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
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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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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/53Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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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 (2) 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
PE20201501A1 (es) 2018-01-12 2020-12-29 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
EP3799602A4 (en) 2018-05-09 2022-02-16 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
WO2020172559A1 (en) * 2019-02-22 2020-08-27 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
WO2020245233A1 (en) * 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
EP4061945A1 (en) * 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
EP4151237A1 (en) 2020-05-12 2023-03-22 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
CN115702243A (zh) * 2020-06-09 2023-02-14 罗氏创新中心哥本哈根有限公司 用于治疗性多核苷酸的鸟苷类似物
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

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