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 (enExample) | 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 (enExample) |
| EP (1) | EP3728281A1 (enExample) |
| JP (2) | JP7348185B2 (enExample) |
| AU (2) | AU2018392782B2 (enExample) |
| CA (1) | CA3086485A1 (enExample) |
| WO (1) | WO2019126651A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102712879B1 (ko) | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| KR20210018267A (ko) | 2018-05-07 | 2021-02-17 | 알닐람 파마슈티칼스 인코포레이티드 | 간외 전달 |
| US20230203484A1 (en) * | 2020-05-22 | 2023-06-29 | Wave Life Sciences Ltd. | Double stranded oligonucleotide compositions and methods relating thereto |
| WO2022011214A1 (en) | 2020-07-10 | 2022-01-13 | Alnylam Pharmaceuticals, Inc. | Circular sirnas |
| US20250270562A1 (en) | 2021-07-09 | 2025-08-28 | Alnylam Pharmaceuticals, Inc. | Bis-rnai compounds for cns delivery |
| AU2022352668A1 (en) * | 2021-09-21 | 2024-03-28 | Wave Life Sciences Ltd. | Double stranded oligonucleotide compositions and methods relating thereto |
| WO2023091644A2 (en) * | 2021-11-19 | 2023-05-25 | Wave Life Sciences Ltd. | Hsd17b13-related double stranded oligonucleotide compositions and methods relating thereto |
| 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 |
| CA3246872A1 (en) | 2022-03-28 | 2023-10-05 | Empirico Inc. | MODIFIED OLIGONUCLEOTIDES |
| EP4522742A2 (en) | 2022-05-13 | 2025-03-19 | Alnylam Pharmaceuticals, Inc. | Single-stranded loop oligonucleotides |
| WO2023235817A1 (en) * | 2022-06-02 | 2023-12-07 | 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 |
Family Cites Families (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| 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 |
| DE69126530T2 (de) | 1990-07-27 | 1998-02-05 | Isis Pharmaceutical, Inc., Carlsbad, Calif. | Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren |
| 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 |
| CA2135313A1 (en) | 1992-06-18 | 1994-01-06 | Theodore Choi | Methods for producing transgenic non-human animals harboring a yeast artificial chromosome |
| 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 |
| CA2220950A1 (en) | 1995-05-26 | 1996-11-28 | Somatix Therapy Corporation | Delivery vehicles comprising stable lipid/nucleic acid complexes |
| 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 |
| WO2005004794A2 (en) | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| CA2930393C (en) | 2007-12-04 | 2022-11-29 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| WO2012018881A2 (en) * | 2010-08-03 | 2012-02-09 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for the regulation of rna |
| WO2012037254A1 (en) | 2010-09-15 | 2012-03-22 | Alnylam Pharmaceuticals, Inc. | MODIFIED iRNA AGENTS |
| KR102712879B1 (ko) * | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| EP4137572A1 (en) | 2014-01-16 | 2023-02-22 | Wave Life Sciences Ltd. | Chiral design |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| JOP20200092A1 (ar) * | 2014-11-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| US20190256845A1 (en) * | 2016-06-10 | 2019-08-22 | Alnylam Pharmaceuticals, Inc. | COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) |
| CN110023321A (zh) | 2016-08-17 | 2019-07-16 | 索尔斯蒂斯生物有限公司 | 多核苷酸构建体 |
| 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 JP JP2020534493A patent/JP7348185B2/ja active Active
- 2018-12-21 US US16/956,271 patent/US11597932B2/en active Active
- 2018-12-21 EP EP18836775.9A patent/EP3728281A1/en active Pending
- 2018-12-21 WO PCT/US2018/067103 patent/WO2019126651A1/en not_active Ceased
-
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
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