JP2018525357A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- composition
- oligonucleotide
- composition according
- pattern
- backbone
- 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.)
- Pending
Links
- 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。
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
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 |
Applications Claiming Priority (7)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
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 |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2021170541A Division 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 |
Publications (2)
Publication Number | Publication Date |
---|---|
JP2018525357A JP2018525357A (ja) | 2018-09-06 |
JP2018525357A5 true JP2018525357A5 (ja) | 2019-09-05 |
Family
ID=57835219
Family Applications (5)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2018502647A Pending JP2018525357A (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 |
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 |
JP2023207761A Pending JP2024028927A (ja) | 2015-07-22 | 2023-12-08 | オリゴヌクレオチド組成物およびその方法 |
Family Applications After (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
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 |
JP2023207761A Pending JP2024028927A (ja) | 2015-07-22 | 2023-12-08 | オリゴヌクレオチド組成物およびその方法 |
Country Status (24)
Country | Link |
---|---|
US (5) | US20180216107A1 (ja) |
EP (3) | EP4344744A2 (ja) |
JP (5) | JP2018525357A (ja) |
KR (1) | KR20180028516A (ja) |
CN (2) | 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) |
Families Citing this family (98)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8158768B2 (en) * | 2002-12-23 | 2012-04-17 | Dynavax Technologies Corporation | Immunostimulatory sequence oligonucleotides and methods of using the same |
KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
IN2012DN00720A (ja) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
EP2831231A1 (en) | 2012-03-30 | 2015-02-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
BR112015000784A8 (pt) | 2012-07-13 | 2018-04-03 | Wave Life Sciences Japan | Grupo auxiliar assimétrico |
TWI657819B (zh) | 2013-07-19 | 2019-05-01 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
KR20230152178A (ko) | 2014-01-16 | 2023-11-02 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
US10815481B2 (en) * | 2014-12-16 | 2020-10-27 | Roche Innovation Center Copenhagen A/S | Chiral library screen |
RU2711506C2 (ru) | 2014-12-17 | 2020-01-17 | ПРОКЬЮЭР ТЕРАПЬЮТИКС II Би.Ви. | Редактирование целевой рнк |
PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
MA43072A (fr) * | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
MX2018003992A (es) | 2015-10-09 | 2018-08-01 | Wave Life Sciences Ltd | Composiciones oligonucleotidicas y sus metodos. |
LT3386511T (lt) | 2015-12-10 | 2021-08-25 | Ptc Therapeutics, Inc. | Hantingtono ligos gydymo būdai |
EP3408391A4 (en) | 2016-01-31 | 2019-08-28 | University of Massachusetts | BRANCHED OLIGONUCLEOTIDES |
CA3015823A1 (en) | 2016-03-13 | 2017-09-21 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
MA45188A (fr) | 2016-06-03 | 2019-04-10 | Wave Life Sciences Ltd | Oligonucléotides, compositions et méthodes associées |
JP7074345B2 (ja) | 2016-06-22 | 2022-05-24 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 一本鎖rna編集オリゴヌクレオチド |
CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
JP7244922B2 (ja) | 2016-09-01 | 2023-03-23 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 化学修飾された一本鎖rna編集オリゴヌクレオチド |
JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
MD3554553T2 (ro) | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
MX2019006989A (es) | 2016-12-19 | 2019-08-16 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
BR112019012664A2 (pt) | 2016-12-19 | 2020-01-21 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
