JP2022173554A5 - - Google Patents
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- Publication number
- 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
Links
- 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 | ||
| GBGB1707203.4A GB201707203D0 (en) | 2017-05-05 | 2017-05-05 | Modified positions |
| GB1707203.4 | 2017-05-05 | ||
| GBGB1708397.3A GB201708397D0 (en) | 2017-05-25 | 2017-05-25 | Modified positions |
| GB1708397.3 | 2017-05-25 | ||
| EP17201352 | 2017-11-13 | ||
| EP17201352.6 | 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 (3) | EP4219713A3 (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) | IL268828B2 (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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| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| EP3483269A1 (en) * | 2017-11-13 | 2019-05-15 | Silence Therapeutics GmbH | Products and compositions |
| US11015198B2 (en) * | 2017-04-05 | 2021-05-25 | Silence Therapeutics Gmbh | Products and compositions |
| EP4219714A3 (en) * | 2017-04-05 | 2023-08-30 | Silence Therapeutics GmbH | Rna interference mediated inhibition of tmprss6 |
| 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 |
| WO2018237245A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS |
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| CN119120476A (zh) | 2017-11-13 | 2024-12-13 | 赛伦斯治疗有限责任公司 | 用于抑制细胞中lpa的表达的核酸 |
| HRP20250277T1 (hr) | 2017-12-01 | 2025-05-09 | Suzhou Ribo Life Science Co., Ltd. | Dvolančani oligonukleotid, sastav i konjugat koji sadrži dvolančani oligonukleotid, postupak njihove pripreme i upotreba |
| EP3710588A4 (en) | 2018-01-16 | 2021-08-18 | Dicerna Pharmaceuticals, Inc. | COMPOSITIONS AND METHODS TO INHIBIT ALDH2 EXPRESSION |
| EP3598995A1 (en) * | 2018-07-26 | 2020-01-29 | Silence Therapeutics GmbH | Products and compositions |
| BR112021002440A2 (pt) | 2018-08-10 | 2021-05-04 | University Of Massachusetts | oligonucleotídeos modificados visando snps |
| WO2020041769A1 (en) * | 2018-08-23 | 2020-02-27 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| AU2019380628B2 (en) | 2018-11-13 | 2024-10-31 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of LPA in a cell |
| CA3126933A1 (en) * | 2019-01-16 | 2020-07-23 | Genzyme Corporation | Serpinc1 irna compositions and methods of use thereof |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| AU2020252560A1 (en) * | 2019-04-04 | 2021-10-28 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting gene expression in the central nervous system |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| US20210115442A1 (en) * | 2019-08-23 | 2021-04-22 | 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 |
| CN114616339A (zh) * | 2019-10-24 | 2022-06-10 | 盖纳万科学有限公司 | 用于治疗肢端肥大症的缀合物和方法 |
| JP2023515664A (ja) * | 2020-03-06 | 2023-04-13 | アリゴス セラピューティクス，インコーポレーテッド | 修飾された短い干渉核酸（ｓｉＮＡ）分子およびそれらの使用 |
| CA3174079A1 (en) | 2020-05-26 | 2021-12-02 | Anastasia Khvorova | Synthetic oligonucleotides having regions of block and cluster modifications |
| WO2022060871A1 (en) | 2020-09-15 | 2022-03-24 | Verve Therapeutics, Inc. | Lipid formulations for gene editing |
| CA3197198A1 (en) | 2020-11-04 | 2022-05-12 | Ute SCHAEPER | Nucleic acids for inhibiting expression of pros1 in a cell |
| CN116761616A (zh) * | 2021-01-22 | 2023-09-15 | 阿耐拉姆制药公司 | 经修饰的双链寡核苷酸 |
| WO2022184852A1 (en) * | 2021-03-03 | 2022-09-09 | Silence Therapeutics Gmbh | Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell |
| EP4313075A4 (en) * | 2021-03-24 | 2025-06-18 | Atalanta Therapeutics, Inc. | DOUBLE-STRANDED SIRNA WITH PATTERNED CHEMICAL MODIFICATIONS |
| IL309334A (en) | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| KR20240103025A9 (ko) | 2021-11-16 | 2025-12-10 | 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. | 안지오텐시노겐(agt) 단백질의 발현을 저해하는 조성물 및 방법 |
| IL315546A (en) | 2022-03-16 | 2024-11-01 | Empirico Inc | GALNAC compositions to improve siRNA bioavailability |
| EP4499834A2 (en) | 2022-03-28 | 2025-02-05 | Empirico Inc. | Modified oligonucleotides |
| TW202430637A (zh) | 2022-11-16 | 2024-08-01 | 美商戴瑟納製藥股份有限公司 | Stat3靶向性寡核苷酸及其用途 |
| 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 |
| WO2024245151A1 (zh) * | 2023-05-26 | 2024-12-05 | 维亚臻生物技术（苏州）有限公司 | 一种用于代谢疾病的双链核苷酸化合物及其应用 |
| TW202515586A (zh) * | 2023-08-09 | 2025-04-16 | 大陸商北京福元醫藥股份有限公司 | 抑制DGAT2基因表達的siRNA、siRNA綴合物或其前藥和藥物組合物及用途 |
| WO2025196505A2 (en) | 2024-03-22 | 2025-09-25 | 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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-
2018
- 2018-04-05 US US16/497,000 patent/US11015198B2/en active Active
- 2018-04-05 DK DK18715706.0T patent/DK3607069T3/da active
- 2018-04-05 RS RS20221162A patent/RS63836B1/sr unknown
- 2018-04-05 ES ES18715706T patent/ES2932831T3/es active Active
- 2018-04-05 SI SI201830836T patent/SI3607069T1/sl unknown
- 2018-04-05 JP JP2020504436A patent/JP7155239B2/ja active Active
- 2018-04-05 HU HUE18715706A patent/HUE061247T2/hu unknown
- 2018-04-05 IL IL268828A patent/IL268828B2/en unknown
- 2018-04-05 PL PL18715706.0T patent/PL3607069T3/pl unknown
- 2018-04-05 IL IL321221A patent/IL321221A/en unknown
- 2018-04-05 EP EP22202294.9A patent/EP4219713A3/en active Pending
- 2018-04-05 EP EP18715706.0A patent/EP3607069B1/en active Active
- 2018-04-05 CN CN201880021538.7A patent/CN110520531A/zh active Pending
- 2018-04-05 FI FIEP18715706.0T patent/FI3607069T3/fi active
- 2018-04-05 WO PCT/EP2018/058766 patent/WO2018185241A1/en not_active Ceased
- 2018-04-05 AU AU2018247925A patent/AU2018247925B2/en active Active
- 2018-04-05 EP EP26171795.3A patent/EP4748382A3/en active Pending
- 2018-04-05 HR HRP20221400TT patent/HRP20221400T1/hr unknown
- 2018-04-05 LT LTEPPCT/EP2018/058766T patent/LT3607069T/lt unknown
-
2021
- 2021-03-31 US US17/219,265 patent/US12084656B2/en active Active
-
2022
- 2022-10-04 JP JP2022160357A patent/JP7583005B2/ja active Active
-
2024
- 2024-07-26 JP JP2024121088A patent/JP7823127B2/ja active Active
- 2024-08-07 US US18/797,143 patent/US20250043288A1/en active Pending
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