BR112021008953A2 - partículas de ácido nucleico-lipídio e de ácido-lipídio, composições, métodos para introduzir um ácido nucleico em uma célula, para distribuição in vivo de um ácido nucleico, para tratar uma doença ou um distúrbio e para preparar um composto, usos de uma partícula, compostos e nanopartícula de lipídio - Google Patents

partículas de ácido nucleico-lipídio e de ácido-lipídio, composições, métodos para introduzir um ácido nucleico em uma célula, para distribuição in vivo de um ácido nucleico, para tratar uma doença ou um distúrbio e para preparar um composto, usos de uma partícula, compostos e nanopartícula de lipídio Download PDF

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Publication number
BR112021008953A2
BR112021008953A2 BR112021008953-5A BR112021008953A BR112021008953A2 BR 112021008953 A2 BR112021008953 A2 BR 112021008953A2 BR 112021008953 A BR112021008953 A BR 112021008953A BR 112021008953 A2 BR112021008953 A2 BR 112021008953A2
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BR
Brazil
Prior art keywords
particle
nucleic acid
lipid
mol
lipidium
Prior art date
Application number
BR112021008953-5A
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English (en)
Portuguese (pt)
Inventor
Michael J. Abrams
James Heyes
Richard J. Holland
Kieu Mong LAM
Mark Wood
Original Assignee
Arbutus Biopharma Corporation
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 Corporation filed Critical Arbutus Biopharma Corporation
Publication of BR112021008953A2 publication Critical patent/BR112021008953A2/pt

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    • 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
    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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

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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
BR112021008953-5A 2018-11-09 2019-11-08 partículas de ácido nucleico-lipídio e de ácido-lipídio, composições, métodos para introduzir um ácido nucleico em uma célula, para distribuição in vivo de um ácido nucleico, para tratar uma doença ou um distúrbio e para preparar um composto, usos de uma partícula, compostos e nanopartícula de lipídio BR112021008953A2 (pt)

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 (1)

Publication Number Publication Date
BR112021008953A2 true BR112021008953A2 (pt) 2021-08-10

Family

ID=70612265

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021008953-5A BR112021008953A2 (pt) 2018-11-09 2019-11-08 partículas de ácido nucleico-lipídio e de ácido-lipídio, composições, métodos para introduzir um ácido nucleico em uma célula, para distribuição in vivo de um ácido nucleico, para tratar uma doença ou um distúrbio e para preparar um composto, usos de uma partícula, compostos e nanopartícula de lipídio

Country Status (14)

Country Link
US (2) US12350383B2 (https=)
EP (1) EP3877001A4 (https=)
JP (2) JP2022501064A (https=)
KR (1) KR20210105888A (https=)
CN (1) CN113286622A (https=)
AU (1) AU2019376006A1 (https=)
BR (1) BR112021008953A2 (https=)
CA (1) CA3119051A1 (https=)
EA (1) EA202191315A1 (https=)
IL (1) IL282957A (https=)
MX (1) MX2021005462A (https=)
SG (1) SG11202104682PA (https=)
WO (1) WO2020097520A1 (https=)
ZA (1) ZA202103229B (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
CA3112837A1 (en) * 2018-10-19 2020-04-23 Translate Bio, Inc. Pumpless encapsulation of messenger rna
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
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
CN116096886A (zh) 2020-03-11 2023-05-09 欧米茄治疗公司 用于调节叉头框p3(foxp3)基因表达的组合物和方法
AU2021305214A1 (en) * 2020-07-10 2023-02-23 Genevant Sciences Gmbh Lipid nanoparticles for delivering therapeutics to lungs
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
EP4367242A2 (en) 2021-07-07 2024-05-15 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
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, 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
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
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
WO2026022656A2 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Lipid particle formulations with permanently charged cationic lipids
WO2026022677A1 (en) 2024-07-22 2026-01-29 Genevant Sciences Gmbh Permanently charged cationic lipids

Family Cites Families (22)

* 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
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
WO2006113679A2 (en) * 2005-04-15 2006-10-26 Board Of Regents, The University Of Texas System Delivery of sirna by neutral lipid compositions
US20070292688A1 (en) * 2005-08-18 2007-12-20 Eastman Kodak Company Silylamine modified nanoparticulate carriers
ITMI20061766A1 (it) * 2006-09-15 2008-03-16 N P T S R L Prepolimeri silano-terminati e relativi formulati adesivo-sigillanti
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
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
MY156621A (en) * 2009-08-21 2016-03-15 China Petroleum & Chemical A catalyst component for ethylene polymerization, preparation thereof and a catalyst comprising the catalyst component
WO2012000104A1 (en) 2010-06-30 2012-01-05 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
US10517822B2 (en) * 2013-11-06 2019-12-31 The University Of Chicago Nanoscale carriers for the delivery or co-delivery of chemotherapeutics, nucleic acids and photosensitizers
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
AU2020262437A1 (en) 2019-04-26 2021-12-23 Genevant Sciences Gmbh Lipid nanoparticles
AU2021305214A1 (en) * 2020-07-10 2023-02-23 Genevant Sciences Gmbh Lipid nanoparticles for delivering therapeutics to lungs
AU2021403156A1 (en) * 2020-12-18 2023-07-13 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles

Also Published As

Publication number Publication date
KR20210105888A (ko) 2021-08-27
CA3119051A1 (en) 2020-05-14
WO2020097520A1 (en) 2020-05-14
EA202191315A1 (ru) 2021-09-08
AU2019376006A1 (en) 2021-06-03
IL282957A (en) 2021-06-30
CN113286622A (zh) 2021-08-20
US12350383B2 (en) 2025-07-08
SG11202104682PA (en) 2021-06-29
NZ776058A (en) 2025-07-25
JP2022501064A (ja) 2022-01-06
EP3877001A4 (en) 2022-08-17
US20220133636A1 (en) 2022-05-05
MX2021005462A (es) 2021-08-24
JP2025019119A (ja) 2025-02-06
US20250281422A1 (en) 2025-09-11
ZA202103229B (en) 2025-08-27
EP3877001A1 (en) 2021-09-15

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