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 (enExample) | 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 (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 |
|---|---|---|---|---|
| ES2373246T3 (es) | 2006-08-11 | 2012-02-01 | Prosensa Technologies B.V. | Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn. |
| HUE028662T2 (en) | 2007-10-26 | 2016-12-28 | Academisch Ziekenhuis Leiden | Preparations and methods for controlling muscle disorders |
| US9574191B2 (en) * | 2010-02-03 | 2017-02-21 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
| US20130059902A1 (en) * | 2010-02-08 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| AU2011282243B2 (en) * | 2010-07-19 | 2016-09-22 | Ionis Pharmaceuticals, Inc. | Modulation of nuclear-retained RNA |
| EP3260540A1 (en) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | 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 |
| CN111893117B (zh) | 2012-01-27 | 2024-06-04 | 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 |
| EP2839008A4 (en) * | 2012-03-12 | 2015-12-02 | Roche Innovation Ct Copenhagen As | COMPOSITIONS AND METHODS FOR MODULATING ATXN3 EXPRESSION |
| HRP20171254T1 (hr) | 2012-04-23 | 2018-01-12 | Biomarin Technologies B.V. | Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja |
| CA2873769A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating hemoglobin gene family expression |
| KR20160074368A (ko) | 2012-05-16 | 2016-06-28 | 라나 테라퓨틱스, 인크. | Utrn 발현을 조절하기 위한 조성물 및 방법 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| EP2850182A4 (en) | 2012-05-16 | 2016-01-20 | Rana Therapeutics Inc | COMPOSITIONS AND METHODS FOR MODULATING ATP2A2 GENE EXPRESSION |
| AU2013262709A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating MECP2 expression |
| DK2850186T3 (en) | 2012-05-16 | 2019-04-08 | Translate Bio Ma Inc | COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY 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 |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| PL3119888T3 (pl) | 2014-03-19 | 2021-12-20 | Ionis Pharmaceuticals, Inc. | Kompozycje do modulacji ekspresji ataksyny 2 |
| CA2966044A1 (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 |
| EP3271460A4 (en) | 2015-03-17 | 2019-03-13 | The General Hospital Corporation | RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1) |
| EP3353303B1 (en) * | 2015-09-25 | 2023-08-02 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating ataxin 3 expression |
| AU2016344384A1 (en) | 2015-10-26 | 2018-05-17 | 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 |
| UY38225A (es) | 2018-05-09 | 2019-11-29 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir de la expresión de atxn3 |
| 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 |
| EP3826645A4 (en) | 2018-07-25 | 2023-05-17 | 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 |
| AU2021246046A1 (en) * | 2020-03-30 | 2022-10-13 | Neubase Therapeutics, Inc. | Modified peptide nucleic acid compositions |
| AU2022246786A1 (en) | 2021-03-29 | 2023-10-05 | 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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| AU2008286771B2 (en) * | 2007-08-15 | 2013-08-15 | Isis Pharmaceuticals, Inc. | Tetrahydropyran nucleic acid analogs |
| CA2931725C (en) | 2009-09-11 | 2021-10-19 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| US9574191B2 (en) | 2010-02-03 | 2017-02-21 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
-
2009
- 2009-07-28 CA CA2732343A patent/CA2732343C/en active Active
- 2009-07-28 EP EP09803468.9A patent/EP2317847B1/en active Active
- 2009-07-28 AU AU2009276763A patent/AU2009276763B2/en active Active
- 2009-07-28 JP JP2011521239A patent/JP2011529686A/ja not_active Withdrawn
- 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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