JP2018529732A5 - - Google Patents
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- JP2018529732A5 JP2018529732A5 JP2018516738A JP2018516738A JP2018529732A5 JP 2018529732 A5 JP2018529732 A5 JP 2018529732A5 JP 2018516738 A JP2018516738 A JP 2018516738A JP 2018516738 A JP2018516738 A JP 2018516738A JP 2018529732 A5 JP2018529732 A5 JP 2018529732A5
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- JP
- Japan
- Prior art keywords
- seq
- sequence
- strand comprises
- rnai agent
- sense strand
- Prior art date
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- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims description 80
- 230000009368 gene silencing by RNA Effects 0.000 claims description 80
- 239000003795 chemical substances by application Substances 0.000 claims description 76
- 125000003729 nucleotide group Chemical group 0.000 claims description 72
- 230000000692 anti-sense effect Effects 0.000 claims description 61
- 108091081021 Sense strand Proteins 0.000 claims description 57
- 239000002773 nucleotide Substances 0.000 claims description 55
- BXJSAMKIFDDLGI-UHFFFAOYSA-N 2-(dimethylamino)-N-[2-[3-[[5-[3-(dimethylcarbamoyl)phenyl]-2-methoxyphenyl]sulfonylamino]anilino]ethyl]benzamide Chemical compound COc1ccc(cc1S(=O)(=O)Nc1cccc(NCCNC(=O)c2ccccc2N(C)C)c1)-c1cccc(c1)C(=O)N(C)C BXJSAMKIFDDLGI-UHFFFAOYSA-N 0.000 claims description 4
- 102000005427 Asialoglycoprotein Receptor Human genes 0.000 claims description 4
- 108010006523 asialoglycoprotein receptor Proteins 0.000 claims description 4
- 239000003446 ligand Substances 0.000 claims description 4
- 229930182830 galactose Natural products 0.000 claims description 3
- 230000014509 gene expression Effects 0.000 claims description 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 3
- 208000035657 Abasia Diseases 0.000 claims description 2
- 101100239890 Candida albicans (strain SC5314 / ATCC MYA-2876) NAG4 gene Proteins 0.000 claims description 2
- 101001024442 Cellulomonas fimi Beta-N-acetylglucosaminidase/beta-glucosidase Proteins 0.000 claims description 2
- 230000002401 inhibitory effect Effects 0.000 claims description 2
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- 238000011282 treatment Methods 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 208000024172 Cardiovascular disease Diseases 0.000 claims 3
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound 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 2
- 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 2
- 101150091521 lpa gene Proteins 0.000 claims 2
- 210000002966 serum Anatomy 0.000 claims 2
- MSWZFWKMSRAUBD-GASJEMHNSA-N 2-amino-2-deoxy-D-galactopyranose Chemical compound N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O MSWZFWKMSRAUBD-GASJEMHNSA-N 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- WQZGKKKJIJFFOK-PHYPRBDBSA-N alpha-D-galactose Chemical compound OC[C@H]1O[C@H](O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-PHYPRBDBSA-N 0.000 claims 1
- MSWZFWKMSRAUBD-UHFFFAOYSA-N beta-D-galactosamine Natural products NC1C(O)OC(CO)C(O)C1O MSWZFWKMSRAUBD-UHFFFAOYSA-N 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 150000002256 galaktoses Chemical class 0.000 claims 1
- 230000005764 inhibitory process Effects 0.000 claims 1
- 125000001921 locked nucleotide group Chemical group 0.000 claims 1
- RHLMXWCISNJNDH-UHFFFAOYSA-N n-[2-[3-[[5-[3-(dimethylcarbamoyl)phenyl]-2-methoxyphenyl]sulfonylamino]anilino]ethyl]-3-methylbenzamide Chemical compound COC1=CC=C(C=2C=C(C=CC=2)C(=O)N(C)C)C=C1S(=O)(=O)NC(C=1)=CC=CC=1NCCNC(=O)C1=CC=CC(C)=C1 RHLMXWCISNJNDH-UHFFFAOYSA-N 0.000 claims 1
- 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 description 4
- 238000000034 method Methods 0.000 description 3
- 210000004027 cell Anatomy 0.000 description 2
- 239000003814 drug Substances 0.000 description 2
