DK2906696T3 - Fremgangsmåder til modulering af c9orf72-ekspression - Google Patents
Fremgangsmåder til modulering af c9orf72-ekspression Download PDFInfo
- Publication number
- DK2906696T3 DK2906696T3 DK13846313T DK13846313T DK2906696T3 DK 2906696 T3 DK2906696 T3 DK 2906696T3 DK 13846313 T DK13846313 T DK 13846313T DK 13846313 T DK13846313 T DK 13846313T DK 2906696 T3 DK2906696 T3 DK 2906696T3
- Authority
- DK
- Denmark
- Prior art keywords
- methods
- c9orf72 expression
- modulating
- modulating c9orf72
- expression
- Prior art date
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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
- 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
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
-
- 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
-
- 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
- C12N2310/3233—Morpholino-type ring
-
- 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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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Veterinary Medicine (AREA)
- Psychiatry (AREA)
- Public Health (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Steroid Compounds (AREA)
- Liquid Crystal Substances (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261714139P | 2012-10-15 | 2012-10-15 | |
| PCT/US2013/065067 WO2014062686A1 (en) | 2012-10-15 | 2013-10-15 | Methods for modulating c9orf72 expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DK2906696T3 true DK2906696T3 (da) | 2019-11-25 |
| DK2906696T4 DK2906696T4 (da) | 2023-02-27 |
Family
ID=50488699
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK13846313.8T DK2906696T4 (da) | 2012-10-15 | 2013-10-15 | Fremgangsmåder til modulering af c9orf72-ekspression |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US9963699B2 (da) |
| EP (1) | EP2906696B2 (da) |
| DK (1) | DK2906696T4 (da) |
| ES (1) | ES2762326T5 (da) |
| FI (1) | FI2906696T4 (da) |
| PT (1) | PT2906696T (da) |
| WO (1) | WO2014062686A1 (da) |
Families Citing this family (46)
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| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9963699B2 (en) | 2012-10-15 | 2018-05-08 | Ionis Pharmaceuticals, Inc. | Methods for modulating C9ORF72 expression |
| RU2730677C2 (ru) | 2012-10-15 | 2020-08-24 | Ионис Фармасьютикалз, Инк. | Соединение для модуляции экспрессии гена c9orf72 и его применение |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| SG10201808903UA (en) * | 2013-10-11 | 2018-11-29 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
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| RU2730677C2 (ru) | 2012-10-15 | 2020-08-24 | Ионис Фармасьютикалз, Инк. | Соединение для модуляции экспрессии гена c9orf72 и его применение |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| US9963699B2 (en) | 2012-10-15 | 2018-05-08 | Ionis Pharmaceuticals, Inc. | Methods for modulating C9ORF72 expression |
| RS59283B1 (sr) | 2013-01-22 | 2019-10-31 | Deutsches Zentrum Fuer Neurodegenerative Erkrankungen E V | Proteini sa dipeptidnim ponovkom kao terapeutsko ciljno mesto kod neurodegenerativnih bolesti sa ekspanzijom heksanukleotidnog ponovka |
| SG10201808903UA (en) | 2013-10-11 | 2018-11-29 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
| EP2868746A1 (en) | 2013-10-29 | 2015-05-06 | Deutsches Krebsforschungszentrum Stiftung des Öffentlichen Rechts | Micrornas modulating the effect of glucocorticoid signaling |
| DE102014204360A1 (de) | 2014-03-10 | 2015-09-10 | Ford Global Technologies, Llc | Verfahren sowie Vorrichtung zur Abschätzung des Abstandes eines in Bewegung befindlichen Fahrzeuges von einem Objekt |
| WO2016024205A1 (en) | 2014-08-15 | 2016-02-18 | Pfizer Inc. | Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene |
| US10538762B2 (en) | 2014-10-14 | 2020-01-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat |
| SI3283080T1 (sl) | 2015-04-16 | 2020-06-30 | Ionis Pharmaceuticals, Inc. | Sestavki za moduliranje izražanja C9ORF72 |
| US9778708B1 (en) | 2016-07-18 | 2017-10-03 | Lenovo Enterprise Solutions (Singapore) Pte. Ltd. | Dual sided latching retainer for computer modules |
-
2013
- 2013-10-15 US US14/436,030 patent/US9963699B2/en active Active
- 2013-10-15 EP EP13846313.8A patent/EP2906696B2/en active Active
- 2013-10-15 WO PCT/US2013/065067 patent/WO2014062686A1/en not_active Ceased
- 2013-10-15 PT PT138463138T patent/PT2906696T/pt unknown
- 2013-10-15 FI FIEP13846313.8T patent/FI2906696T4/fi active
- 2013-10-15 DK DK13846313.8T patent/DK2906696T4/da active
- 2013-10-15 ES ES13846313T patent/ES2762326T5/es active Active
Also Published As
| Publication number | Publication date |
|---|---|
| EP2906696A4 (en) | 2016-06-08 |
| EP2906696B2 (en) | 2022-12-14 |
| PT2906696T (pt) | 2019-12-16 |
| US9963699B2 (en) | 2018-05-08 |
| ES2762326T3 (es) | 2020-05-22 |
| DK2906696T4 (da) | 2023-02-27 |
| US20150267197A1 (en) | 2015-09-24 |
| WO2014062686A1 (en) | 2014-04-24 |
| EP2906696B1 (en) | 2019-10-09 |
| ES2762326T5 (es) | 2023-04-27 |
| EP2906696A1 (en) | 2015-08-19 |
| FI2906696T4 (fi) | 2023-03-18 |
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