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
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- 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 |
|---|---|---|---|---|
| SI2049664T1 (sl) | 2006-08-11 | 2012-04-30 | Prosensa Technologies Bv | Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev |
| ES2639852T3 (es) | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| WO2011097388A1 (en) * | 2010-02-03 | 2011-08-11 | Alnylam Pharmaceuticals, Inc. | Selective inhibition of polyglutamine protein expression |
| JP2013518603A (ja) * | 2010-02-08 | 2013-05-23 | アイシス ファーマシューティカルズ， インコーポレーテッド | 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 |
| EP2595663A4 (en) * | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| CA2817256A1 (en) | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| WO2013033223A1 (en) | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| US20150315595A1 (en) * | 2012-03-12 | 2015-11-05 | Santaris Pharma A/S | Compositions and Methods for Modulation of ATXN3 Expression |
| LT2841578T (lt) | 2012-04-23 | 2017-09-25 | Biomarin Technologies B.V. | Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui |
| AU2013262699A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating ATP2A2 expression |
| SG11201407483YA (en) | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| EA201492122A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| BR112014028631A2 (pt) | 2012-05-16 | 2017-10-17 | Rana Therapeutics Inc | composições e métodos para modulação da expressão da família de genes da hemoglobina |
| 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 |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| EP3119888B1 (en) * | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| US10758558B2 (en) | 2015-02-13 | 2020-09-01 | Translate Bio Ma, 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) |
| EP4285912A3 (en) * | 2015-09-25 | 2024-07-10 | 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 |
| AU2016381174A1 (en) | 2015-12-31 | 2018-05-31 | 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 |
| EP4663758A3 (en) | 2018-05-09 | 2026-04-29 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing atxn3 expression |
| JP7379387B2 (ja) | 2018-06-05 | 2023-11-14 | エフ． ホフマン－ラ ロシュ アーゲー | Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド |
| AR115847A1 (es) | 2018-07-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir la expresión de la atxn2 |
| US11286485B2 (en) | 2019-04-04 | 2022-03-29 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating ATXN2 expression |
| AU2021246046A1 (en) * | 2020-03-30 | 2022-10-13 | Neubase Therapeutics, Inc. | Modified peptide nucleic acid compositions |
| EP4314296A2 (en) | 2021-03-29 | 2024-02-07 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
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| SI2049664T1 (sl) * | 2006-08-11 | 2012-04-30 | Prosensa Technologies Bv | Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev |
| KR101654007B1 (ko) * | 2007-08-15 | 2016-09-05 | 아이오니스 파마수티컬즈, 인코포레이티드 | 테트라하이드로피란 핵산 유사체 |
| NZ714887A (en) * | 2009-09-11 | 2019-07-26 | 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 CA CA2732343A patent/CA2732343C/en active Active
- 2009-07-28 US US13/056,556 patent/US8901095B2/en active Active
- 2009-07-28 EP EP09803468.9A patent/EP2317847B1/en active Active
- 2009-07-28 WO PCT/US2009/051938 patent/WO2010014592A1/en not_active Ceased
- 2009-07-28 AU AU2009276763A patent/AU2009276763B2/en active Active
- 2009-07-28 JP JP2011521239A patent/JP2011529686A/ja not_active Withdrawn
-
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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