MX2020011911A - Compuestos y metodos para reducir de la expresion de atxn3. - Google Patents

Compuestos y metodos para reducir de la expresion de atxn3.

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Publication number
MX2020011911A
MX2020011911A MX2020011911A MX2020011911A MX2020011911A MX 2020011911 A MX2020011911 A MX 2020011911A MX 2020011911 A MX2020011911 A MX 2020011911A MX 2020011911 A MX2020011911 A MX 2020011911A MX 2020011911 A MX2020011911 A MX 2020011911A
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MX
Mexico
Prior art keywords
compounds
methods
reducing
animal
cell
Prior art date
Application number
MX2020011911A
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English (en)
Inventor
Susan M Freier
Original Assignee
Ionis Pharmaceuticals Inc
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
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of MX2020011911A publication Critical patent/MX2020011911A/es

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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
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  • Medicinal Chemistry (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o la actividad del ARN de ATXN3 en una célula o animal, y en determinadas modalidades para reducir la cantidad de proteína ATXN3 en una célula o animal. Dichos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o característica de una enfermedad neurodegenerativa. Dichos síntomas y características incluyen disfunción motriz, formación de agregados y muerte neuronal. Dichas enfermedades neurodegenerativas incluyen ataxia espinocerebelosa tipo 3 (SCA3).
MX2020011911A 2018-05-09 2019-05-09 Compuestos y metodos para reducir de la expresion de atxn3. MX2020011911A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862669238P 2018-05-09 2018-05-09
PCT/US2019/031562 WO2019217708A1 (en) 2018-05-09 2019-05-09 Compounds and methods for reducing atxn3 expression

Publications (1)

Publication Number Publication Date
MX2020011911A true MX2020011911A (es) 2021-01-29

Family

ID=68468406

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020011911A MX2020011911A (es) 2018-05-09 2019-05-09 Compuestos y metodos para reducir de la expresion de atxn3.

Country Status (21)

Country Link
US (3) US11434488B2 (es)
EP (1) EP3799602A4 (es)
JP (1) JP2021522800A (es)
KR (1) KR20210008497A (es)
CN (1) CN112189053B (es)
AR (1) AR115375A1 (es)
AU (1) AU2019266307A1 (es)
BR (1) BR112020021253A2 (es)
CA (1) CA3098144A1 (es)
CL (1) CL2020002888A1 (es)
CO (1) CO2020013433A2 (es)
CR (1) CR20200604A (es)
EA (1) EA202092500A1 (es)
JO (1) JOP20200283A1 (es)
MX (1) MX2020011911A (es)
PE (1) PE20212131A1 (es)
PH (1) PH12020500668A1 (es)
SG (1) SG11202010215TA (es)
TW (1) TW202003541A (es)
UY (1) UY38225A (es)
WO (1) WO2019217708A1 (es)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
US11447775B2 (en) 2018-01-12 2022-09-20 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
KR20210008497A (ko) 2018-05-09 2021-01-22 아이오니스 파마수티컬즈, 인코포레이티드 Atxn3 발현 감소용 화합물 및 방법
US20220195431A1 (en) * 2019-02-22 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
JP7155302B2 (ja) 2019-06-06 2022-10-18 エフ．ホフマン－ラ ロシュ アーゲー Ａｔｘｎ３を標的とするアンチセンスオリゴヌクレオチド
EP4061945A1 (en) * 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
EP4151237A4 (en) * 2020-05-12 2024-08-14 Mitsubishi Tanabe Pharma Corp COMPOUND, METHOD AND PHARMACEUTICAL COMPOSITION FOR REGULATING THE EXPRESSION OF ATAXIN 3
EP4162047A1 (en) * 2020-06-09 2023-04-12 Roche Innovation Center Copenhagen A/S Guanosine analogues for use in therapeutic polynucleotides
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
AR124229A1 (es) * 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3

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