JP2019513770A5 - - Google Patents
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- JP2019513770A5 JP2019513770A5 JP2018553397A JP2018553397A JP2019513770A5 JP 2019513770 A5 JP2019513770 A5 JP 2019513770A5 JP 2018553397 A JP2018553397 A JP 2018553397A JP 2018553397 A JP2018553397 A JP 2018553397A JP 2019513770 A5 JP2019513770 A5 JP 2019513770A5
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- JP
- Japan
- Prior art keywords
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- alkyl
- absent
- compound according
- 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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- 150000001875 compounds Chemical class 0.000 claims 19
- 150000003839 salts Chemical class 0.000 claims 19
- 125000005647 linker group Chemical group 0.000 claims 12
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 8
- 125000001475 halogen functional group Chemical group 0.000 claims 7
- 125000000304 alkynyl group Chemical group 0.000 claims 6
- 229910052739 hydrogen Inorganic materials 0.000 claims 6
- 239000001257 hydrogen Substances 0.000 claims 6
- 125000006545 (C1-C9) alkyl group Chemical group 0.000 claims 4
- 125000003342 alkenyl group Chemical group 0.000 claims 4
- 125000003545 alkoxy group Chemical group 0.000 claims 4
- 150000002431 hydrogen Chemical class 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 150000007523 nucleic acids Chemical class 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 239000007787 solid Substances 0.000 claims 4
- 235000000346 sugar Nutrition 0.000 claims 4
- 150000008163 sugars Chemical class 0.000 claims 4
- 230000008685 targeting Effects 0.000 claims 4
- 125000004178 (C1-C4) alkyl group Chemical group 0.000 claims 3
- 125000004429 atom Chemical group 0.000 claims 3
- 125000000753 cycloalkyl group Chemical group 0.000 claims 3
- 239000003446 ligand Substances 0.000 claims 3
- 125000004767 (C1-C4) haloalkoxy group Chemical group 0.000 claims 2
- 125000004765 (C1-C4) haloalkyl group Chemical group 0.000 claims 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 2
- 125000003118 aryl group Chemical group 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 2
- 125000006239 protecting group Chemical group 0.000 claims 2
- 125000000229 (C1-C4)alkoxy group Chemical group 0.000 claims 1
- 125000004209 (C1-C8) alkyl group Chemical group 0.000 claims 1
- 125000006648 (C1-C8) haloalkyl group Chemical group 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000000623 heterocyclic group Chemical group 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022130754A JP7604427B2 (ja) | 2016-04-11 | 2022-08-18 | 標的核酸コンジュゲート組成物 |
| JP2024216389A JP2025031736A (ja) | 2016-04-11 | 2024-12-11 | 標的核酸コンジュゲート組成物 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662321034P | 2016-04-11 | 2016-04-11 | |
| US62/321,034 | 2016-04-11 | ||
| US201662417156P | 2016-11-03 | 2016-11-03 | |
| US62/417,156 | 2016-11-03 | ||
| US201662438310P | 2016-12-22 | 2016-12-22 | |
| US62/438,310 | 2016-12-22 | ||
| PCT/CA2017/050447 WO2017177326A1 (en) | 2016-04-11 | 2017-04-11 | Targeted nucleic acid conjugate compositions |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022130754A Division JP7604427B2 (ja) | 2016-04-11 | 2022-08-18 | 標的核酸コンジュゲート組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019513770A JP2019513770A (ja) | 2019-05-30 |
| JP2019513770A5 true JP2019513770A5 (https=) | 2020-05-21 |
Family
ID=60041316
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018553397A Pending JP2019513770A (ja) | 2016-04-11 | 2017-04-11 | 標的核酸コンジュゲート組成物 |
| JP2022130754A Active JP7604427B2 (ja) | 2016-04-11 | 2022-08-18 | 標的核酸コンジュゲート組成物 |
