JP2016195616A5 - - Google Patents
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- JP2016195616A5 JP2016195616A5 JP2016167575A JP2016167575A JP2016195616A5 JP 2016195616 A5 JP2016195616 A5 JP 2016195616A5 JP 2016167575 A JP2016167575 A JP 2016167575A JP 2016167575 A JP2016167575 A JP 2016167575A JP 2016195616 A5 JP2016195616 A5 JP 2016195616A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- base
- exon
- pharmaceutically acceptable
- thymine
- 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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- 229920000272 Oligonucleotide Polymers 0.000 claims 29
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 27
- 239000000074 antisense oligonucleotide Substances 0.000 claims 27
- RWQNBRDOKXIBIV-UHFFFAOYSA-N Thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 16
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 16
- 239000000203 mixture Substances 0.000 claims 9
- 229940113082 Thymine Drugs 0.000 claims 8
- 229940035893 Uracil Drugs 0.000 claims 8
- 150000003839 salts Chemical class 0.000 claims 7
- 239000011780 sodium chloride Substances 0.000 claims 7
- 102000001039 Dystrophin Human genes 0.000 claims 6
- 108010069091 Dystrophin Proteins 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 206010013801 Duchenne muscular dystrophy Diseases 0.000 claims 4
- 208000003627 Muscular Dystrophy Diseases 0.000 claims 4
- ZIBGPFATKBEMQZ-UHFFFAOYSA-N Triethylene glycol Chemical group OCCOCCOCCO ZIBGPFATKBEMQZ-UHFFFAOYSA-N 0.000 claims 4
- 230000000295 complement Effects 0.000 claims 4
- 201000006938 muscular dystrophy Diseases 0.000 claims 4
- 125000004193 piperazinyl group Chemical group 0.000 claims 4
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 3
- 108020004999 Messenger RNA Proteins 0.000 claims 2
- 230000001939 inductive effect Effects 0.000 claims 2
- 229920002106 messenger RNA Polymers 0.000 claims 2
- 230000000692 anti-sense Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 101710031453 groL2 Proteins 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
Claims (15)
- ヒトジストロフィン遺伝子のエキソン４４に対して１００％相補的な塩基配列を含む２０〜３５塩基のアンチセンスオリゴヌクレオチドであって、該塩基配列は、配列番号１〜２０から選択される配列の少なくとも１５塩基を含み、チミン塩基は任意でウラシル塩基であり、該アンチセンスオリゴヌクレオチドは、モルフォリノアンチセンスオリゴヌクレオチドであり、該アンチセンスオリゴヌクレオチドは、エキソン４４スキッピングを誘導する、アンチセンスオリゴヌクレオチドまたは薬学的に受容可能なその塩。
- チミン塩基（Ｔ）の代わりにウラシル塩基（Ｕ）を有する、請求項１に記載のアンチセンスオリゴヌクレオチドまたは薬学的に受容可能なその塩。
- 前記アンチセンスオリゴヌクレオチドが、ピペラジニル部分を介してトリエチレングリコール部分に結合体化されている、請求項１または２に記載のアンチセンスオリゴヌクレオチドまたは薬学的に受容可能なその塩。
- 筋ジストロフィーの処置における使用のための、請求項１〜３のいずれか１項に記載のアンチセンスオリゴヌクレオチドまたは薬学的に受容可能なその塩を含む組成物。
- 前記筋ジストロフィーが、デュシェンヌ型筋ジストロフィー（ＤＭＤ）である、請求項４に記載の組成物。
- 筋ジストロフィーの処置における使用のための薬学的組成物であって、
（ｉ）ヒトジストロフィン遺伝子のエキソン４４に対して１００％相補的な塩基配列を含む、２０〜３５塩基のアンチセンスオリゴヌクレオチドであって、該塩基配列は、配列番号１〜２０から選択される配列の少なくとも１５塩基を含み、チミン塩基は任意でウラシル塩基であり、該アンチセンスオリゴヌクレオチドは、モルフォリノアンチセンスオリゴヌクレオチドであり、該アンチセンスオリゴヌクレオチドは、エキソン４４スキッピングを誘導する、アンチセンスオリゴヌクレオチドまたは薬学的に受容可能なその塩、および
（ｉｉ）薬学的に受容可能なキャリア
を含む、薬学的組成物。 - 前記アンチセンスオリゴヌクレオチドが、チミン塩基（Ｔ）の代わりにウラシル塩基（Ｕ）を有する、請求項６に記載の薬学的組成物。
- 前記アンチセンスオリゴヌクレオチドが、ピペラジニル部分を介してトリエチレングリコール部分に結合体化されている、請求項６または７に記載の薬学的組成物。
- 前記筋ジストロフィーが、デュシェンヌ型筋ジストロフィー（ＤＭＤ）である、請求項６〜８のいずれか１項に記載の薬学的組成物。
- ヒトジストロフィン遺伝子のプロセスされたｍＲＮＡ転写物のエキソン４４スキッピングを誘導するための医薬の製造における使用のための、ヒトジストロフィン遺伝子のエキソン４４に対して１００％相補的な塩基配列を含む、２０〜３５塩基のアンチセンスオリゴヌクレオチドであって、該塩基配列は、配列番号１〜２０から選択される配列の少なくとも１５塩基を含み、チミン塩基は任意でウラシル塩基であり、該アンチセンスオリゴヌクレオチドは、モルフォリノアンチセンスオリゴヌクレオチドであり、該アンチセンスオリゴヌクレオチドは、エキソン４４スキッピングを誘導する、アンチセンスオリゴヌクレオチドまたは薬学的に受容可能なその塩を含む、組成物。
- 前記アンチセンスオリゴヌクレオチドが、チミン塩基（Ｔ）の代わりにウラシル塩基（Ｕ）を有する、請求項１０に記載の組成物。
- 前記アンチセンスオリゴヌクレオチドが、ピペラジニル部分を介してトリエチレングリコール部分に結合体化されている、請求項１０または１１に記載の組成物。
- ヒトジストロフィン遺伝子のプレプロセスされたｍＲＮＡ転写物のエキソン４４スキッピングを誘導するための組成物であって、該ヒトジストロフィン遺伝子のエキソン４４に１００％相補的な塩基配列を含む２０〜３５塩基のアンチセンスオリゴヌクレオチドを含み、該塩基配列は、配列番号１〜２０から選択される配列の少なくとも１５塩基を含み、チミン塩基は、必要に応じてウラシル塩基であり、該アンチセンスオリゴヌクレオチドは、モルフォリノアンチセンスオリゴヌクレオチドであり、該アンチセンスオリゴヌクレオチドは、エキソン４４スキッピングを誘導する、アンチセンスオリゴヌクレオチドまたはその薬学的に受容可能な塩を含む、組成物。
- 前記アンチセンスオリゴヌクレオチドが、チミン塩基（Ｔ）の代わりにウラシル塩基（Ｕ）を有する、請求項１３に記載の組成物。
