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MX2019005470A - Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm. - Google Patents

Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm.

Info

Publication number
MX2019005470A
MX2019005470A MX2019005470A MX2019005470A MX2019005470A MX 2019005470 A MX2019005470 A MX 2019005470A MX 2019005470 A MX2019005470 A MX 2019005470A MX 2019005470 A MX2019005470 A MX 2019005470A MX 2019005470 A MX2019005470 A MX 2019005470A
Authority
MX
Mexico
Prior art keywords
mrna
delivery
lipid nanoparticle
nanoparticle formulation
based lipid
Prior art date
Application number
MX2019005470A
Other languages
English (en)
Inventor
Karve Shrirang
Derosa Frank
Heartlein Michael
Original Assignee
Translate Bio 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 Translate Bio Inc filed Critical Translate Bio Inc
Publication of MX2019005470A publication Critical patent/MX2019005470A/es

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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
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    • 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
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    • 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
    • A61K48/0041—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 polymeric
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
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    • 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
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  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
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  • Biochemistry (AREA)
  • Pulmonology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Organic Chemistry (AREA)
  • Otolaryngology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
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  • Botany (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente invencion proporciona, entre otras cosas, composiciones y metodos para formular nanoparticulas que contienen acidos nucleicos que comprenden no mas de tres componentes de lipidos distintos, un componente lipidico distinto que es un lipido cationico basado en esteroles; en algunas modalidades, la presente invencion proporciona composiciones y métodos en los cuales las nanoparticulas lipidicas comprenden ademas lipidos auxiliares y lipidos modificados con PEG; la formulacion resultante comprende un porcentaje de encapsulacion alto de acidos nucleicos.
MX2019005470A 2016-11-10 2017-11-10 Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm. MX2019005470A (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662420421P 2016-11-10 2016-11-10
US201662420428P 2016-11-10 2016-11-10
US201662421021P 2016-11-11 2016-11-11
US201662421007P 2016-11-11 2016-11-11
US201762464327P 2017-02-27 2017-02-27
US201762464330P 2017-02-27 2017-02-27
PCT/US2017/061100 WO2018089790A1 (en) 2016-11-10 2017-11-10 Improved ice-based lipid nanoparticle formulation for delivery of mrna

Publications (1)

Publication Number Publication Date
MX2019005470A true MX2019005470A (es) 2019-11-21

Family

ID=62065903

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019005470A MX2019005470A (es) 2016-11-10 2017-11-10 Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm.

Country Status (10)

Country Link
US (5) US10471153B2 (es)
EP (1) EP3538068A1 (es)
KR (1) KR20190120160A (es)
CN (2) CN110114058B (es)
AU (2) AU2017357748B2 (es)
CA (1) CA3043033A1 (es)
IL (2) IL310050A (es)
MA (1) MA46756A (es)
MX (1) MX2019005470A (es)
WO (1) WO2018089790A1 (es)

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