PH12016500603A1 - Compositions for modulating c9orf72 expression - Google Patents
Compositions for modulating c9orf72 expressionInfo
- Publication number
- PH12016500603A1 PH12016500603A1 PH12016500603A PH12016500603A PH12016500603A1 PH 12016500603 A1 PH12016500603 A1 PH 12016500603A1 PH 12016500603 A PH12016500603 A PH 12016500603A PH 12016500603 A PH12016500603 A PH 12016500603A PH 12016500603 A1 PH12016500603 A1 PH 12016500603A1
- Authority
- PH
- Philippines
- Prior art keywords
- c90rf72
- compositions
- methods
- mrna
- exon
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 108020004999 messenger RNA Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 2
- 239000003112 inhibitor Substances 0.000 abstract 2
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 230000004770 neurodegeneration Effects 0.000 abstract 1
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 1
- 230000001717 pathogenic effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000008685 targeting Effects 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
- 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
- G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
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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
- C12N2310/32—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
- C12N2310/323—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
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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
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- Pathology (AREA)
- Analytical Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Physics & Mathematics (AREA)
- Food Science & Technology (AREA)
- Psychiatry (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361890108P | 2013-10-11 | 2013-10-11 | |
| US201361891313P | 2013-10-15 | 2013-10-15 | |
| US201361919540P | 2013-12-20 | 2013-12-20 | |
| US201461927903P | 2014-01-15 | 2014-01-15 | |
| US201461980502P | 2014-04-16 | 2014-04-16 | |
| PCT/US2014/060194 WO2015054676A2 (en) | 2013-10-11 | 2014-10-11 | Compositions for modulating c9orf72 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PH12016500603A1 true PH12016500603A1 (en) | 2016-06-13 |
Family
ID=52813758
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PH12016500603A PH12016500603A1 (en) | 2013-10-11 | 2016-04-04 | Compositions for modulating c9orf72 expression |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US10221414B2 (enExample) |
| EP (2) | EP3055414A4 (enExample) |
| JP (2) | JP6679476B2 (enExample) |
| KR (1) | KR20160062069A (enExample) |
| CN (2) | CN105637090B (enExample) |
| AU (2) | AU2014331652B2 (enExample) |
| BR (1) | BR112016007751A2 (enExample) |
| CA (1) | CA2926408A1 (enExample) |
| CL (3) | CL2016000839A1 (enExample) |
| IL (1) | IL244759A0 (enExample) |
| MX (2) | MX2016004651A (enExample) |
| MY (1) | MY192689A (enExample) |
| PH (1) | PH12016500603A1 (enExample) |
| RU (1) | RU2748426C2 (enExample) |
| SG (2) | SG11201602597YA (enExample) |
| WO (1) | WO2015054676A2 (enExample) |
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| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| EP2906696B2 (en) | 2012-10-15 | 2022-12-14 | Ionis Pharmaceuticals, Inc. | Methods for modulating c9orf72 expression |
| EP2906697A4 (en) | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| RU2730677C2 (ru) | 2012-10-15 | 2020-08-24 | Ионис Фармасьютикалз, Инк. | Соединение для модуляции экспрессии гена c9orf72 и его применение |
| BR112016007751A2 (pt) | 2013-10-11 | 2017-09-12 | Ionis Pharmaceuticals Inc | composições para modulação de expressão de c9orf72 |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| MX366128B (es) * | 2015-04-16 | 2019-06-28 | Ionis Pharmaceuticals Inc | Composiciones para modular la expresion de c90rf72. |
| ES2917181T3 (es) | 2015-07-10 | 2022-07-07 | Ionis Pharmaceuticals Inc | Moduladores de diacilglicerol aciltransferasa 2 (DGAT2) |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| CN105663129B (zh) * | 2015-12-29 | 2020-05-15 | 山东大学 | 用于治疗肌萎缩侧索硬化和额颞叶痴呆的化合物与应用 |
| AU2017248637A1 (en) * | 2016-04-13 | 2018-09-27 | Ionis Pharmaceuticals, Inc. | Methods for reducing C9ORF72 expression |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| US11859179B2 (en) | 2017-05-09 | 2024-01-02 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (ALS) |
| CN110996968A (zh) * | 2017-08-08 | 2020-04-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| WO2019060686A1 (en) | 2017-09-22 | 2019-03-28 | University Of Massachusetts | NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF |
| EP3701030A4 (en) * | 2017-10-23 | 2022-04-20 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR NEURODEGENERATIVE DISEASES |
| WO2019094694A1 (en) | 2017-11-10 | 2019-05-16 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| WO2020055917A1 (en) * | 2018-09-10 | 2020-03-19 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating cln3 expression |
| WO2020072428A1 (en) * | 2018-10-01 | 2020-04-09 | United Neuroscience | Peptide immunogen constructs directed against dipeptide repeat proteins from c9orf72 |
| EP3890752A1 (en) | 2018-12-06 | 2021-10-13 | Biogen MA Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| EP3918073A4 (en) * | 2019-01-29 | 2023-11-22 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION |
| WO2020205605A2 (en) * | 2019-03-29 | 2020-10-08 | University Of Massachusetts | OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72 |
| WO2020227395A2 (en) | 2019-05-06 | 2020-11-12 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| BR112022011417A2 (pt) * | 2019-12-13 | 2022-08-30 | Alnylam Pharmaceuticals Inc | Composições do agente de irna da fase de leitura aberta 72 do cromossomo humano 9 (c9orf72) e métodos de uso das mesmas |
| WO2021159008A2 (en) | 2020-02-07 | 2021-08-12 | Maze Therapeutics, Inc. | Compositions and methods for treating neurodegenerative diseases |
| WO2021174031A2 (en) * | 2020-02-28 | 2021-09-02 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating splicing of pre-mrna |
| WO2021195510A1 (en) | 2020-03-27 | 2021-09-30 | University Of Massachusetts | Dual-acting sirna based modulation of c9orf72 |
| BR112022020412A2 (pt) * | 2020-04-09 | 2023-05-02 | Association Inst De Myologie | Sequências antissenso para tratar esclerose lateral amiotrófica |
| BR112023001624A2 (pt) | 2020-07-28 | 2023-02-23 | Ionis Pharmaceuticals Inc | Compostos e métodos para reduzir a expressão de app |
| WO2022216759A1 (en) | 2021-04-06 | 2022-10-13 | Maze Therapeutics, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
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| WO2025107038A1 (en) * | 2023-11-23 | 2025-05-30 | Murdoch University | Method of treating motor neurone disease |
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