WO2018145009A1 (en) * | 2017-02-06 | 2018-08-09 | University Of Tennessee Research Foundation | Dna-zyme based methods & compositions for treating huntington's disease |
GB2574769A (en) | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
IL300875A (en) | 2017-06-05 | 2023-04-01 | Ptc Therapeutics Inc | Compounds for the treatment of Huntington's disease |
WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | COMPOUNDS, COMPOSITIONS AND METHODS OF SYNTHESIS |
US11395822B2 (en) | 2017-06-28 | 2022-07-26 | Ptc Therapeutics, Inc. | Methods for treating Huntington's disease |
MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
CN110996968A (zh) | 2017-08-08 | 2020-04-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
SG11202000276YA (en) | 2017-09-18 | 2020-04-29 | Wave Life Sciences Ltd | Technologies for oligonucleotide preparation |
EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
US20210145852A1 (en) | 2017-09-28 | 2021-05-20 | Sarepta Therapeutics, Inc. | Combination Therapies for Treating Muscular Dystrophy |
US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
JP7455746B2 (ja) | 2018-01-12 | 2024-03-26 | ブリストル-マイヤーズ スクイブ カンパニー | アルファ-シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用 |
CN111902537A (zh) | 2018-01-15 | 2020-11-06 | Ionis制药公司 | Dnm2表达的调节剂 |
US11332733B2 (en) | 2018-02-12 | 2022-05-17 | lonis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
WO2019183440A1 (en) * | 2018-03-22 | 2019-09-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating fmr1 expression |
WO2019191092A1 (en) | 2018-03-27 | 2019-10-03 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
CA3096667A1 (en) * | 2018-04-12 | 2019-10-17 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
CU20200082A7 (es) | 2018-05-09 | 2021-06-08 | Ionis Pharmaceuticals Inc | Compuestos y métodos para la reducción de la expresión de fxi |
CA3098624A1 (en) * | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
GB201808146D0 (en) * | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY |
SG11202012674PA (en) | 2018-06-27 | 2021-01-28 | Ptc Therapeutics Inc | Heterocyclic and heteroaryl compounds for treating huntington's disease |
CN109030440B (zh) * | 2018-07-18 | 2020-12-04 | 西北农林科技大学 | 一种基于三氧化钼量子点检测单宁酸含量的方法 |
AR115847A1 (es) | 2018-07-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir la expresión de la atxn2 |
TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
SG11202101288TA (en) | 2018-08-10 | 2021-03-30 | Univ Massachusetts | Modified oligonucleotides targeting snps |
TW202023573A (zh) | 2018-09-19 | 2020-07-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
WO2020072883A1 (en) * | 2018-10-05 | 2020-04-09 | Ionis Pharmaceuticals, Inc. | Chirally enriched oligomeric compounds |
AU2019380940A1 (en) | 2018-11-15 | 2021-06-03 | Ionis Pharmaceuticals, Inc. | Modulators of IRF5 expression |
CA3117694A1 (en) | 2018-11-21 | 2020-05-28 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing prion expression |
EA202191601A1 (ru) | 2018-12-13 | 2022-01-19 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
MA54875A (fr) * | 2019-02-01 | 2021-12-08 | Wave Life Sciences Ltd | Compositions oligonucléotidiques et procédés associés |
WO2020191252A1 (en) * | 2019-03-20 | 2020-09-24 | Wave Life Sciences Ltd. | Technologies useful for oligonucleotide preparation |
WO2020205463A1 (en) | 2019-03-29 | 2020-10-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating ube3a-ats |
US20220193246A1 (en) | 2019-04-18 | 2022-06-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
SG11202111387YA (en) * | 2019-04-25 | 2021-11-29 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
WO2020243292A1 (en) | 2019-05-28 | 2020-12-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
EP3956450A4 (en) | 2019-07-26 | 2022-11-16 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR MODULATION OF GFAP |
