JP2020534253A - オリゴヌクレオチド組成物及びその方法 - Google Patents
オリゴヌクレオチド組成物及びその方法 Download PDFInfo
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- JP2020534253A JP2020534253A JP2020506776A JP2020506776A JP2020534253A JP 2020534253 A JP2020534253 A JP 2020534253A JP 2020506776 A JP2020506776 A JP 2020506776A JP 2020506776 A JP2020506776 A JP 2020506776A JP 2020534253 A JP2020534253 A JP 2020534253A
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- oligonucleotide
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- c9orf72
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- 0 CCC(C(CC*1)=O)C1=C Chemical compound CCC(C(CC*1)=O)C1=C 0.000 description 20
- WAUJZUGAXSBUDL-UHFFFAOYSA-N C(CC1)CC2C1POCC2 Chemical compound C(CC1)CC2C1POCC2 WAUJZUGAXSBUDL-UHFFFAOYSA-N 0.000 description 1
- QDKBRJLPOWPPRK-GDNLROHBSA-N CCCNCCCCCC[U]C([U])[U]C(C)/C=C(\C)/C=C Chemical compound CCCNCCCCCC[U]C([U])[U]C(C)/C=C(\C)/C=C QDKBRJLPOWPPRK-GDNLROHBSA-N 0.000 description 1
- DINHQTMZNSGPJQ-UHFFFAOYSA-N CCN(C=CC(C(C)=N)=N1)C1=O Chemical compound CCN(C=CC(C(C)=N)=N1)C1=O DINHQTMZNSGPJQ-UHFFFAOYSA-N 0.000 description 1
- IKWLQGUEMFAUPE-UHFFFAOYSA-O CNPN1C(C[OH2+])CCCC1 Chemical compound CNPN1C(C[OH2+])CCCC1 IKWLQGUEMFAUPE-UHFFFAOYSA-O 0.000 description 1
- GSPXYMMIPMXPBZ-UHFFFAOYSA-N C[Pm](C)OCC1CCCCC1 Chemical compound C[Pm](C)OCC1CCCCC1 GSPXYMMIPMXPBZ-UHFFFAOYSA-N 0.000 description 1
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- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Crystallography & Structural Chemistry (AREA)
- Bioinformatics & Computational Biology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762542778P | 2017-08-08 | 2017-08-08 | |
| US62/542,778 | 2017-08-08 | ||
| PCT/US2018/045653 WO2019032607A1 (en) | 2017-08-08 | 2018-08-07 | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020534253A true JP2020534253A (ja) | 2020-11-26 |
| JP2020534253A5 JP2020534253A5 (enExample) | 2021-09-16 |
Family
ID=65271538
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020506776A Pending JP2020534253A (ja) | 2017-08-08 | 2018-08-07 | オリゴヌクレオチド組成物及びその方法 |
| JP2020506814A Active JP7422068B2 (ja) | 2017-08-08 | 2018-08-07 | オリゴヌクレオチド組成物及びその方法 |
| JP2023161596A Pending JP2024009824A (ja) | 2017-08-08 | 2023-09-25 | オリゴヌクレオチド組成物及びその方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020506814A Active JP7422068B2 (ja) | 2017-08-08 | 2018-08-07 | オリゴヌクレオチド組成物及びその方法 |
| JP2023161596A Pending JP2024009824A (ja) | 2017-08-08 | 2023-09-25 | オリゴヌクレオチド組成物及びその方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11739325B2 (enExample) |
| EP (2) | EP3664815A4 (enExample) |
| JP (3) | JP2020534253A (enExample) |
| KR (1) | KR20200035301A (enExample) |
| CN (2) | CN111032057A (enExample) |
| AR (1) | AR112779A1 (enExample) |
| AU (1) | AU2018316159A1 (enExample) |
| CA (1) | CA3072076A1 (enExample) |
| SG (1) | SG11202000274RA (enExample) |
| TW (1) | TW201920672A (enExample) |
| WO (2) | WO2019032607A1 (enExample) |
Families Citing this family (61)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| CN106068325B (zh) | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| JP7511326B2 (ja) | 2015-10-09 | 2024-07-05 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物およびその方法 |
| MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| JP2019520339A (ja) | 2016-06-03 | 2019-07-18 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | オリゴヌクレオチド、その組成物および方法 |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| NZ751483A (en) | 2016-09-01 | 2022-07-01 | Proqr Therapeutics Ii Bv | Chemically modified single-stranded rna-editing oligonucleotides |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| US11723912B2 (en) | 2016-12-08 | 2023-08-15 | University Of Utah Research Foundation | Staufen1 agents and associated methods |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| CN111051281A (zh) | 2017-06-21 | 2020-04-21 | 波涛生命科学有限公司 | 用于合成的化合物、组合物和方法 |
