JP6893785B2 - ｍＲＮＡのＣＮＳ送達及びその使用方法 - Google Patents

ｍＲＮＡのＣＮＳ送達及びその使用方法 Download PDF

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JP6893785B2
JP6893785B2 JP2016522098A JP2016522098A JP6893785B2 JP 6893785 B2 JP6893785 B2 JP 6893785B2 JP 2016522098 A JP2016522098 A JP 2016522098A JP 2016522098 A JP2016522098 A JP 2016522098A JP 6893785 B2 JP6893785 B2 JP 6893785B2
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lipid
protein
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フランク デローザ，
フランク デローザ，
マイケル ハートレイン，
マイケル ハートレイン，
シュリラン カーブ，
シュリラン カーブ，
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トランスレイト バイオ， インコーポレイテッド
トランスレイト バイオ， インコーポレイテッド
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • A61K48/005—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical

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JP2016522098A 2013-10-22 2014-10-22 ｍＲＮＡのＣＮＳ送達及びその使用方法 Active JP6893785B2 (ja)

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US201361894246P 2013-10-22 2013-10-22
US61/894,246 2013-10-22
US201462020161P 2014-07-02 2014-07-02
US62/020,161 2014-07-02
PCT/US2014/061786 WO2015061461A1 (en) 2013-10-22 2014-10-22 Cns delivery of mrna and uses thereof

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JP2019015455A Division JP2019065047A (ja) 2013-10-22 2019-01-31 ｍＲＮＡのＣＮＳ送達及びその使用方法
JP2021020597A Division JP2021073313A (ja) 2013-10-22 2021-02-12 ｍＲＮＡのＣＮＳ送達及びその使用方法

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JP2016539916A JP2016539916A (ja) 2016-12-22
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JP2021020597A Pending JP2021073313A (ja) 2013-10-22 2021-02-12 ｍＲＮＡのＣＮＳ送達及びその使用方法
JP2022103427A Active JP7586858B2 (ja) 2013-10-22 2022-06-28 ｍＲＮＡのＣＮＳ送達及びその使用方法
JP2023016013A Pending JP2023041951A (ja) 2013-10-22 2023-02-06 ｍＲＮＡのＣＮＳ送達及びその使用方法
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JP2022103427A Active JP7586858B2 (ja) 2013-10-22 2022-06-28 ｍＲＮＡのＣＮＳ送達及びその使用方法
JP2023016013A Pending JP2023041951A (ja) 2013-10-22 2023-02-06 ｍＲＮＡのＣＮＳ送達及びその使用方法
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US (4) US20150110857A1 (https=)
EP (2) EP4036241A1 (https=)
JP (6) JP6893785B2 (https=)
CN (1) CN105658800A (https=)
AU (3) AU2014340149B2 (https=)
BR (1) BR112016008832A2 (https=)
CA (1) CA2928040A1 (https=)
EA (1) EA201690588A1 (https=)
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