| JP2024216389A Pending JP2025031736A (ja) | 2016-04-11 | 2024-12-11 | 標的核酸コンジュゲート組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022130754A Active JP7604427B2 (ja) | 2016-04-11 | 2022-08-18 | 標的核酸コンジュゲート組成物 |
| JP2024216389A Pending JP2025031736A (ja) | 2016-04-11 | 2024-12-11 | 標的核酸コンジュゲート組成物 |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US11896672B2 (https=) |
| EP (1) | EP3443092A4 (https=) |
| JP (3) | JP2019513770A (https=) |
| KR (3) | KR102905848B1 (https=) |
| CN (1) | CN109312342A (https=) |
| AU (3) | AU2017251107B9 (https=) |
| CA (1) | CA3020585A1 (https=) |
| CL (1) | CL2018002892A1 (https=) |
| IL (4) | IL307915B2 (https=) |
| MA (1) | MA45478A (https=) |
| MX (2) | MX2018012460A (https=) |
| NZ (1) | NZ747138A (https=) |
| SG (2) | SG10202008301UA (https=) |
| TW (2) | TW202527991A (https=) |
| UA (1) | UA126378C2 (https=) |
| WO (1) | WO2017177326A1 (https=) |
| ZA (1) | ZA201806861B (https=) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HRP20181564T1 (hr) | 2011-06-30 | 2018-11-30 | Arrowhead Pharmaceuticals, Inc. | Pripravci i postupci za inhibiciju ekspresije gena virusa hepatitisa b |
| KR20180038465A (ko) | 2015-08-07 | 2018-04-16 | 애로우헤드 파마슈티컬스 인코포레이티드 | Ｂ형 간염 바이러스 감염에 대한 ｒｎａｉ 치료법 |
| JOP20190015A1 (ar) | 2016-08-04 | 2019-02-04 | Arrowhead Pharmaceuticals Inc | عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب |
| WO2018191278A2 (en) * | 2017-04-11 | 2018-10-18 | Arbutus Biopharma Corporation | Targeted compositions |
| TWI869058B (zh) | 2018-07-27 | 2025-01-01 | 加拿大商愛彼特生物製藥公司 | 經取代四氫環戊[c]吡咯、經取代二氫吡咯，其類似物及使用其之方法 |
| KR20210120988A (ko) * | 2018-11-02 | 2021-10-07 | 제네반트 사이언시즈 게엠베하 | 치료 방법 |
| CA3118305A1 (en) * | 2018-11-02 | 2020-05-07 | Arbutus Biopharma Corporation | Bivalent targeted conjugates |
| CA3133629A1 (en) * | 2019-03-21 | 2020-09-24 | Mitotherapeutix Llc | Multivalent ligand clusters for targeted delivery of therapeutic agents |
| JP2023505434A (ja) * | 2019-12-06 | 2023-02-09 | ジェネヴァント サイエンシズ ゲーエムベーハー | 肝線維症を治療するためのコンジュゲート及び方法 |
| CN111041025B (zh) | 2019-12-17 | 2021-06-18 | 深圳市瑞吉生物科技有限公司 | 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法 |
| AU2020408039A1 (en) * | 2019-12-20 | 2022-08-18 | Arbutus Biopharma Corporation | Synthetic processes and intermediates |
| MX2022011805A (es) | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| AU2021244559A1 (en) | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| CN111744019B (zh) | 2020-07-01 | 2023-08-04 | 深圳瑞吉生物科技有限公司 | 基于甘露糖的mRNA靶向递送系统及其应用 |
| CN115516092B (zh) * | 2020-09-30 | 2025-05-16 | 亿腾医药（苏州）有限公司 | 血管生成素样3(ANGPTL3)的siRNA及其用途 |
| CN116546989A (zh) * | 2020-10-09 | 2023-08-04 | 阿达尔克斯制药有限公司 | N-乙酰半乳糖胺（GalNAc）来源的化合物和寡核苷酸 |
| EP4225322A4 (en) * | 2020-10-09 | 2024-12-11 | Adarx Pharmaceuticals, Inc. | N-acetylgalactosamine(galnac)-derived compounds and oligonucleotides |
| IL302530A (en) * | 2020-11-06 | 2023-07-01 | Arbutus Biopharma Corp | Targeted conjugates involving modified siRNA |
| EP4329884A1 (en) | 2021-04-27 | 2024-03-06 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
| CN117881786A (zh) | 2021-04-27 | 2024-04-12 | 世代生物公司 | 表达治疗性抗体的非病毒dna载体及其用途 |
| JP2024523237A (ja) | 2021-06-18 | 2024-06-28 | ホンジーン バイオテック コーポレイション | 官能化されたｎ－アセチルガラクトサミンヌクレオシド |
| JP2024532271A (ja) | 2021-08-30 | 2024-09-05 | ホンジーン バイオテック コーポレイション | 官能化ｎ－アセチルガラクトサミンアナログ |
| KR20240082358A (ko) * | 2021-09-23 | 2024-06-10 | 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. | 치료제의 표적화된 전달을 위한 디아민 스캐폴드를 갖는 다가 리간드 클러스터 |
| CN118076621A (zh) * | 2021-10-05 | 2024-05-24 | 美国圣因生物股份有限公司 | 多羟基化的环戊烷衍生物及使用方法 |
| IL312811A (en) | 2021-11-16 | 2024-07-01 | Shanghai Argo Biopharmaceutical Co Ltd | Composition and method for inhibiting angiotensinogen (agt) protein expression |
