JP2022173554A5 - - Google Patents
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- JP2022173554A5 JP2022173554A5 JP2022160357A JP2022160357A JP2022173554A5 JP 2022173554 A5 JP2022173554 A5 JP 2022173554A5 JP 2022160357 A JP2022160357 A JP 2022160357A JP 2022160357 A JP2022160357 A JP 2022160357A JP 2022173554 A5 JP2022173554 A5 JP 2022173554A5
- Authority
- JP
- Japan
- Prior art keywords
- strand
- nucleic acid
- positions
- modified
- nucleotides
- 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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- 108020004707 nucleic acids Proteins 0.000 claims 27
- 150000007523 nucleic acids Chemical class 0.000 claims 27
- 102000039446 nucleic acids Human genes 0.000 claims 27
- 125000003729 nucleotide group Chemical group 0.000 claims 25
- 239000002773 nucleotide Substances 0.000 claims 24
- 230000004048 modification Effects 0.000 claims 11
- 238000012986 modification Methods 0.000 claims 11
- 125000001153 fluoro group Chemical group F* 0.000 claims 5
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims 4
- 229910052799 carbon Inorganic materials 0.000 claims 4
- 239000003446 ligand Substances 0.000 claims 3
- 229910019142 PO4 Inorganic materials 0.000 claims 2
- 125000002947 alkylene group Chemical group 0.000 claims 2
- 150000001720 carbohydrates Chemical class 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- 150000003013 phosphoric acid derivatives Chemical class 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical group CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims 1
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims 1
- -1 alkylene ether Chemical group 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
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- RTZKZFJDLAIYFH-UHFFFAOYSA-N ether Substances CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024121088A JP7823127B2 (ja) | 2017-04-05 | 2024-07-26 | 産物及び組成物 |
Applications Claiming Priority (10)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17165129 | 2017-04-05 | ||
| EP17165129.2 | 2017-04-05 | ||
| GB1707203.4 | 2017-05-05 | ||
| GBGB1707203.4A GB201707203D0 (en) | 2017-05-05 | 2017-05-05 | Modified positions |
| GB1708397.3 | 2017-05-25 | ||
| GBGB1708397.3A GB201708397D0 (en) | 2017-05-25 | 2017-05-25 | Modified positions |
| EP17201352.6 | 2017-11-13 | ||
| EP17201352 | 2017-11-13 | ||
| JP2020504436A JP7155239B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
| PCT/EP2018/058766 WO2018185241A1 (en) | 2017-04-05 | 2018-04-05 | Products and compositions |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020504436A Division JP7155239B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024121088A Division JP7823127B2 (ja) | 2017-04-05 | 2024-07-26 | 産物及び組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022173554A JP2022173554A (ja) | 2022-11-18 |
| JP2022173554A5 true JP2022173554A5 (enExample) | 2022-12-06 |
| JP7583005B2 JP7583005B2 (ja) | 2024-11-13 |
Family
ID=61899311
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020504436A Active JP7155239B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
| JP2022160357A Active JP7583005B2 (ja) | 2017-04-05 | 2022-10-04 | 産物及び組成物 |
| JP2024121088A Active JP7823127B2 (ja) | 2017-04-05 | 2024-07-26 | 産物及び組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020504436A Active JP7155239B2 (ja) | 2017-04-05 | 2018-04-05 | 産物及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024121088A Active JP7823127B2 (ja) | 2017-04-05 | 2024-07-26 | 産物及び組成物 |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US11015198B2 (enExample) |
| EP (2) | EP3607069B1 (enExample) |
| JP (3) | JP7155239B2 (enExample) |
| CN (1) | CN110520531A (enExample) |
| AU (1) | AU2018247925B2 (enExample) |
| DK (1) | DK3607069T3 (enExample) |
| ES (1) | ES2932831T3 (enExample) |
| FI (1) | FI3607069T3 (enExample) |
| HR (1) | HRP20221400T1 (enExample) |
| HU (1) | HUE061247T2 (enExample) |
| IL (2) | IL321221A (enExample) |
| LT (1) | LT3607069T (enExample) |
| PL (1) | PL3607069T3 (enExample) |
| RS (1) | RS63836B1 (enExample) |
| SI (1) | SI3607069T1 (enExample) |
| WO (1) | WO2018185241A1 (enExample) |
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| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| DK3607069T3 (da) * | 2017-04-05 | 2022-11-21 | Silence Therapeutics Gmbh | Produkter og sammensætninger |
| EP3483269A1 (en) * | 2017-11-13 | 2019-05-15 | Silence Therapeutics GmbH | Products and compositions |
| IL269517B2 (en) | 2017-04-05 | 2025-01-01 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of a target gene in a cell, conjugates thereof, compositions containing them, and medical uses thereof |
| SI3607068T1 (sl) * | 2017-04-05 | 2023-02-28 | Silence Therapeutics Gmbh | Z RNA interferenco posredovano inhibiranje TMPRSS6 |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法 |
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| HRP20230127T1 (hr) | 2017-11-13 | 2023-03-31 | Silence Therapeutics Gmbh | Nukleinske kiseline za inhibiranje ekspresije lpa u stanici |
