JP2021505129A5 - - Google Patents
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- Publication number
- JP2021505129A5 JP2021505129A5 JP2020529238A JP2020529238A JP2021505129A5 JP 2021505129 A5 JP2021505129 A5 JP 2021505129A5 JP 2020529238 A JP2020529238 A JP 2020529238A JP 2020529238 A JP2020529238 A JP 2020529238A JP 2021505129 A5 JP2021505129 A5 JP 2021505129A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- seq
- oligonucleotide
- nucleic acid
- group
- 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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- 239000008194 pharmaceutical composition Substances 0.000 claims 16
- 108091034117 Oligonucleotide Proteins 0.000 claims 11
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 6
- 239000000074 antisense oligonucleotide Substances 0.000 claims 6
- 238000012230 antisense oligonucleotides Methods 0.000 claims 6
- 150000007523 nucleic acids Chemical group 0.000 claims 6
- 125000003835 nucleoside group Chemical group 0.000 claims 5
- 239000002777 nucleoside Substances 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- -1 LNA nucleoside Chemical class 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 239000000969 carrier Substances 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000002904 solvent Substances 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023093643A JP7601441B2 (ja) | 2017-12-01 | 2023-06-07 | アンジェルマン症候群アンチセンス治療 |
| JP2024206905A JP7863362B2 (ja) | 2017-12-01 | 2024-11-28 | アンジェルマン症候群アンチセンス治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762593431P | 2017-12-01 | 2017-12-01 | |
| US62/593,431 | 2017-12-01 | ||
| US201862676034P | 2018-05-24 | 2018-05-24 | |
| US62/676,034 | 2018-05-24 | ||
| PCT/US2018/063416 WO2019109001A1 (en) | 2017-12-01 | 2018-11-30 | Angelman syndrome antisense treatment |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023093643A Division JP7601441B2 (ja) | 2017-12-01 | 2023-06-07 | アンジェルマン症候群アンチセンス治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021505129A JP2021505129A (ja) | 2021-02-18 |
| JP2021505129A5 true JP2021505129A5 (enExample) | 2022-01-06 |
| JP7297320B2 JP7297320B2 (ja) | 2023-06-26 |
Family
ID=66664231
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020529238A Active JP7297320B2 (ja) | 2017-12-01 | 2018-11-30 | アンジェルマン症候群アンチセンス治療 |
| JP2023093643A Active JP7601441B2 (ja) | 2017-12-01 | 2023-06-07 | アンジェルマン症候群アンチセンス治療 |
| JP2024206905A Active JP7863362B2 (ja) | 2017-12-01 | 2024-11-28 | アンジェルマン症候群アンチセンス治療 |
| JP2026075038A Active JP7897667B1 (ja) | 2017-12-01 | 2026-04-28 | アンジェルマン症候群アンチセンス治療 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023093643A Active JP7601441B2 (ja) | 2017-12-01 | 2023-06-07 | アンジェルマン症候群アンチセンス治療 |
| JP2024206905A Active JP7863362B2 (ja) | 2017-12-01 | 2024-11-28 | アンジェルマン症候群アンチセンス治療 |
| JP2026075038A Active JP7897667B1 (ja) | 2017-12-01 | 2026-04-28 | アンジェルマン症候群アンチセンス治療 |
Country Status (27)
| Country | Link |
|---|---|
| US (5) | US11198869B2 (enExample) |
| EP (2) | EP3717646B1 (enExample) |
| JP (4) | JP7297320B2 (enExample) |
| KR (2) | KR102723989B1 (enExample) |
| CN (2) | CN111433361B (enExample) |
| AU (1) | AU2018375807B2 (enExample) |
| BR (1) | BR112020009431A2 (enExample) |
| CA (1) | CA3079755A1 (enExample) |
| CL (1) | CL2020001344A1 (enExample) |
| CO (1) | CO2020006361A2 (enExample) |
| DK (1) | DK3717646T3 (enExample) |
| ES (1) | ES3032315T3 (enExample) |
| FI (1) | FI3717646T3 (enExample) |
| HR (1) | HRP20250804T1 (enExample) |
| HU (1) | HUE072645T2 (enExample) |
| IL (1) | IL274146B2 (enExample) |
| LT (1) | LT3717646T (enExample) |
| MX (2) | MX2020005680A (enExample) |
