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
Links
- 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) | EP4328306A3 (https=) |
| JP (3) | JP7297320B2 (https=) |
| KR (2) | KR20240161203A (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 |
|---|---|---|---|---|
| HUE067905T2 (hu) | 2015-11-12 | 2024-11-28 | Hoffmann La Roche | Oligonukleotidok paternális UBE3A expresszió indukálására |
| CN111770758A (zh) * | 2018-02-27 | 2020-10-13 | 北卡罗来纳-查佩尔山大学 | 用于治疗Angelman综合征的方法和组合物 |
| FI3947684T3 (fi) | 2019-03-29 | 2025-05-27 | Ionis Pharmaceuticals Inc | Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi |
| BR112022024206A2 (pt) * | 2020-06-29 | 2023-01-03 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular plp1 |
| US20230416851A1 (en) * | 2020-10-06 | 2023-12-28 | University Of Maryland, Baltimore | Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides |
| IL315359A (en) * | 2022-03-07 | 2024-11-01 | Univ Connecticut | shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME |
| WO2024162126A1 (ja) | 2023-01-31 | 2024-08-08 | 富士フイルム株式会社 | 硬化性組成物、硬化物、光学材料、回折光学素子及び化合物 |
| 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 |
|---|---|---|---|---|
| WO1993007883A1 (en) | 1991-10-24 | 1993-04-29 | Isis Pharmaceuticals, Inc. | Derivatized oligonucleotides having improved uptake and other properties |
| US20040259247A1 (en) * | 2000-12-01 | 2004-12-23 | Thomas Tuschl | Rna interference mediating small rna molecules |
| 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 |
| WO2008113832A2 (en) | 2007-03-22 | 2008-09-25 | Santaris Pharma A/S | SHORT RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF TARGET mRNA |
| DK2285819T3 (da) | 2008-04-04 | 2013-12-02 | Isis Pharmaceuticals Inc | Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider |
| RU2572826C2 (ru) | 2008-12-02 | 2016-01-20 | Чиралджен, Лтд. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| US20120282176A1 (en) | 2011-04-20 | 2012-11-08 | Roche Glycart Ag | Method and Constructs for the pH Dependent Passage of the Blood-brain-barrier |
| EP3453761A1 (en) | 2011-08-29 | 2019-03-13 | 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 |
| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| JP2017536366A (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 | Ｌｎａキラルホスホロチオエート |
| WO2016086104A1 (en) | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| CN107208092B (zh) | 2014-12-16 | 2021-09-10 | 罗氏创新中心哥本哈根有限公司 | 手性毒性筛选方法 |
| AU2016215155A1 (en) * | 2015-02-04 | 2017-08-17 | 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 |
| HUE067905T2 (hu) | 2015-11-12 | 2024-11-28 | Hoffmann La Roche | Oligonukleotidok paternális UBE3A expresszió indukálására |
| 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 |
| US10414747B2 (en) | 2016-10-04 | 2019-09-17 | Merck Sharp & Dohme Corp. | Benzo[b]thiophene compounds as sting agonists |
| JP7164540B2 (ja) | 2017-03-29 | 2022-11-01 | 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 | 立体的に規定されたホスホロチオエートオリゴヌクレオチドの調製のための直交保護基 |
| 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 PT PT188831028T patent/PT3717646T/pt unknown
- 2018-11-30 DK DK18883102.8T patent/DK3717646T3/da active
- 2018-11-30 LT LTEPPCT/US2018/063416T patent/LT3717646T/lt unknown
- 2018-11-30 US US16/767,916 patent/US11198869B2/en active Active
- 2018-11-30 EP EP23214708.2A patent/EP4328306A3/en active Pending
- 2018-11-30 SM SM20250334T patent/SMT202500334T1/it unknown
- 2018-11-30 RU RU2020121752A patent/RU2020121752A/ru unknown
- 2018-11-30 IL IL274146A patent/IL274146B2/en unknown
- 2018-11-30 SI SI201831246T patent/SI3717646T1/sl unknown
- 2018-11-30 EP EP18883102.8A patent/EP3717646B1/en active Active
- 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 HU HUE18883102A patent/HUE072645T2/hu unknown
- 2018-11-30 CA CA3079755A patent/CA3079755A1/en active Pending
- 2018-11-30 JP JP2020529238A patent/JP7297320B2/ja active Active
- 2018-11-30 ES ES18883102T patent/ES3032315T3/es active Active
- 2018-11-30 CN CN201880077892.1A patent/CN111433361B/zh active Active
- 2018-11-30 SG SG11202003596WA patent/SG11202003596WA/en unknown
- 2018-11-30 CN CN202410312340.1A patent/CN118291458A/zh active Pending
- 2018-11-30 PE PE2020000649A patent/PE20211238A1/es unknown
- 2018-11-30 KR KR1020247035783A patent/KR20240161203A/ko active Pending
- 2018-11-30 KR KR1020207016107A patent/KR102723989B1/ko active Active
- 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 HR HRP20250804TT patent/HRP20250804T1/hr unknown
- 2018-11-30 AU AU2018375807A patent/AU2018375807B2/en active Active
- 2018-11-30 RS RS20250666A patent/RS66986B1/sr unknown
- 2018-11-30 FI FIEP18883102.8T patent/FI3717646T3/fi 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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