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 JP2025041632A (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 (3)
| 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 Pending JP2025041632A (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 |
Family Applications After (2)
| 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 Pending JP2025041632A (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 |
Country Status (27)
| Country | Link |
|---|---|
| US (5) | US11198869B2 (enExample) |
| EP (2) | EP4328306A3 (enExample) |
| JP (3) | JP7297320B2 (enExample) |
| KR (2) | KR20240161203A (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 (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL281211B2 (en) | 2015-11-12 | 2024-01-01 | Hoffmann La Roche | Oligonucleotides for inducing paternal ube3a expression |
| US20210054370A1 (en) * | 2018-02-27 | 2021-02-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
| MD3947684T2 (ro) | 2019-03-29 | 2025-10-31 | Ionis Pharmaceuticals Inc | Compuși și metode pentru modularea UBE3A-ATS |
| JP7799640B2 (ja) * | 2020-06-29 | 2026-01-15 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｐｌｐ１を調節するための化合物及び方法 |
| US20230416851A1 (en) * | 2020-10-06 | 2023-12-28 | University Of Maryland, Baltimore | Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides |
| US20230332150A1 (en) * | 2022-03-07 | 2023-10-19 | University Of Connecticut | shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME |
| EP4660207A1 (en) | 2023-01-31 | 2025-12-10 | FUJIFILM Corporation | Curable composition, cured product, optical material, diffractive optical element, and compound |
| KR20250068540A (ko) * | 2023-11-08 | 2025-05-16 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| TW202548015A (zh) * | 2024-04-11 | 2025-12-16 | 美商奧崔基尼克斯製藥公司 | 天使症候群反義治療的給藥方案 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE239484T1 (de) | 1991-10-24 | 2003-05-15 | Isis Pharmaceuticals Inc | Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen |
| KR100909681B1 (ko) * | 2000-12-01 | 2009-07-29 | 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. | Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자 |
| EP2397563A3 (en) * | 2004-09-17 | 2012-07-18 | 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 |
| WO2009124238A1 (en) | 2008-04-04 | 2009-10-08 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2011017521A2 (en) | 2009-08-06 | 2011-02-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| KR20140031217A (ko) | 2011-04-20 | 2014-03-12 | 로슈 글리카트 아게 | 혈액-뇌 차단벽의 pH 의존적 통과를 위한 방법 및 구축물 |
| 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 |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| BR112014028634A2 (pt) * | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| ES2809199T3 (es) * | 2012-06-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Modulación de la expresión de UBE3A-ATS |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| JP2017536119A (ja) | 2014-11-19 | 2017-12-07 | ロシュ イノベーション センター コペンハーゲン エーエス | キラルホスホロチオエート連結を含むｌｎａギャップマーオリゴヌクレオチド |
| US10400243B2 (en) | 2014-11-25 | 2019-09-03 | Ionis Pharmaceuticals, Inc. | Modulation of UBE3A-ATS expression |
| EP3234131B1 (en) | 2014-12-16 | 2020-04-22 | Roche Innovation Center Copenhagen A/S | Chiral toxicity screening method |
| UY36550A (es) * | 2015-02-04 | 2016-08-31 | Roche Innovation Ct Copenhagen As | Oligómeros antisentido de tau y sus usos |
| JP2018512876A (ja) * | 2015-04-22 | 2018-05-24 | ミナ セラピューティクス リミテッド | ｓａＲＮＡ組成物および使用方法 |
| IL281211B2 (en) * | 2015-11-12 | 2024-01-01 | Hoffmann La Roche | Oligonucleotides for inducing paternal ube3a expression |
| CN109311925B (zh) | 2016-05-12 | 2022-06-03 | 罗氏创新中心哥本哈根有限公司 | 立体限定的氧杂氮杂磷杂环戊烷亚磷酰胺单体与核苷或寡核苷酸的增强的偶联 |
| RS62410B1 (sr) | 2016-10-04 | 2021-10-29 | Merck Sharp & Dohme | Benzo[b]tiofenska jedinjenja kao agonisti sting |
| US11591362B2 (en) | 2017-03-29 | 2023-02-28 | Roche Innovation Center Copenhagen A/S | Orthogonal protecting groups for the preparation of stereodefined phosphorothioate oligonucleotides |
| US20200172483A1 (en) | 2017-06-22 | 2020-06-04 | Curadev Pharma Limited | Heterocyclic small molecule modulators of human sting |
-
2018
- 2018-11-30 HR HRP20250804TT patent/HRP20250804T1/hr unknown
- 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 PL PL18883102.8T patent/PL3717646T3/pl unknown
- 2018-11-30 SM SM20250334T patent/SMT202500334T1/it unknown
- 2018-11-30 AU AU2018375807A patent/AU2018375807B2/en active Active
- 2018-11-30 IL IL274146A patent/IL274146B2/en unknown
- 2018-11-30 BR BR112020009431-5A patent/BR112020009431A2/pt active Search and Examination
- 2018-11-30 PT PT188831028T patent/PT3717646T/pt 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 SG SG11202003596WA patent/SG11202003596WA/en unknown
- 2018-11-30 FI FIEP18883102.8T patent/FI3717646T3/fi active
- 2018-11-30 ES ES18883102T patent/ES3032315T3/es active Active
- 2018-11-30 CA CA3079755A patent/CA3079755A1/en active Pending
- 2018-11-30 JP JP2020529238A patent/JP7297320B2/ja active Active
- 2018-11-30 RS RS20250666A patent/RS66986B1/sr unknown
- 2018-11-30 EP EP23214708.2A patent/EP4328306A3/en active Pending
- 2018-11-30 CN CN201880077892.1A patent/CN111433361B/zh active Active
- 2018-11-30 MX MX2020005680A patent/MX2020005680A/es unknown
- 2018-11-30 KR KR1020247035783A patent/KR20240161203A/ko not_active Ceased
- 2018-11-30 KR KR1020207016107A patent/KR102723989B1/ko active Active
- 2018-11-30 EP EP18883102.8A patent/EP3717646B1/en active Active
- 2018-11-30 HU HUE18883102A patent/HUE072645T2/hu unknown
- 2018-11-30 US US16/767,916 patent/US11198869B2/en active Active
- 2018-11-30 CN CN202410312340.1A patent/CN118291458A/zh active Pending
- 2018-11-30 DK DK18883102.8T patent/DK3717646T3/da active
-
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
-
2021
- 2021-11-10 US US17/523,456 patent/US11685920B2/en active Active
-
2022
- 2022-11-14 US US18/055,212 patent/US11739328B2/en active Active
-
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/JP2025041632A/ja active Pending
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