JP2021507709A5 - - Google Patents
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- Publication number
- JP2021507709A5 JP2021507709A5 JP2020534493A JP2020534493A JP2021507709A5 JP 2021507709 A5 JP2021507709 A5 JP 2021507709A5 JP 2020534493 A JP2020534493 A JP 2020534493A JP 2020534493 A JP2020534493 A JP 2020534493A JP 2021507709 A5 JP2021507709 A5 JP 2021507709A5
- Authority
- JP
- Japan
- Prior art keywords
- chiral
- terminal
- modified
- phosphorus atom
- configuration
- 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.)
- Granted
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- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 50
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 50
- 239000003795 chemical substances by application Substances 0.000 claims description 48
- 238000012986 modification Methods 0.000 claims description 43
- 230000004048 modification Effects 0.000 claims description 43
- 125000004437 phosphorous atom Chemical group 0.000 claims description 40
- 229910052698 phosphorus Inorganic materials 0.000 claims description 40
- 230000000692 anti-sense effect Effects 0.000 claims description 38
- 108091081021 Sense strand Proteins 0.000 claims description 23
- 125000003729 nucleotide group Chemical group 0.000 claims description 17
- 239000002773 nucleotide Substances 0.000 claims description 16
- 101150075175 Asgr1 gene Proteins 0.000 claims description 10
- 239000003446 ligand Substances 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 5
- 125000002015 acyclic group Chemical group 0.000 claims description 4
- 230000002401 inhibitory effect Effects 0.000 claims description 4
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical class CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 238000007920 subcutaneous administration Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 2
- 102000005427 Asialoglycoprotein Receptor Human genes 0.000 claims 1
- 108010006523 asialoglycoprotein receptor Proteins 0.000 claims 1
- 238000000034 method Methods 0.000 description 2
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023145038A JP2023171757A (ja) | 2017-12-21 | 2023-09-07 | キラル富化二本鎖ｒｎａ剤 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762609093P | 2017-12-21 | 2017-12-21 | |
| US62/609,093 | 2017-12-21 | ||
| PCT/US2018/067103 WO2019126651A1 (en) | 2017-12-21 | 2018-12-21 | Chirally-enriched double-stranded rna agents |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023145038A Division JP2023171757A (ja) | 2017-12-21 | 2023-09-07 | キラル富化二本鎖ｒｎａ剤 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021507709A JP2021507709A (ja) | 2021-02-25 |
| JP2021507709A5 true JP2021507709A5 (https=) | 2021-11-25 |
| JP7348185B2 JP7348185B2 (ja) | 2023-09-20 |
Family
ID=65041929
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020534493A Active JP7348185B2 (ja) | 2017-12-21 | 2018-12-21 | キラル富化二本鎖ｒｎａ剤 |
| JP2023145038A Pending JP2023171757A (ja) | 2017-12-21 | 2023-09-07 | キラル富化二本鎖ｒｎａ剤 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023145038A Pending JP2023171757A (ja) | 2017-12-21 | 2023-09-07 | キラル富化二本鎖ｒｎａ剤 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11597932B2 (https=) |
| EP (1) | EP3728281A1 (https=) |
| JP (2) | JP7348185B2 (https=) |
| AU (3) | AU2018392782B2 (https=) |
| CA (1) | CA3086485A1 (https=) |
| WO (1) | WO2019126651A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| WO2019217459A1 (en) | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| JP2023526533A (ja) * | 2020-05-22 | 2023-06-21 | ウェイブ ライフ サイエンシズ リミテッド | 二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 |
| US20230257745A1 (en) | 2020-07-10 | 2023-08-17 | Alnylam Pharmaceuticals, Inc. | Circular siRNAs |
| WO2023283403A2 (en) | 2021-07-09 | 2023-01-12 | Alnylam Pharmaceuticals, Inc. | Bis-rnai compounds for cns delivery |
| CA3232068A1 (en) * | 2021-09-21 | 2023-03-30 | Chandra Vargeese | Double stranded oligonucleotide compositions and methods relating thereto |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| JP2024545602A (ja) * | 2021-11-19 | 2024-12-10 | ウェイブ ライフ サイエンシズ リミテッド | Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 |
| CN114230624A (zh) * | 2021-12-22 | 2022-03-25 | 上海兆维科技发展有限公司 | 一种核苷二聚体亚膦酰胺的合成方法 |
