JP6126009B2 - α−シヌクレイン発現の調節 - Google Patents
α−シヌクレイン発現の調節 Download PDFInfo
- Publication number
- JP6126009B2 JP6126009B2 JP2013540028A JP2013540028A JP6126009B2 JP 6126009 B2 JP6126009 B2 JP 6126009B2 JP 2013540028 A JP2013540028 A JP 2013540028A JP 2013540028 A JP2013540028 A JP 2013540028A JP 6126009 B2 JP6126009 B2 JP 6126009B2
- Authority
- JP
- Japan
- Prior art keywords
- modified
- compound
- certain embodiments
- synuclein
- modified oligonucleotide
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
-
- 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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- 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
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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
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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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Neurology (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US41484810P | 2010-11-17 | 2010-11-17 | |
| US61/414,848 | 2010-11-17 | ||
| PCT/US2011/061245 WO2012068405A2 (en) | 2010-11-17 | 2011-11-17 | Modulation of alpha synuclein expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017004099A Division JP2017079786A (ja) | 2010-11-17 | 2017-01-13 | α−シヌクレイン発現の調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014501507A JP2014501507A (ja) | 2014-01-23 |
| JP2014501507A5 JP2014501507A5 (enExample) | 2016-07-07 |
| JP6126009B2 true JP6126009B2 (ja) | 2017-05-10 |
Family
ID=46084657
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013540028A Active JP6126009B2 (ja) | 2010-11-17 | 2011-11-17 | α−シヌクレイン発現の調節 |
| JP2017004099A Withdrawn JP2017079786A (ja) | 2010-11-17 | 2017-01-13 | α−シヌクレイン発現の調節 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017004099A Withdrawn JP2017079786A (ja) | 2010-11-17 | 2017-01-13 | α−シヌクレイン発現の調節 |
Country Status (6)
| Country | Link |
|---|---|
| US (4) | US9663783B2 (enExample) |
| EP (2) | EP2640853B1 (enExample) |
| JP (2) | JP6126009B2 (enExample) |
| AU (2) | AU2011329777B2 (enExample) |
| CA (2) | CA3077910A1 (enExample) |
| WO (1) | WO2012068405A2 (enExample) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11359197B2 (en) | 2018-01-12 | 2022-06-14 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| US12241065B2 (en) | 2018-01-12 | 2025-03-04 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT2560687T (lt) * | 2010-04-19 | 2017-09-25 | Nlife Therapeutics S.L. | Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams |
| WO2012068405A2 (en) | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| JP6294876B2 (ja) | 2012-06-25 | 2018-03-14 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | Ube3a−ats発現の調節 |
| ES2967530T3 (es) * | 2012-10-26 | 2024-04-30 | Palomo Ltd | Composiciones y métodos para el tratamiento de la enfermedad de Parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas |
| US10762374B2 (en) * | 2014-12-30 | 2020-09-01 | Blinkfire Analytics, Inc. | High accuracy image identification system |
| SI3283080T1 (sl) * | 2015-04-16 | 2020-06-30 | Ionis Pharmaceuticals, Inc. | Sestavki za moduliranje izražanja C9ORF72 |
| UA125963C2 (uk) | 2015-11-12 | 2022-07-20 | Ф. Хоффманн-Ля Рош Аг | Олігонуклеотид для індукції батьківської експресії ube3a |
| 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 |
| US9840710B2 (en) | 2015-11-18 | 2017-12-12 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease |
| WO2017119463A1 (ja) * | 2016-01-07 | 2017-07-13 | 国立大学法人大阪大学 | α-シヌクレイン発現抑制剤 |
| IL263913B2 (en) * | 2016-06-29 | 2025-01-01 | Univ California | Structure-based peptides that inhibit alpha-synuclein aggregation |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| WO2019009298A1 (ja) * | 2017-07-05 | 2019-01-10 | 国立大学法人大阪大学 | α-シヌクレイン発現抑制剤 |
| EP3628737A4 (en) * | 2017-07-05 | 2021-01-20 | Osaka University | ENA-ANTISENSE-OLIGONUCLEOTIDE TO INHIBIT ALPHA-SYNUCLEIN EXPRESSION |
| 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 |
| MA51634A (fr) | 2018-01-12 | 2020-11-18 | Roche Innovation Ct Copenhagen As | Oligonucléotides antisens d'alpha-synucléine et leurs utilisations |
| CA3108526A1 (en) * | 2018-08-03 | 2020-02-06 | Genzyme Corporation | Variant rnai against alpha-synuclein |
| BR112022018475A2 (pt) * | 2020-03-18 | 2022-11-29 | Univ Massachusetts | Oligonucleotídeos para modulação de snca |
| AU2021353935A1 (en) * | 2020-10-01 | 2023-05-25 | Alnylam Pharmaceuticals, Inc. | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
| PE20240696A1 (es) | 2021-03-08 | 2024-04-10 | Servier Lab | Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina |
| US20250382611A1 (en) * | 2021-06-21 | 2025-12-18 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| WO2023215863A2 (en) * | 2022-05-06 | 2023-11-09 | Ionis Pharmaceuticals, Inc. | Rnai agents for modulating snca |
| IL322701A (en) * | 2023-02-17 | 2025-10-01 | Ionis Pharmaceuticals Inc | Compounds and methods for regulating alpha-synuclein expression |
| TW202528540A (zh) * | 2023-09-20 | 2025-07-16 | 日商第一三共股份有限公司 | 抑制α-突觸核蛋白之表現的siRNA |
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2011
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Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11359197B2 (en) | 2018-01-12 | 2022-06-14 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| US11447775B2 (en) | 2018-01-12 | 2022-09-20 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| US12180478B2 (en) | 2018-01-12 | 2024-12-31 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| US12241065B2 (en) | 2018-01-12 | 2025-03-04 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
Also Published As
| Publication number | Publication date |
|---|---|
| AU2016225852B2 (en) | 2018-09-27 |
| JP2017079786A (ja) | 2017-05-18 |
| WO2012068405A3 (en) | 2012-07-12 |
| US20250136982A1 (en) | 2025-05-01 |
| CA3077910A1 (en) | 2012-05-24 |
| US20210087562A1 (en) | 2021-03-25 |
| EP2640853A2 (en) | 2013-09-25 |
| AU2016225852A1 (en) | 2016-09-29 |
| EP2640853B1 (en) | 2018-12-26 |
| US10815480B2 (en) | 2020-10-27 |
| CA2817960A1 (en) | 2012-05-24 |
| US20140005252A1 (en) | 2014-01-02 |
| JP2014501507A (ja) | 2014-01-23 |
| US12043831B2 (en) | 2024-07-23 |
| US20180073022A1 (en) | 2018-03-15 |
| AU2011329777A1 (en) | 2013-05-02 |
| EP2640853A4 (en) | 2015-01-07 |
| AU2011329777B2 (en) | 2016-06-09 |
| US9663783B2 (en) | 2017-05-30 |
| WO2012068405A2 (en) | 2012-05-24 |
| EP3521451A1 (en) | 2019-08-07 |
| CA2817960C (en) | 2020-06-09 |
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