DK3461895T3 - Modulation af ube3a-ats-ekspression - Google Patents

Modulation af ube3a-ats-ekspression Download PDF

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Publication number
DK3461895T3
DK3461895T3 DK18188705.0T DK18188705T DK3461895T3 DK 3461895 T3 DK3461895 T3 DK 3461895T3 DK 18188705 T DK18188705 T DK 18188705T DK 3461895 T3 DK3461895 T3 DK 3461895T3
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DK
Denmark
Prior art keywords
ube3a
modulation
ats
expression
ats expression
Prior art date
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DK18188705.0T
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English (en)
Inventor
Frank Rigo
Amanda Ward
Arthur L Beaudet
Linyan Meng
Original Assignee
Ionis Pharmaceuticals Inc
Baylor College Medicine
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49784000&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK3461895(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc, Baylor College Medicine filed Critical Ionis Pharmaceuticals Inc
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Publication of DK3461895T3 publication Critical patent/DK3461895T3/da

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    • 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
    • C12N15/11—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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
DK18188705.0T 2012-06-25 2013-06-25 Modulation af ube3a-ats-ekspression DK3461895T3 (da)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201261664083P 2012-06-25 2012-06-25
US201261738959P 2012-12-18 2012-12-18
US201361750939P 2013-01-10 2013-01-10
US201361755617P 2013-01-23 2013-01-23
US201361772925P 2013-03-05 2013-03-05
EP13809694.6A EP2864479B1 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression

Publications (1)

Publication Number Publication Date
DK3461895T3 true DK3461895T3 (da) 2020-07-20

Family

ID=49784000

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DK18188705.0T DK3461895T3 (da) 2012-06-25 2013-06-25 Modulation af ube3a-ats-ekspression
DK13809694.6T DK2864479T3 (da) 2012-06-25 2013-06-25 Modulation af ube3a-ats-ekspression

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK13809694.6T DK2864479T3 (da) 2012-06-25 2013-06-25 Modulation af ube3a-ats-ekspression

Country Status (13)

Country Link
US (1) US9617539B2 (da)
EP (3) EP3461895B1 (da)
JP (1) JP6294876B2 (da)
AU (1) AU2013280474B2 (da)
CA (1) CA2877905A1 (da)
DK (2) DK3461895T3 (da)
ES (2) ES2688831T3 (da)
HU (1) HUE051698T2 (da)
LT (1) LT3461895T (da)
PL (1) PL3461895T3 (da)
PT (1) PT3461895T (da)
SI (1) SI3461895T1 (da)
WO (1) WO2014004572A2 (da)

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MX2018004978A (es) 2015-11-12 2018-07-06 Hoffmann La Roche Oligonucleotidos para inducir la expresion paterna de la ligasa de la proteina ubiquitina e3a (ube3a).
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CN113164624A (zh) * 2018-09-21 2021-07-23 康涅狄格大学 恢复人类快乐木偶综合征中父系ube3a基因表达的组合物和方法
US20220091137A1 (en) * 2019-01-17 2022-03-24 Hoffmann-La Roche Inc. E3 ubiquitin ligase (ube3a) protein targets
CN117431244A (zh) * 2019-03-29 2024-01-23 Ionis制药公司 用于调节ube3a-ats的化合物和方法
WO2020237130A1 (en) * 2019-05-22 2020-11-26 The University Of North Carolina At Chapel Hill Ube3a genes and expression cassettes and their use
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JP2023524247A (ja) * 2020-04-28 2023-06-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用
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WO2023168000A1 (en) * 2022-03-03 2023-09-07 The Regents Of The University Of California Compositions and methods for treatment of angelman syndrome

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CA2877905A1 (en) 2014-01-03
EP2864479B1 (en) 2018-08-15
EP3461895B1 (en) 2020-07-01
LT3461895T (lt) 2020-09-10
EP3770258A1 (en) 2021-01-27
EP3461895A1 (en) 2019-04-03
SI3461895T1 (sl) 2020-10-30
US9617539B2 (en) 2017-04-11
WO2014004572A3 (en) 2014-05-01
JP6294876B2 (ja) 2018-03-14
AU2013280474A1 (en) 2015-01-22
ES2688831T3 (es) 2018-11-07
PL3461895T3 (pl) 2021-01-11
DK2864479T3 (da) 2018-10-22
JP2015529635A (ja) 2015-10-08
US20150191723A1 (en) 2015-07-09
ES2809199T3 (es) 2021-03-03
WO2014004572A2 (en) 2014-01-03
HUE051698T2 (hu) 2021-03-29
AU2013280474B2 (en) 2019-01-03
EP2864479A4 (en) 2016-06-08
PT3461895T (pt) 2020-09-08
EP2864479A2 (en) 2015-04-29

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