JP2011529686A5 - - Google Patents
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- JP2011529686A5 JP2011529686A5 JP2011521239A JP2011521239A JP2011529686A5 JP 2011529686 A5 JP2011529686 A5 JP 2011529686A5 JP 2011521239 A JP2011521239 A JP 2011521239A JP 2011521239 A JP2011521239 A JP 2011521239A JP 2011529686 A5 JP2011529686 A5 JP 2011529686A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- pharmaceutical composition
- repeat region
- acid analog
- 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.)
- Withdrawn
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- 108020004707 nucleic acids Proteins 0.000 claims description 41
- 102000039446 nucleic acids Human genes 0.000 claims description 41
- 150000007523 nucleic acids Chemical class 0.000 claims description 41
- 238000000034 method Methods 0.000 claims description 40
- 201000010099 disease Diseases 0.000 claims description 20
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 20
- 102000004169 proteins and genes Human genes 0.000 claims description 20
- 108090000623 proteins and genes Proteins 0.000 claims description 20
- 108020004999 messenger RNA Proteins 0.000 claims description 18
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 17
- 239000000203 mixture Substances 0.000 claims description 11
- 125000002091 cationic group Chemical group 0.000 claims description 10
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 10
- 230000005764 inhibitory process Effects 0.000 claims description 8
- 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
- 238000004519 manufacturing process Methods 0.000 claims description 4
- 150000002632 lipids Chemical class 0.000 claims description 3
- -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
- 230000008685 targeting Effects 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 19
- 238000001361 intraarterial administration Methods 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US8435008P | 2008-07-29 | 2008-07-29 | |
| US61/084,350 | 2008-07-29 | ||
| PCT/US2009/051938 WO2010014592A1 (en) | 2008-07-29 | 2009-07-28 | Selective inhibition of polyglutamine protein expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015098107A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2011529686A JP2011529686A (ja) | 2011-12-15 |
| JP2011529686A5 true JP2011529686A5 (enExample) | 2012-09-13 |
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 After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US8901095B2 (enExample) |
| EP (1) | EP2317847B1 (enExample) |
| JP (2) | JP2011529686A (enExample) |
| AU (3) | AU2009276763B2 (enExample) |
| CA (1) | CA2732343C (enExample) |
| WO (1) | WO2010014592A1 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6047270B2 (ja) | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー．フェー． | Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
| NZ584793A (en) | 2007-10-26 | 2012-05-25 | 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 |
| ES2756326T3 (es) * | 2010-07-19 | 2020-04-27 | Ionis Pharmaceuticals Inc | Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK) |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP3702460A1 (en) | 2010-11-12 | 2020-09-02 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| EP2751269B1 (en) | 2011-08-29 | 2016-03-23 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CN104254610A (zh) * | 2012-03-12 | 2014-12-31 | 桑塔里斯制药公司 | 调节atxn3表达的组合物和方法 |
| PT2841578T (pt) | 2012-04-23 | 2017-08-10 | Biomarin Tech Bv | Resumo |
| CN104540946A (zh) | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节utrn表达的组合物和方法 |
| CN104583402A (zh) | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节mecp2表达的组合物和方法 |
| US10174315B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating hemoglobin gene family expression |
| EA201492120A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии atp2a2 |
| 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 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| EP3027617B1 (en) | 2013-07-31 | 2026-02-18 | 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 |
| SI3119888T1 (sl) * | 2014-03-19 | 2021-11-30 | Ionis Pharmaceuticals, Inc. | Sestavki za moduliranje izražanja ataksina 2 |
| WO2015143245A1 (en) * | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| WO2016070060A1 (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 |
| US10900036B2 (en) | 2015-03-17 | 2021-01-26 | The General Hospital Corporation | RNA interactome of polycomb repressive complex 1 (PRC1) |
| WO2017053781A1 (en) * | 2015-09-25 | 2017-03-30 | 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 |
| WO2017117496A1 (en) | 2015-12-31 | 2017-07-06 | 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 |
| JP2021522800A (ja) | 2018-05-09 | 2021-09-02 | 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． | Ａｔｘｎ３発現を低減するための化合物及び方法 |
| EP3802824A1 (en) | 2018-06-05 | 2021-04-14 | F. Hoffmann-La Roche AG | Oligonucleotides for modulating atxn2 expression |
| AU2019310097A1 (en) | 2018-07-25 | 2021-02-04 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing ATXN2 expression |
| US11286485B2 (en) | 2019-04-04 | 2022-03-29 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating ATXN2 expression |
| JP7796038B2 (ja) * | 2020-03-30 | 2026-01-08 | ニューベース セラピューティクス， インコーポレイテッド | 改変ペプチド核酸組成物 |
| BR112023019981A2 (pt) | 2021-03-29 | 2023-12-12 | Alnylam Pharmaceuticals Inc | Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
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| JP6047270B2 (ja) * | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー．フェー． | Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
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| EP3626823A1 (en) * | 2009-09-11 | 2020-03-25 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| WO2011097388A1 (en) | 2010-02-03 | 2011-08-11 | Alnylam Pharmaceuticals, Inc. | Selective inhibition of polyglutamine protein expression |
-
2009
- 2009-07-28 AU AU2009276763A patent/AU2009276763B2/en active Active
- 2009-07-28 JP JP2011521239A patent/JP2011529686A/ja not_active Withdrawn
- 2009-07-28 CA CA2732343A patent/CA2732343C/en active Active
- 2009-07-28 EP EP09803468.9A patent/EP2317847B1/en active Active
- 2009-07-28 US US13/056,556 patent/US8901095B2/en active Active
- 2009-07-28 WO PCT/US2009/051938 patent/WO2010014592A1/en not_active Ceased
-
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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