| PE (1) | PE20211238A1 (enExample) |
| PL (1) | PL3717646T3 (enExample) |
| PT (1) | PT3717646T (enExample) |
| RS (1) | RS66986B1 (enExample) |
| RU (1) | RU2020121752A (enExample) |
| SG (1) | SG11202003596WA (enExample) |
| SI (1) | SI3717646T1 (enExample) |
| SM (1) | SMT202500334T1 (enExample) |
| WO (1) | WO2019109001A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HK1258434A1 (zh) | 2015-11-12 | 2019-11-08 | F. Hoffmann-La Roche Ag | 来自灵长类物种的标准化神经元细胞测定试验 |
| EP3758714A4 (en) * | 2018-02-27 | 2021-12-01 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME |
| TW202536179A (zh) | 2019-03-29 | 2025-09-16 | 美商Ionis製藥公司 | 用於調節ube3a-ats之化合物及方法 |
| TW202216996A (zh) * | 2020-06-29 | 2022-05-01 | 美商Ionis製藥公司 | 調節ｐｌｐ１之化合物及方法 |
| CA3197664A1 (en) * | 2020-10-06 | 2022-04-14 | University Of Maryland, Baltimore | Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides |
| CN119384504A (zh) * | 2022-03-07 | 2025-01-28 | 康涅狄格大学 | 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA |
| KR20250107262A (ko) | 2023-01-31 | 2025-07-11 | 후지필름 가부시키가이샤 | 경화성 조성물, 경화물, 광학 재료, 회절 광학 소자 및 화합물 |
| WO2025100980A1 (ko) * | 2023-11-08 | 2025-05-15 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2025217466A1 (en) * | 2024-04-11 | 2025-10-16 | Ultragenyx Pharmaceutical Inc. | Dosing regimens for antisense treatment of angelman syndrome |
| CN121992041A (zh) * | 2024-11-06 | 2026-05-08 | 中国科学院脑科学与智能技术卓越创新中心 | 靶向ube3a等位基因建立天使综合征动物模型的方法和应用 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2823959B2 (ja) | 1991-10-24 | 1998-11-11 | アイシス・ファーマシューティカルス・インコーポレーテッド | 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド |
| CN100523215C (zh) * | 2000-12-01 | 2009-08-05 | 马普科技促进协会 | 介导rna干涉的小rna分子 |
| EP1799859B1 (en) * | 2004-09-17 | 2014-07-02 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| EP1676834A1 (en) | 2004-12-30 | 2006-07-05 | Sanofi-Aventis Deutschland GmbH | Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| US8178503B2 (en) * | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| US20080299659A1 (en) * | 2007-03-02 | 2008-12-04 | Nastech Pharmaceutical Company Inc. | Nucleic acid compounds for inhibiting apob gene expression and uses thereof |
| US8580756B2 (en) | 2007-03-22 | 2013-11-12 | Santaris Pharma A/S | Short oligomer antagonist compounds for the modulation of target mRNA |
| US9290534B2 (en) | 2008-04-04 | 2016-03-22 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside |
| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| RU2013150331A (ru) | 2011-04-20 | 2015-05-27 | Рош Гликарт Аг | СПОСОБ И УСТРОЙСТВА ДЛЯ рН-ЗАВИСИМОГО ПРОХОЖДЕНИЯ ГЕМАТОЭНЦЕФАЛИЧЕСКОГО БАРЬЕРА |
| US10023861B2 (en) | 2011-08-29 | 2018-07-17 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| CN104540946A (zh) * | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节utrn表达的组合物和方法 |
| WO2013173637A1 (en) * | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| ES2809199T3 (es) * | 2012-06-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Modulación de la expresión de UBE3A-ATS |
| DK2872147T3 (da) | 2012-07-13 | 2023-02-20 | Wave Life Sciences Ltd | Fremgangsmåde til fremstilling af kirale oligonukleotider |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| EP3221329A1 (en) | 2014-11-19 | 2017-09-27 | Roche Innovation Center Copenhagen A/S | Lna gapmer oligonucleotides comprising chiral phosphorothioate linkages |
| WO2016086104A1 (en) | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| DK3234131T3 (da) | 2014-12-16 | 2020-06-29 | Roche Innovation Ct Copenhagen As | Fremgangsmåde til screening af kiral toksicitet |
