BR112015022507A2 - ácidos ribonucleicos com nucleotídeos 4'-tio-modificados, composição compreendendo o mesmo e usos relacionados - Google Patents

ácidos ribonucleicos com nucleotídeos 4'-tio-modificados, composição compreendendo o mesmo e usos relacionados

Info

Publication number
BR112015022507A2
BR112015022507A2 BR112015022507A BR112015022507A BR112015022507A2 BR 112015022507 A2 BR112015022507 A2 BR 112015022507A2 BR 112015022507 A BR112015022507 A BR 112015022507A BR 112015022507 A BR112015022507 A BR 112015022507A BR 112015022507 A2 BR112015022507 A2 BR 112015022507A2
Authority
BR
Brazil
Prior art keywords
thio
ribonucleic acids
composition
same
modified nucleotide
Prior art date
Application number
BR112015022507A
Other languages
English (en)
Inventor
Derosa Frank
Heartlein Michael
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 BR112015022507A2 publication Critical patent/BR112015022507A2/pt

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (AREA)
  • Dispersion Chemistry (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicinal Preparation (AREA)

Abstract

resumo ácidos ribonucleicos com nucleotídeos 4'-tio-modificados e métodos relacionados são divulgadas moléculas de rna mensageiro e composições relacionadas incorporando uma modificação 4'-tio no anel furanose de pelo menos um resíduo de nucleotídeo e métodos para usar estes mrnas para produzir uma proteína terapêutica codificada in vivo e para tratar ou prevenir doenças ou distúrbios. em certas modalidades, o mrna 4'-tio modificado fornece estabilidade potencializada e/ou imunogenicidade reduzida em terapias in vivo.
BR112015022507A 2013-03-14 2014-03-14 ácidos ribonucleicos com nucleotídeos 4'-tio-modificados, composição compreendendo o mesmo e usos relacionados BR112015022507A2 (pt)

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
BR112015022507A2 true BR112015022507A2 (pt) 2017-10-24

Family

ID=50639964

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015022507A BR112015022507A2 (pt) 2013-03-14 2014-03-14 ácidos ribonucleicos com nucleotídeos 4'-tio-modificados, composição compreendendo o mesmo e usos relacionados

Country Status (16)

Country Link
US (4) US10266559B2 (pt)
EP (3) EP2970351B1 (pt)
JP (1) JP6567494B2 (pt)
KR (1) KR20150127582A (pt)
CN (1) CN105026411A (pt)
AU (2) AU2014239562B2 (pt)
BR (1) BR112015022507A2 (pt)
CA (1) CA2902884C (pt)
EA (1) EA201591281A1 (pt)
ES (2) ES2647832T3 (pt)
HK (1) HK1219955A1 (pt)
IL (1) IL240465A0 (pt)
MX (1) MX2015011943A (pt)
SG (1) SG11201507474QA (pt)
WO (1) WO2014152513A1 (pt)
ZA (1) ZA201507605B (pt)

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Also Published As

Publication number Publication date
EP3301102A1 (en) 2018-04-04
US10266559B2 (en) 2019-04-23
ES2797974T3 (es) 2020-12-04
US11447520B2 (en) 2022-09-20
EP3301102B1 (en) 2020-04-15
CA2902884C (en) 2021-05-25
CN105026411A (zh) 2015-11-04
AU2018203985B2 (en) 2019-09-19
US20160031928A1 (en) 2016-02-04
IL240465A0 (en) 2015-09-24
HK1219955A1 (zh) 2017-04-21
EP3750903A1 (en) 2020-12-16
CA2902884A1 (en) 2014-09-25
US20230192753A1 (en) 2023-06-22
EP2970351A1 (en) 2016-01-20
SG11201507474QA (en) 2015-10-29
ES2647832T3 (es) 2017-12-26
US20190263850A1 (en) 2019-08-29
US20210009629A1 (en) 2021-01-14
EP2970351B1 (en) 2017-09-13
AU2014239562B2 (en) 2018-07-05
AU2018203985A1 (en) 2018-06-21
JP2016513470A (ja) 2016-05-16
US10822368B2 (en) 2020-11-03
KR20150127582A (ko) 2015-11-17
ZA201507605B (en) 2017-01-25
MX2015011943A (es) 2015-12-01
EA201591281A1 (ru) 2016-02-29
JP6567494B2 (ja) 2019-08-28
AU2014239562A1 (en) 2015-08-27
WO2014152513A1 (en) 2014-09-25

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2505 DE 08-01-2019 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.