AU2019376006B2 - Cationic lipids containing silicon - Google Patents

Cationic lipids containing silicon

Info

Publication number
AU2019376006B2
AU2019376006B2 AU2019376006A AU2019376006A AU2019376006B2 AU 2019376006 B2 AU2019376006 B2 AU 2019376006B2 AU 2019376006 A AU2019376006 A AU 2019376006A AU 2019376006 A AU2019376006 A AU 2019376006A AU 2019376006 B2 AU2019376006 B2 AU 2019376006B2
Authority
AU
Australia
Prior art keywords
mol
lipid
nucleic acid
particle
sirna
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.)
Active
Application number
AU2019376006A
Other languages
English (en)
Other versions
AU2019376006A1 (en
Inventor
Michael J. Abrams
James Heyes
Richard J. Holland
Kieu Mong LAM
Mark Wood
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.)
Arbutus Biopharma Corp
Original Assignee
Arbutus Biopharma Corp
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 Arbutus Biopharma Corp filed Critical Arbutus Biopharma Corp
Publication of AU2019376006A1 publication Critical patent/AU2019376006A1/en
Application granted granted Critical
Publication of AU2019376006B2 publication Critical patent/AU2019376006B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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

Landscapes

  • 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)
AU2019376006A 2018-11-09 2019-11-08 Cationic lipids containing silicon Active AU2019376006B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2019376006A1 AU2019376006A1 (en) 2021-06-03
AU2019376006B2 true AU2019376006B2 (en) 2026-07-02

Family

ID=70612265

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019376006A Active AU2019376006B2 (en) 2018-11-09 2019-11-08 Cationic lipids containing silicon

Country Status (13)

Country Link
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)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108368028B (zh) 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
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)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007021946A2 (en) * 2005-08-18 2007-02-22 Carestream Health, Inc. Silylamine modified nanoparticulate carriers

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4618689A (en) 1985-07-18 1986-10-21 General Electric Company Novel aminofunctional silicone compositions
JPH08166679A (ja) * 1994-12-13 1996-06-25 Fuji Xerox Co Ltd 電子写真感光体およびのその製造方法
DE10058620A1 (de) * 2000-11-25 2002-05-29 Degussa Verfahren zur Herstellung von Aminoalkylsilanen
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
AU2004257373B2 (en) 2003-07-16 2011-03-24 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US8895717B2 (en) * 2005-04-15 2014-11-25 The Board Of Regents Of The University Of Texas System Delivery of siRNA by neutral lipid compositions
ITMI20061766A1 (it) * 2006-09-15 2008-03-16 N P T S R L Prepolimeri silano-terminati e relativi formulati adesivo-sigillanti
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Novel lipid formulations for nucleic acid delivery
AU2009303345B2 (en) * 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
ES2472668T3 (es) * 2009-08-21 2014-07-02 China Petroleum & Chemical Corporation Componente de catalizador para la polimerizaci�n de etileno, preparación del mismo y catalizador que comprende el componente de catalizador
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US9028603B2 (en) * 2011-06-09 2015-05-12 The Research Foundation Of State University Of New York Anti-fouling coating compositions and methods for preventing the fouling of surfaces
CN105873569B (zh) * 2013-11-06 2020-07-28 芝加哥大学 用于化疗剂、核酸和光敏剂的递送或共递送的纳米级载体
CN108350455A (zh) * 2015-07-29 2018-07-31 阿布特斯生物制药公司 用于使b型肝炎病毒基因表达沉默的组合物和方法
CN106834355B (zh) * 2015-12-07 2021-02-23 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
EP3269785A1 (de) * 2016-07-13 2018-01-17 Nitrochemie Aschau GmbH Zusammensetzung für silikonkautschukmassen
AU2017379901B2 (en) * 2016-12-21 2024-02-15 Arbutus Biopharma Corporation Methods for ameliorating infusion reactions
WO2019051257A2 (en) * 2017-09-11 2019-03-14 Arbutus Biopharma Corporation METHODS OF TREATING HEPATITIS B TYPE INFECTIONS
WO2020219941A1 (en) * 2019-04-26 2020-10-29 Genevant Sciences Gmbh Lipid nanoparticles
MX2023000511A (es) * 2020-07-10 2023-02-13 Genevant Sciences Gmbh Nanoparticulas lipidicas para administrar agentes terapeuticos en los pulmones.
AU2021403156A1 (en) * 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007021946A2 (en) * 2005-08-18 2007-02-22 Carestream Health, Inc. Silylamine modified nanoparticulate carriers

Also Published As

Publication number Publication date
CN113286622A (zh) 2021-08-20
EP3877001A1 (en) 2021-09-15
JP2022501064A (ja) 2022-01-06
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
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