JP2018525357A5 - - Google Patents
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- JP2018525357A5 JP2018525357A5 JP2018502647A JP2018502647A JP2018525357A5 JP 2018525357 A5 JP2018525357 A5 JP 2018525357A5 JP 2018502647 A JP2018502647 A JP 2018502647A JP 2018502647 A JP2018502647 A JP 2018502647A JP 2018525357 A5 JP2018525357 A5 JP 2018525357A5
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- 239000000203 mixture Substances 0.000 claims 57
- 229920000272 Oligonucleotide Polymers 0.000 claims 40
- 238000006011 modification reaction Methods 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 5
- 230000004048 modification Effects 0.000 claims 4
- 238000003776 cleavage reaction Methods 0.000 claims 3
- 230000000295 complement Effects 0.000 claims 3
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 3
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K [O-]P([O-])([O-])=O Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- 229920000642 polymer Polymers 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 239000011780 sodium chloride Substances 0.000 claims 2
- 210000001175 Cerebrospinal Fluid Anatomy 0.000 claims 1
- 101710003775 ERVK-10 Proteins 0.000 claims 1
- 101710037030 ERVK-11 Proteins 0.000 claims 1
- 101710009283 ERVK-18 Proteins 0.000 claims 1
- 101710009286 ERVK-19 Proteins 0.000 claims 1
- 102000033147 ERVK-25 Human genes 0.000 claims 1
- 101710035700 ERVK-25 Proteins 0.000 claims 1
- 101710038044 ERVK-6 Proteins 0.000 claims 1
- 101710014468 ERVK-7 Proteins 0.000 claims 1
- 101710014482 ERVK-8 Proteins 0.000 claims 1
- 101710043924 HERVK_113 Proteins 0.000 claims 1
- 201000001971 Huntington's disease Diseases 0.000 claims 1
- 108020004999 Messenger RNA Proteins 0.000 claims 1
- 101710006375 RNASEH1 Proteins 0.000 claims 1
- 101700078434 RT67 Proteins 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000002619 bicyclic group Chemical group 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000004615 ingredient Substances 0.000 claims 1
- 230000001404 mediated Effects 0.000 claims 1
- 229920002106 messenger RNA Polymers 0.000 claims 1
- 239000000546 pharmaceutic aid Substances 0.000 claims 1
- 101700086982 rnh Proteins 0.000 claims 1
- 159000000000 sodium salts Chemical class 0.000 claims 1
- 230000001629 suppression Effects 0.000 claims 1
Claims (34)
- 1)共通塩基配列および長さ;
2)骨格結合の共通パターン;および
3)骨格キラル中心の共通パターン;
によって特徴づけられる特定のオリゴヌクレオチドタイプのオリゴヌクレオチドを含む、キラル制御されたオリゴヌクレオチド組成物であって、
前記組成物は、同じ塩基配列および長さを有するオリゴヌクレオチドの実質的にラセミの調製物に比べて、前記特定のオリゴヌクレオチドタイプのオリゴヌクレオチドに関して濃縮されるという点でキラル制御された
組成物。 - 請求項1に記載の組成物であって,
1)共通塩基配列および長さ;
2)骨格結合の共通パターン;および
3)骨格キラル中心の共通パターン
によって特徴づけられる特定のオリゴヌクレオチドタイプのオリゴヌクレオチドを含む、キラル制御されたオリゴヌクレオチド組成物であって、