- 前記アンチセンスオリゴヌクレオチドが、ピペラジニル部分を介してトリエチレングリコール部分に結合体化されている、請求項１３または１４に記載の組成物。
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US10841608P | 2008-10-24 | 2008-10-24 | |
| US61/108,416 | 2008-10-24 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014214204A Division JP2015017131A (ja) | 2008-10-24 | 2014-10-21 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017028390A Division JP2017086087A (ja) | 2008-10-24 | 2017-02-17 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016195616A JP2016195616A (ja) | 2016-11-24 |
| JP2016195616A5 true JP2016195616A5 (ja) | 2017-03-23 |
| JP6429285B2 JP6429285B2 (ja) | 2018-11-28 |
Family
ID=41510869
Family Applications (10)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011533392A Active JP5864257B2 (ja) | 2008-10-24 | 2009-10-23 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2014214204A Withdrawn JP2015017131A (ja) | 2008-10-24 | 2014-10-21 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2016167575A Active JP6429285B2 (ja) | 2008-10-24 | 2016-08-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2017028390A Withdrawn JP2017086087A (ja) | 2008-10-24 | 2017-02-17 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2018084126A Active JP6406782B2 (ja) | 2008-10-24 | 2018-04-25 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2019121399A Withdrawn JP2019178163A (ja) | 2008-10-24 | 2019-06-28 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021031641A Withdrawn JP2021088595A (ja) | 2008-10-24 | 2021-03-01 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021066507A Pending JP2021100439A (ja) | 2008-10-24 | 2021-04-09 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023019132A Pending JP2023055956A (ja) | 2008-10-24 | 2023-02-10 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023055590A Pending JP2023073441A (ja) | 2008-10-24 | 2023-03-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
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|---|---|---|---|
| JP2011533392A Active JP5864257B2 (ja) | 2008-10-24 | 2009-10-23 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2014214204A Withdrawn JP2015017131A (ja) | 2008-10-24 | 2014-10-21 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Family Applications After (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017028390A Withdrawn JP2017086087A (ja) | 2008-10-24 | 2017-02-17 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2018084126A Active JP6406782B2 (ja) | 2008-10-24 | 2018-04-25 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2019121399A Withdrawn JP2019178163A (ja) | 2008-10-24 | 2019-06-28 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021031641A Withdrawn JP2021088595A (ja) | 2008-10-24 | 2021-03-01 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021066507A Pending JP2021100439A (ja) | 2008-10-24 | 2021-04-09 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023019132A Pending JP2023055956A (ja) | 2008-10-24 | 2023-02-10 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023055590A Pending JP2023073441A (ja) | 2008-10-24 | 2023-03-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Country Status (22)
| Country | Link |
|---|---|
| US (14) | US8871918B2 (ja) |
| EP (7) | EP4174178A1 (ja) |
| JP (10) | JP5864257B2 (ja) |
| KR (5) | KR20110082576A (ja) |
| CN (3) | CN102203253B (ja) |
| AU (1) | AU2009308167B2 (ja) |
| BR (3) | BRPI0920276B1 (ja) |
| CA (2) | CA2740328A1 (ja) |
| CY (1) | CY1122369T1 (ja) |
| DK (1) | DK3133160T3 (ja) |
| ES (2) | ES2714787T3 (ja) |
| HK (1) | HK1200868A1 (ja) |
| HR (1) | HRP20190387T1 (ja) |
| HU (1) | HUE042979T2 (ja) |
| IL (6) | IL313162A (ja) |
| LT (1) | LT3133160T (ja) |
| PL (1) | PL3133160T3 (ja) |
| PT (1) | PT3133160T (ja) |
| SI (1) | SI3133160T1 (ja) |
| TR (1) | TR201902952T4 (ja) |
| TW (6) | TWI639700B (ja) |
| WO (1) | WO2010048586A1 (ja) |
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