JP2015171368A5 - - Google Patents
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- Publication number
- JP2015171368A5 JP2015171368A5 JP2015098107A JP2015098107A JP2015171368A5 JP 2015171368 A5 JP2015171368 A5 JP 2015171368A5 JP 2015098107 A JP2015098107 A JP 2015098107A JP 2015098107 A JP2015098107 A JP 2015098107A JP 2015171368 A5 JP2015171368 A5 JP 2015171368A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid analog
- repeat region
- pharmaceutical composition
- mrna
- 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.)
- Granted
Links
- 108020004707 nucleic acids Proteins 0.000 claims description 55
- 102000039446 nucleic acids Human genes 0.000 claims description 55
- 150000007523 nucleic acids Chemical class 0.000 claims description 55
- 239000000203 mixture Substances 0.000 claims description 22
- 108020004999 messenger RNA Proteins 0.000 claims description 21
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 20
- 201000010099 disease Diseases 0.000 claims description 19
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 19
- 102000004169 proteins and genes Human genes 0.000 claims description 19
- 108090000623 proteins and genes Proteins 0.000 claims description 19
- 125000002091 cationic group Chemical group 0.000 claims description 9
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 9
- 230000005764 inhibitory process Effects 0.000 claims description 8
- 238000004519 manufacturing process Methods 0.000 claims description 5
- 102000007372 Ataxin-1 Human genes 0.000 claims description 4
- 108010032963 Ataxin-1 Proteins 0.000 claims description 4
- 102000007371 Ataxin-3 Human genes 0.000 claims description 4
- 108010032947 Ataxin-3 Proteins 0.000 claims description 4
- 102000007370 Ataxin2 Human genes 0.000 claims description 4
- 108010032951 Ataxin2 Proteins 0.000 claims description 4
- 102000004321 Atrophin-1 Human genes 0.000 claims description 4
- 108090000806 Atrophin-1 Proteins 0.000 claims description 4
- 230000002401 inhibitory effect Effects 0.000 claims description 4
- 150000002632 lipids Chemical class 0.000 claims description 4
- -1 aminoethoxy Chemical group 0.000 claims description 2
- 238000009472 formulation Methods 0.000 claims description 2
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 16
- 230000000295 complement effect Effects 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 1
- 238000000034 method Methods 0.000 description 29
- 238000007792 addition Methods 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 238000006467 substitution reaction Methods 0.000 description 2
- 230000008707 rearrangement Effects 0.000 description 1
- 230000008521 reorganization Effects 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US8435008P | 2008-07-29 | 2008-07-29 | |
| US61/084,350 | 2008-07-29 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011521239A Division JP2011529686A (ja) | 2008-07-29 | 2009-07-28 | ポリグルタミンタンパク質発現の選択的阻害 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015171368A JP2015171368A (ja) | 2015-10-01 |
| JP2015171368A5 true JP2015171368A5 (https=) | 2016-03-31 |
| JP6265940B2 JP6265940B2 (ja) | 2018-01-24 |
Family
ID=41610691
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011521239A Withdrawn JP2011529686A (ja) | 2008-07-29 | 2009-07-28 | ポリグルタミンタンパク質発現の選択的阻害 |
| JP2015098107A Active JP6265940B2 (ja) | 2008-07-29 | 2015-05-13 | ポリグルタミンタンパク質発現の選択的阻害 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011521239A Withdrawn JP2011529686A (ja) | 2008-07-29 | 2009-07-28 | ポリグルタミンタンパク質発現の選択的阻害 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US8901095B2 (https=) |
| EP (1) | EP2317847B1 (https=) |
| JP (2) | JP2011529686A (https=) |
| AU (3) | AU2009276763B2 (https=) |
| CA (1) | CA2732343C (https=) |
| WO (1) | WO2010014592A1 (https=) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2007282224B2 (en) | 2006-08-11 | 2013-08-29 | Vico Therapeutics B.V. | Methods and means for treating DNA repeat instability associated genetic disorders |
| AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| WO2011097388A1 (en) * | 2010-02-03 | 2011-08-11 | Alnylam Pharmaceuticals, Inc. | Selective inhibition of polyglutamine protein expression |