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 125000005647 linker group Chemical group 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 229940071643 prefilled syringe Drugs 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562235816P | 2015-10-01 | 2015-10-01 | |
| US62/235,816 | 2015-10-01 | ||
| US201662346304P | 2016-06-06 | 2016-06-06 | |
| US62/346,304 | 2016-06-06 | ||
| US201662383221P | 2016-09-02 | 2016-09-02 | |
| US62/383,221 | 2016-09-02 | ||
| PCT/US2016/054729 WO2017059223A2 (en) | 2015-10-01 | 2016-09-30 | Compositions and methods for inhibiting gene expression of lpa |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021039949A Division JP7116212B2 (ja) | 2015-10-01 | 2021-03-12 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018529732A JP2018529732A (ja) | 2018-10-11 |
| JP2018529732A5 true JP2018529732A5 (enExample) | 2019-11-07 |
| JP6991966B2 JP6991966B2 (ja) | 2022-02-03 |
Family
ID=58424351
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018516738A Active JP6991966B2 (ja) | 2015-10-01 | 2016-09-30 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
| JP2021039949A Active JP7116212B2 (ja) | 2015-10-01 | 2021-03-12 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
| JP2022096415A Active JP7442574B2 (ja) | 2015-10-01 | 2022-06-15 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
| JP2023199857A Withdrawn JP2024009262A (ja) | 2015-10-01 | 2023-11-27 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021039949A Active JP7116212B2 (ja) | 2015-10-01 | 2021-03-12 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
| JP2022096415A Active JP7442574B2 (ja) | 2015-10-01 | 2022-06-15 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
| JP2023199857A Withdrawn JP2024009262A (ja) | 2015-10-01 | 2023-11-27 | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 |
Country Status (37)
Families Citing this family (47)
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| US7718133B2 (en) | 2003-10-09 | 2010-05-18 | 3M Innovative Properties Company | Multilayer processing devices and methods |
| US10246709B2 (en) * | 2016-03-07 | 2019-04-02 | Arrowhead Pharmaceuticals, Inc. | Targeting ligands for therapeutic compounds |
| CA3011668A1 (en) | 2016-09-02 | 2018-03-08 | Arrowhead Pharmaceuticals, Inc. | Targeting ligands |
| ES2932831T3 (es) * | 2017-04-05 | 2023-01-26 | Silence Therapeutics Gmbh | Productos y composiciones |
| JP2020526192A (ja) * | 2017-07-06 | 2020-08-31 | アローヘッド ファーマシューティカルズ インコーポレイテッド | アルファ−ＥＮａＣの発現を阻害するためのＲＮＡｉ剤、および使用方法 |
| WO2019051402A1 (en) * | 2017-09-11 | 2019-03-14 | Arrowhead Pharmaceuticals, Inc. | RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3) |
| EP3681513A4 (en) * | 2017-09-14 | 2021-09-22 | Arrowhead Pharmaceuticals, Inc. | RNAI AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF ANGIOPOIETIN-LIKE 3 (ANGPTL3) AND METHOD OF USE |
| KR102822647B1 (ko) | 2017-10-04 | 2025-06-19 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩티드 조성물 및 그의 용도 |
| JP7473472B2 (ja) * | 2017-10-17 | 2024-04-23 | アローヘッド ファーマシューティカルズ インコーポレイテッド | アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物 |
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| PH12021550801B1 (en) * | 2018-11-13 | 2024-04-26 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of lpa in a cell |
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| JP2023504744A (ja) * | 2019-12-09 | 2023-02-06 | アムジエン・インコーポレーテツド | ＬＰＡ発現を阻害するためのＲＮＡｉコンストラクト及び方法 |
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| CN117836307A (zh) | 2021-06-18 | 2024-04-05 | 弘景生物科技有限公司 | 功能化的n-乙酰基半乳糖胺核苷 |
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| US20250283076A1 (en) | 2021-09-14 | 2025-09-11 | Argonaute RNA Limited | Treatment of cardiovascular disease |
| EP4403633A1 (en) * | 2021-09-18 | 2024-07-24 | Genoval Therapeutics Co., Ltd. | Lpa inhibitor and use thereof |
| EP4448540A1 (en) | 2021-12-15 | 2024-10-23 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| JP2024545222A (ja) * | 2021-12-16 | 2024-12-05 | 上海拓界生物医薬科技有限公司 | ＬＰＡを標的とするｓｉＲＮＡ及び複合体 |
