HK1204652A1 - Compositions and methods for modulation of atxn3 expression - Google Patents

Compositions and methods for modulation of atxn3 expression Download PDF

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Publication number
HK1204652A1
HK1204652A1 HK15105081.2A HK15105081A HK1204652A1 HK 1204652 A1 HK1204652 A1 HK 1204652A1 HK 15105081 A HK15105081 A HK 15105081A HK 1204652 A1 HK1204652 A1 HK 1204652A1
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HK
Hong Kong
Prior art keywords
oligonucleotide
atxn3
expression
seq
atxn
Prior art date
Application number
HK15105081.2A
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English (en)
Chinese (zh)
Inventor
．烏斯卡特吉
N．乌斯卡特吉
．黑特耶恩
M．黑特耶恩
．海森
J．B．R．海森
Original Assignee
罗氏创新中心哥本哈根有限公司
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Application filed by 罗氏创新中心哥本哈根有限公司 filed Critical 罗氏创新中心哥本哈根有限公司
Publication of HK1204652A1 publication Critical patent/HK1204652A1/xx

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    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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
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    • 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
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    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33—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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
HK15105081.2A 2012-03-12 2013-03-12 Compositions and methods for modulation of atxn3 expression HK1204652A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261609774P 2012-03-12 2012-03-12
US61/609,774 2012-03-12
PCT/US2013/030553 WO2013138353A2 (en) 2012-03-12 2013-03-12 Compositions and methods for modulation of atxn3 expression

Publications (1)

Publication Number Publication Date
HK1204652A1 true HK1204652A1 (en) 2015-11-27

Family

ID=49161936

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15105081.2A HK1204652A1 (en) 2012-03-12 2013-03-12 Compositions and methods for modulation of atxn3 expression

Country Status (7)

Country Link
US (1) US20150315595A1 (enExample)
EP (1) EP2839008A4 (enExample)
JP (1) JP2015511821A (enExample)
CN (1) CN104254610A (enExample)
BR (1) BR112014021612A2 (enExample)
HK (1) HK1204652A1 (enExample)
WO (1) WO2013138353A2 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
WO2019217708A1 (en) * 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
WO2019233922A1 (en) 2018-06-05 2019-12-12 F. Hoffmann-La Roche Ag Oligonucleotides for modulating atxn2 expression
EP3884050A1 (en) * 2018-11-19 2021-09-29 uniQure IP B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
US20220098592A1 (en) * 2019-01-09 2022-03-31 Universidade De Coimbra Double stranded rna and uses thereof
JP2022520986A (ja) * 2019-02-22 2022-04-04 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ａｔｘｎ３発現を減少させるための化合物及び方法
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
CN113950529A (zh) 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
WO2021102373A1 (en) * 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
EP4151237A4 (en) * 2020-05-12 2024-08-14 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
AR124227A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
AR124229A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
AR127158A1 (es) 2021-09-29 2023-12-27 Hoffmann La Roche Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina
CN116064667B (zh) * 2022-12-09 2024-07-02 中南大学湘雅医院 基于CRISPR/Cas9的人源化ATXN3基因敲入小鼠模型的构建方法及应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
AU2004209599A1 (en) * 2003-02-10 2004-08-19 Santaris Pharma A/S Oligomeric compounds for the modulation of ras expression
JP4463608B2 (ja) * 2004-04-19 2010-05-19 独立行政法人科学技術振興機構 変異ＭＪＤ遺伝子の発現を特異的に抑制するｓｉＲＮＡ
JP5665317B2 (ja) * 2006-10-18 2015-02-04 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物
JP2011516489A (ja) * 2008-03-31 2011-05-26 ユニバーシティ・オブ・サウス・フロリダ 疾患誘発性運動失調症および非運動失調性平衡異常の治療法
CA2732343C (en) * 2008-07-29 2017-05-09 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
WO2010120508A2 (en) * 2009-03-31 2010-10-21 The General Hospital Corporation Regulation of mir-33 micrornas in the treatment of cholesterol-related disorders
JP6006120B2 (ja) * 2010-02-08 2016-10-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 対立遺伝子多様体の選択的低減
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates

Also Published As

Publication number Publication date
EP2839008A4 (en) 2015-12-02
US20150315595A1 (en) 2015-11-05
EP2839008A2 (en) 2015-02-25
WO2013138353A3 (en) 2013-11-14
BR112014021612A2 (pt) 2017-07-18
JP2015511821A (ja) 2015-04-23
WO2013138353A2 (en) 2013-09-19
CN104254610A (zh) 2014-12-31

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