CA2877905A1 - Modulation of ube3a-ats expression - Google Patents

Modulation of ube3a-ats expression Download PDF

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Publication number
CA2877905A1
CA2877905A1 CA2877905A CA2877905A CA2877905A1 CA 2877905 A1 CA2877905 A1 CA 2877905A1 CA 2877905 A CA2877905 A CA 2877905A CA 2877905 A CA2877905 A CA 2877905A CA 2877905 A1 CA2877905 A1 CA 2877905A1
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Canada
Prior art keywords
ube3a
ats
seq
region
nucleic acid
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Pending
Application number
CA2877905A
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English (en)
French (fr)
Inventor
Frank Rigo
Amanda WARD
Arthur L. Beaudet
Linyan MENG
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Baylor College of Medicine
Ionis Pharmaceuticals Inc
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Baylor College of Medicine
Isis Pharmaceuticals Inc
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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=CA2877905(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Baylor College of Medicine, Isis Pharmaceuticals Inc filed Critical Baylor College of Medicine
Publication of CA2877905A1 publication Critical patent/CA2877905A1/en
Pending legal-status Critical Current

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    • 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
    • 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
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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
    • 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
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    • 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
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • 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)
CA2877905A 2012-06-25 2013-06-25 Modulation of ube3a-ats expression Pending CA2877905A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261664083P 2012-06-25 2012-06-25
US61/664,083 2012-06-25
US201261738959P 2012-12-18 2012-12-18
US61/738,959 2012-12-18
US201361750939P 2013-01-10 2013-01-10
US61/750,939 2013-01-10
US201361755617P 2013-01-23 2013-01-23
US61/755,617 2013-01-23
US201361772925P 2013-03-05 2013-03-05
US61/772,925 2013-03-05
PCT/US2013/047701 WO2014004572A2 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression

Publications (1)

Publication Number Publication Date
CA2877905A1 true CA2877905A1 (en) 2014-01-03

Family

ID=49784000

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2877905A Pending CA2877905A1 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression

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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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
HK1258434A1 (zh) 2015-11-12 2019-11-08 F. Hoffmann-La Roche Ag 来自灵长类物种的标准化神经元细胞测定试验
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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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EP3833398A4 (en) 2018-09-21 2021-12-22 University of Connecticut COMPOSITIONS AND METHODS OF RESTORING PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN'S SYNDROME IN HUMANS
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
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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
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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
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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AU2013280474B2 (en) 2019-01-03
HUE051698T2 (hu) 2021-03-29
WO2014004572A2 (en) 2014-01-03
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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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