| CA2975525A1 (en) * | 2015-02-04 | 2016-08-11 | F. Hoffmann-La Roche Ag | Tau antisense oligomers and uses thereof |
| EP3286318A2 (en) * | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| HK1258434A1 (zh) | 2015-11-12 | 2019-11-08 | F. Hoffmann-La Roche Ag | 来自灵长类物种的标准化神经元细胞测定试验 |
| WO2017194498A1 (en) | 2016-05-12 | 2017-11-16 | Roche Innovation Center Copenhagen A/S | Enhanced coupling of stereodefined oxazaphospholidine phosphoramidite monomers to nucleoside or oligonucleotide |
| JP6636673B2 (ja) | 2016-10-04 | 2020-01-29 | メルク・シャープ・アンド・ドーム・コーポレーションＭｅｒｃｋ Ｓｈａｒｐ ＆ Ｄｏｈｍｅ Ｃｏｒｐ． | ＳＴＩＮＧアゴニストとしてのベンゾ［ｂ］チオフェン化合物 |
| KR20190135494A (ko) | 2017-03-29 | 2019-12-06 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | 입체한정된 포스포로티오에이트 올리고뉴클레오티드의 제조를 위한 직교 보호기 |
| JP2020524718A (ja) | 2017-06-22 | 2020-08-20 | キュラデブ・ファーマ・リミテッドＣｕｒａｄｅｖ Ｐｈａｒｍａ Ｌｉｍｉｔｅｄ | ヒトｓｔｉｎｇの複素環式小分子調節因子 |
-
2018
- 2018-11-30 PL PL18883102.8T patent/PL3717646T3/pl unknown
- 2018-11-30 CA CA3079755A patent/CA3079755A1/en active Pending
- 2018-11-30 KR KR1020207016107A patent/KR102723989B1/ko active Active
- 2018-11-30 SG SG11202003596WA patent/SG11202003596WA/en unknown
- 2018-11-30 US US16/767,916 patent/US11198869B2/en active Active
- 2018-11-30 SM SM20250334T patent/SMT202500334T1/it unknown
- 2018-11-30 PT PT188831028T patent/PT3717646T/pt unknown
- 2018-11-30 CN CN201880077892.1A patent/CN111433361B/zh active Active
- 2018-11-30 IL IL274146A patent/IL274146B2/en unknown
- 2018-11-30 FI FIEP18883102.8T patent/FI3717646T3/fi active
- 2018-11-30 RS RS20250666A patent/RS66986B1/sr unknown
- 2018-11-30 RU RU2020121752A patent/RU2020121752A/ru unknown
- 2018-11-30 WO PCT/US2018/063416 patent/WO2019109001A1/en not_active Ceased
- 2018-11-30 MX MX2020005680A patent/MX2020005680A/es unknown
- 2018-11-30 HU HUE18883102A patent/HUE072645T2/hu unknown
- 2018-11-30 KR KR1020247035783A patent/KR102967878B1/ko active Active
- 2018-11-30 EP EP18883102.8A patent/EP3717646B1/en active Active
- 2018-11-30 BR BR112020009431-5A patent/BR112020009431A2/pt active IP Right Grant
- 2018-11-30 ES ES18883102T patent/ES3032315T3/es active Active
- 2018-11-30 SI SI201831246T patent/SI3717646T1/sl unknown
- 2018-11-30 PE PE2020000649A patent/PE20211238A1/es unknown
- 2018-11-30 LT LTEPPCT/US2018/063416T patent/LT3717646T/lt unknown
- 2018-11-30 DK DK18883102.8T patent/DK3717646T3/da active
- 2018-11-30 JP JP2020529238A patent/JP7297320B2/ja active Active
- 2018-11-30 HR HRP20250804TT patent/HRP20250804T1/hr unknown
- 2018-11-30 EP EP23214708.2A patent/EP4328306A3/en active Pending
- 2018-11-30 AU AU2018375807A patent/AU2018375807B2/en active Active
- 2018-11-30 CN CN202410312340.1A patent/CN118291458A/zh active Pending
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2020
- 2020-05-20 CL CL2020001344A patent/CL2020001344A1/es unknown
- 2020-05-26 CO CONC2020/0006361A patent/CO2020006361A2/es unknown
- 2020-07-13 MX MX2025009778A patent/MX2025009778A/es unknown
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2021
- 2021-11-10 US US17/523,456 patent/US11685920B2/en active Active
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2022
- 2022-11-14 US US18/055,212 patent/US11739328B2/en active Active
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2023
- 2023-06-07 JP JP2023093643A patent/JP7601441B2/ja active Active
- 2023-06-30 US US18/345,452 patent/US12180480B2/en active Active
-
2024
- 2024-11-12 US US18/944,351 patent/US20250066786A1/en active Pending
- 2024-11-28 JP JP2024206905A patent/JP7863362B2/ja active Active
-
2026
- 2026-04-28 JP JP2026075038A patent/JP7897667B1/ja active Active
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