| WO2023114746A1 (en) | 2021-12-15 | 2023-06-22 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| EP4455155A1 (en) * | 2022-01-20 | 2024-10-30 | Chengdu Xinzhenghe Pharmaceutical Technology Co. Ltd | Targeting ligand and use thereof |
| WO2023177655A1 (en) | 2022-03-14 | 2023-09-21 | Generation Bio Co. | Heterologous prime boost vaccine compositions and methods of use |
| WO2024118503A1 (en) | 2022-11-28 | 2024-06-06 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| CN118993970B (zh) * | 2023-05-17 | 2025-12-23 | 苏州富士莱医药股份有限公司 | 一种l96侧链化合物的制备方法 |
| CN116903684B (zh) * | 2023-06-08 | 2025-12-12 | 弘亮(上海)生物医药科技有限公司 | 一种肝靶向化合物和寡核苷酸缀合物及其应用 |
| GB202311334D0 (en) * | 2023-07-24 | 2023-09-06 | Astrazeneca Ab | Multivalent cargo-carrying complexes and uses thereof |
| CN116655715B (zh) * | 2023-07-27 | 2023-10-20 | 北京炫景瑞医药科技有限公司 | 一种GalNAc衍生物、缀合物、组合物以及它们的用途 |
| CN117624266A (zh) * | 2023-10-19 | 2024-03-01 | 弘亮(上海)生物医药科技有限公司 | 一种肝靶向化合物及其寡核苷酸缀合物、偶联方法与应用 |
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| WO2003070918A2 (en) | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | Rna interference by modified short interfering nucleic acid |
| US7833992B2 (en) * | 2001-05-18 | 2010-11-16 | Merck Sharpe & Dohme | Conjugates and compositions for cellular delivery |
| US9657294B2 (en) * | 2002-02-20 | 2017-05-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| US8258288B2 (en) * | 2002-02-20 | 2012-09-04 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA) |
| WO2005026165A1 (en) * | 2003-09-12 | 2005-03-24 | Warner-Lambert Company Llc | Quinolone antibacterial agents |
| EP3486653A1 (en) * | 2007-05-24 | 2019-05-22 | The United States Government as represented by The Department of Veterans Affairs | Treatment of skeletal muscle disorder using ent2 |
| WO2009082607A2 (en) * | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| EP2231194B1 (en) | 2007-12-04 | 2017-02-22 | Alnylam Pharmaceuticals Inc. | Folate-irna conjugates |
| EP2291200A4 (en) * | 2008-05-22 | 2012-05-30 | Isis Pharmaceuticals Inc | PROCESS FOR MODULATING THE EXPRESSION OF RBP4 |
| US9273283B2 (en) * | 2009-10-29 | 2016-03-01 | The Trustees Of Dartmouth College | Method of producing T cell receptor-deficient T cells expressing a chimeric receptor |
| US9101643B2 (en) * | 2009-11-03 | 2015-08-11 | Alnylam Pharmaceuticals, Inc. | Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR) |
| DK2539451T3 (da) * | 2010-02-24 | 2016-04-04 | Arrowhead Res Corp | Sammensætninger til målrettet tilførsel af siRNA |
| EP2652134B1 (en) | 2010-12-17 | 2017-03-01 | Arrowhead Pharmaceuticals, Inc. | Galactose cluster-pharmacokinetic modulator targeting moiety for sirna |
| EP3453761A1 (en) | 2011-08-29 | 2019-03-13 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| EP2780454A2 (en) * | 2011-11-18 | 2014-09-24 | Alnylam Pharmaceuticals, Inc. | Modified rnai agents |
| HUE048622T2 (hu) * | 2011-11-18 | 2020-08-28 | Alnylam Pharmaceuticals Inc | RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére |
| US20160184458A1 (en) * | 2013-03-14 | 2016-06-30 | Shire Human Genetic Therapies, Inc. | Mrna therapeutic compositions and use to treat diseases and disorders |
| MX374090B (es) * | 2013-03-14 | 2025-03-05 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico. |
| DK2991656T3 (da) * | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
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| TW201610151A (zh) * | 2013-09-23 | 2016-03-16 | 阿尼拉製藥公司 | 治療或預防與甲狀腺素運載蛋白相關疾病之方法 |
| EP3862362A3 (en) * | 2014-05-01 | 2021-10-27 | Ionis Pharmaceuticals, Inc. | Conjugates of modified antisense oligonucleotides and their use for modulating pkk expression |
| WO2015191693A2 (en) * | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