| JP2020534253A (ja) | 2017-08-08 | 2020-11-26 | ウェーブ ライフ サイエンシーズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| KR20200052369A (ko) | 2017-09-18 | 2020-05-14 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 제조 기술 |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| US11332733B2 (en) | 2018-02-12 | 2022-05-17 | lonis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| AU2019265904A1 (en) | 2018-05-11 | 2020-11-12 | 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 |
| JP2021527649A (ja) * | 2018-06-14 | 2021-10-14 | ユニバーシティー オブ ユタ リサーチ ファウンデーションUniversity of Utah Research Foundation | Staufen1調節剤および関連する方法 |
| CA3121749A1 (en) | 2018-12-06 | 2020-06-11 | Biogen Ma Inc. | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
| CA3126845A1 (en) * | 2019-02-01 | 2020-08-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2020191212A1 (en) * | 2019-03-20 | 2020-09-24 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| CN114072501A (zh) | 2019-05-06 | 2022-02-18 | 马萨诸塞大学 | 抗c9orf72寡核苷酸及相关方法 |
| 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 |
| WO2021007110A1 (en) * | 2019-07-05 | 2021-01-14 | University Of Florida Research Foundation, Incorporated | Methods for treating ran protein-associated neurological diseases |
| US20240175016A1 (en) * | 2019-10-06 | 2024-05-30 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| EP4097236A4 (en) * | 2020-01-28 | 2024-03-27 | University of Southern California | COMPOSITIONS AND METHODS FOR TREATING NEUROLOGICAL DISEASES |
| WO2021159008A2 (en) | 2020-02-07 | 2021-08-12 | Maze Therapeutics, Inc. | Compositions and methods for treating neurodegenerative diseases |
| EP4133073A2 (en) * | 2020-04-09 | 2023-02-15 | Association Institut de Myologie | Antisense sequences for treating amyotrophic lateral sclerosis |
| EP4153747A2 (en) | 2020-05-22 | 2023-03-29 | Wave Life Sciences Ltd. | Double stranded oligonucleotide compositions and methods relating thereto |
| MX2023011794A (es) | 2021-04-06 | 2024-01-08 | Maze Therapeutics Inc | Composiciones y métodos para el tratamiento de la proteinopatía de proteina de union a acido desoxirribonucleico tar 43 (tdp-43). |
| CN117561334A (zh) * | 2021-06-04 | 2024-02-13 | 阿尔尼拉姆医药品有限公司 | 人染色体9开放阅读框72(C9ORF72)iRNA药剂组合物和其使用方法 |
| EP4479535A1 (en) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| JP2025516680A (ja) | 2022-05-13 | 2025-05-30 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | 一本鎖ループオリゴヌクレオチド |
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| JP2025524566A (ja) | 2022-07-15 | 2025-07-30 | 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 | Adar媒介rna編集のためのオリゴヌクレオチドおよびその使用 |
| JP2025535055A (ja) | 2022-10-05 | 2025-10-22 | トレース ニューロサイエンス, インコーポレイテッド | Unc13aアンチセンスオリゴヌクレオチド及びその使用 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| KR20250113455A (ko) | 2022-11-24 | 2025-07-25 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 유전성 hfe-혈색소침착증의 치료를 위한 안티센스 올리고뉴클레오티드 |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| KR20250115388A (ko) | 2022-12-09 | 2025-07-30 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 심혈관 질환 치료용 안티센스 올리고뉴클레오타이드 |
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| EP3664816A4 (en) | 2021-05-19 |
| US20200362337A1 (en) | 2020-11-19 |
| WO2019032612A1 (en) | 2019-02-14 |
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