WO2014004572A3 - Modulation of ube3a-ats expression - Google Patents

Modulation of ube3a-ats expression Download PDF

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Publication number
WO2014004572A3
WO2014004572A3 PCT/US2013/047701 US2013047701W WO2014004572A3 WO 2014004572 A3 WO2014004572 A3 WO 2014004572A3 US 2013047701 W US2013047701 W US 2013047701W WO 2014004572 A3 WO2014004572 A3 WO 2014004572A3
Authority
WO
WIPO (PCT)
Prior art keywords
ube3a
modulation
ats
compounds
methods
Prior art date
Application number
PCT/US2013/047701
Other languages
French (fr)
Other versions
WO2014004572A2 (en
Inventor
Frank Rigo
Amanda WARD
Arthur L. Beaudet
Linyan MENG
Original Assignee
Isis 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=WO2014004572(A3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority to CA2877905A priority Critical patent/CA2877905A1/en
Priority to ES13809694.6T priority patent/ES2688831T3/en
Priority to EP20182895.1A priority patent/EP3770258A1/en
Priority to EP18188705.0A priority patent/EP3461895B1/en
Priority to US14/409,348 priority patent/US9617539B2/en
Application filed by Isis Pharmaceuticals, Inc., Baylor College Of Medicine filed Critical Isis Pharmaceuticals, Inc.
Priority to PL18188705T priority patent/PL3461895T3/en
Priority to DK13809694.6T priority patent/DK2864479T3/en
Priority to EP13809694.6A priority patent/EP2864479B1/en
Priority to AU2013280474A priority patent/AU2013280474B2/en
Priority to JP2015520410A priority patent/JP6294876B2/en
Publication of WO2014004572A2 publication Critical patent/WO2014004572A2/en
Publication of WO2014004572A3 publication Critical patent/WO2014004572A3/en

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

Abstract

Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.
PCT/US2013/047701 2012-06-25 2013-06-25 Modulation of ube3a-ats expression WO2014004572A2 (en)

Priority Applications (10)

Application Number Priority Date Filing Date Title
JP2015520410A JP6294876B2 (en) 2012-06-25 2013-06-25 Regulation of UBE3A-ATS expression
ES13809694.6T ES2688831T3 (en) 2012-06-25 2013-06-25 UBE3A-ATS expression modulation
EP20182895.1A EP3770258A1 (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
US14/409,348 US9617539B2 (en) 2012-06-25 2013-06-25 Modulation of UBE3A-ATS expression
CA2877905A CA2877905A1 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression
PL18188705T PL3461895T3 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression
DK13809694.6T DK2864479T3 (en) 2012-06-25 2013-06-25 MODULATION OF UBE3A-ATS EXPRESSION
EP13809694.6A EP2864479B1 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression
AU2013280474A AU2013280474B2 (en) 2012-06-25 2013-06-25 Modulation of UBE3A-ATS expression

Applications Claiming Priority (10)

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

Publications (2)

Publication Number Publication Date
WO2014004572A2 WO2014004572A2 (en) 2014-01-03
WO2014004572A3 true WO2014004572A3 (en) 2014-05-01

Family

ID=49784000

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2013/047701 WO2014004572A2 (en) 2012-06-25 2013-06-25 Modulation of ube3a-ats expression

Country Status (13)

Country Link
US (1) US9617539B2 (en)
EP (3) EP2864479B1 (en)
JP (1) JP6294876B2 (en)
AU (1) AU2013280474B2 (en)
CA (1) CA2877905A1 (en)
DK (2) DK3461895T3 (en)
ES (2) ES2688831T3 (en)
HU (1) HUE051698T2 (en)
LT (1) LT3461895T (en)
PL (1) PL3461895T3 (en)
PT (1) PT3461895T (en)
SI (1) SI3461895T1 (en)
WO (1) WO2014004572A2 (en)

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US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
PL3291843T3 (en) 2015-05-07 2023-07-17 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
ES2852998T3 (en) 2015-11-12 2021-09-14 Hoffmann La Roche Oligonucleotides to induce expression of paternal UBE3A
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
EP3717646A4 (en) * 2017-12-01 2021-12-01 The Texas A&M University System Angelman syndrome antisense treatment
CN111770758A (en) * 2018-02-27 2020-10-13 北卡罗来纳-查佩尔山大学 Methods and compositions for treating Angelman syndrome
CN112739353A (en) 2018-06-14 2021-04-30 奥维德医疗公司 Use of MIR-92A or MIR-145 for treating angeman syndrome
KR20210065125A (en) * 2018-09-21 2021-06-03 유니버시티 오브 코네티컷 Compositions and methods for restoring paternal UBE3A gene expression in Angelman syndrome in humans
CN113316722A (en) * 2019-01-17 2021-08-27 豪夫迈·罗氏有限公司 E3 ubiquitin ligase (UBE3A) protein targets
AU2020253821A1 (en) * 2019-03-29 2021-10-28 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating UBE3A-ATS
KR20210158859A (en) * 2019-05-22 2021-12-31 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 UBE3A gene and expression cassette and uses thereof
US20220265607A1 (en) * 2019-07-16 2022-08-25 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
EP4076471A4 (en) * 2019-12-18 2023-12-27 National University of Singapore Method for treating angelman syndrome and related disorders
WO2021222232A1 (en) * 2020-04-28 2021-11-04 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
WO2022236296A1 (en) * 2021-05-05 2022-11-10 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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