| US20170304459A1 (en) * | 2014-10-10 | 2017-10-26 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for inhalation delivery of conjugated oligonucleotide |
| WO2016055601A1 (en) | 2014-10-10 | 2016-04-14 | F. Hoffmann-La Roche Ag | Galnac phosphoramidites, nucleic acid conjugates thereof and their use |
| TWI710633B (zh) | 2014-11-10 | 2020-11-21 | 美商阿尼拉製藥公司 | Ｂ型肝炎病毒（ＨＢＶ）ｉＲＮＡ組成物及其用途方法 |
| EP3323893A4 (en) | 2015-07-16 | 2019-03-27 | Kyowa Hakko Kirin Co., Ltd. | BETA2GPI GENE EXPRESSION-INHIBITING NUCLEIC ACID COMPLEX |
| EP3228326A1 (en) * | 2016-04-05 | 2017-10-11 | Silence Therapeutics GmbH | Nucleic acid linked to a trivalent glycoconjugate |
| EP3319098A1 (en) * | 2016-11-02 | 2018-05-09 | Abiomed Europe GmbH | Intravascular blood pump comprising corrosion resistant permanent magnet |
| WO2018191278A2 (en) | 2017-04-11 | 2018-10-18 | Arbutus Biopharma Corporation | Targeted compositions |
| KR20210120988A (ko) * | 2018-11-02 | 2021-10-07 | 제네반트 사이언시즈 게엠베하 | 치료 방법 |
| EP3941921A4 (en) * | 2019-03-20 | 2023-05-03 | Arbutus Biopharma Corporation | Therapeutic methods for treating hepatitis b |
| EP4048801A4 (en) * | 2019-10-24 | 2023-11-22 | Genevant Sciences Gmbh | Conjugates and methods for treating acromegaly |
| JP2023505434A (ja) * | 2019-12-06 | 2023-02-09 | ジェネヴァント サイエンシズ ゲーエムベーハー | 肝線維症を治療するためのコンジュゲート及び方法 |
| AU2020408039A1 (en) * | 2019-12-20 | 2022-08-18 | Arbutus Biopharma Corporation | Synthetic processes and intermediates |
| IL302530A (en) * | 2020-11-06 | 2023-07-01 | Arbutus Biopharma Corp | Targeted conjugates involving modified siRNA |
| US20250215042A1 (en) * | 2022-04-01 | 2025-07-03 | Genevant Sciences Gmbh | Mannose-targeted compositions |
-
2017
- 2017-04-11 SG SG10202008301UA patent/SG10202008301UA/en unknown
- 2017-04-11 AU AU2017251107A patent/AU2017251107B9/en active Active
- 2017-04-11 CN CN201780034659.0A patent/CN109312342A/zh active Pending
- 2017-04-11 WO PCT/CA2017/050447 patent/WO2017177326A1/en not_active Ceased
- 2017-04-11 MX MX2018012460A patent/MX2018012460A/es unknown
- 2017-04-11 TW TW113135344A patent/TW202527991A/zh unknown
- 2017-04-11 SG SG11201808912QA patent/SG11201808912QA/en unknown
- 2017-04-11 UA UAA201811057A patent/UA126378C2/uk unknown
- 2017-04-11 KR KR1020237033350A patent/KR102905848B1/ko active Active
- 2017-04-11 US US16/092,685 patent/US11896672B2/en active Active
- 2017-04-11 NZ NZ747138A patent/NZ747138A/en unknown
- 2017-04-11 MA MA045478A patent/MA45478A/fr unknown
- 2017-04-11 KR KR1020257043150A patent/KR20260008191A/ko active Pending
- 2017-04-11 CA CA3020585A patent/CA3020585A1/en active Pending
- 2017-04-11 EP EP17781679.0A patent/EP3443092A4/en active Pending
- 2017-04-11 IL IL307915A patent/IL307915B2/en unknown
- 2017-04-11 TW TW106112079A patent/TWI859114B/zh active
- 2017-04-11 IL IL288461A patent/IL288461B2/en unknown
- 2017-04-11 KR KR1020187032569A patent/KR102585288B1/ko active Active
- 2017-04-11 JP JP2018553397A patent/JP2019513770A/ja active Pending
- 2017-04-11 IL IL318896A patent/IL318896A/en unknown
-
2018
- 2018-10-09 IL IL262242A patent/IL262242B/en unknown
- 2018-10-10 CL CL2018002892A patent/CL2018002892A1/es unknown
- 2018-10-11 MX MX2024011712A patent/MX2024011712A/es unknown
- 2018-10-15 ZA ZA2018/06861A patent/ZA201806861B/en unknown
-
2022
- 2022-08-18 JP JP2022130754A patent/JP7604427B2/ja active Active
-
2023
- 2023-12-01 AU AU2023274208A patent/AU2023274208A1/en not_active Abandoned
-
2024
- 2024-01-10 US US18/409,330 patent/US20240245784A1/en active Pending
- 2024-09-12 US US18/883,520 patent/US20250099594A1/en active Pending
- 2024-12-11 JP JP2024216389A patent/JP2025031736A/ja active Pending
-
2025
- 2025-12-09 AU AU2025279606A patent/AU2025279606A1/en active Pending
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