| EP4493570A2 (en) | 2022-03-16 | 2025-01-22 | Empirico Inc. | Galnac compositions for improving sirna bioavailability |
| KR20240163743A (ko) | 2022-03-28 | 2024-11-19 | 엠피리코 인크. | 변형된 올리고뉴클레오티드 |
| EP4522742A2 (en) | 2022-05-13 | 2025-03-19 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| EP4532724A1 (en) * | 2022-06-02 | 2025-04-09 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds and uses thereof |
| WO2024238385A2 (en) | 2023-05-12 | 2024-11-21 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
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| US1861608A (en) | 1929-12-21 | 1932-06-07 | Emerson Electric Mfg Co | Fan and means for directing the air current therethrough |
| US4469863A (en) | 1980-11-12 | 1984-09-04 | Ts O Paul O P | Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof |
| US5023243A (en) | 1981-10-23 | 1991-06-11 | Molecular Biosystems, Inc. | Oligonucleotide therapeutic agent and method of making same |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| FR2645866B1 (fr) | 1989-04-17 | 1991-07-05 | Centre Nat Rech Scient | Nouvelles lipopolyamines, leur preparation et leur emploi |
| US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
| US5177198A (en) | 1989-11-30 | 1993-01-05 | University Of N.C. At Chapel Hill | Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
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| US5283185A (en) | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| EP0646178A1 (en) | 1992-06-04 | 1995-04-05 | The Regents Of The University Of California | expression cassette with regularoty regions functional in the mammmlian host |
| JPH07508410A (ja) | 1992-06-18 | 1995-09-21 | ジェンファーム インターナショナル インコーポレイテッド | 酵母人工染色体を有するトランスジェニック非ヒト動物の製造方法 |
| US5476925A (en) | 1993-02-01 | 1995-12-19 | Northwestern University | Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups |
| GB9304620D0 (en) | 1993-03-06 | 1993-04-21 | Ciba Geigy Ag | Compounds |
| JPH11508231A (ja) | 1995-05-26 | 1999-07-21 | ソマティックス セラピー コーポレイション | 安定な脂質／核酸複合体を含む送達ビヒクル |
| US6034135A (en) | 1997-03-06 | 2000-03-07 | Promega Biosciences, Inc. | Dimeric cationic lipids |
| DE19956568A1 (de) | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
| US20040259247A1 (en) | 2000-12-01 | 2004-12-23 | Thomas Tuschl | Rna interference mediating small rna molecules |
| US7723509B2 (en) * | 2003-04-17 | 2010-05-25 | Alnylam Pharmaceuticals | IRNA agents with biocleavable tethers |
| EP2508608A1 (en) * | 2003-06-09 | 2012-10-10 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| WO2012018881A2 (en) * | 2010-08-03 | 2012-02-09 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for the regulation of rna |
| CA2812046A1 (en) * | 2010-09-15 | 2012-03-22 | Alnylam Pharmaceuticals, Inc. | Modified irna agents |
| JP6453212B2 (ja) * | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| ES2917473T3 (es) * | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| TWI710633B (zh) * | 2014-11-10 | 2020-11-21 | 美商阿尼拉製藥公司 | Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法 |
| 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 |
| EP3469083A1 (en) * | 2016-06-10 | 2019-04-17 | Alnylam Pharmaceuticals, Inc. | COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) |
| JP2019532027A (ja) * | 2016-08-17 | 2019-11-07 | ソルスティス バイオロジクス，リミティッド | ポリヌクレオチド構築物 |
| KR102318555B1 (ko) | 2020-03-19 | 2021-10-29 | 한국과학기술연구원 | 광소자용 역나노콘과 그 제조방법 |
-
2018
- 2018-12-21 CA CA3086485A patent/CA3086485A1/en active Pending
- 2018-12-21 AU AU2018392782A patent/AU2018392782B2/en active Active
- 2018-12-21 US US16/956,271 patent/US11597932B2/en active Active
- 2018-12-21 WO PCT/US2018/067103 patent/WO2019126651A1/en not_active Ceased
- 2018-12-21 EP EP18836775.9A patent/EP3728281A1/en active Pending
- 2018-12-21 JP JP2020534493A patent/JP7348185B2/ja active Active
-
2022
- 2022-11-08 US US17/982,819 patent/US20230122244A1/en active Pending
-
2023
- 2023-09-07 JP JP2023145038A patent/JP2023171757A/ja active Pending
- 2023-11-30 AU AU2023274165A patent/AU2023274165B2/en active Active
-
2025
- 2025-12-15 AU AU2025279812A patent/AU2025279812A1/en active Pending
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