BR112015005091A2 - polinucleotídeo antissenso isolado, composição farmacêutica, métodos para induzir salto de éxon de um rna, para melhorar distrofia muscular, para inibir a progressão de patologia distrófica, e para melhorar função muscular, e, kit. - Google Patents

polinucleotídeo antissenso isolado, composição farmacêutica, métodos para induzir salto de éxon de um rna, para melhorar distrofia muscular, para inibir a progressão de patologia distrófica, e para melhorar função muscular, e, kit.

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Publication number
BR112015005091A2
BR112015005091A2 BR112015005091A BR112015005091A BR112015005091A2 BR 112015005091 A2 BR112015005091 A2 BR 112015005091A2 BR 112015005091 A BR112015005091 A BR 112015005091A BR 112015005091 A BR112015005091 A BR 112015005091A BR 112015005091 A2 BR112015005091 A2 BR 112015005091A2
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BR
Brazil
Prior art keywords
improve
progression
inducing
inhibit
kit
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BR112015005091A
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English (en)
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BR112015005091B1 (pt
Inventor
Mcnally Elizabeth
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Univ Chicago
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Application filed by Univ Chicago filed Critical Univ Chicago
Publication of BR112015005091A2 publication Critical patent/BR112015005091A2/pt
Publication of BR112015005091B1 publication Critical patent/BR112015005091B1/pt

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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1138—Non-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 against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112015005091-3A 2012-09-06 2013-09-06 uso de um polinucleotídeo antissenso isolado BR112015005091B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261697766P 2012-09-06 2012-09-06
US61/697766 2012-09-06
PCT/US2013/058636 WO2014039916A1 (en) 2012-09-06 2013-09-06 Antisense polynucleotides to induce exon skipping and methods of treating dystrophies

Publications (2)

Publication Number Publication Date
BR112015005091A2 true BR112015005091A2 (pt) 2017-09-26
BR112015005091B1 BR112015005091B1 (pt) 2021-07-06

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Application Number Title Priority Date Filing Date
BR112015005091-3A BR112015005091B1 (pt) 2012-09-06 2013-09-06 uso de um polinucleotídeo antissenso isolado

Country Status (17)

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US (12) US9499817B2 (pt)
EP (2) EP3421602B1 (pt)
BR (1) BR112015005091B1 (pt)
CA (1) CA2884245C (pt)
CY (2) CY1121037T1 (pt)
DK (2) DK2892617T3 (pt)
ES (2) ES2686727T3 (pt)
HK (1) HK1212272A1 (pt)
HR (2) HRP20181458T1 (pt)
HU (2) HUE054260T2 (pt)
LT (1) LT3421602T (pt)
PL (1) PL3421602T3 (pt)
PT (2) PT3421602T (pt)
RS (2) RS61985B1 (pt)
SI (2) SI2892617T1 (pt)
TN (1) TN2015000079A1 (pt)
WO (1) WO2014039916A1 (pt)

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SI3283500T1 (sl) 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov
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WO2020132647A1 (en) 2018-12-21 2020-06-25 Northwestern University Use of annexins in preventing and treating muscle membrane injury
AU2019417700A1 (en) 2018-12-23 2021-07-08 Csl Behring L.L.C. Donor T-cells with kill switch
CN113518825A (zh) 2018-12-23 2021-10-19 美国杰特贝林生物制品有限公司 Wiskott-aldrich综合征的造血干细胞基因疗法
MX2021014566A (es) 2019-05-28 2022-03-22 Selecta Biosciences Inc Métodos y composiciones para la respuesta inmune atenuada anti-vector de transferencia viral.
CN115997015A (zh) 2020-06-26 2023-04-21 美国杰特贝林生物制品有限公司 具有杀伤开关的供体t细胞
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Publication number Publication date
US20180037889A1 (en) 2018-02-08
US20190194659A1 (en) 2019-06-27
EP2892617B1 (en) 2018-06-13
CY1121037T1 (el) 2019-12-11
US20160369277A1 (en) 2016-12-22
PL3421602T3 (pl) 2021-10-25
SI3421602T1 (sl) 2021-08-31
PT3421602T (pt) 2021-05-31
ES2686727T3 (es) 2018-10-19
EP2892617A4 (en) 2016-06-22
US20180327743A1 (en) 2018-11-15
HK1212272A1 (en) 2016-06-10
LT3421602T (lt) 2021-05-25
HRP20210877T1 (hr) 2021-07-23
HUE039676T2 (hu) 2019-01-28
SI2892617T1 (sl) 2018-11-30
HRP20181458T1 (hr) 2018-11-02
DK3421602T3 (da) 2021-05-10
US20200040340A1 (en) 2020-02-06
CY1124197T1 (el) 2022-05-27
EP3421602B1 (en) 2021-03-03
ES2878650T3 (es) 2021-11-19
US20220251559A1 (en) 2022-08-11
HUE054260T2 (hu) 2021-08-30
PT2892617T (pt) 2018-10-18
RS61985B1 (sr) 2021-07-30
US20150225718A1 (en) 2015-08-13
DK2892617T3 (en) 2018-09-03
BR112015005091B1 (pt) 2021-07-06
US20210403910A1 (en) 2021-12-30
US20200291403A1 (en) 2020-09-17
CA2884245A1 (en) 2014-03-13
EP2892617A1 (en) 2015-07-15
EP3421602A1 (en) 2019-01-02
US9499817B2 (en) 2016-11-22
WO2014039916A1 (en) 2014-03-13
TN2015000079A1 (en) 2016-06-29
US20210147845A1 (en) 2021-05-20
US9777271B2 (en) 2017-10-03
US20230183700A1 (en) 2023-06-15
RS57789B1 (sr) 2018-12-31
CA2884245C (en) 2023-03-14
US20240076670A1 (en) 2024-03-07

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