| DK3719128T3 (da) * | 2017-12-01 | 2025-03-03 | Suzhou Ribo Life Science Co Ltd | Dobbelt-strenget oligonukleotid, sammensætning og konjugat bestaende dobbelt-strenget oligonukleotid, fremgangsmåde herfor og anvendelse heraf |
| KR20200109311A (ko) | 2018-01-16 | 2020-09-22 | 다이서나 파마수이티컬, 인크. | Aldh2 발현을 억제하기 위한 조성물 및 방법 |
| EP3598995A1 (en) * | 2018-07-26 | 2020-01-29 | Silence Therapeutics GmbH | Products and compositions |
| US11279930B2 (en) * | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| PH12021550801B1 (en) | 2018-11-13 | 2024-04-26 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of lpa in a cell |
| MX2021008628A (es) * | 2019-01-16 | 2021-11-17 | Genzyme Corp | Composiciones de arni para serpinc1 y metodos de uso de las mismas. |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| BR112021019793A2 (pt) * | 2019-04-04 | 2021-12-07 | Dicerna Pharmaceuticals Inc | Composições e métodos para inibir expressão de gene no sistema nervoso central |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| WO2021041247A1 (en) * | 2019-08-23 | 2021-03-04 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| CA3158917A1 (en) * | 2019-10-24 | 2021-04-29 | Genevant Sciences Gmbh | Conjugates and methods for treating acromegaly |
| PE20221578A1 (es) | 2020-03-06 | 2022-10-06 | Aligos Therapeutics Inc | Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| JP2023549011A (ja) | 2020-09-15 | 2023-11-22 | ヴァーヴ・セラピューティクス，インコーポレーテッド | 遺伝子編集のための脂質製剤 |
| JP2023547512A (ja) | 2020-11-04 | 2023-11-10 | ウニベルジテート ベルン | 細胞におけるｐｒｏｓ１の発現を阻害するための核酸 |
| MX2023008469A (es) * | 2021-01-22 | 2023-11-22 | Alnylam Pharmaceuticals Inc | Oligonucleotidos de doble hebra modificados. |
| WO2022184852A1 (en) * | 2021-03-03 | 2022-09-09 | Silence Therapeutics Gmbh | Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell |
| WO2022204430A1 (en) * | 2021-03-24 | 2022-09-29 | Atalanta Therapeutics, Inc. | Double-stranded sirna having patterned chemical modifications |
| CN117677699A (zh) | 2021-06-23 | 2024-03-08 | 马萨诸塞大学 | 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物 |
| IL312811A (en) | 2021-11-16 | 2024-07-01 | Shanghai Argo Biopharmaceutical Co Ltd | Composition and method for inhibiting angiotensinogen (agt) protein expression |
| US11879125B2 (en) | 2022-03-16 | 2024-01-23 | Empirico Inc. | GalNAc compositions for improving siRNA bioavailability |
| AU2023245603A1 (en) | 2022-03-28 | 2024-11-07 | Empirico Inc. | Modified oligonucleotides |
| CN120981247A (zh) | 2023-03-27 | 2025-11-18 | 赛伦斯治疗有限责任公司 | 用于干细胞移植的化合物和组合物 |
| EP4722365A1 (en) * | 2023-05-26 | 2026-04-08 | Visirna Therapeutics (Suzhou) Co., Ltd. | Double-stranded nucleotide compound for metabolic diseases and use thereof |
| CN119464281A (zh) * | 2023-08-09 | 2025-02-18 | 北京福元医药股份有限公司 | 抑制DGAT2基因表达的siRNA、siRNA缀合物或其前药和药物组合物及用途 |
| US12553050B2 (en) | 2024-03-22 | 2026-02-17 | Takeda Pharmaceutical Company Limited | Compositions and methods for inhibiting cytochrome P450 family 7 subfamily a member 1 (CYP7A1) expression |
| CN118460651B (zh) * | 2024-07-10 | 2025-03-18 | 凯莱英医药集团（天津）股份有限公司 | 制备Cemdisiran的方法 |
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| DK3607069T3 (da) | 2017-04-05 | 2022-11-21 | Silence Therapeutics Gmbh | Produkter og sammensætninger |
| KR20240161202A (ko) | 2017-09-11 | 2024-11-12 | 애로우헤드 파마슈티컬스 인코포레이티드 | 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물 |
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-
2018
- 2018-04-05 DK DK18715706.0T patent/DK3607069T3/da active
- 2018-04-05 US US16/497,000 patent/US11015198B2/en active Active
- 2018-04-05 WO PCT/EP2018/058766 patent/WO2018185241A1/en not_active Ceased
- 2018-04-05 FI FIEP18715706.0T patent/FI3607069T3/fi active
- 2018-04-05 SI SI201830836T patent/SI3607069T1/sl unknown
- 2018-04-05 ES ES18715706T patent/ES2932831T3/es active Active
- 2018-04-05 CN CN201880021538.7A patent/CN110520531A/zh active Pending
- 2018-04-05 EP EP18715706.0A patent/EP3607069B1/en active Active
- 2018-04-05 HU HUE18715706A patent/HUE061247T2/hu unknown
- 2018-04-05 EP EP22202294.9A patent/EP4219713A3/en active Pending
- 2018-04-05 RS RS20221162A patent/RS63836B1/sr unknown
- 2018-04-05 IL IL321221A patent/IL321221A/en unknown
- 2018-04-05 JP JP2020504436A patent/JP7155239B2/ja active Active
- 2018-04-05 HR HRP20221400TT patent/HRP20221400T1/hr unknown
- 2018-04-05 LT LTEPPCT/EP2018/058766T patent/LT3607069T/lt unknown
- 2018-04-05 PL PL18715706.0T patent/PL3607069T3/pl unknown
- 2018-04-05 IL IL268828A patent/IL268828B2/en unknown
- 2018-04-05 AU AU2018247925A patent/AU2018247925B2/en active Active
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2021
- 2021-03-31 US US17/219,265 patent/US12084656B2/en active Active
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2022
- 2022-10-04 JP JP2022160357A patent/JP7583005B2/ja active Active
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2024
- 2024-07-26 JP JP2024121088A patent/JP7823127B2/ja active Active
- 2024-08-07 US US18/797,143 patent/US20250043288A1/en active Pending