CO2019003729A2 - Compuestos y métodos para reducir de la expresión de atxn3 - Google Patents

Compuestos y métodos para reducir de la expresión de atxn3

Info

Publication number
CO2019003729A2
CO2019003729A2 CONC2019/0003729A CO2019003729A CO2019003729A2 CO 2019003729 A2 CO2019003729 A2 CO 2019003729A2 CO 2019003729 A CO2019003729 A CO 2019003729A CO 2019003729 A2 CO2019003729 A2 CO 2019003729A2
Authority
CO
Colombia
Prior art keywords
methods
reduce
compounds
atxn3
expression
Prior art date
Application number
CONC2019/0003729A
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 CO2019003729A2 publication Critical patent/CO2019003729A2/es

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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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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    • 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
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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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    • C12N2310/30—Chemical structure
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • 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

RESUMEN 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.
CONC2019/0003729A 2016-11-10 2019-04-12 Compuestos y métodos para reducir de la expresión de atxn3 CO2019003729A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662420294P 2016-11-10 2016-11-10
PCT/US2017/061121 WO2018089805A1 (en) 2016-11-10 2017-11-10 Compounds and methods for reducing atxn3 expression

Publications (1)

Publication Number Publication Date
CO2019003729A2 true CO2019003729A2 (es) 2019-06-28

Family

ID=62109841

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2019/0003729A CO2019003729A2 (es) 2016-11-10 2019-04-12 Compuestos y métodos para reducir de 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)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3353303T (pt) 2015-09-25 2023-10-10 Academisch Ziekenhuis Leiden Composições e métodos para modulação da expressão de ataxina 3
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EA202092500A1 (ru) 2018-05-09 2021-03-01 Ионис Фармасьютикалз, Инк. Соединения и способы для снижения экспрессии 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
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
MX2021005886A (es) * 2018-11-21 2021-06-23 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de priones.
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
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
JPWO2021230286A1 (es) 2020-05-12 2021-11-18
WO2022117747A2 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
CN118019848A (zh) 2021-09-29 2024-05-10 豪夫迈·罗氏有限公司 Rna编辑

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