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)
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)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (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 (pl)
EP (4) EP4458361A3 (pl)
JP (1) JP6294876B2 (pl)
AU (1) AU2013280474B2 (pl)
CA (1) CA2877905A1 (pl)
DK (2) DK3461895T3 (pl)
ES (3) ES2688831T3 (pl)
HU (1) HUE051698T2 (pl)
LT (1) LT3461895T (pl)
PL (1) PL3461895T3 (pl)
PT (1) PT3461895T (pl)
SI (1) SI3461895T1 (pl)
WO (1) WO2014004572A2 (pl)

Families Citing this family (22)

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PL3461895T3 (pl) 2012-06-25 2021-01-11 Ionis Pharmaceuticals, Inc. Modulacja ekspresji ube3a-ats
US10400243B2 (en) 2014-11-25 2019-09-03 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
SI4220360T1 (sl) * 2015-11-12 2024-10-30 F. Hoffmann - La Roche Ag Oligonukleotidi za indukcijo očetovske ekspresije UBE3A
EP3548005A4 (en) 2016-11-29 2020-06-17 Puretech Health LLC Exosomes for delivery of therapeutic agents
PT3717646T (pt) * 2017-12-01 2025-07-28 Texas A & M Univ Sys Tratamento antisense da síndrome de angelman
CN111770758A (zh) * 2018-02-27 2020-10-13 北卡罗来纳-查佩尔山大学 用于治疗Angelman综合征的方法和组合物
KR20210040358A (ko) 2018-06-14 2021-04-13 오비드 테라퓨틱스 인크. 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용
AU2019344006B2 (en) * 2018-09-21 2025-11-27 University Of Connecticut Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome
CN113316722A (zh) 2019-01-17 2021-08-27 豪夫迈·罗氏有限公司 E3泛素连接酶(ube3a)蛋白质靶标
CN113728104B (zh) * 2019-03-29 2023-10-27 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
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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US20230167438A1 (en) * 2020-04-28 2023-06-01 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
WO2022169861A2 (en) * 2021-02-03 2022-08-11 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
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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EP3461895B1 (en) 2020-07-01
HUE051698T2 (hu) 2021-03-29
AU2013280474B2 (en) 2019-01-03
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EP2864479A2 (en) 2015-04-29
WO2014004572A2 (en) 2014-01-03
EP4458361A3 (en) 2025-01-22
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CA2877905A1 (en) 2014-01-03
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WO2014004572A3 (en) 2014-05-01
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US9617539B2 (en) 2017-04-11
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DK2864479T3 (en) 2018-10-22
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