JP6606557B2 - C9orf72発現を調節するための組成物 - Google Patents
C9orf72発現を調節するための組成物 Download PDFInfo
- Publication number
- JP6606557B2 JP6606557B2 JP2017546618A JP2017546618A JP6606557B2 JP 6606557 B2 JP6606557 B2 JP 6606557B2 JP 2017546618 A JP2017546618 A JP 2017546618A JP 2017546618 A JP2017546618 A JP 2017546618A JP 6606557 B2 JP6606557 B2 JP 6606557B2
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- JP
- Japan
- Prior art keywords
- c9orf72
- certain embodiments
- antisense
- compound
- modified
- 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.)
- Expired - Fee Related
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- 0 C*C(C)C(C(C)(N)OC)(C1(C)OCIC)OC(*)(*2C3CC3)C2(*C)C1(N)P Chemical compound C*C(C)C(C(C)(N)OC)(C1(C)OCIC)OC(*)(*2C3CC3)C2(*C)C1(N)P 0.000 description 2
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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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- 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
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- 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
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- 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
- A61P25/22—Anxiolytics
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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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- 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
- C07H21/04—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 deoxyribosyl as saccharide radical
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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
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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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- 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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- 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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- 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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- 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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- 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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- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- 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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- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Neurology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Psychiatry (AREA)
- Pain & Pain Management (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019190648A JP6924242B2 (ja) | 2015-04-16 | 2019-10-18 | C9orf72発現を調節するための組成物 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562148691P | 2015-04-16 | 2015-04-16 | |
| US62/148,691 | 2015-04-16 | ||
| US201562232941P | 2015-09-25 | 2015-09-25 | |
| US62/232,941 | 2015-09-25 | ||
| US201562239400P | 2015-10-09 | 2015-10-09 | |
| US62/239,400 | 2015-10-09 | ||
| PCT/US2016/027747 WO2016168592A2 (en) | 2015-04-16 | 2016-04-15 | Compositions for modulating c9orf72 expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019190648A Division JP6924242B2 (ja) | 2015-04-16 | 2019-10-18 | C9orf72発現を調節するための組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018512127A JP2018512127A (ja) | 2018-05-17 |
| JP2018512127A5 JP2018512127A5 (enExample) | 2019-05-23 |
| JP6606557B2 true JP6606557B2 (ja) | 2019-11-13 |
Family
ID=57126261
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017546618A Expired - Fee Related JP6606557B2 (ja) | 2015-04-16 | 2016-04-15 | C9orf72発現を調節するための組成物 |
| JP2019190648A Expired - Fee Related JP6924242B2 (ja) | 2015-04-16 | 2019-10-18 | C9orf72発現を調節するための組成物 |
| JP2021124953A Ceased JP2021184720A (ja) | 2015-04-16 | 2021-07-30 | C9orf72発現を調節するための組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019190648A Expired - Fee Related JP6924242B2 (ja) | 2015-04-16 | 2019-10-18 | C9orf72発現を調節するための組成物 |
| JP2021124953A Ceased JP2021184720A (ja) | 2015-04-16 | 2021-07-30 | C9orf72発現を調節するための組成物 |
Country Status (30)
| Country | Link |
|---|---|
| US (4) | US9605263B2 (enExample) |
| EP (2) | EP3722424A1 (enExample) |
| JP (3) | JP6606557B2 (enExample) |
| KR (2) | KR102258516B1 (enExample) |
| CN (2) | CN113265404A (enExample) |
| AU (2) | AU2016249002B2 (enExample) |
