CA2667055C - Antisense compounds - Google Patents
Antisense compounds Download PDFInfo
- Publication number
- CA2667055C CA2667055C CA2667055A CA2667055A CA2667055C CA 2667055 C CA2667055 C CA 2667055C CA 2667055 A CA2667055 A CA 2667055A CA 2667055 A CA2667055 A CA 2667055A CA 2667055 C CA2667055 C CA 2667055C
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- CA
- Canada
- Prior art keywords
- nucleoside
- bicyclic
- wing
- substituted
- 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.)
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- 150000001875 compounds Chemical class 0.000 title claims abstract description 302
- 230000000692 anti-sense effect Effects 0.000 title description 146
- 241001465754 Metazoa Species 0.000 claims abstract description 19
- 239000002777 nucleoside Substances 0.000 claims description 246
- 125000003835 nucleoside group Chemical group 0.000 claims description 122
- 108091034117 Oligonucleotide Proteins 0.000 claims description 120
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 86
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 86
- -1 bicyclic nucleoside Chemical class 0.000 claims description 85
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 85
- 125000002619 bicyclic group Chemical group 0.000 claims description 46
- 230000014509 gene expression Effects 0.000 claims description 38
- 125000000217 alkyl group Chemical group 0.000 claims description 31
- 230000000295 complement effect Effects 0.000 claims description 29
- 125000000623 heterocyclic group Chemical group 0.000 claims description 19
- 229910052739 hydrogen Inorganic materials 0.000 claims description 17
- 239000001257 hydrogen Substances 0.000 claims description 17
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- 125000005699 methyleneoxy group Chemical group [H]C([H])([*:1])O[*:2] 0.000 claims description 14
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- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 13
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 9
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- A—HUMAN NECESSITIES
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/351—Conjugate
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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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
Landscapes
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
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- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
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- Wood Science & Technology (AREA)
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- Epidemiology (AREA)
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- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (3)
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| US85289406P | 2006-10-18 | 2006-10-18 | |
| US60/852,894 | 2006-10-18 | ||
| PCT/US2007/081850 WO2008049085A1 (en) | 2006-10-18 | 2007-10-18 | Antisense compounds |
Publications (2)
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| CA2667055A1 CA2667055A1 (en) | 2008-04-24 |
| CA2667055C true CA2667055C (en) | 2017-05-09 |
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|---|---|---|---|
| CA2667055A Active CA2667055C (en) | 2006-10-18 | 2007-10-18 | Antisense compounds |
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| WO (1) | WO2008049085A1 (cg-RX-API-DMAC7.html) |
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| EP2015758B1 (en) | 2006-05-05 | 2014-04-02 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression apob |
| ES2620472T5 (es) * | 2006-10-18 | 2020-07-09 | Ionis Pharmaceuticals Inc | Compuestos antisentido |
| US8580756B2 (en) * | 2007-03-22 | 2013-11-12 | Santaris Pharma A/S | Short oligomer antagonist compounds for the modulation of target mRNA |
| WO2009046426A2 (en) * | 2007-10-04 | 2009-04-09 | Isis Pharmaceuticals, Inc. | Compounds and methods for improving cellular uptake of oligomeric compounds |
| EP2274423A2 (en) * | 2008-04-04 | 2011-01-19 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
| DK2379084T3 (en) | 2008-10-15 | 2018-01-22 | Ionis Pharmaceuticals Inc | MODULATION OF FACTOR 11 EXPRESSION |
| AU2009323766B2 (en) | 2008-12-02 | 2016-10-06 | Wave Life Sciences Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| SG177564A1 (en) | 2009-07-06 | 2012-02-28 | Ontorii Inc | Novel nucleic acid prodrugs and methods of use thereof |
| WO2011097644A2 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| EP3321361B1 (en) | 2010-02-08 | 2019-03-27 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| KR101900770B1 (ko) | 2010-07-19 | 2018-09-20 | 아이오니스 파마수티컬즈, 인코포레이티드 | 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법 |
| US8648053B2 (en) | 2010-10-20 | 2014-02-11 | Rosalind Franklin University Of Medicine And Science | Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| DK2638163T3 (en) | 2010-11-12 | 2017-07-24 | Massachusetts Gen Hospital | POLYCOMB-ASSOCIATED NON-CODING RNAs |
| WO2012068405A2 (en) | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| WO2012161806A1 (en) * | 2011-02-25 | 2012-11-29 | Isis Pharmaceuticals, Inc. | Modulation of stat3 expression |
| UA118951C2 (uk) * | 2011-04-21 | 2019-04-10 | Айоніс Фармасьютікалз, Інк. | Модулювання експресії вірусу гепатиту b (hbv) |
| WO2012151324A1 (en) | 2011-05-02 | 2012-11-08 | Isis Pharmaceuticals, Inc. | Antisense compounds targeting genes associated with usher syndrome |
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| DK2742135T4 (da) * | 2011-08-11 | 2020-07-13 | Ionis Pharmaceuticals Inc | Bindingsmodificerede gapped oligomeriske forbindelser og anvendelser deraf |
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| CA2873779A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating mecp2 expression |
| BR112014028631A2 (pt) | 2012-05-16 | 2017-10-17 | Rana Therapeutics Inc | composições e métodos para modulação da expressão da família de genes da hemoglobina |
| EA201492118A1 (ru) * | 2012-05-16 | 2015-04-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии pten |
| CN104583401A (zh) | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节atp2a2表达的组合物和方法 |
| EP2850185A4 (en) | 2012-05-16 | 2015-12-30 | Rana Therapeutics Inc | COMPOSITIONS AND METHOD FOR MODULATING UTRN GENE EXPRESSION |
| KR20150030205A (ko) | 2012-05-16 | 2015-03-19 | 라나 테라퓨틱스, 인크. | Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법 |
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| US9487780B2 (en) | 2012-06-01 | 2016-11-08 | Ionis Pharmaceuticals, Inc. | Antisense compounds targeting genes associated with fibronectin |
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| JP6246121B2 (ja) | 2012-07-13 | 2017-12-13 | 株式会社新日本科学 | キラル核酸アジュバント |
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| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ | 不斉補助基 |
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| KR102138781B1 (ko) | 2013-05-01 | 2020-07-28 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지질단백질 c-iii 발현을 조절하는 조성물 및 방법 |
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2014
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- 2014-07-24 JP JP2014150706A patent/JP2014221817A/ja active Pending
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2016
- 2016-09-27 AU AU2016231660A patent/AU2016231660A1/en not_active Abandoned
- 2016-12-09 US US15/373,860 patent/US10493092B2/en active Active
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2019
- 2019-10-21 US US16/659,368 patent/US20200276221A1/en not_active Abandoned
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2023
- 2023-01-05 US US18/150,395 patent/US20240058370A1/en active Pending
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