR20210105888A (ko) 2021-08-27
WO2020097520A1 (en) 2020-05-14
ZA202103229B (en) 2025-08-27
CA3119051A1 (en) 2020-05-14
NZ776058A (en) 2025-07-25
AU2019376006A1 (en) 2021-06-03
EA202191315A1 (ru) 2021-09-08
US20250281422A1 (en) 2025-09-11
AU2019376006B2 (en) 2026-07-02
US20220133636A1 (en) 2022-05-05
EP3877001A4 (en) 2022-08-17
IL282957A (en) 2021-06-30
MX2021005462A (es) 2021-08-24
JP2025019119A (ja) 2025-02-06
US12350383B2 (en) 2025-07-08
SG11202104682PA (en) 2021-06-29

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