CN114728017A (zh) | 2019-10-14 | 2022-07-08 | 阿斯利康(瑞典)有限公司 | Pnpla3表达的调节剂 |
KR20220093335A (ko) | 2019-11-01 | 2022-07-05 | 노파르티스 아게 | 헌팅턴병의 진행을 늦추는 치료를 위한 스플라이싱 조절제의 용도 |
AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
BR112022023465A2 (pt) | 2020-05-22 | 2023-01-10 | Wave Life Sciences Ltd | Agente de rnai de fita dupla (dsrnai), composição de oligonucleotídeo quiralmente controlada, oligonucleotídeo de fita dupla, método para reduzir o nível e/ou a atividade de um transcrito ou uma proteína codificada pelo mesmo, e método para supressão específica quanto ao alelo de um transcrito de uma sequência de ácidos nucleicos |
AR122534A1 (es) | 2020-06-03 | 2022-09-21 | Triplet Therapeutics Inc | Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3 |
TW202227102A (zh) | 2020-09-22 | 2022-07-16 | 瑞典商阿斯特捷利康公司 | 治療脂肪肝病之方法 |
US11447521B2 (en) | 2020-11-18 | 2022-09-20 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating angiotensinogen expression |
GB2603454A (en) | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
WO2022189363A1 (en) | 2021-03-08 | 2022-09-15 | Les Laboratoires Servier | Antisense oligonucleotides for inhibiting alpha-synuclein expression |
TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
CN113122615A (zh) * | 2021-05-24 | 2021-07-16 | 广州赛哲生物科技股份有限公司 | 一种应用于绝对定量高通量测序的多重pcr扩增的单分子标签引物及其应用 |
EP4359532A2 (en) | 2021-06-22 | 2024-05-01 | AcuraStem Incorporated | Pikfyve antisense oligonucleotides |
CA3174095A1 (en) | 2021-06-23 | 2022-12-29 | Vignesh Narayan HARIHARAN | Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders |
CA3227115A1 (en) | 2021-07-21 | 2023-01-26 | Wen-Hsuan Chang | Unc13a antisense oligonucleotides |
CA3233242A1 (en) | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
GB202117758D0 (en) | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
WO2023212625A1 (en) | 2022-04-28 | 2023-11-02 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
WO2024035946A1 (en) * | 2022-08-11 | 2024-02-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
Family Cites Families (193)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US513030A (en) | 1894-01-16 | Machine for waxing or coating paper | ||
US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
US4415732A (en) | 1981-03-27 | 1983-11-15 | University Patents, Inc. | Phosphoramidite compounds and processes |
DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
US4659774A (en) | 1985-11-01 | 1987-04-21 | American Hoechst Corporation | Support for solid-phase oligonucleotide synthesis |
US5130300A (en) | 1986-03-07 | 1992-07-14 | Monsanto Company | Method for enhancing growth of mammary parenchyma |
DE3851889T2 (de) | 1987-06-24 | 1995-04-13 | Florey Howard Inst | Nukleosid-derivate. |
US4923901A (en) | 1987-09-04 | 1990-05-08 | Millipore Corporation | Membranes with bound oligonucleotides and peptides |
US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
US5141813A (en) | 1989-08-28 | 1992-08-25 | Clontech Laboratories, Inc. | Multifunctional controlled pore glass reagent for solid phase oligonucleotide synthesis |
US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
CA2029273A1 (en) | 1989-12-04 | 1991-06-05 | Christine L. Brakel | Modified nucleotide compounds |
US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
ATE154246T1 (de) | 1990-07-27 | 1997-06-15 | Isis Pharmaceuticals Inc | Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren |
US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
US5512668A (en) * | 1991-03-06 | 1996-04-30 | Polish Academy Of Sciences | Solid phase oligonucleotide synthesis using phospholane intermediates |
US20020183502A1 (en) | 1991-05-21 | 2002-12-05 | Mesmaeker Alain De | Backbone-modified oligonucleotide analogs and methods for using same |
US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
EP0655088B1 (en) | 1991-10-15 | 2002-07-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having chiral phosphorus linkages |
EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
US6235887B1 (en) | 1991-11-26 | 2001-05-22 | Isis Pharmaceuticals, Inc. | Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines |
US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
EP1695979B1 (en) | 1991-12-24 | 2011-07-06 | Isis Pharmaceuticals, Inc. | Gapped modified oligonucleotides |
US6015886A (en) | 1993-05-24 | 2000-01-18 | Chemgenes Corporation | Oligonucleotide phosphate esters |
US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
EP0729474A4 (en) | 1993-11-16 | 1998-10-21 | Genta Inc | SYNTHETIC OLIGOMERS THAT HAVE CHIRALITY-PURE PHOSPHONATE INTERNUCLEOSIDYL BINDINGS MIXED WITH NON-PHOSPHONATE INTERNUKLEOSIDYL BINDINGS |
US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
US6166197A (en) | 1995-03-06 | 2000-12-26 | Isis Pharmaceuticals, Inc. | Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions |
US6222025B1 (en) | 1995-03-06 | 2001-04-24 | Isis Pharmaceuticals, Inc. | Process for the synthesis of 2′-O-substituted pyrimidines and oligomeric compounds therefrom |
US6172209B1 (en) | 1997-02-14 | 2001-01-09 | Isis Pharmaceuticals Inc. | Aminooxy-modified oligonucleotides and methods for making same |
US6639062B2 (en) | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
WO1999005160A2 (en) | 1997-07-25 | 1999-02-04 | Hybridon, Inc. | Oligonuclotides having 3' terminal stereospecific phosphorothioates |
US6617438B1 (en) | 1997-11-05 | 2003-09-09 | Sirna Therapeutics, Inc. | Oligoribonucleotides with enzymatic activity |
US6528640B1 (en) | 1997-11-05 | 2003-03-04 | Ribozyme Pharmaceuticals, Incorporated | Synthetic ribonucleic acids with RNAse activity |
PT1054693E (pt) * | 1998-02-20 | 2009-01-22 | Genentech Inc | Inibidores da activação do complemento |
US7045610B2 (en) | 1998-04-03 | 2006-05-16 | Epoch Biosciences, Inc. | Modified oligonucleotides for mismatch discrimination |
US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
US6867294B1 (en) * | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
US6066500A (en) | 1999-06-25 | 2000-05-23 | Isis Pharmaceuticals Inc. | Antisense modulation of Beta catenin expression |
US6147200A (en) | 1999-08-19 | 2000-11-14 | Isis Pharmaceuticals, Inc. | 2'-O-acetamido modified monomers and oligomers |
US6949520B1 (en) | 1999-09-27 | 2005-09-27 | Coley Pharmaceutical Group, Inc. | Methods related to immunostimulatory nucleic acid-induced interferon |
US20020082227A1 (en) | 1999-09-30 | 2002-06-27 | Scott Henry | Use of oligonucleotides for inhibition of complement activation |
US20010055761A1 (en) | 1999-10-29 | 2001-12-27 | Agilent Technologies | Small scale dna synthesis using polymeric solid support with functionalized regions |
DE10019756A1 (de) | 2000-04-20 | 2001-10-25 | Bayer Ag | Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen |
US6492171B2 (en) | 2000-05-16 | 2002-12-10 | Isis Pharmaceuticals, Inc. | Antisense modulation of TERT expression |
GB0024752D0 (en) | 2000-10-10 | 2000-11-22 | Univ Belfast | Oxidative halogenation of aromatic compounds |
US20050277133A1 (en) | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
US6440739B1 (en) | 2001-07-17 | 2002-08-27 | Isis Pharmaceuticals, Inc. | Antisense modulation of glioma-associated oncogene-2 expression |
JP4348044B2 (ja) | 2002-02-12 | 2009-10-21 | 株式会社キラルジェン | 立体規則性の高いジヌクレオシドホスホロチオエートの製造法 |
US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
US8232383B2 (en) | 2002-02-20 | 2012-07-31 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
WO2003085110A2 (en) | 2002-04-05 | 2003-10-16 | Santaris Pharma A/S | Oligomeric compounds for the modulation hif-1alpha expression |
EP2333062A1 (en) | 2002-07-10 | 2011-06-15 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | RNA-interference by single-stranded RNA molecules |
US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
