DK3022217T3 - Sammensætninger til modulering af tau-ekspression - Google Patents
Sammensætninger til modulering af tau-ekspression Download PDFInfo
- Publication number
- DK3022217T3 DK3022217T3 DK14826839.4T DK14826839T DK3022217T3 DK 3022217 T3 DK3022217 T3 DK 3022217T3 DK 14826839 T DK14826839 T DK 14826839T DK 3022217 T3 DK3022217 T3 DK 3022217T3
- Authority
- DK
- Denmark
- Prior art keywords
- modulation
- compositions
- tau expression
- tau
- expression
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
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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
- 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
- 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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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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
- A61P25/08—Antiepileptics; Anticonvulsants
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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
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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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
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/33—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
- C12N2310/341—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
- C12N2310/345—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
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Steroid Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
- Thiazole And Isothizaole Compounds (AREA)
- Pyridine Compounds (AREA)
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361856551P | 2013-07-19 | 2013-07-19 | |
US201361879621P | 2013-09-18 | 2013-09-18 | |
US201361885371P | 2013-10-01 | 2013-10-01 | |
US201462014486P | 2014-06-19 | 2014-06-19 | |
PCT/US2014/047486 WO2015010135A2 (en) | 2013-07-19 | 2014-07-21 | Compositions for modulating tau expression |
Publications (1)
Publication Number | Publication Date |
---|---|
DK3022217T3 true DK3022217T3 (da) | 2021-06-28 |
Family
ID=52346860
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK14826839.4T DK3022217T3 (da) | 2013-07-19 | 2014-07-21 | Sammensætninger til modulering af tau-ekspression |
Country Status (25)
Country | Link |
---|---|
US (4) | US9683235B2 (da) |
EP (2) | EP3022217B1 (da) |
JP (4) | JP6684707B2 (da) |
KR (4) | KR102273926B1 (da) |
CN (2) | CN108064227B (da) |
AU (2) | AU2014290395B2 (da) |
BR (1) | BR112016000654B1 (da) |
CA (2) | CA2918600C (da) |
CY (1) | CY1124292T1 (da) |
DK (1) | DK3022217T3 (da) |
ES (1) | ES2871533T3 (da) |
HK (1) | HK1223626A1 (da) |
HR (1) | HRP20211051T1 (da) |
HU (1) | HUE055144T2 (da) |
IL (4) | IL279572B2 (da) |
LT (1) | LT3022217T (da) |
MX (2) | MX2016000782A (da) |
NZ (2) | NZ715828A (da) |
PL (1) | PL3022217T3 (da) |
PT (1) | PT3022217T (da) |
RS (1) | RS62054B1 (da) |
RU (1) | RU2735551C2 (da) |
SI (1) | SI3022217T1 (da) |
TW (4) | TW202246503A (da) |
WO (1) | WO2015010135A2 (da) |
Families Citing this family (37)
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BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
KR101885383B1 (ko) | 2009-07-06 | 2018-08-03 | 웨이브 라이프 사이언시스 리미티드 | 신규한 핵산 프로드러그 및 그의 사용 방법 |
JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
WO2013148260A1 (en) | 2012-03-30 | 2013-10-03 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
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EP4219516A3 (en) | 2012-07-13 | 2024-01-10 | Wave Life Sciences Ltd. | Chiral control |
SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
EP2873674B1 (en) | 2012-07-13 | 2020-05-06 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
US9644207B2 (en) | 2013-03-14 | 2017-05-09 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating Tau expression |
TW202246503A (zh) | 2013-07-19 | 2022-12-01 | 美商百健Ma公司 | 用於調節τ蛋白表現之組合物 |
WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
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KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
EP3254104B1 (en) | 2015-02-04 | 2021-08-25 | Bristol-Myers Squibb Company | Methods of selecting therapeutic molecules |
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US10513701B2 (en) | 2015-03-25 | 2019-12-24 | Università Degli Studi Di Trento | RNA interference mediated therapy for neurodegenerative diseases |
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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 |
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 |
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 |
WO2018102397A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
KR20190085529A (ko) | 2016-12-01 | 2019-07-18 | 상가모 테라퓨틱스, 인코포레이티드 | Tau 조절제 및 그것의 전달을 위한 방법 및 조성물 |
MX2020009532A (es) * | 2018-03-13 | 2020-10-05 | Janssen Pharmaceutica Nv | Oligonucleotidos modificados para uso en el tratamiento de tauopatias. |
MA53924A (fr) | 2018-05-22 | 2021-08-25 | Ionis Pharmaceuticals Inc | Modulateurs de l'expression d'apol1 |
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UY38282A (es) * | 2018-06-27 | 2019-12-31 | Ionis Pharmaceuticals Inc | Compuestos y métodos para la reducción de la expresión de lrrk2 |
KR20230143199A (ko) * | 2018-07-03 | 2023-10-11 | 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 |
TW202023573A (zh) | 2018-09-19 | 2020-07-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
JP2023515862A (ja) * | 2020-03-01 | 2023-04-14 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
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