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 |
|---|---|---|---|
| JP2020212528A JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2020212527A JP7370311B2 (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 |
|---|---|---|---|
| JP2020212528A Division JP7672220B2 (ja) | 2014-08-20 | 2020-12-22 | 修飾二本鎖ｒｎａ剤 |
| JP2020212527A Division JP7370311B2 (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) | EP3808846A1 (enExample) |
| JP (5) | JP7289610B2 (enExample) |
| KR (3) | KR102494171B1 (enExample) |
| CN (3) | CN107075516A (enExample) |
| CA (1) | CA2958758A1 (enExample) |
| EA (1) | EA201790420A1 (enExample) |
| IL (3) | IL316808A (enExample) |
| MX (3) | MX2017002144A (enExample) |
| NZ (1) | NZ730296A (enExample) |
| SG (3) | SG10201903290YA (enExample) |
| WO (1) | WO2016028649A1 (enExample) |
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| CN106834291B (zh) | 2008-10-20 | 2020-09-29 | 阿尔尼拉姆医药品有限公司 | 抑制运甲状腺素蛋白表达的组合物和方法 |
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| RS57418B1 (sr) | 2013-05-22 | 2018-09-28 | Alnylam Pharmaceuticals Inc | Serpina1 irnk sastavi i postupci njihove primene |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ | 十分に安定化された非対称ｓｉｒｎａ |
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