US7045306B2 (en) | 2003-04-28 | 2006-05-16 | The General Hospital Corporation | Method for identifying compounds in vitro that modulate the dysregulation of transcription of transcription mediated by mutant huntingtin protein |
AU2004239114B2 (en) | 2003-05-14 | 2008-03-13 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
JP2005089441A (ja) | 2003-08-08 | 2005-04-07 | Toudai Tlo Ltd | 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法 |
US7427672B2 (en) | 2003-08-28 | 2008-09-23 | Takeshi Imanishi | Artificial nucleic acids of n-o bond crosslinkage type |
WO2005023828A1 (ja) | 2003-09-02 | 2005-03-17 | Takeshi Wada | リボヌクレオチド又はリボヌクレオチド誘導体の製造方法 |
JP4616175B2 (ja) | 2003-09-02 | 2011-01-19 | 株式会社キラルジェン | 5’−ホスフィチル化モノマーおよびh−ホスホネートオリゴヌクレオチド誘導体の製造方法 |
US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
ES2808561T3 (es) | 2003-09-12 | 2021-03-01 | Univ Massachusetts | Interferencia por ARN para el tratamiento de trastornos de ganancia de función |
WO2005070859A1 (ja) | 2004-01-27 | 2005-08-04 | Takeshi Wada | フルオラス担体およびそれを用いたオリゴヌクレオチド誘導体の製造方法 |
WO2005085272A1 (ja) | 2004-03-05 | 2005-09-15 | Takeshi Wada | ボラノホスフェートモノマーおよびそれを用いたオリゴヌクレオチド誘導体の製造方法 |
WO2005092909A1 (ja) | 2004-03-25 | 2005-10-06 | Toudai Tlo, Ltd. | 立体規則性の高いリボヌクレオチド類縁体及びデオキシリボヌクレオチド類縁体の製造法 |
US20060019916A1 (en) | 2004-04-02 | 2006-01-26 | Coley Pharmaceutical Group, Inc. | Immunostimulatory nucleic acids for inducing IL-10 responses |
WO2006034348A2 (en) | 2004-09-17 | 2006-03-30 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
WO2006066260A2 (en) | 2004-12-17 | 2006-06-22 | Thiosense, Inc. | Compositions of and methods for producing phosphorus-chiral monomers and oligomers |
WO2006121960A2 (en) | 2005-05-06 | 2006-11-16 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
EP2062980B1 (en) | 2005-06-28 | 2011-08-31 | Medtronic, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin gene. |
US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
EP1937312B1 (en) | 2005-08-30 | 2016-06-29 | Ionis Pharmaceuticals, Inc. | Chimeric oligomeric compounds for modulation of splicing |
AU2006305886C1 (en) * | 2005-10-28 | 2011-03-17 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
PT2161038E (pt) | 2006-01-26 | 2014-03-10 | Isis Pharmaceuticals Inc | Composições e suas utilizações dirigidas à huntingtina |
EP2314594B1 (en) | 2006-01-27 | 2014-07-23 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
US20090326041A1 (en) | 2006-05-05 | 2009-12-31 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression of sglt2 |
DK2066684T3 (da) | 2006-05-11 | 2012-10-22 | Isis Pharmaceuticals Inc | 5´-Modificerede bicycliske nukleinsyreanaloge |
EP2034015B1 (en) | 2006-05-31 | 2012-07-11 | Toray Industries, Inc. | Immunostimulatory oligonucleotide and pharmaceutical application thereof |
CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
AU2007310989B2 (en) | 2006-10-18 | 2014-05-29 | Isis Pharmaceuticals, Inc. | Antisense compounds |
US20100298280A1 (en) | 2007-06-13 | 2010-11-25 | Petra Kioschis-Schneider | Compounds for the Modulation of Huntingtin Aggregation, Methods and Means for Identifying Such Compounds |
EP2014769B1 (en) | 2007-06-18 | 2010-03-31 | Commissariat à l'Energie Atomique | Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease |
CN101795715A (zh) | 2007-07-09 | 2010-08-04 | 艾德拉药物股份有限公司 | 稳定化免疫调控性rna(simra)化合物 |
EP2224912B1 (en) * | 2008-01-02 | 2016-05-11 | TEKMIRA Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
EP2240768A1 (en) | 2008-02-04 | 2010-10-20 | Galapagos N.V. | Target sequences and methods to identify the same, useful in treatment of neurodegenerative diseases |
US8679750B2 (en) * | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
AU2009244013B2 (en) | 2008-05-09 | 2015-06-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington's disease |