| BR (1) | BR112017016068A2 (enExample) |
| CA (1) | CA2978103A1 (enExample) |
| CL (4) | CL2017002567A1 (enExample) |
| CO (1) | CO2017007671A2 (enExample) |
| CY (1) | CY1122981T1 (enExample) |
| DK (1) | DK3283080T3 (enExample) |
| ES (1) | ES2791995T3 (enExample) |
| HR (1) | HRP20200843T1 (enExample) |
| HU (1) | HUE049233T2 (enExample) |
| IL (2) | IL253557B (enExample) |
| LT (1) | LT3283080T (enExample) |
| MX (2) | MX366128B (enExample) |
| MY (1) | MY181815A (enExample) |
| NZ (1) | NZ733882A (enExample) |
| PH (2) | PH12017501875B1 (enExample) |
| PL (1) | PL3283080T3 (enExample) |
| PT (1) | PT3283080T (enExample) |
| RS (1) | RS60230B1 (enExample) |
| RU (1) | RU2736574C2 (enExample) |
| SA (3) | SA517390168B1 (enExample) |
| SG (2) | SG10202001856WA (enExample) |
| SI (1) | SI3283080T1 (enExample) |
| SM (1) | SMT202000165T1 (enExample) |
| WO (1) | WO2016168592A2 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2616051T3 (es) | 2008-12-02 | 2017-06-09 | Wave Life Sciences Japan, Inc. | Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| JP6128529B2 (ja) | 2011-07-19 | 2017-05-17 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | 官能化核酸の合成のための方法 |
| US9598458B2 (en) | 2012-07-13 | 2017-03-21 | Wave Life Sciences Japan, Inc. | Asymmetric auxiliary group |
| EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| US9963699B2 (en) | 2012-10-15 | 2018-05-08 | Ionis Pharmaceuticals, Inc. | Methods for modulating C9ORF72 expression |
| KR20150070278A (ko) | 2012-10-15 | 2015-06-24 | 아이시스 파마수티컬즈 인코포레이티드 | C9orf72 발현 조절용 조성물 |
| EP3055414A4 (en) * | 2013-10-11 | 2017-07-19 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| DK3094728T3 (da) | 2014-01-16 | 2022-05-16 | Wave Life Sciences Ltd | Kiralt design |
| WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| CA3017532A1 (en) * | 2016-04-13 | 2017-10-19 | Ionis Pharmaceuticals, Inc. | Methods for reducing c9orf72 expression |
| WO2018081728A1 (en) * | 2016-10-31 | 2018-05-03 | Emendobio Inc. | Compositions for genome editing |
| EP3664816A4 (en) | 2017-08-08 | 2021-05-19 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES |
| US10905707B2 (en) | 2017-11-10 | 2021-02-02 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
| CA3088112A1 (en) | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| MX2020007439A (es) | 2018-01-12 | 2020-09-14 | Bristol Myers Squibb Co | Oligonucleotidos antisentido que actuan sobre alfa-sinucleina y usos de estos. |
| CN113164557A (zh) * | 2018-07-23 | 2021-07-23 | 因柯利尔疗法公司 | 治疗神经性病症的方法 |
| WO2020027311A1 (ja) | 2018-08-03 | 2020-02-06 | 国立大学法人東京医科歯科大学 | Tdp-43凝集阻害剤 |
| JP7455831B2 (ja) * | 2018-11-21 | 2024-03-26 | 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 | プリオン発現を低減するための化合物及び方法 |
| EP3890752A1 (en) | 2018-12-06 | 2021-10-13 | Biogen MA Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| WO2020205605A2 (en) * | 2019-03-29 | 2020-10-08 | University Of Massachusetts | OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72 |
| CN114072501A (zh) | 2019-05-06 | 2022-02-18 | 马萨诸塞大学 | 抗c9orf72寡核苷酸及相关方法 |
| CN114585370A (zh) * | 2019-10-06 | 2022-06-03 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| WO2024036186A2 (en) * | 2022-08-09 | 2024-02-15 | The University Of Massachusetts | Identification of allele-specific target sequences for c9orf72 |
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| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
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| DK0942000T3 (da) | 1989-10-24 | 2004-11-01 | Isis Pharmaceuticals Inc | 2'-modificerede oligonukleotider |
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| GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
| ATE167523T1 (de) | 1990-05-11 | 1998-07-15 | Microprobe Corp | Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden |
| US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| ES2103918T3 (es) | 1991-10-17 | 1997-10-01 | Ciba Geigy Ag | Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios. |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| CA2126691C (en) | 1991-12-24 | 2003-05-06 | Phillip Dan Cook | Gapped 2' modified oligonucleotides |
| FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
| EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
| WO1994022864A1 (en) | 1993-03-30 | 1994-10-13 | Sterling Winthrop Inc. | Acyclic nucleoside analogs and oligonucleotide sequences containing them |
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