JP2018184423A5 - - Google Patents
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- JP2018184423A5 JP2018184423A5 JP2018128926A JP2018128926A JP2018184423A5 JP 2018184423 A5 JP2018184423 A5 JP 2018184423A5 JP 2018128926 A JP2018128926 A JP 2018128926A JP 2018128926 A JP2018128926 A JP 2018128926A JP 2018184423 A5 JP2018184423 A5 JP 2018184423A5
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- 239000008194 pharmaceutical composition Substances 0.000 claims 29
- 230000000692 anti-sense Effects 0.000 claims 16
- 150000001875 compounds Chemical class 0.000 claims 16
- 229920000272 Oligonucleotide Polymers 0.000 claims 13
- 208000002320 Spinal Muscular Atrophy Diseases 0.000 claims 10
- 239000002777 nucleoside Substances 0.000 claims 9
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 4
- 239000000074 antisense oligonucleotide Substances 0.000 claims 4
- 125000003835 nucleoside group Chemical group 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 210000002161 Motor Neurons Anatomy 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 2
- 230000004083 survival Effects 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-Methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- -1 Methoxyethyl nucleoside Chemical class 0.000 claims 1
- 208000008425 Protein Deficiency Diseases 0.000 claims 1
- 102100002720 SNRPN Human genes 0.000 claims 1
- 230000001058 adult Effects 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 230000002068 genetic Effects 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
Claims (23)
- ヒト被験体の脊髄性筋萎縮症（ＳＭＡ）を治療するための医薬組成物であって、アンチセンス化合物を含み、ここで、当該医薬組成物は、ヒト被験体のくも膜下腔内へ投与され、当該アンチセンス化合物は、１８個の結合したヌクレオシドから成るアンチセンスオリゴヌクレオチドを含み、ここで、当該オリゴヌクレオチドは、配列番号１の核酸塩基配列から成る核酸塩基配列を有し、当該オリゴヌクレオチドのそれぞれのヌクレオシド間結合は、ホスホロチオエート結合であり、そして、当該オリゴヌクレオチドのそれぞれのヌクレオシドは、２’−Ｏ−メトキシエチルヌクレオシドである、前記医薬組成物。
- ＳＭＡがＩ型ＳＭＡである、請求項１に記載の医薬組成物。
- ＳＭＡがＩＩ型ＳＭＡである、請求項１に記載の医薬組成物。
- ＳＭＡがＩＩＩ型ＳＭＡである、請求項１に記載の医薬組成物。
- サバイバル運動ニューロン１（ＳＭＮ１）遺伝子の両方の機能的コピーの喪失を有するヒト被検体における、サバイバル運動ニューロン２（ＳＭＮ２）メッセンジャーリボ核酸（ｍＲＮＡ）転写物中のエクソン７の含有量を増加させるための医薬組成物であって、アンチセンス化合物を含み、当該医薬組成物はヒト被検体のくも膜下腔内へ投与され、そして当該アンチセンス化合物は１８個の結合したヌクレオシドから成るアンチセンスオリゴヌクレオチドを含み、当該オリゴヌクレオチドは配列番号１の核酸塩基配列から成る核酸塩基配列を有し、当該オリゴヌクレオチドのそれぞれのヌクレオシド間結合はホスホロチオエート結合であり、そして当該オリゴヌクレオチドのそれぞれのヌクレオシドは２’−Ｏ−メトキシエチルヌクレオシドである、前記医薬組成物。
- ヒト被検体が、ＳＭＮ１遺伝子中に変異を有することが遺伝子試験により特定される、請求項５に記載の医薬組成物。
- 機能的ＳＭＮタンパク質欠損につながるＳＭＮ１遺伝子中の変異を有するヒト被検体における、ＳＭＮ２ ｍＲＮＡ転写物中のエクソン７の含有量を増加させるための医薬組成物であって、アンチセンス化合物を含み、ここで当該医薬組成物はヒト被検体のくも膜下腔内へ投与され、当該アンチセンス化合物は１８個の結合したヌクレオシドから成るアンチセンスオリゴヌクレオチドを含み、当該オリゴヌクレオチドは配列番号１の核酸塩基配列から成る核酸塩基配列を有し、当該オリゴヌクレオチドのそれぞれのヌクレオシド間結合はホスホロチオエート結合であり、そして当該オリゴヌクレオチドのそれぞれのヌクレオシドは２’−Ｏ−メトキシエチルヌクレオシドである、前記医薬組成物。
