IL277238A - Modified oligonucleotides for use in the treatment of tauopathy - Google Patents
Modified oligonucleotides for use in the treatment of tauopathyInfo
- Publication number
- IL277238A IL277238A IL277238A IL27723820A IL277238A IL 277238 A IL277238 A IL 277238A IL 277238 A IL277238 A IL 277238A IL 27723820 A IL27723820 A IL 27723820A IL 277238 A IL277238 A IL 277238A
- Authority
- IL
- Israel
- Prior art keywords
- tauopathies
- treatment
- modified oligonucleotides
- oligonucleotides
- modified
- Prior art date
Links
Classifications
-
- 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
-
- 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
- A61P25/28—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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- 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 ===---===
-
- 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
-
- 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
- C12N2310/3521—Methyl
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862642499P | 2018-03-13 | 2018-03-13 | |
| PCT/EP2019/056312 WO2019175260A2 (en) | 2018-03-13 | 2019-03-13 | Modified oligonucleotides and methods of use in tauopathies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL277238A true IL277238A (en) | 2020-10-29 |
Family
ID=65861258
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL277238A IL277238A (en) | 2018-03-13 | 2020-09-09 | Modified oligonucleotides for use in the treatment of tauopathy |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US11453881B2 (enExample) |
| EP (2) | EP3922637A1 (enExample) |
| JP (1) | JP2021515784A (enExample) |
| KR (1) | KR20200131263A (enExample) |
| CN (1) | CN111868068A (enExample) |
| AU (1) | AU2019233612A1 (enExample) |
| BR (1) | BR112020018365A2 (enExample) |
| CA (1) | CA3091979A1 (enExample) |
| EA (1) | EA202092156A1 (enExample) |
| ES (1) | ES3063421T3 (enExample) |
| IL (1) | IL277238A (enExample) |
| MA (1) | MA52149A (enExample) |
| MX (1) | MX2020009532A (enExample) |
| SG (1) | SG11202008895WA (enExample) |
| WO (1) | WO2019175260A2 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2866392C (en) | 2012-03-30 | 2023-10-24 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| TWI702046B (zh) | 2013-07-19 | 2020-08-21 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
| US20230250125A1 (en) * | 2020-04-01 | 2023-08-10 | Janssen Biopharma, Inc. | Nucleic acid polymers |
| US20250320493A1 (en) * | 2021-07-23 | 2025-10-16 | Biogen Ma Inc. | Methods for reducing tau expression |
| AU2024313057A1 (en) * | 2023-06-22 | 2025-10-30 | Denali Therapeutics Inc. | Compositions and methods for modulating tau expression |
| WO2025212467A1 (en) | 2024-03-31 | 2025-10-09 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of microtubule associated protein tau (mapt), compositions thereof, and methods of use |
| WO2026025082A1 (en) * | 2024-07-25 | 2026-01-29 | Biogen Ma Inc. | Methods for treating alzheimer's disease |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| BRPI0008131B8 (pt) | 1999-02-12 | 2021-05-25 | Daiichi Sankyo Co Ltd | composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| ATE356824T1 (de) | 1999-05-04 | 2007-04-15 | Santaris Pharma As | L-ribo-lna analoge |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| JP4151751B2 (ja) | 1999-07-22 | 2008-09-17 | 第一三共株式会社 | 新規ビシクロヌクレオシド類縁体 |
| HK1049339B (en) * | 1999-09-10 | 2008-06-27 | Geron Corporation | Oligonucleotide n3'-p5' thiophosphoramidates: their synthesis and use |
| US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma A/S | Synthesis of locked nucleic acid derivatives |
| CA2504694C (en) | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| ES2415665T3 (es) * | 2004-06-21 | 2013-07-26 | Proteome Sciences Plc | Procedimientos de cribado usando c-abl, fyn y sky en combinación con la proteína tau |
| GB0605337D0 (en) | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
| US9663784B2 (en) * | 2012-05-26 | 2017-05-30 | Bonac Corporation | Single-stranded nucleic acid molecule for regulating expression of gene having delivering function |
| US9228189B2 (en) * | 2012-10-26 | 2016-01-05 | Geron Corporation | C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders |
| BR112015022156A2 (pt) * | 2013-03-14 | 2017-11-14 | Isis Pharmaceuticals Inc | composições e métodos para modular a expressão de tau |
| TWI702046B (zh) * | 2013-07-19 | 2020-08-21 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
| HK1244030A1 (en) * | 2014-12-29 | 2018-07-27 | 东丽株式会社 | Composition containing nucleic acid molecule stably |
| CA2975525A1 (en) * | 2015-02-04 | 2016-08-11 | F. Hoffmann-La Roche Ag | Tau antisense oligomers and uses thereof |
| KR20250112919A (ko) | 2015-12-21 | 2025-07-24 | 노파르티스 아게 | 타우 발현을 감소시키기 위한 조성물 및 방법 |
-
2019
- 2019-03-13 SG SG11202008895WA patent/SG11202008895WA/en unknown
- 2019-03-13 MA MA052149A patent/MA52149A/fr unknown
- 2019-03-13 KR KR1020207027936A patent/KR20200131263A/ko not_active Ceased
- 2019-03-13 WO PCT/EP2019/056312 patent/WO2019175260A2/en not_active Ceased
- 2019-03-13 EP EP21170299.8A patent/EP3922637A1/en active Pending
- 2019-03-13 US US16/980,389 patent/US11453881B2/en active Active
- 2019-03-13 JP JP2020547392A patent/JP2021515784A/ja active Pending
- 2019-03-13 CN CN201980018910.3A patent/CN111868068A/zh active Pending
- 2019-03-13 MX MX2020009532A patent/MX2020009532A/es unknown
- 2019-03-13 CA CA3091979A patent/CA3091979A1/en active Pending
- 2019-03-13 ES ES19712535T patent/ES3063421T3/es active Active
- 2019-03-13 AU AU2019233612A patent/AU2019233612A1/en not_active Abandoned
- 2019-03-13 EA EA202092156A patent/EA202092156A1/ru unknown
- 2019-03-13 BR BR112020018365-2A patent/BR112020018365A2/pt not_active Application Discontinuation
- 2019-03-13 EP EP19712535.4A patent/EP3765476B1/en active Active
-
2020
- 2020-09-09 IL IL277238A patent/IL277238A/en unknown
-
2022
- 2022-09-26 US US17/935,518 patent/US20240011029A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3765476A2 (en) | 2021-01-20 |
| WO2019175260A3 (en) | 2019-11-14 |
| CN111868068A (zh) | 2020-10-30 |
| JP2021515784A (ja) | 2021-06-24 |
| US20240011029A1 (en) | 2024-01-11 |
| BR112020018365A2 (pt) | 2020-12-29 |
| US20210095284A1 (en) | 2021-04-01 |
| EP3765476B1 (en) | 2025-11-26 |
| MA52149A (fr) | 2021-01-20 |
| AU2019233612A1 (en) | 2020-09-10 |
| CA3091979A1 (en) | 2019-09-19 |
| KR20200131263A (ko) | 2020-11-23 |
| EA202092156A1 (ru) | 2021-04-01 |
| SG11202008895WA (en) | 2020-10-29 |
| EP3922637A1 (en) | 2021-12-15 |
| US11453881B2 (en) | 2022-09-27 |
| ES3063421T3 (en) | 2026-04-16 |
| WO2019175260A2 (en) | 2019-09-19 |
| MX2020009532A (es) | 2020-10-05 |
| EP3765476C0 (en) | 2025-11-26 |
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