PL3461895T3 - Modulacja ekspresji ube3a-ats - Google Patents

Modulacja ekspresji ube3a-ats

Info

Publication number
PL3461895T3
PL3461895T3 PL18188705T PL18188705T PL3461895T3 PL 3461895 T3 PL3461895 T3 PL 3461895T3 PL 18188705 T PL18188705 T PL 18188705T PL 18188705 T PL18188705 T PL 18188705T PL 3461895 T3 PL3461895 T3 PL 3461895T3
Authority
PL
Poland
Prior art keywords
ube3a
modulation
ats
expression
ats expression
Prior art date
Application number
PL18188705T
Other languages
English (en)
Polish (pl)
Inventor
Frank Rigo
Amanda WARD
Arthur L. Beaudet
Linyan MENG
Original Assignee
Ionis Pharmaceuticals, Inc.
Baylor College Of Medicine
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
Family has litigation
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=PL3461895(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 Of Medicine filed Critical Ionis Pharmaceuticals, Inc.
Publication of PL3461895T3 publication Critical patent/PL3461895T3/pl

Links

Classifications

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
PL18188705T 2012-06-25 2013-06-25 Modulacja ekspresji ube3a-ats PL3461895T3 (pl)

Applications Claiming Priority (8)

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
PCT/US2013/047701 WO2014004572A2 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression
EP18188705.0A EP3461895B1 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression

Publications (1)

Publication Number Publication Date
PL3461895T3 true PL3461895T3 (pl) 2021-01-11

Family

ID=49784000

Family Applications (1)

Application Number Title Priority Date Filing Date
PL18188705T PL3461895T3 (pl) 2012-06-25 2013-06-25 Modulacja ekspresji ube3a-ats

Country Status (13)

Country Link
US (1) US9617539B2 (enExample)
EP (4) EP3461895B1 (enExample)
JP (1) JP6294876B2 (enExample)
AU (1) AU2013280474B2 (enExample)
CA (1) CA2877905A1 (enExample)
DK (2) DK2864479T3 (enExample)
ES (3) ES2809199T3 (enExample)
HU (1) HUE051698T2 (enExample)
LT (1) LT3461895T (enExample)
PL (1) PL3461895T3 (enExample)
PT (1) PT3461895T (enExample)
SI (1) SI3461895T1 (enExample)
WO (1) WO2014004572A2 (enExample)

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ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
WO2016086104A1 (en) * 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
CA2984629C (en) 2015-05-07 2024-06-18 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
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PL3717646T3 (pl) * 2017-12-01 2025-10-27 The Texas A&M University System Leczenie antysensowe zespołu angelmana
EP3758714A4 (en) * 2018-02-27 2021-12-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME
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EP3911957A1 (en) * 2019-01-17 2021-11-24 F. Hoffmann-La Roche AG E3 ubiquitin ligase (ube3a) protein targets
TW202536179A (zh) * 2019-03-29 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之化合物及方法
EA202193214A1 (ru) * 2019-05-22 2022-03-14 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Гены ube3a и кассеты экспрессии и их использование
WO2021011802A1 (en) * 2019-07-16 2021-01-21 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
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WO2021222232A1 (en) * 2020-04-28 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
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US20240318198A1 (en) * 2021-02-03 2024-09-26 The Board Of Regents Of The University Of Texas System Gene therapy for angelman syndrome
US20240240179A1 (en) * 2021-05-05 2024-07-18 The General Hospital Corporation Therapy for treatment of prader-willi syndrome
WO2022266351A2 (en) * 2021-06-17 2022-12-22 Q-State Biosciences, Inc. Therapeutic oligonucleotide methods
WO2023020980A1 (en) * 2021-08-16 2023-02-23 F. Hoffmann-La Roche Ag E3 ubiquitin ligase (ube3a) protein targets
WO2023168000A1 (en) * 2022-03-03 2023-09-07 The Regents Of The University Of California Compositions and methods for treatment of angelman syndrome
JP7904923B2 (ja) 2022-05-27 2026-08-13 ゼロ カーボン システムズ インコーポレイテッド ハイスループット可動パネル直接空気回収システム
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TW202536178A (zh) * 2023-11-09 2025-09-16 美商Ionis製藥公司 用於調節ｕｂｅ３ａ－ａｔｓ之方法
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DK2864479T3 (en) 2018-10-22
EP4458361A3 (en) 2025-01-22
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WO2014004572A3 (en) 2014-05-01
AU2013280474B2 (en) 2019-01-03
HUE051698T2 (hu) 2021-03-29
WO2014004572A2 (en) 2014-01-03
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US9617539B2 (en) 2017-04-11
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EP3461895B1 (en) 2020-07-01
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AU2013280474A1 (en) 2015-01-22
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SI3461895T1 (sl) 2020-10-30
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