前記組成物は、同じ塩基配列および長さを有するオリゴヌクレオチドの実質的にラセミの調製物に比べて、前記特定のオリゴヌクレオチドタイプのオリゴヌクレオチドに関して濃縮されるという点でキラル制御され,
前記オリゴヌクレオチドは変異ハンチンチン遺伝子を標的にし、かつ前記長さは約10〜約50個のヌクレオチドであり、前記骨格結合は、少なくとも1つのホスホロチオエートを含み、かつ骨格キラル中心の前記パターンは、少なくとも1つのRpキラル中心および少なくとも1つのSpキラル中心を含む
組成物。 - 請求項1又は2に記載の組成物であって、
前記オリゴヌクレオチドが、1つまたは複数の翼領域および共通コア領域を含み:
各翼領域が独立して、2塩基以上の長さを有し、かつ独立して任意選択で、1個または複数のキラルなインターヌクレオチド結合を含み;かつ
前記コア領域が独立して、2塩基以上の長さを有し、かつ独立して、1個または複数のキラルなインターヌクレオチド結合を含む、
組成物。 - 請求項1〜3のいずれか1項に記載の組成物であって、
前記オリゴヌクレオチドタイプのオリゴヌクレオチドが、少なくとも1つの翼領域およびコア領域を含み:
各翼領域が独立して、2塩基以上の長さを有し、かつ独立して任意選択で、1個または複数のキラルなインターヌクレオチド結合を含み;
前記コア領域が独立して、2塩基以上の長さを有し、かつ独立して、1個または複数のキラルなインターヌクレオチド結合を含み;かつ
翼領域内の少なくとも1つのヌクレオチドが、前記コア領域の少なくとも1つのヌクレオチドとは異なり、前記差異が:
1)骨格結合;
2)骨格キラル中心のパターン;
3)糖修飾
のうちの1つまたは複数である、
組成物。 - 請求項1〜4のいずれか1項に記載の組成物であって、
同じオリゴヌクレオチドタイプのオリゴヌクレオチドが、同一の構造を有する、
組成物。 - 請求項1〜6のいずれか1項に記載の組成物であって、
前記オリゴヌクレオチドが、翼−コア−翼の構造を含む、
組成物。 - 請求項1〜8のいずれか1項に記載の組成物であって、
前記コアが、1個または複数のホスホロチオエート結合を含む、
組成物。 - 請求項1〜9のいずれか1項に記載の組成物であって、 それぞれの翼が独立に修飾糖部分を含む、
組成物。 - 請求項10に記載の組成物であって、
前記修飾糖部分が2’−修飾を有する、
組成物。 - 請求項10に記載の組成物であって、 前記修飾糖部分が二環式糖修飾を含む、
組成物。 - 請求項10に記載の組成物であって、
前記修飾糖部分が、2’−OR1(式中、R1は、任意選択で置換されたC1−6アルキルである。)である2’−修飾を含む、
組成物。 - 請求項10に記載の組成物であって、 前記修飾糖部分が、2’−MOEである2’−修飾を含む、
組成物。 - 請求項10に記載の組成物であって、
前記修飾糖部分が、2’−OMeである2’−修飾を含む、
組成物。 - 請求項1〜15のいずれか1項に記載の組成物であって、
前記コア領域は(Sp)t(Rp)n(Sp)m(式中、tは1〜10であり、nは1であり、mは2〜50である)を含む骨格キラル中心のパターンを有する、
組成物。 - 請求項6に記載の組成物であって,
前記オリゴヌクレオチドが、SSR、RSS、SSRSS、SSRSSR、RSSSRSRRRS、RSSSSSSSSS、SRRSRSSSSR、SRSRSSRSSR、RRRSSSRSSS、RRRSRSSRSR、RRSSSRSRSR、SRSSSRSSSS、SSRRSSRSRS、SSSSSSRRSS、RRRSSRRRSR、RRRRSSSSRS、SRRSRRRRRR、RSSRSSRRRR、RSRRSRRSRR、RRSRSSRSRS、SSRRRRRSRR、RSRRSRSSSR、RRSSRSRRRR、RRSRSRRSSS、RRSRSSSRRR、RSRRRRSRSR、SSRSSSRRRS、RSSRSRSRSR、RSRSRSSRSS、RRRSSRRSRS、SRRSSRRSRS、RRRRSRSRRRまたはSSSSRRRRSRを含む骨格キラル中心のパターンを含む、
組成物。 - 請求項1〜17のいずれか1項に記載の組成物であって、
前記共通塩基配列が、ハンチンチン病と関連する一塩基多型(SNP)と相補的である配列を含む、
組成物。 - 請求項1〜18のいずれか1項に記載の組成物であって、 前記一塩基多型が、rs362307、rs7685686、rs362268、rs2530595、rs362331およびrs362306から選択される、
組成物。 - 請求項1〜19のいずれか1項に記載の組成物であって、
前記オリゴヌクレオチドが、変異ハンチンチン遺伝子mRNAのRNaseH媒介切断に関与できる、組成物。 - 請求項1に記載の組成物であって、
前記オリゴヌクレオチドが、表N1A、表N2A、表N3A、表N4Aおよび表8から選択される構造を有する、
組成物。 - 請求項1に記載の組成物であって、
前記オリゴヌクレオチドが、mG*SmGmCmAmC*SA*SA*SG*SG*SG*SC*SA*SC*RA*SG*SmAmCmUmU*SmC、又はその薬学的に許容される塩を含み、
ここで、*SはSpホスホロチオエート結合を表し、
*RはRpホスホロチオエート結合を表し、
mは、塩基を含むヌクレオシドへの2’−OMe修飾を表す組成物。 - 請求項1に記載の組成物であって、前記塩基配列、骨格結合のパターン、および/又は骨格キラル中心のパターンは、請求項22に記載の塩基配列、骨格結合のパターン、および/又は骨格キラル中心のパターンを含むか、のみからなる、組成物。
- オリゴヌクレオチドであって、前記オリゴヌクレオチドは、請求項1〜23のいずれかに記載のオリゴヌクレオチドである、オリゴヌクレオチド。
- 請求項24に記載のオリゴヌクレオチドであって、
前記オリゴヌクレオチドが、mG*SmGmCmAmC*SA*SA*SG*SG*SG*SC*SA*SC*RA*SG*SmAmCmUmU*SmC、又はその薬学的に許容される塩を含み、
ここで、*SはSpホスホロチオエート結合を表し、
