BR112013020273A2 - oligonucleotídeos antissenso - Google Patents

oligonucleotídeos antissenso

Info

Publication number
BR112013020273A2
BR112013020273A2 BR112013020273A BR112013020273A BR112013020273A2 BR 112013020273 A2 BR112013020273 A2 BR 112013020273A2 BR 112013020273 A BR112013020273 A BR 112013020273A BR 112013020273 A BR112013020273 A BR 112013020273A BR 112013020273 A2 BR112013020273 A2 BR 112013020273A2
Authority
BR
Brazil
Prior art keywords
oligonucleotide molecules
antisense
antisense oligonucleotide
antisense oligonucleotides
dystrophy
Prior art date
Application number
BR112013020273A
Other languages
English (en)
Inventor
Qi Long Lu
Original Assignee
Charlotte Mecklenburg Hospital
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 Charlotte Mecklenburg Hospital filed Critical Charlotte Mecklenburg Hospital
Publication of BR112013020273A2 publication Critical patent/BR112013020273A2/pt

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Abstract

resumo patente de invenção: "oligonucleotídeos antissenso". a presente invenção refere-se a compostos antissenso e composições para o tratamento de distrofia muscular e, em particular, distrofia muscular de duchenne (dmd). em uma modalidade, a invenção é dirigida a moléculas de oligonucleotídeo antissenso, composições farmacêuticas e formulações que compreendem moléculas de oligonucleotídeo antissenso e métodos de tratamento de doenças musculares e distúrbios relacionados à distrofia em que as moléculas de oligonucleotídeo antissenso compreendem uma sequência de base selecionada do grupo que consiste em seq id nos: 5-8, 10, 12, 14, 16, 24, 27, 28, 34, 35, 37, 40, 42, 44-46, 79, 97, 100, 101 e 116 e combinações das mesmas.
BR112013020273A 2011-02-08 2012-02-08 oligonucleotídeos antissenso BR112013020273A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161440603P 2011-02-08 2011-02-08
PCT/US2012/024230 WO2012109296A1 (en) 2011-02-08 2012-02-08 Antisense oligonucleotides

Publications (1)

Publication Number Publication Date
BR112013020273A2 true BR112013020273A2 (pt) 2016-10-18

Family

ID=45755532

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112013020273A BR112013020273A2 (pt) 2011-02-08 2012-02-08 oligonucleotídeos antissenso

Country Status (9)

Country Link
US (1) US9078911B2 (pt)
EP (1) EP2672977A1 (pt)
JP (1) JP2014507143A (pt)
KR (1) KR20140052963A (pt)
CN (1) CN103501793A (pt)
BR (1) BR112013020273A2 (pt)
CA (1) CA2826836A1 (pt)
MX (1) MX2013009191A (pt)
WO (1) WO2012109296A1 (pt)

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Also Published As

Publication number Publication date
MX2013009191A (es) 2013-11-04
CN103501793A (zh) 2014-01-08
EP2672977A1 (en) 2013-12-18
KR20140052963A (ko) 2014-05-07
JP2014507143A (ja) 2014-03-27
US9078911B2 (en) 2015-07-14
WO2012109296A1 (en) 2012-08-16
US20120202752A1 (en) 2012-08-09
CA2826836A1 (en) 2012-08-16

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Legal Events

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B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]
B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]