BR112015022156A2 - composições e métodos para modular a expressão de tau - Google Patents
composições e métodos para modular a expressão de tauInfo
- Publication number
- BR112015022156A2 BR112015022156A2 BR112015022156A BR112015022156A BR112015022156A2 BR 112015022156 A2 BR112015022156 A2 BR 112015022156A2 BR 112015022156 A BR112015022156 A BR 112015022156A BR 112015022156 A BR112015022156 A BR 112015022156A BR 112015022156 A2 BR112015022156 A2 BR 112015022156A2
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- tau
- compounds
- compositions
- antisense
- Prior art date
Links
- 102000013498 tau Proteins Human genes 0.000 title abstract 7
- 108010026424 tau Proteins Proteins 0.000 title abstract 7
- 238000000034 method Methods 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 3
- 201000011240 Frontotemporal dementia Diseases 0.000 abstract 2
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 108020004999 messenger RNA Proteins 0.000 abstract 2
- 230000004770 neurodegeneration Effects 0.000 abstract 2
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 2
- 208000024827 Alzheimer disease Diseases 0.000 abstract 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 208000004051 Chronic Traumatic Encephalopathy Diseases 0.000 abstract 1
- 201000007547 Dravet syndrome Diseases 0.000 abstract 1
- 208000024658 Epilepsy syndrome Diseases 0.000 abstract 1
- 208000002877 Epileptic Syndromes Diseases 0.000 abstract 1
- 208000036572 Myoclonic epilepsy Diseases 0.000 abstract 1
- 206010073677 Severe myoclonic epilepsy of infancy Diseases 0.000 abstract 1
- 230000001668 ameliorated effect Effects 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 208000017004 dementia pugilistica Diseases 0.000 abstract 1
- 206010015037 epilepsy Diseases 0.000 abstract 1
- 201000002212 progressive supranuclear palsy Diseases 0.000 abstract 1
Classifications
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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
- 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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- 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
- A61P25/28—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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- 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
- C12N2310/10—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
- 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
- C12N2310/31—Chemical structure of the backbone
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/31—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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- 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
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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
- C12N2310/321—2'-O-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
- C12N2310/322—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
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/32—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
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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
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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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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- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (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)
- Neurosurgery (AREA)
- Neurology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Epidemiology (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361785177P | 2013-03-14 | 2013-03-14 | |
| US201461943931P | 2014-02-24 | 2014-02-24 | |
| PCT/US2014/029752 WO2014153236A1 (en) | 2013-03-14 | 2014-03-14 | Compositions and methods for modulating tau expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112015022156A2 true BR112015022156A2 (pt) | 2017-11-14 |
Family
ID=51581461
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112015022156A BR112015022156A2 (pt) | 2013-03-14 | 2014-03-14 | composições e métodos para modular a expressão de tau |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US9644207B2 (enExample) |
| EP (2) | EP2971142B1 (enExample) |
| JP (2) | JP6629716B2 (enExample) |
| KR (1) | KR20150130430A (enExample) |
| CN (2) | CN105264091B (enExample) |
| AU (1) | AU2014236156C1 (enExample) |
| BR (1) | BR112015022156A2 (enExample) |
| CA (1) | CA2906119A1 (enExample) |
| ES (1) | ES2807379T3 (enExample) |
| IL (1) | IL241439B (enExample) |
| MX (1) | MX2015012621A (enExample) |
| RU (1) | RU2745324C2 (enExample) |
| WO (1) | WO2014153236A1 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013202595B2 (en) | 2012-03-30 | 2016-04-21 | Biogen Ma Inc. | Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| KR20150130430A (ko) | 2013-03-14 | 2015-11-23 | 아이시스 파마수티컬즈 인코포레이티드 | 타우 발현을 조절하는 조성물 및 방법 |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ma公司 | 用於調節τ蛋白表現之組合物 |
| TW201641691A (zh) | 2015-02-04 | 2016-12-01 | 必治妥美雅史谷比公司 | Tau反義寡聚物及其用途 |
| CN115181778A (zh) | 2015-02-04 | 2022-10-14 | 百时美施贵宝公司 | 选择治疗性分子的方法 |
| EP3274456B1 (en) * | 2015-03-25 | 2020-06-24 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| EP3377629B1 (en) | 2015-11-18 | 2023-07-05 | Rosalind Franklin University of Medicine and Science | Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease |
| US10370667B2 (en) | 2015-11-18 | 2019-08-06 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease |
| JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| AU2017367722B2 (en) * | 2016-12-01 | 2024-02-01 | Sangamo Therapeutics, Inc. | Tau modulators and methods and compositions for delivery thereof |
| TWI809004B (zh) * | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
| CN111868068A (zh) | 2018-03-13 | 2020-10-30 | 詹森药业有限公司 | 用于在tau蛋白病变治疗中使用的经修饰的寡核苷酸 |
| KR102585973B1 (ko) | 2018-07-03 | 2023-10-10 | 에프. 호프만-라 로슈 아게 | 타우 발현을 조절하기 위한 올리고뉴클레오티드 |
| JP7340794B2 (ja) * | 2018-07-04 | 2023-09-08 | 学校法人 愛知医科大学 | タウのスプライシングを制御するアンチセンスオリゴヌクレオチド及びその用途 |
| CN113226333A (zh) | 2018-10-02 | 2021-08-06 | 桑格摩生物治疗股份有限公司 | 用于调控Tau蛋白的方法和组合物 |
| BR112021007476A2 (pt) * | 2018-11-21 | 2021-11-03 | Ionis Pharmaceuticals Inc | Compostos e métodos para reduzir a expressão de príon |
| US20220333105A1 (en) * | 2019-06-03 | 2022-10-20 | Quralis Corporation | Oligonucleotides and methods of use for treating neurological diseases |
| MX2022012293A (es) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de acido ribonucleico de interferencia (arni) de la proteina tau asociada a microtubulos (mapt) y sus metodos de uso. |
| EP4373938A2 (en) * | 2021-07-23 | 2024-05-29 | University Of Kentucky Research Foundation | Inhibition of neurofibrillary tangles using oligonucleotides against circular rnas from the microtubule associated protein tau (mapt) locus |
| US11761284B2 (en) | 2021-07-26 | 2023-09-19 | Benton Frederick Baugh | Method for BOP stack structure |
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| KR20150130430A (ko) | 2015-11-23 |
| JP2019141097A (ja) | 2019-08-29 |
| CN105264091B (zh) | 2020-02-28 |
| IL241439B (en) | 2020-03-31 |
| RU2745324C2 (ru) | 2021-03-23 |
| CN111254145A (zh) | 2020-06-09 |
| MX2015012621A (es) | 2016-05-31 |
| EP2971142B1 (en) | 2020-06-24 |
| JP6629716B2 (ja) | 2020-01-15 |
| EP3766974A1 (en) | 2021-01-20 |
| JP2016513976A (ja) | 2016-05-19 |
| CN105264091A (zh) | 2016-01-20 |
| AU2014236156B2 (en) | 2020-05-28 |
| HK1218659A1 (en) | 2017-03-03 |
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