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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    • 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
    • A61K31/7115—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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    • C12N15/00—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
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
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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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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

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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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EP4285912A2 (en) 2015-09-25 2023-12-06 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
KR20210008497A (ko) 2018-05-09 2021-01-22 아이오니스 파마수티컬즈, 인코포레이티드 Atxn3 발현 감소용 화합물 및 방법
JOP20200334A1 (ar) 2018-06-27 2020-12-20 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير الوراثي عن lrrk2
CA3121010A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
JP7455831B2 (ja) 2018-11-21 2024-03-26 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 プリオン発現を低減するための化合物及び方法
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
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
WO2021053018A1 (en) * 2019-09-16 2021-03-25 Uniqure Ip B.V. Targeting misspliced transcripts in genetic disorders
WO2021230286A1 (ja) * 2020-05-12 2021-11-18 田辺三菱製薬株式会社 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
AR127158A1 (es) 2021-09-29 2023-12-27 Hoffmann La Roche Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina

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