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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- 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
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- 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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- 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
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- 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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- 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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- Engineering & Computer Science (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- 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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| EP2023940B1 (en) | 2006-05-05 | 2011-06-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of sglt2 |
| ES2377327T5 (es) * | 2006-10-18 | 2020-04-28 | 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 |
| US8318496B2 (en) * | 2007-10-04 | 2012-11-27 | 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 |
| US8334372B2 (en) | 2008-10-15 | 2012-12-18 | Isis Pharmaceuticals, Inc. | Modulation of factor 11 expression |
| WO2010064146A2 (en) | 2008-12-02 | 2010-06-10 | Chiralgen, Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| AU2011213562B2 (en) | 2010-02-08 | 2016-07-07 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| EP2534262B1 (en) | 2010-02-08 | 2016-12-14 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| DK3031920T3 (da) | 2010-07-19 | 2019-10-14 | Ionis Pharmaceuticals Inc | Modulation af dystrofi myotonica-protein kinase (dmpk) ekspression |
| 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 |
| JP6336755B2 (ja) | 2010-11-12 | 2018-06-06 | ザ ジェネラル ホスピタル コーポレイション | ポリコームに関連する非コードｒｎａ |
| JP6126009B2 (ja) | 2010-11-17 | 2017-05-10 | 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． | α−シヌクレイン発現の調節 |
| EP3067421B1 (en) | 2011-02-08 | 2018-10-10 | 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 |
| CR20190207A (es) | 2011-04-21 | 2019-06-26 | Ionis Pharmaceuticals Inc | MODULACIÓN DE LA EXPRESIÓN DEL VIRUS DE LA HEPATITIS B (VHB) (Divisional 2013-0551) |
| US9353371B2 (en) | 2011-05-02 | 2016-05-31 | Ionis Pharmaceuticals, Inc. | Antisense compounds targeting genes associated with usher syndrome |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2013022966A1 (en) * | 2011-08-11 | 2013-02-14 | Isis Pharmaceuticals, Inc. | Linkage modified gapped oligomeric compounds and uses thereof |
| EP3533873A1 (en) | 2011-09-14 | 2019-09-04 | Translate Bio MA, Inc. | Multimeric oligonucleotide compounds |
| US9243291B1 (en) * | 2011-12-01 | 2016-01-26 | Isis Pharmaceuticals, Inc. | Methods of predicting toxicity |
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| DK2850186T3 (en) | 2012-05-16 | 2019-04-08 | Translate Bio Ma Inc | COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY 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 |
| CA2873797A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating utrn expression |
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| CN104583400A (zh) * | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节pten表达的组合物和方法 |
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| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
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| AU2014222150A1 (en) | 2013-03-01 | 2015-09-10 | National University Corporation Tokyo Medical And Dental University | Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent |
| ME03043B (me) | 2013-03-14 | 2018-10-20 | Alnylam Pharmaceuticals Inc | Irnk sastavi komponente komplementa c5 i metode za njihovu upotrebu |
| RU2670614C9 (ru) | 2013-05-01 | 2018-11-23 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии hbv и ttr |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
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