JP2017525705A5 - - Google Patents
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- JP2017525705A5 JP2017525705A5 JP2017509743A JP2017509743A JP2017525705A5 JP 2017525705 A5 JP2017525705 A5 JP 2017525705A5 JP 2017509743 A JP2017509743 A JP 2017509743A JP 2017509743 A JP2017509743 A JP 2017509743A JP 2017525705 A5 JP2017525705 A5 JP 2017525705A5
- Authority
- JP
- Japan
- Prior art keywords
- dsrna agent
- nucleotides
- agent according
- administration
- dsrna
- 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.)
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- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 31
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims 30
- 239000003795 chemical substances by application Substances 0.000 claims 28
- 239000002773 nucleotide Substances 0.000 claims 18
- 125000003729 nucleotide group Chemical group 0.000 claims 18
- 230000000692 anti-sense effect Effects 0.000 claims 12
- 108091081021 Sense strand Proteins 0.000 claims 9
- 238000000034 method Methods 0.000 claims 7
- 238000012986 modification Methods 0.000 claims 5
- 230000004048 modification Effects 0.000 claims 5
- 101150075175 Asgr1 gene Proteins 0.000 claims 4
- 239000003446 ligand Substances 0.000 claims 4
- 102000040430 polynucleotide Human genes 0.000 claims 3
- 108091033319 polynucleotide Proteins 0.000 claims 3
- 239000002157 polynucleotide Substances 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 238000007920 subcutaneous administration Methods 0.000 claims 3
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 2
- IQFYYKKMVGJFEH-BIIVOSGPSA-N 2'-deoxythymidine Natural products O=C1NC(=O)C(C)=CN1[C@@H]1O[C@@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-BIIVOSGPSA-N 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 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 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 125000002015 acyclic group Chemical group 0.000 claims 1
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 claims 1
- 230000000368 destabilizing effect Effects 0.000 claims 1
- NAGJZTKCGNOGPW-UHFFFAOYSA-K dioxido-sulfanylidene-sulfido-$l^{5}-phosphane Chemical compound [O-]P([O-])([S-])=S NAGJZTKCGNOGPW-UHFFFAOYSA-K 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000001475 halogen functional group Chemical group 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 238000001361 intraarterial administration Methods 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 230000001926 lymphatic effect Effects 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-N methylphosphonic acid Chemical compound CP(O)(O)=O YACKEPLHDIMKIO-UHFFFAOYSA-N 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Priority Applications (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020212527A JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2020212528A JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2023178574A JP7654744B2 (ja) | 2014-08-20 | 2023-10-17 | 修飾二本鎖ｒｎａ剤 |
| JP2025044214A JP2025111426A (ja) | 2014-08-20 | 2025-03-19 | 修飾二本鎖ｒｎａ剤 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462039507P | 2014-08-20 | 2014-08-20 | |
| US62/039,507 | 2014-08-20 | ||
| US201462083744P | 2014-11-24 | 2014-11-24 | |
| US62/083,744 | 2014-11-24 | ||
| US201462093919P | 2014-12-18 | 2014-12-18 | |
| US62/093,919 | 2014-12-18 | ||
| PCT/US2015/045407 WO2016028649A1 (en) | 2014-08-20 | 2015-08-14 | Modified double-stranded rna agents |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020212527A Division JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2020212528A Division JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017525705A JP2017525705A (ja) | 2017-09-07 |
| JP2017525705A5 true JP2017525705A5 (enExample) | 2018-09-20 |
| JP7289610B2 JP7289610B2 (ja) | 2023-06-12 |
Family
ID=53969461
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017509743A Active JP7289610B2 (ja) | 2014-08-20 | 2015-08-14 | 修飾二本鎖ｒｎａ剤 |
