RU2020127664A - Композиции для модуляции экспрессии гена c9orf72 - Google Patents
Композиции для модуляции экспрессии гена c9orf72 Download PDFInfo
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- RU2020127664A RU2020127664A RU2020127664A RU2020127664A RU2020127664A RU 2020127664 A RU2020127664 A RU 2020127664A RU 2020127664 A RU2020127664 A RU 2020127664A RU 2020127664 A RU2020127664 A RU 2020127664A RU 2020127664 A RU2020127664 A RU 2020127664A
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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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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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
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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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
- 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
-
- 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
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- 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)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261714132P | 2012-10-15 | 2012-10-15 | |
| US61/714,132 | 2012-10-15 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2015117680A Division RU2730677C2 (ru) | 2012-10-15 | 2013-10-15 | Соединение для модуляции экспрессии гена c9orf72 и его применение |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2020127664A true RU2020127664A (ru) | 2020-09-17 |
Family
ID=50488880
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020127664A RU2020127664A (ru) | 2012-10-15 | 2013-10-15 | Композиции для модуляции экспрессии гена c9orf72 |
| RU2015117680A RU2730677C2 (ru) | 2012-10-15 | 2013-10-15 | Соединение для модуляции экспрессии гена c9orf72 и его применение |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2015117680A RU2730677C2 (ru) | 2012-10-15 | 2013-10-15 | Соединение для модуляции экспрессии гена c9orf72 и его применение |
Country Status (13)
| Country | Link |
|---|---|
| US (4) | US10443052B2 (enExample) |
| EP (1) | EP2906258A4 (enExample) |
| JP (3) | JP6570447B2 (enExample) |
| KR (1) | KR20150070278A (enExample) |
| CN (2) | CN110951731A (enExample) |
| AU (2) | AU2013331434B2 (enExample) |
| BR (1) | BR112015008399A8 (enExample) |
| CA (1) | CA2888486A1 (enExample) |
| HK (1) | HK1212597A1 (enExample) |
| IL (1) | IL238266A0 (enExample) |
| MX (2) | MX370924B (enExample) |
| RU (2) | RU2020127664A (enExample) |
| WO (1) | WO2014062691A2 (enExample) |
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| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| US10443052B2 (en) | 2012-10-15 | 2019-10-15 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C9ORF72 expression |
| FI2906696T4 (fi) | 2012-10-15 | 2023-03-18 | Menetelmiä c9orf72:n ilmentymisen moduloimiseksi | |
| EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| RS59283B1 (sr) * | 2013-01-22 | 2019-10-31 | Deutsches Zentrum Fuer Neurodegenerative Erkrankungen E V | Proteini sa dipeptidnim ponovkom kao terapeutsko ciljno mesto kod neurodegenerativnih bolesti sa ekspanzijom heksanukleotidnog ponovka |
| MY192689A (en) | 2013-10-11 | 2022-09-01 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| SI3119888T1 (sl) | 2014-03-19 | 2021-11-30 | Ionis Pharmaceuticals, Inc. | Sestavki za moduliranje izražanja ataksina 2 |
| WO2016024205A1 (en) * | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene |
| US10538762B2 (en) | 2014-10-14 | 2020-01-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat |
| US10793855B2 (en) | 2015-01-06 | 2020-10-06 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of C9ORF72 antisense transcript |
| WO2016167780A1 (en) * | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| WO2016168592A2 (en) * | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
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| AU2016335032B2 (en) * | 2015-10-05 | 2022-04-14 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
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| WO2017180835A1 (en) | 2016-04-13 | 2017-10-19 | Ionis Pharmaceuticals, Inc. | Methods for reducing c9orf72 expression |
| MX381216B (es) * | 2016-09-30 | 2025-03-12 | Regeneron Pharma | Animales no humanos que tienen una expansion de repeticiones de hexanucleotidos en un locus c9orf72. |
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| AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
| WO2018212271A1 (ja) * | 2017-05-18 | 2018-11-22 | 国立大学法人京都大学 | 脊髄小脳変性症３６型の予防又は治療用組成物 |
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| WO2019094694A1 (en) | 2017-11-10 | 2019-05-16 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
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| AU2020252020A1 (en) * | 2019-03-29 | 2021-11-11 | University Of Massachusetts | Oligonucleotide-based modulation of C90RF72 |
| EP3966328A4 (en) | 2019-05-06 | 2023-10-18 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| IL293824A (en) | 2019-12-13 | 2022-08-01 | Alnylam Pharmaceuticals Inc | Compositions of RNA material (c9orf72) human chromosome 9 open reading frame 72 and methods of using them |
| US12344839B2 (en) | 2020-03-27 | 2025-07-01 | University Of Massachusetts | Dual-acting siRNA based modulation of C9orf72 |
| JP7787094B2 (ja) * | 2020-04-09 | 2025-12-16 | アソシエーション・アンスティトゥート・ドゥ・マイオロジー | 筋萎縮性側索硬化症を治療するためのアンチセンス配列 |
| US20230272401A1 (en) * | 2020-06-11 | 2023-08-31 | Genetic Intelligence, Inc | Compositions for flcn gene modulation and methods thereof |
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| IL238266A0 (en) | 2015-06-30 |
| US10443052B2 (en) | 2019-10-15 |
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