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 |
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US21803109P | 2009-06-17 | 2009-06-17 | |
US61/218,031 | 2009-06-17 |
Related Parent Applications (1)
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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)
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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 |
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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)
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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 |
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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 |
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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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