WO2009143391A2 (en) | 2008-05-22 | 2009-11-26 | Isis Pharmaceuticals, Inc | Methods for modulation expression of creb |
WO2009148605A2 (en) | 2008-06-04 | 2009-12-10 | Isis Pharmaceuticals, Inc. | Methods for treating hypercholesterolemia |
US8703730B2 (en) | 2008-07-10 | 2014-04-22 | Regenesance B.V. | Complement antagonists and uses thereof |
KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
US20120264806A1 (en) | 2009-02-06 | 2012-10-18 | Bennett C Frank | Oligomeric compounds and excipients |
US8987222B2 (en) * | 2009-04-08 | 2015-03-24 | University Of Massachusetts | Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease |
IN2012DN00720A (ja) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
WO2011015573A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
WO2011015572A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
US8927553B2 (en) | 2009-08-10 | 2015-01-06 | Daljit Singh Dhanoa | Deuterium-enriched alkyl sulfonamides and uses thereof |
KR102279458B1 (ko) | 2009-09-11 | 2021-07-21 | 아이오니스 파마수티컬즈, 인코포레이티드 | 헌팅틴 발현의 조절 |
CN102574888A (zh) | 2009-09-16 | 2012-07-11 | 株式会社启拉坚 | 用于rna及其衍生物的合成的新型保护基 |
EP2519632B1 (en) | 2009-12-31 | 2018-04-11 | CuRNA, Inc. | Treatment of insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to irs2 and transcription factor e3 (tfe3) |
EP2534262B1 (en) | 2010-02-08 | 2016-12-14 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
US8957040B2 (en) | 2010-02-08 | 2015-02-17 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
JP2011184318A (ja) | 2010-03-05 | 2011-09-22 | Univ Of Tokyo | リボヌクレシドh−ボラノホスホネート |
JP5847700B2 (ja) | 2010-03-05 | 2016-01-27 | 株式会社Wave Life Sciences Japan | リボヌクレオシドホスホロチオエートの製造方法 |
RU2664452C2 (ru) | 2010-04-19 | 2018-08-17 | Нлифе Терапеутикс, С.Л. | Конъюгат, медицинское средство и способы лечения и/или профилактики депрессии и болезни, связанной с отложением телец Леви |
JP5756858B2 (ja) * | 2010-08-20 | 2015-07-29 | セルリアン・ファーマ・インコーポレイテッド | 複合体、粒子、組成物および関連の方法 |
AU2011296268A1 (en) | 2010-08-31 | 2013-02-21 | Merck Sharp & Dohme Corp. | Novel single chemical entities and methods for delivery of oligonucleotides |
US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
EP2638163B1 (en) | 2010-11-12 | 2017-05-17 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
WO2012073857A1 (ja) | 2010-11-30 | 2012-06-07 | 株式会社キラルジェン | 2'-o-修飾rna |
US10017764B2 (en) | 2011-02-08 | 2018-07-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
US9181544B2 (en) | 2011-02-12 | 2015-11-10 | University Of Iowa Research Foundation | Therapeutic compounds |
EP2699271A4 (en) * | 2011-04-20 | 2015-10-07 | Larry J Smith | METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION USING COMPONENTS THAT SELF-ASSEMBLED IN CELLS AND PRODUCE RNAI ACTIVITY |
BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
US10202599B2 (en) | 2011-08-11 | 2019-02-12 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
ES2907250T3 (es) * | 2012-01-27 | 2022-04-22 | Biomarin Tech Bv | Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker |
EP2873674B1 (en) | 2012-07-13 | 2020-05-06 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
BR112015000784A8 (pt) | 2012-07-13 | 2018-04-03 | Wave Life Sciences Japan | Grupo auxiliar assimétrico |
WO2014059356A2 (en) | 2012-10-12 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
CA2889608A1 (en) | 2012-10-26 | 2014-05-01 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to cell types |
WO2014064257A1 (en) | 2012-10-26 | 2014-05-01 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
RU2649367C2 (ru) | 2013-01-30 | 2018-04-02 | Ф. Хоффманн-Ля Рош Аг | Конъюгаты углевода и lna-олигонуклеотида |
US10260069B2 (en) | 2013-02-04 | 2019-04-16 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
EA031393B1 (ru) | 2013-05-01 | 2018-12-28 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии hbv и ttr |
AU2014329452B2 (en) | 2013-10-03 | 2019-06-20 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor |
NZ719477A (en) | 2013-11-11 | 2022-05-27 | Sangamo Therapeutics Inc | Methods and compositions for treating huntington’s disease |
WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
KR20230152178A (ko) | 2014-01-16 | 2023-11-02 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
EP3647318B1 (en) | 2014-04-28 | 2021-06-30 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
KR102380324B1 (ko) | 2014-05-08 | 2022-03-30 | 상가모 테라퓨틱스, 인코포레이티드 | 헌팅턴병을 치료하기 위한 방법 및 조성물 |
EP3146051B8 (en) | 2014-05-20 | 2019-11-27 | University of Iowa Research Foundation | Huntington's disease therapeutic compounds |
US20160017327A1 (en) | 2014-07-11 | 2016-01-21 | The Johns Hopkins University | Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity |
CA2955250A1 (en) | 2014-07-16 | 2016-01-21 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
US20180016575A1 (en) | 2014-11-19 | 2018-01-18 | Roche Innovation Center Copenhagen A/S | LNA Gapmer Oligonucleotides Comprising Chiral Phosphorothioate Linkages |
US10815481B2 (en) | 2014-12-16 | 2020-10-27 | Roche Innovation Center Copenhagen A/S | Chiral library screen |
CN108064292B (zh) | 2014-12-24 | 2021-05-04 | 尤尼克尔生物制药股份有限公司 | RNAi诱导的亨廷顿蛋白基因抑制 |
ES2893114T3 (es) | 2015-02-04 | 2022-02-08 | Bristol Myers Squibb Co | Métodos para seleccionar moléculas terapéuticas |
WO2016127002A1 (en) | 2015-02-04 | 2016-08-11 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
KR20170110149A (ko) | 2015-02-10 | 2017-10-10 | 젠자임 코포레이션 | 변이형 RNAi |
PL3277814T3 (pl) | 2015-04-03 | 2020-11-30 | University Of Massachusetts | Związki oligonukleotydowe ukierunkowane na mrna huntingtyny |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
MX2018003992A (es) | 2015-10-09 | 2018-08-01 | Wave Life Sciences Ltd | Composiciones oligonucleotidicas y sus metodos. |
WO2017067970A1 (en) | 2015-10-22 | 2017-04-27 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
CA3015823A1 (en) | 2016-03-13 | 2017-09-21 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
MA45290A (fr) | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
MA45188A (fr) | 2016-06-03 | 2019-04-10 | Wave Life Sciences Ltd | Oligonucléotides, compositions et méthodes associées |
US20190264267A1 (en) | 2016-07-25 | 2019-08-29 | Wave Life Sciences Ltd. | Phasing |
US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
CN111164091A (zh) | 2017-06-02 | 2020-05-15 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
US11597927B2 (en) | 2017-06-02 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | COMPOUNDS, COMPOSITIONS AND METHODS OF SYNTHESIS |
CN110996968A (zh) | 2017-08-08 | 2020-04-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
SG11202000276YA (en) | 2017-09-18 | 2020-04-29 | Wave Life Sciences Ltd | Technologies for oligonucleotide preparation |
US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
CA3096667A1 (en) | 2018-04-12 | 2019-10-17 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
CA3122271A1 (en) | 2018-12-06 | 2020-06-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
MA54875A (fr) | 2019-02-01 | 2021-12-08 | Wave Life Sciences Ltd | Compositions oligonucléotidiques et procédés associés |
WO2020191252A1 (en) | 2019-03-20 | 2020-09-24 | Wave Life Sciences Ltd. | Technologies useful for oligonucleotide preparation |
JPWO2020196662A1 (ja) | 2019-03-25 | 2020-10-01 | ||
SG11202111387YA (en) | 2019-04-25 | 2021-11-29 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
SG11202111386UA (en) | 2019-04-25 | 2021-11-29 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
CN114502177A (zh) | 2019-05-09 | 2022-05-13 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
CA3156176A1 (en) | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
CA3154768A1 (en) | 2019-10-06 | 2021-04-15 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