| JP2020212527A Active JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2020212528A Active JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2023178574A Active JP7654744B2 (ja) | 2014-08-20 | 2023-10-17 | 修飾二本鎖ｒｎａ剤 |
| JP2025044214A Pending JP2025111426A (ja) | 2014-08-20 | 2025-03-19 | 修飾二本鎖ｒｎａ剤 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020212527A Active JP7370311B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2020212528A Active JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2023178574A Active JP7654744B2 (ja) | 2014-08-20 | 2023-10-17 | 修飾二本鎖ｒｎａ剤 |
| JP2025044214A Pending JP2025111426A (ja) | 2014-08-20 | 2025-03-19 | 修飾二本鎖ｒｎａ剤 |
Country Status (12)
| Country | Link |
|---|---|
| US (11) | US10233448B2 (enExample) |
| EP (4) | EP4585686A3 (enExample) |
| JP (5) | JP7289610B2 (enExample) |
| KR (3) | KR20240010762A (enExample) |
| CN (3) | CN118652888A (enExample) |
| CA (1) | CA2958758A1 (enExample) |
| EA (1) | EA201790420A1 (enExample) |
| IL (3) | IL316808A (enExample) |
| MX (3) | MX2017002144A (enExample) |
| NZ (1) | NZ730296A (enExample) |
| SG (3) | SG11201701166UA (enExample) |
| WO (1) | WO2016028649A1 (enExample) |
Families Citing this family (143)
| Publication number | Priority date | Publication date | Assignee | Title |
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| MX344354B (es) | 2008-10-20 | 2016-12-14 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion de transtiretina. |
| EP3656860B1 (en) | 2011-06-21 | 2022-04-20 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| KR102853694B1 (ko) | 2011-11-18 | 2025-09-03 | 알닐람 파마슈티칼스 인코포레이티드 | 변형된 ＲＮＡｉ 제제 |
| US9982257B2 (en) | 2012-07-13 | 2018-05-29 | Wave Life Sciences Ltd. | Chiral control |
| PT2999785T (pt) | 2013-05-22 | 2018-07-09 | Alnylam Pharmaceuticals Inc | Composições de irna de serpina1 e métodos de uso das mesmas |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
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| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| HK1258758A1 (zh) | 2016-01-31 | 2019-11-22 | University Of Massachusetts | 分支的寡核苷酸 |
| RU2018127537A (ru) * | 2016-02-01 | 2020-03-03 | Арракис Терапьютикс, Инк. | Соединения и способы лечения phk-опосредованных заболеваний |
| MA45349A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques egfr et leurs utilisations |
| MA45470A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques kras et leurs utilisations |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| MA45469A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Acides nucléiques de bêta-caténine et leurs utilisations |
| TWI815794B (zh) | 2016-06-06 | 2023-09-21 | 美商愛羅海德製藥公司 | 5’-環-磷酸修飾之核苷酸 |
| EP3496758A4 (en) | 2016-08-12 | 2020-11-11 | University of Massachusetts | CONJUGATE OLIGONUCLEOTIDES |
| 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 |
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| US11447519B2 (en) | 2016-11-10 | 2022-09-20 | San Diego State University Research Foundation | Compounds for fluorescence sensing of duplex formation |
| KR20240035907A (ko) * | 2016-11-23 | 2024-03-18 | 알닐람 파마슈티칼스 인코포레이티드 | 표적외 효과가 감소된 변형 rna 작용제 |
| TW202313978A (zh) * | 2016-11-23 | 2023-04-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法 |
| CN110225770B (zh) | 2016-12-14 | 2022-04-26 | 杨森生物科技公司 | 结合cd8a的纤连蛋白iii型结构域 |
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| IL269576B2 (en) * | 2017-04-05 | 2024-06-01 | Silence Therapeutics Gmbh | Products and compositions |
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| CN111378657B (zh) * | 2018-12-28 | 2024-03-15 | 苏州瑞博生物技术股份有限公司 | 抑制COL1A1基因表达的siRNA、含有该siRNA的药物组合物及其用途 |
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| CN118256498A (zh) * | 2019-05-22 | 2024-06-28 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与缀合物及制备方法和用途 |
| CN111973619B (zh) * | 2019-05-23 | 2024-01-30 | 苏州瑞博生物技术股份有限公司 | 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
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