*RはRpホスホロチオエート結合を表し、
mは、塩基を含むヌクレオシドへの2’−OMe修飾を表す、オリゴヌクレオチド。 - 請求項25に記載のオリゴヌクレオチドであって、
前記オリゴヌクレオチドはナトリウム塩である、オリゴヌクレオチド。 - 医薬組成物であって、
有効量の請求項24〜26のいずれか1項に記載のオリゴヌクレオチドと、
薬学的に許容される希釈剤、薬学的に許容される賦形剤、及び薬学的に許容される担体から選ばれる少なくとも一つの薬学的に許容される不活性原料を含む、医薬組成物。 - 請求項1〜27のいずれか1項に記載の組成物であって、人工脳脊髄液をさらに含む組成物。
- 請求項1〜28のいずれか1項に記載の組成物であって、
前記組成物中の前記オリゴヌクレオチドの少なくとも約50%が、前記共通塩基配列および長さ、骨格結合の前記共通パターンおよび骨格キラル中心の前記共通パターンを有する、組成物。 - 核酸高分子の制御された切断のための方法であって:
ヌクレオチド配列が標的配列を含む核酸高分子と、請求項1〜29のいずれかに記載のオリゴヌクレオチド組成物とを接触させるステップを含む
方法。 - 標的配列を含む塩基配列を有する核酸の切断のための方法であって:
標的配列を含む塩基配列を有する核酸と、請求項2〜23のいずれか1項に記載のオリゴヌクレオチド組成物とを接触させるステップを含み、
前記共通塩基配列は、前記核酸内の前記標的配列に対して相補的である配列であるか、前記核酸内の前記標的配列に対して相補的である配列を含む、方法。 - 対象のハンチントン病を予防かつ/または治療するための方法であって、
有効量の請求項1から31のいずれかに記載の組成物又はオリゴヌクレオチドを前記対象に投与する工程を含む方法。 - 請求項1〜32のいずれか1項に記載の方法であって、疾患を惹き起こすアレルからの発現が選択的に抑圧される、方法。
- 実施形態1〜606から選択される、
オリゴヌクレオチド、オリゴヌクレオチド組成物または方法。
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JP2021170541A JP2022009217A (ja) | 2015-07-22 | 2021-10-18 | オリゴヌクレオチド組成物およびその方法 |
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US201562195779P | 2015-07-22 | 2015-07-22 | |
US62/195,779 | 2015-07-22 | ||
US201562236847P | 2015-10-02 | 2015-10-02 | |
US62/236,847 | 2015-10-02 | ||
US201662331960P | 2016-05-04 | 2016-05-04 | |
US62/331,960 | 2016-05-04 | ||
PCT/US2016/043542 WO2017015555A1 (en) | 2015-07-22 | 2016-07-22 | Oligonucleotide compositions and methods thereof |
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JP2018525357A5 true JP2018525357A5 (ja) | 2019-09-05 |
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JP2018502637A Active JP7296724B2 (ja) | 2015-07-22 | 2016-07-22 | オリゴヌクレオチド組成物およびその方法 |
JP2021134582A Active JP7441816B2 (ja) | 2015-07-22 | 2021-08-20 | オリゴヌクレオチド組成物およびその方法 |
JP2021170541A Pending JP2022009217A (ja) | 2015-07-22 | 2021-10-18 | オリゴヌクレオチド組成物およびその方法 |
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US (6) | US20180216107A1 (ja) |
EP (3) | EP4344744A2 (ja) |
JP (5) | JP2018525357A (ja) |
KR (1) | KR20180028516A (ja) |
CN (3) | CN108135921B (ja) |
AU (2) | AU2016297155B2 (ja) |
BR (1) | BR112017028636A2 (ja) |
CA (1) | CA2989682A1 (ja) |
CL (1) | CL2018000145A1 (ja) |
CO (1) | CO2018001484A2 (ja) |
CR (1) | CR20180107A (ja) |
EC (1) | ECSP18010463A (ja) |
HK (2) | HK1255369A1 (ja) |
IL (1) | IL256603A (ja) |
MA (1) | MA43072A (ja) |
MX (1) | MX2018000796A (ja) |
NI (1) | NI201800009A (ja) |
PE (1) | PE20181174A1 (ja) |
PH (1) | PH12018500145A1 (ja) |
RU (1) | RU2017146349A (ja) |
SG (1) | SG10201912900XA (ja) |
SV (1) | SV2018005619A (ja) |
TW (1) | TW201707711A (ja) |
WO (2) | WO2017015575A1 (ja) |
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