- ヒト被検体が、ＳＭＡの１以上の指標を有すると診断されている、請求項１〜７のいずれか１項に記載の医薬組成物。
- ヒト被検体がＳＭＡに関連する少なくとも１つの症状を有する、請求項１〜７のいずれか１項に記載の医薬組成物。
- アンチセンスオリゴヌクレオチドの少なくとも１つのヌクレオチドが修飾核塩基を含む、請求項１〜７のいずれか１項に記載の医薬組成物。
- 修飾核塩基が５−メチルシトシンである、請求項１０に記載の医薬組成物。
- アンチセンス化合物を、約１ｍｇ／ｍｌ〜約５ｍｇ／ｍｌの濃度でヒト被検体に投与する、請求項１〜１１のいずれか１項に記載の医薬組成物。
- アンチセンス化合物を、ヒト被検体の体重ｋｇ当たりアンチセンス化合物０．０１〜１０ｍｇの用量で投与する、請求項１〜１１のいずれか１項に記載の医薬組成物。
- アンチセンス化合物５ｍｇ〜２０ｍｇの用量を投与する、請求項１〜１１のいずれか１項に記載の医薬組成物。
- アンチセンス化合物の第１の用量を、ヒト被験体の生後１週間以内に投与する、請求項１〜１４のいずれか１項に記載の医薬組成物。
- アンチセンス化合物の第１の用量を、ヒト被験体の生後１ヵ月以内に投与する、請求項１〜１４のいずれか１項に記載の医薬組成物。
- アンチセンス化合物の第１の用量を、ヒト被験体の生後３ヵ月以内に投与する、請求項１〜１４のいずれか１項に記載の医薬組成物。
- アンチセンス化合物の第１の用量を、ヒト被験体の生後６ヵ月以内に投与する、請求項１〜１４のいずれか１項に記載の医薬組成物。
- アンチセンス化合物の第１の用量を、ヒト被験体が１〜２歳であるときに投与する、請求項１〜１４のいずれか１項に記載の医薬組成物。
- アンチセンス化合物の第１の用量を、ヒト被験体が１〜１５歳であるときに投与する、請求項１〜１４のいずれか１項に記載の医薬組成物。
- ヒト被検体が幼児である、請求項１〜２０のいずれか１項に記載の医薬組成物。
- ヒト被検体が成人である、請求項１〜２０のいずれか１項に記載の医薬組成物。
- ボーラス注入によりヒト被検体のくも膜下腔内に投与される、請求項１〜２２のいずれか１項に記載の医薬組成物。
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21803109P | 2009-06-17 | 2009-06-17 | |
| US61/218,031 | 2009-06-17 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016216200A Division JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019227997A Division JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018184423A JP2018184423A (ja) | 2018-11-22 |
| JP2018184423A5 true JP2018184423A5 (ja) | 2019-05-16 |
| JP6637121B2 JP6637121B2 (ja) | 2020-01-29 |
Family
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Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2018128926A Active JP6637121B2 (ja) | 2009-06-17 | 2018-07-06 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Country Status (24)
| Country | Link |
|---|---|
| US (5) | US8980853B2 (ja) |
| EP (4) | EP3643783A1 (ja) |
| JP (7) | JP5707396B2 (ja) |
| KR (8) | KR20120093138A (ja) |
| CN (5) | CN115227710A (ja) |
| AU (2) | AU2010262862C1 (ja) |
| CA (1) | CA2765396C (ja) |
| CY (2) | CY1120807T1 (ja) |
| DK (2) | DK3305302T3 (ja) |
| ES (2) | ES2699827T3 (ja) |
| HK (1) | HK1246648B (ja) |
| HR (2) | HRP20181712T1 (ja) |
| HU (2) | HUE046966T2 (ja) |
| IL (2) | IL217014A (ja) |
| LT (2) | LT3449926T (ja) |
| MX (3) | MX361732B (ja) |
| NO (1) | NO2020008I1 (ja) |
| NZ (2) | NZ624712A (ja) |
| PL (2) | PL3305302T3 (ja) |
| PT (2) | PT3449926T (ja) |
| RU (2) | RU2566724C9 (ja) |
| SI (2) | SI3305302T1 (ja) |
| TR (1) | TR201816256T4 (ja) |
| WO (1) | WO2010148249A1 (ja) |
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