KR20210008497A - Atxn3 발현 감소용 화합물 및 방법 - Google Patents
Atxn3 발현 감소용 화합물 및 방법 Download PDFInfo
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- KR20210008497A KR20210008497A KR1020207035007A KR20207035007A KR20210008497A KR 20210008497 A KR20210008497 A KR 20210008497A KR 1020207035007 A KR1020207035007 A KR 1020207035007A KR 20207035007 A KR20207035007 A KR 20207035007A KR 20210008497 A KR20210008497 A KR 20210008497A
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- modified
- certain embodiments
- seq
- oligomeric compound
- nucleobase
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- 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
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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
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- 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
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- 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
- 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
- C12N2310/311—Phosphotriesters
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- 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
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- 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)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862669238P | 2018-05-09 | 2018-05-09 | |
| US62/669,238 | 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 |
|---|---|
| KR20210008497A true KR20210008497A (ko) | 2021-01-22 |
Family
ID=68468406
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020207035007A Withdrawn KR20210008497A (ko) | 2018-05-09 | 2019-05-09 | Atxn3 발현 감소용 화합물 및 방법 |
Country Status (21)
| Country | Link |
|---|---|
| US (2) | US11434488B2 (https=) |
| EP (2) | EP4663758A3 (https=) |
| JP (1) | JP2021522800A (https=) |
| KR (1) | KR20210008497A (https=) |
| CN (1) | CN112189053B (https=) |
| AR (1) | AR115375A1 (https=) |
| AU (1) | AU2019266307A1 (https=) |
| BR (1) | BR112020021253A2 (https=) |
| CA (1) | CA3098144A1 (https=) |
| CL (1) | CL2020002888A1 (https=) |
| CO (1) | CO2020013433A2 (https=) |
| CR (1) | CR20200604A (https=) |
| EA (1) | EA202092500A1 (https=) |
| JO (1) | JOP20200283A1 (https=) |
| MX (1) | MX2020011911A (https=) |
| PE (1) | PE20212131A1 (https=) |
| PH (1) | PH12020500668A1 (https=) |
| SG (1) | SG11202010215TA (https=) |
| TW (1) | TW202003541A (https=) |
| UY (1) | UY38225A (https=) |
| WO (1) | WO2019217708A1 (https=) |
Families Citing this family (13)
| 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 |
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| EP4663758A3 (en) | 2018-05-09 | 2026-04-29 | 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 |
| 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 |
| US12565653B2 (en) | 2019-11-22 | 2026-03-03 | Alnylam Pharmaceuticals, Inc. | ATAXIN3 (ATXN3) RNAi agent compositions and methods of use thereof |
| CN120505310A (zh) * | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
| JP7776420B2 (ja) * | 2020-05-12 | 2025-11-26 | 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 |
| US20230212572A1 (en) * | 2020-06-09 | 2023-07-06 | Roche Innovation Center Copenhagen A/S | Guanosine Analogues for Use in Therapeutics 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 |
| WO2025252669A1 (en) | 2024-06-03 | 2025-12-11 | Evotec International Gmbh | Modified oligonucleotides for reducing atxn3 expression |
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