MX2015011943A - Acidos ribonucleicos con nucleotidos modificados por 4'-tio y metodos relacionados. - Google Patents

Acidos ribonucleicos con nucleotidos modificados por 4'-tio y metodos relacionados.

Info

Publication number
MX2015011943A
MX2015011943A MX2015011943A MX2015011943A MX2015011943A MX 2015011943 A MX2015011943 A MX 2015011943A MX 2015011943 A MX2015011943 A MX 2015011943A MX 2015011943 A MX2015011943 A MX 2015011943A MX 2015011943 A MX2015011943 A MX 2015011943A
Authority
MX
Mexico
Prior art keywords
thio
modified nucleotides
related methods
ribonucleic acids
vivo
Prior art date
Application number
MX2015011943A
Other languages
English (en)
Inventor
Michael Heartlein
Frank Derosa
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of MX2015011943A publication Critical patent/MX2015011943A/es

Links

Classifications

    • 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
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Abstract

Se describen moléculas de ARN mensajero y composiciones relacionadas que incorporan una modificación de 4'-tio en el anillo de furanosa de al menos un residuo de nucleótidos y métodos para utilizar estos ARNm para producir una proteína terapéutica codificada in vivo y para tratar o prevenir enfermedades o trastornos; en determinadas modalidades, el ARNm modificado por 4'-tio proporciona estabilidad mejorada y/o inmunogenicidad reducida en terapias in vivo.
MX2015011943A 2013-03-14 2014-03-14 Acidos ribonucleicos con nucleotidos modificados por 4'-tio y metodos relacionados. MX2015011943A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361785098P 2013-03-14 2013-03-14
PCT/US2014/027422 WO2014152513A1 (en) 2013-03-14 2014-03-14 RIBONUCLEIC ACIDs WITH 4'-THIO-MODIFIED NUCLEOTIDES AND RELATED METHODS

Publications (1)

Publication Number Publication Date
MX2015011943A true MX2015011943A (es) 2015-12-01

Family

ID=50639964

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2015011943A MX2015011943A (es) 2013-03-14 2014-03-14 Acidos ribonucleicos con nucleotidos modificados por 4'-tio y metodos relacionados.

Country Status (16)

Country Link
US (4) US10266559B2 (es)
EP (3) EP2970351B1 (es)
JP (1) JP6567494B2 (es)
KR (1) KR20150127582A (es)
CN (1) CN105026411A (es)
AU (2) AU2014239562B2 (es)
BR (1) BR112015022507A2 (es)
CA (1) CA2902884C (es)
EA (1) EA201591281A1 (es)
ES (2) ES2797974T3 (es)
HK (1) HK1219955A1 (es)
IL (1) IL240465A0 (es)
MX (1) MX2015011943A (es)
SG (1) SG11201507474QA (es)
WO (1) WO2014152513A1 (es)
ZA (1) ZA201507605B (es)

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Also Published As

Publication number Publication date
WO2014152513A1 (en) 2014-09-25
BR112015022507A2 (pt) 2017-10-24
JP6567494B2 (ja) 2019-08-28
US20160031928A1 (en) 2016-02-04
CA2902884A1 (en) 2014-09-25
EP2970351B1 (en) 2017-09-13
AU2018203985A1 (en) 2018-06-21
KR20150127582A (ko) 2015-11-17
ES2797974T3 (es) 2020-12-04
EP2970351A1 (en) 2016-01-20
US20230192753A1 (en) 2023-06-22
AU2014239562B2 (en) 2018-07-05
JP2016513470A (ja) 2016-05-16
CA2902884C (en) 2021-05-25
US10266559B2 (en) 2019-04-23
US20190263850A1 (en) 2019-08-29
EP3301102A1 (en) 2018-04-04
EP3750903A1 (en) 2020-12-16
ZA201507605B (en) 2017-01-25
US20210009629A1 (en) 2021-01-14
AU2014239562A1 (en) 2015-08-27
US10822368B2 (en) 2020-11-03
AU2018203985B2 (en) 2019-09-19
EA201591281A1 (ru) 2016-02-29
EP3301102B1 (en) 2020-04-15
SG11201507474QA (en) 2015-10-29
US11447520B2 (en) 2022-09-20
IL240465A0 (en) 2015-09-24
ES2647832T3 (es) 2017-12-26
CN105026411A (zh) 2015-11-04
HK1219955A1 (zh) 2017-04-21

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