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 (https=) | 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 (https=) |
| EP (2) | EP3717646B1 (https=) |
| JP (3) | JP7297320B2 (https=) |
| KR (2) | KR102723989B1 (https=) |
| CN (2) | CN111433361B (https=) |
| AU (1) | AU2018375807B2 (https=) |
| BR (1) | BR112020009431A2 (https=) |
| CA (1) | CA3079755A1 (https=) |
| CL (1) | CL2020001344A1 (https=) |
| CO (1) | CO2020006361A2 (https=) |
| DK (1) | DK3717646T3 (https=) |
| ES (1) | ES3032315T3 (https=) |
| FI (1) | FI3717646T3 (https=) |
| HR (1) | HRP20250804T1 (https=) |
| HU (1) | HUE072645T2 (https=) |
| IL (1) | IL274146B2 (https=) |
| LT (1) | LT3717646T (https=) |
| MX (2) | MX2020005680A (https=) |
| PE (1) | PE20211238A1 (https=) |
| PL (1) | PL3717646T3 (https=) |
| PT (1) | PT3717646T (https=) |
| RS (1) | RS66986B1 (https=) |
| RU (1) | RU2020121752A (https=) |
| SG (1) | SG11202003596WA (https=) |
| SI (1) | SI3717646T1 (https=) |
| SM (1) | SMT202500334T1 (https=) |
| WO (1) | WO2019109001A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT3374509T (lt) | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| WO2019168950A1 (en) * | 2018-02-27 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
| PT3947684T (pt) | 2019-03-29 | 2025-05-19 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular ube3a‑ats |
| EP4172338A4 (en) * | 2020-06-29 | 2025-06-11 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR MODULATING PLP1 |
| US20230416851A1 (en) * | 2020-10-06 | 2023-12-28 | 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 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE69233046T2 (de) | 1991-10-24 | 2004-03-04 | Isis Pharmaceuticals, Inc., Carlsfeld | Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen |
| CA2429814C (en) * | 2000-12-01 | 2014-02-18 | Thomas Tuschl | Rna interference mediating small rna molecules |
| US7919472B2 (en) | 2004-09-17 | 2011-04-05 | 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 |
| DK2149605T3 (da) | 2007-03-22 | 2013-09-30 | Santaris Pharma As | Korte RNA antagonist forbindelser til modulering af det ønskede mRNA |
| US9290534B2 (en) | 2008-04-04 | 2016-03-22 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside |
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| US9012421B2 (en) | 2009-08-06 | 2015-04-21 | 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 |
| WO2013033230A1 (en) | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| EA201492122A1 (ru) * | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| WO2014004572A2 (en) * | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| WO2016079183A1 (en) | 2014-11-19 | 2016-05-26 | Roche Innovation Center Copenhagen A/S | Lna chiral phosphorothioates |
| US10400243B2 (en) | 2014-11-25 | 2019-09-03 | Ionis Pharmaceuticals, Inc. | Modulation of UBE3A-ATS expression |
| US10815481B2 (en) | 2014-12-16 | 2020-10-27 | Roche Innovation Center Copenhagen A/S | Chiral library screen |
| US11077132B2 (en) * | 2015-02-04 | 2021-08-03 | F. Hoffmann-La Roche Ag | Tau antisense oligomers and uses thereof |
| US20180305689A1 (en) * | 2015-04-22 | 2018-10-25 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| LT3374509T (lt) * | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| EP3455232B1 (en) | 2016-05-12 | 2020-05-06 | Roche Innovation Center Copenhagen A/S | Enhanced coupling of stereodefined oxazaphospholidine phosphoramidite monomers to nucleoside or oligonucleotide |
| PT3523287T (pt) | 2016-10-04 | 2021-10-06 | Merck Sharp & Dohme | Compostos de benzo[b]tiofeno como agonistas de sting |
| KR20190135494A (ko) | 2017-03-29 | 2019-12-06 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | 입체한정된 포스포로티오에이트 올리고뉴클레오티드의 제조를 위한 직교 보호기 |
| WO2018234807A1 (en) | 2017-06-22 | 2018-12-27 | Curadev Pharma Limited | Heterocyclic small molecule modulators of human sting |
-
2018
- 2018-11-30 KR KR1020207016107A patent/KR102723989B1/ko active Active
- 2018-11-30 AU AU2018375807A patent/AU2018375807B2/en active Active
- 2018-11-30 RS RS20250666A patent/RS66986B1/sr unknown
- 2018-11-30 MX MX2020005680A patent/MX2020005680A/es unknown
- 2018-11-30 PT PT188831028T patent/PT3717646T/pt unknown
- 2018-11-30 SI SI201831246T patent/SI3717646T1/sl unknown
- 2018-11-30 IL IL274146A patent/IL274146B2/en unknown
- 2018-11-30 JP JP2020529238A patent/JP7297320B2/ja active Active
- 2018-11-30 CN CN201880077892.1A patent/CN111433361B/zh active Active
- 2018-11-30 SM SM20250334T patent/SMT202500334T1/it unknown
- 2018-11-30 HR HRP20250804TT patent/HRP20250804T1/hr unknown
- 2018-11-30 RU RU2020121752A patent/RU2020121752A/ru unknown
- 2018-11-30 CA CA3079755A patent/CA3079755A1/en active Pending
- 2018-11-30 EP EP18883102.8A patent/EP3717646B1/en active Active
- 2018-11-30 US US16/767,916 patent/US11198869B2/en active Active
- 2018-11-30 WO PCT/US2018/063416 patent/WO2019109001A1/en not_active Ceased
- 2018-11-30 FI FIEP18883102.8T patent/FI3717646T3/fi active
- 2018-11-30 LT LTEPPCT/US2018/063416T patent/LT3717646T/lt unknown
- 2018-11-30 EP EP23214708.2A patent/EP4328306A3/en active Pending
- 2018-11-30 DK DK18883102.8T patent/DK3717646T3/da active
- 2018-11-30 KR KR1020247035783A patent/KR20240161203A/ko active Pending
- 2018-11-30 CN CN202410312340.1A patent/CN118291458A/zh active Pending
- 2018-11-30 PE PE2020000649A patent/PE20211238A1/es unknown
- 2018-11-30 SG SG11202003596WA patent/SG11202003596WA/en unknown
- 2018-11-30 PL PL18883102.8T patent/PL3717646T3/pl unknown
- 2018-11-30 BR BR112020009431-5A patent/BR112020009431A2/pt active Search and Examination
- 2018-11-30 ES ES18883102T patent/ES3032315T3/es active Active
- 2018-11-30 HU HUE18883102A patent/HUE072645T2/hu unknown
-
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
-
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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