CL2023003077A1 - Composiciones de nanopartículas lipídicas - Google Patents

Composiciones de nanopartículas lipídicas

Info

Publication number
CL2023003077A1
CL2023003077A1 CL2023003077A CL2023003077A CL2023003077A1 CL 2023003077 A1 CL2023003077 A1 CL 2023003077A1 CL 2023003077 A CL2023003077 A CL 2023003077A CL 2023003077 A CL2023003077 A CL 2023003077A CL 2023003077 A1 CL2023003077 A1 CL 2023003077A1
Authority
CL
Chile
Prior art keywords
lipids
methods
biologically active
lipid nanoparticle
delivery
Prior art date
Application number
CL2023003077A
Other languages
English (en)
Inventor
Swami Archana
Rakshe Vishal
Prodeus Aaron
Maetani Micah
Giare Parmar Rubina
Original Assignee
Intellia Therapeutics Inc
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 Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of CL2023003077A1 publication Critical patent/CL2023003077A1/es

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Classifications

    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K48/0033—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 the non-active part being non-polymeric
    • 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
    • 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
    • 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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

La descripción proporciona composiciones de nanopartículas lipídicas (LNP) de lípidos ionizables, lípidos auxiliares, lípidos neutros y lípidos PEG útiles para la administración de agentes biológicamente activos, por ejemplo, la administración de agentes biológicamente activos a las células para preparar células modificadas genéticamente. Las composiciones de LNP descritas en la presente son útiles en métodos de edición de genes y métodos para administrar un agente biológicamente activo y métodos para modificar o escindir ADN.
CL2023003077A 2021-04-17 2023-10-16 Composiciones de nanopartículas lipídicas CL2023003077A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163176227P 2021-04-17 2021-04-17
US202163254948P 2021-10-12 2021-10-12
US202163274153P 2021-11-01 2021-11-01
US202263316568P 2022-03-04 2022-03-04

Publications (1)

Publication Number Publication Date
CL2023003077A1 true CL2023003077A1 (es) 2024-04-12

Family

ID=81580499

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2023003077A CL2023003077A1 (es) 2021-04-17 2023-10-16 Composiciones de nanopartículas lipídicas

Country Status (12)

Country Link
EP (1) EP4322920A1 (es)
JP (1) JP2024515650A (es)
KR (1) KR20240017792A (es)
AU (1) AU2022257050A1 (es)
BR (1) BR112023021477A2 (es)
CA (1) CA3216877A1 (es)
CL (1) CL2023003077A1 (es)
CR (1) CR20230536A (es)
IL (1) IL307738A (es)
MX (1) MX2023012235A (es)
TW (1) TW202308597A (es)
WO (1) WO2022221697A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3234127A1 (en) * 2021-10-08 2023-04-13 Suzhou Abogen Biosciences Co., Ltd. Lipid compounds and lipid nanoparticle compositions
WO2024138189A2 (en) 2022-12-22 2024-06-27 Intellia Therapeutics, Inc. Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies
WO2024199282A1 (zh) * 2023-03-28 2024-10-03 苏州艾博生物科技有限公司 脂质化合物和脂质纳米颗粒组合物

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US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
SG10201407996PA (en) 2009-12-23 2015-01-29 Novartis Ag Lipids, lipid compositions, and methods of using them
WO2011091324A2 (en) 2010-01-22 2011-07-28 The Scripps Research Institute Methods of generating zinc finger nucleases having altered activity
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
AU2013363194B2 (en) 2012-12-17 2019-05-16 President And Fellows Of Harvard College RNA-guided human genome engineering
AU2014224205C1 (en) 2013-03-08 2019-04-04 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3872066A1 (en) 2013-12-19 2021-09-01 Novartis AG Lipids and lipid compositions for the delivery of active agents
JP6778175B2 (ja) 2014-07-16 2020-10-28 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
ES2910425T3 (es) 2015-09-17 2022-05-12 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes terapéuticos
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
US20190307795A1 (en) 2018-01-26 2019-10-10 The Board Of Trustees Of The Leland Stanford Junior University Regulatory t cells targeted with chimeric antigen receptors
CA3134544A1 (en) * 2019-03-28 2020-10-01 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis comprising a corticosteroid or use thereof
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Also Published As

Publication number Publication date
BR112023021477A2 (pt) 2024-01-30
EP4322920A1 (en) 2024-02-21
WO2022221697A1 (en) 2022-10-20
IL307738A (en) 2023-12-01
JP2024515650A (ja) 2024-04-10
MX2023012235A (es) 2024-01-23
CA3216877A1 (en) 2022-10-20
KR20240017792A (ko) 2024-02-08
AU2022257050A1 (en) 2023-11-30
CR20230536A (es) 2024-03-08
TW202308597A (zh) 2023-03-01

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