CL2019001166A1 - Compuestos y métodos para reducir la expresión de atxn3. - Google Patents

Compuestos y métodos para reducir la expresión de atxn3.

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Publication number
CL2019001166A1
CL2019001166A1 CL2019001166A CL2019001166A CL2019001166A1 CL 2019001166 A1 CL2019001166 A1 CL 2019001166A1 CL 2019001166 A CL2019001166 A CL 2019001166A CL 2019001166 A CL2019001166 A CL 2019001166A CL 2019001166 A1 CL2019001166 A1 CL 2019001166A1
Authority
CL
Chile
Prior art keywords
compounds
methods
reduce
atxn3
expression
Prior art date
Application number
CL2019001166A
Other languages
English (en)
Inventor
Susan M Freier
Holly Kordasiewicz
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 CL2019001166A1 publication Critical patent/CL2019001166A1/es

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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
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    • 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
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

SE PROPORCIONAN COMPUESTOS, MÉTODOS Y COMPOSICIONES FARMACÉUTICAS PARA REDUCIR LA CANTIDAD O LA ACTIVIDAD DEL ARNM DE ATXN3 EN UNA CÉLULA O ANIMAL, Y EN DETERMINADOS CASOS PARA REDUCIR LA CANTIDAD DE PROTEÍNA ATAXINA-3 EN UNA CÉLULA O ANIMAL. DICHOS COMPUESTOS, MÉTODOS Y COMPOSICIONES FARMACÉUTICAS SON ÚTILES PARA EVITAR O MEJORAR AL MENOS UN SÍNTOMA O CARACTERÍSTICA DE UNA ENFERMEDAD NEURODEGENERATIVA. DICHOS SÍNTOMAS Y CARACTERÍSTICAS INCLUYEN ATAXIA, NEUROPATÍA Y FORMACIÓN DE AGREGADOS. DICHAS ENFERMEDADES NEURODEGENERATIVAS INCLUYEN SCA3.
CL2019001166A 2016-11-10 2019-04-26 Compuestos y métodos para reducir la expresión de atxn3. CL2019001166A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662420294P 2016-11-10 2016-11-10

Publications (1)

Publication Number Publication Date
CL2019001166A1 true CL2019001166A1 (es) 2019-09-23

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
CL2019001166A CL2019001166A1 (es) 2016-11-10 2019-04-26 Compuestos y métodos para reducir la expresión de atxn3.

Country Status (21)

Country Link
US (2) US11583548B2 (es)
EP (1) EP3538656A4 (es)
JP (1) JP2019534009A (es)
KR (1) KR20190076025A (es)
CN (1) CN109923210A (es)
AR (1) AR110878A1 (es)
AU (1) AU2017357760A1 (es)
BR (1) BR112019007383A2 (es)
CA (1) CA3041347A1 (es)
CL (1) CL2019001166A1 (es)
CO (1) CO2019003729A2 (es)
CR (1) CR20190277A (es)
EA (1) EA201990862A1 (es)
IL (1) IL266169A (es)
JO (1) JOP20190104A1 (es)
MA (1) MA46791A (es)
MX (1) MX2019005506A (es)
PE (1) PE20191047A1 (es)
SG (1) SG11201903167QA (es)
TW (1) TW201819397A (es)
WO (1) WO2018089805A1 (es)

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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
UY38225A (es) 2018-05-09 2019-11-29 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir de la expresión de atxn3
TWI833770B (zh) * 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
JP2022507283A (ja) * 2018-11-19 2022-01-18 ユニキュアー アイピー ビー．ブイ． 脊髄小脳失調症３型の治療のためのアタキシン－３のｒｎａｉ誘導低減
SG11202103794QA (en) 2018-11-21 2021-05-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing prion expression
EP3980539A1 (en) 2019-06-06 2022-04-13 F. Hoffmann-La Roche AG Antisense oligonucleotides targeting atxn3
CN110527741A (zh) * 2019-09-11 2019-12-03 东北农业大学 一种与美洲南瓜抗白粉病生理小种2f基因紧密连锁的分子标记、引物及应用
EP4031148A1 (en) * 2019-09-16 2022-07-27 uniQure IP B.V. Targeting misspliced transcripts in genetic disorders
EP4151237A1 (en) * 2020-05-12 2023-03-22 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
US20220177883A1 (en) 2020-12-03 2022-06-09 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
WO2023052317A1 (en) 2021-09-29 2023-04-06 F. Hoffmann-La Roche Ag Rna editing

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