WO2021178237A2 (en) | 2020-03-01 | 2021-09-10 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
US20230203087A1 (en) | 2020-05-22 | 2023-06-29 | Pachamuthu Kandasamy | Oligonucleotide compositions and methods thereof |
-
0
- MA MA43072A patent/MA43072A/fr unknown
-
2016
- 2016-07-22 KR KR1020187004483A patent/KR20180028516A/ko unknown
- 2016-07-22 AU AU2016297155A patent/AU2016297155B2/en active Active
- 2016-07-22 CA CA2989682A patent/CA2989682A1/en active Pending
- 2016-07-22 TW TW105123348A patent/TW201707711A/zh unknown
- 2016-07-22 SG SG10201912900XA patent/SG10201912900XA/en unknown
- 2016-07-22 CN CN201680042413.3A patent/CN108135921B/zh active Active
- 2016-07-22 EP EP23206565.6A patent/EP4344744A2/en active Pending
- 2016-07-22 WO PCT/US2016/043598 patent/WO2017015575A1/en active Application Filing
- 2016-07-22 CR CR20180107A patent/CR20180107A/es unknown
- 2016-07-22 MX MX2018000796A patent/MX2018000796A/es unknown
- 2016-07-22 PE PE2018000099A patent/PE20181174A1/es unknown
- 2016-07-22 BR BR112017028636A patent/BR112017028636A2/pt not_active IP Right Cessation
- 2016-07-22 JP JP2018502647A patent/JP2018525357A/ja active Pending
- 2016-07-22 CN CN201680043299.6A patent/CN108025089A/zh active Pending
- 2016-07-22 JP JP2018502637A patent/JP7296724B2/ja active Active
- 2016-07-22 RU RU2017146349A patent/RU2017146349A/ru unknown
- 2016-07-22 EP EP16828604.5A patent/EP3325017A4/en not_active Withdrawn
- 2016-07-22 US US15/746,199 patent/US20180216107A1/en not_active Abandoned
- 2016-07-22 EP EP16828617.7A patent/EP3324978A4/en active Pending
- 2016-07-22 WO PCT/US2016/043542 patent/WO2017015555A1/en active Application Filing
- 2016-07-22 US US15/746,220 patent/US10479995B2/en active Active
-
2017
- 2017-12-26 IL IL256603A patent/IL256603A/en unknown
-
2018
- 2018-01-15 NI NI201800009A patent/NI201800009A/es unknown
- 2018-01-18 PH PH12018500145A patent/PH12018500145A1/en unknown
- 2018-01-18 CL CL2018000145A patent/CL2018000145A1/es unknown
- 2018-01-22 SV SV2018005619A patent/SV2018005619A/es unknown
- 2018-02-09 EC ECIEPI201810463A patent/ECSP18010463A/es unknown
- 2018-02-15 CO CONC2018/0001484A patent/CO2018001484A2/es unknown
- 2018-11-14 HK HK18114508.6A patent/HK1255369A1/zh unknown
- 2018-11-14 HK HK18114509.5A patent/HK1255370A1/zh unknown
-
2019
- 2019-08-26 US US16/551,503 patent/US20200080083A1/en not_active Abandoned
-
2020
- 2020-02-04 US US16/782,021 patent/US11634710B2/en active Active
-
2021
- 2021-07-14 US US17/375,658 patent/US20220195429A1/en active Pending
- 2021-08-20 JP JP2021134582A patent/JP7441816B2/ja active Active
- 2021-10-18 JP JP2021170541A patent/JP2022009217A/ja active Pending
-
2023
- 2023-02-14 AU AU2023200807A patent/AU2023200807A1/en active Pending
- 2023-12-08 JP JP2023207761A patent/JP2024028927A/ja active Pending
Similar Documents
Publication | Publication Date | Title |
---|---|---|
JP2018525357A5 (ja) | ||
US10337005B2 (en) | Oligonucleotide-based inhibitors comprising locked nucleic acid motif | |
RU2017146349A (ru) | Композиции олигонуклеотидов и способы с ними | |
ES2731460T3 (es) | Alto rendimiento de células individuales con código de barra | |
JP2020188771A (ja) | 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物 | |
US20140066491A1 (en) | Chemical modification motifs for mirna inhibitors and mimetics | |
JP2018512155A5 (ja) | ||
JP2016513976A5 (ja) | ||
TWI762732B (zh) | 用於降低PAPD5及PAPD7 mRNA以治療B型肝炎感染之核酸分子 | |
JP2023533721A (ja) | トリヌクレオチドキャップ類似体、それらの調製、及び使用 | |
CN104540527A (zh) | Mir-15微小rna家族的抑制剂 | |
TW202219273A (zh) | 靶向rna結合蛋白位點之寡核苷酸 | |
JP2023179428A (ja) | メッセンジャーリボ核酸(mRNA)をコードする安定化された核酸 | |
ES2875318T3 (es) | Procedimiento para generar colecciones de ADN circular monocatenario para secuenciación de molécula única | |
JPWO2020171149A1 (ja) | ヘテロ核酸の最適ps修飾パターン | |
WO2024084048A1 (en) | Heteroduplex rna editing oligonucleotide complexes | |
TW202136510A (zh) | 用於抑制scn9a表現之增強型寡核苷酸 |