| EP2536738A4 (en) * | 2010-02-08 | 2014-09-17 | Isis Pharmaceuticals Inc | METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES OR SUFFERING IN CONNECTION WITH REPEAT EXTENSIONS |
| WO2012012467A2 (en) * | 2010-07-19 | 2012-01-26 | Isis Pharmaceuticals, Inc. | Modulation of nuclear-retained rna |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP3260540A1 (en) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9976138B2 (en) | 2011-08-29 | 2018-05-22 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| CN112251436A (zh) | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| JP2015511821A (ja) * | 2012-03-12 | 2015-04-23 | ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブＲｏｃｈｅ Ｉｎｎｏｖａｔｉｏｎ Ｃｅｎｔｅｒ Ｃｏｐｅｎｈａｇｅｎ Ａ／Ｓ | Ａｔｘｎ３発現の調節のための組成物および方法 |
| SMT201700339T1 (it) | 2012-04-23 | 2017-09-07 | Biomarin Tech Bv | Oligonucleotidi di modulazione dell'rna con caratteristiche migliorate per il trattamento dei disturbi neuromuscolari |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| CA2873766A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating atp2a2 expression |
| CA2873769A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating hemoglobin gene family expression |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 |
| BR112014028634A2 (pt) | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| US10435430B2 (en) | 2013-07-31 | 2019-10-08 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| 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 |
| CN106103717A (zh) | 2014-03-19 | 2016-11-09 | Ionis制药公司 | 用于调节共济失调蛋白2表达的组合物 |
| WO2015143245A1 (en) * | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| CA2966044A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| WO2016130943A1 (en) | 2015-02-13 | 2016-08-18 | Rana Therapeutics, Inc. | Hybrid oligonucleotides and uses thereof |
| WO2016149455A2 (en) | 2015-03-17 | 2016-09-22 | The General Hospital Corporation | The rna interactome of polycomb repressive complex 1 (prc1) |
| US10533175B2 (en) * | 2015-09-25 | 2020-01-14 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating Ataxin 3 expression |
| EP3368089B1 (en) | 2015-10-26 | 2025-11-05 | Translate Bio Ma, Inc. | Nanoparticle formulations for delivery of nucleic acid complexes |
| US20190002887A1 (en) | 2015-12-31 | 2019-01-03 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| JP6430999B2 (ja) * | 2016-06-24 | 2018-11-28 | トヨタ自動車株式会社 | 自動車用エンジン冷却液組成物、自動車用エンジン濃縮冷却液組成物及び内燃機関の運転方法 |
| JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
| SG11202010215TA (en) | 2018-05-09 | 2020-11-27 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing atxn3 expression |
| WO2019233922A1 (en) | 2018-06-05 | 2019-12-12 | F. Hoffmann-La Roche Ag | Oligonucleotides for modulating atxn2 expression |
| BR112021000308A2 (pt) | 2018-07-25 | 2021-04-13 | Ionis Pharmaceuticals, Inc. | Compostos e métodos para redução da expressão de atxn2 |
| US11286485B2 (en) | 2019-04-04 | 2022-03-29 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating ATXN2 expression |
| EP4127228A4 (en) * | 2020-03-30 | 2024-05-29 | NeuBase Therapeutics, Inc. | Modified peptide nucleic acid compositions |
| WO2022212231A2 (en) | 2021-03-29 | 2022-10-06 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| EP4396352A4 (en) | 2021-09-01 | 2025-10-01 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION |
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-
2009
- 2009-07-28 WO PCT/US2009/051938 patent/WO2010014592A1/en not_active Ceased
- 2009-07-28 EP EP09803468.9A patent/EP2317847B1/en active Active
- 2009-07-28 CA CA2732343A patent/CA2732343C/en active Active
- 2009-07-28 US US13/056,556 patent/US8901095B2/en active Active
- 2009-07-28 JP JP2011521239A patent/JP2011529686A/ja not_active Withdrawn
- 2009-07-28 AU AU2009276763A patent/AU2009276763B2/en active Active
-
2014
- 2014-11-03 US US14/531,203 patent/US9340785B2/en active Active
-
2015
- 2015-05-13 JP JP2015098107A patent/JP6265940B2/ja active Active
- 2015-10-08 AU AU2015238876A patent/AU2015238876A1/en not_active Abandoned
-
2017
- 2017-08-15 AU AU2017216467A patent/AU2017216467A1/en not_active Abandoned
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