| CN114703184B (zh) * | 2022-03-11 | 2024-06-18 | 厦门甘宝利生物医药有限公司 | Lpa抑制剂及其用途 |
| CN119698469A (zh) * | 2022-08-11 | 2025-03-25 | 益杰立科(上海)生物科技有限公司 | 一种表观编辑靶点的方法及用途 |
| CN115851723B (zh) * | 2022-10-24 | 2023-10-03 | 厦门甘宝利生物医药有限公司 | 一种抑制lpa基因表达的rna抑制剂及其应用 |
| US12116382B2 (en) | 2022-11-28 | 2024-10-15 | Hongene Biotech Corporation | Functionalized N-acetylgalactosamine analogs |
| KR20250130686A (ko) * | 2023-01-13 | 2025-09-02 | 일라이 릴리 앤드 캄파니 | 치료 올리고뉴클레오티드-함유 제약 조성물 및 그를 사용한 투여 요법 |
| CN119384503A (zh) * | 2023-04-03 | 2025-01-28 | 翰森（上海）健康科技有限公司 | 靶向LPA基因的RNAi剂及其用途 |
| WO2024222686A1 (zh) * | 2023-04-24 | 2024-10-31 | 上海京新生物医药有限公司 | 用于抑制LPA表达的RNAi剂及其应用 |
| CN119343449A (zh) * | 2023-05-24 | 2025-01-21 | 云合智药（苏州）生物科技有限公司 | 用于抑制angptl3表达的rnai剂及其应用 |
| WO2024245410A1 (zh) * | 2023-05-31 | 2024-12-05 | 上海拓界生物医药科技有限公司 | 一种靶向LPA的dsRNA及其应用 |
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| DE102023115075A1 (de) * | 2023-06-07 | 2024-12-12 | B.Braun Avitum Ag | Schlauchrollenpumpe |
| TW202530406A (zh) | 2023-09-21 | 2025-08-01 | 美商Ionis製藥公司 | 用於抑制ｌｐａ的化合物及方法 |
| WO2025082058A1 (zh) * | 2023-10-16 | 2025-04-24 | 润佳(上海)医药技术有限公司 | 一种抑制LPA基因表达的dsRNA及其用途 |
| WO2025087442A1 (zh) * | 2023-10-27 | 2025-05-01 | 北京安龙生物医药有限公司 | 靶向脂蛋白a的寡核苷酸及其用途 |
| WO2025103391A1 (zh) * | 2023-11-15 | 2025-05-22 | 北京加科瑞康医药科技有限公司 | 用于抑制lpa的基因表达的sirna，含其的组合物及其用途 |
| CN120020252A (zh) * | 2023-11-17 | 2025-05-20 | 纳肽得(青岛)生物医药有限公司 | 抑制LPA表达的siRNA及其用途 |
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2015
- 2015-10-01 JO JOP/2021/0043A patent/JOP20210043A1/ar unknown
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2016
- 2016-09-28 JO JOP/2016/0211A patent/JOP20160211B1/ar active
- 2016-09-30 MX MX2018003833A patent/MX2018003833A/es unknown
- 2016-09-30 EP EP21191363.7A patent/EP4029941A1/en active Pending
- 2016-09-30 KR KR1020247036942A patent/KR20240162596A/ko active Pending
- 2016-09-30 MY MYPI2018000440A patent/MY195796A/en unknown
- 2016-09-30 AU AU2016331084A patent/AU2016331084B2/en active Active
- 2016-09-30 EA EA201890864A patent/EA038478B1/ru unknown
- 2016-09-30 US US15/281,309 patent/US9932586B2/en active Active
- 2016-09-30 TW TW111141660A patent/TWI836693B/zh active
- 2016-09-30 CR CR20180231A patent/CR20180231A/es unknown
- 2016-09-30 LT LTEPPCT/US2016/054729T patent/LT3356529T/lt unknown
- 2016-09-30 TW TW113106862A patent/TWI880645B/zh active
- 2016-09-30 CN CN201680057095.8A patent/CN108368506A/zh active Pending
- 2016-09-30 CA CA3000397A patent/CA3000397A1/en active Pending
- 2016-09-30 UY UY0001036926A patent/UY36926A/es active IP Right Grant
- 2016-09-30 HU HUE16852695A patent/HUE055942T2/hu unknown
- 2016-09-30 SG SG10202008530TA patent/SG10202008530TA/en unknown
- 2016-09-30 ES ES16852695T patent/ES2896298T3/es active Active
- 2016-09-30 JP JP2018516738A patent/JP6991966B2/ja active Active
- 2016-09-30 RS RS20211351A patent/RS62523B1/sr unknown
- 2016-09-30 SI SI201631374T patent/SI3356529T1/sl unknown
- 2016-09-30 TN TNP/2018/000094A patent/TN2018000094A1/en unknown
- 2016-09-30 EP EP16852695.2A patent/EP3356529B1/en active Active
- 2016-09-30 HR HRP20211410TT patent/HRP20211410T1/hr unknown
- 2016-09-30 SM SM20210622T patent/SMT202100622T1/it unknown
- 2016-09-30 BR BR112018006489-0A patent/BR112018006489A2/pt active Search and Examination
- 2016-09-30 PL PL16852695T patent/PL3356529T3/pl unknown
- 2016-09-30 TW TW105131823A patent/TWI784934B/zh active
- 2016-09-30 KR KR1020187010095A patent/KR102728481B1/ko active Active
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- 2016-09-30 WO PCT/US2016/054729 patent/WO2017059223A2/en not_active Ceased
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2017
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2018
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2020
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