JP2020530487A5 - - Google Patents
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- JP2020530487A5 JP2020530487A5 JP2020529113A JP2020529113A JP2020530487A5 JP 2020530487 A5 JP2020530487 A5 JP 2020530487A5 JP 2020529113 A JP2020529113 A JP 2020529113A JP 2020529113 A JP2020529113 A JP 2020529113A JP 2020530487 A5 JP2020530487 A5 JP 2020530487A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutically acceptable
- solvate
- substituted
- acceptable salt
- unsubstituted
- 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 21
- 150000003839 salts Chemical class 0.000 claims 18
- 239000012453 solvate Substances 0.000 claims 18
- 125000004093 cyano group Chemical group *C#N 0.000 claims 3
- 125000000753 cycloalkyl group Chemical group 0.000 claims 3
- 229910052736 halogen Inorganic materials 0.000 claims 3
- 229910052739 hydrogen Inorganic materials 0.000 claims 3
- 125000005913 (C3-C6) cycloalkyl group Chemical group 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 2
- 125000001559 cyclopropyl group Chemical group [H]C1([H])C([H])([H])C1([H])* 0.000 claims 2
- 150000002367 halogens Chemical group 0.000 claims 2
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 2
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 2
- 125000003545 alkoxy group Chemical group 0.000 claims 1
- 229910052799 carbon Inorganic materials 0.000 claims 1
- 125000001995 cyclobutyl group Chemical group [H]C1([H])C([H])([H])C([H])(*)C1([H])[H] 0.000 claims 1
- 125000000113 cyclohexyl group Chemical group [H]C1([H])C([H])([H])C([H])([H])C([H])(*)C([H])([H])C1([H])[H] 0.000 claims 1
- 125000001511 cyclopentyl group Chemical group [H]C1([H])C([H])([H])C([H])([H])C([H])(*)C1([H])[H] 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000005843 halogen group Chemical group 0.000 claims 1
- 125000004404 heteroalkyl group Chemical group 0.000 claims 1
- 125000005842 heteroatom Chemical group 0.000 claims 1
- -1 hydroxy, amino, methoxy Chemical group 0.000 claims 1
- 229910052760 oxygen Inorganic materials 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 125000006413 ring segment Chemical group 0.000 claims 1
- 125000001424 substituent group Chemical group 0.000 claims 1
- 229910052717 sulfur Inorganic materials 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762541202P | 2017-08-04 | 2017-08-04 | |
| US62/541,202 | 2017-08-04 | ||
| US201762562948P | 2017-09-25 | 2017-09-25 | |
| US201762562927P | 2017-09-25 | 2017-09-25 | |
| US62/562,927 | 2017-09-25 | ||
| US62/562,948 | 2017-09-25 | ||
| PCT/US2018/045282 WO2019028440A1 (en) | 2017-08-04 | 2018-08-03 | METHODS AND COMPOSITIONS FOR MODULATING SPLICING |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020078335A Division JP7135026B2 (ja) | 2017-08-04 | 2020-04-27 | スプライシングをモジュレートする方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020530487A JP2020530487A (ja) | 2020-10-22 |
| JP2020530487A5 true JP2020530487A5 (enExample) | 2021-09-16 |
| JP7312749B2 JP7312749B2 (ja) | 2023-07-21 |
Family
ID=65233135
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020529113A Active JP7312749B2 (ja) | 2017-08-04 | 2018-08-03 | スプライシングをモジュレートする方法および組成物 |
| JP2020078335A Active JP7135026B2 (ja) | 2017-08-04 | 2020-04-27 | スプライシングをモジュレートする方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020078335A Active JP7135026B2 (ja) | 2017-08-04 | 2020-04-27 | スプライシングをモジュレートする方法および組成物 |
Country Status (15)
| Country | Link |
|---|---|
| US (8) | US11008572B2 (enExample) |
| EP (2) | EP3689863A1 (enExample) |
| JP (2) | JP7312749B2 (enExample) |
| KR (2) | KR102636383B1 (enExample) |
| CN (2) | CN111499615B (enExample) |
| AU (2) | AU2018309187C1 (enExample) |
| CA (1) | CA3072205A1 (enExample) |
| GB (2) | GB2579747B8 (enExample) |
| IL (2) | IL272413B (enExample) |
| MX (2) | MX2020001425A (enExample) |
| MY (1) | MY201938A (enExample) |
| RU (1) | RU2020105929A (enExample) |
| SG (2) | SG10202002990XA (enExample) |
| WO (1) | WO2019028440A1 (enExample) |
| ZA (2) | ZA202000695B (enExample) |
Families Citing this family (74)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110946865B (zh) | 2015-12-10 | 2024-01-26 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| WO2018098446A1 (en) | 2016-11-28 | 2018-05-31 | Ptc Therapeutics, Inc | Methods for modulating rna splicing |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| BR112019026508A2 (pt) | 2017-06-14 | 2020-07-14 | Ptc Therapeutics, Inc. | métodos para modificar o splicing do rna |
| CN111163838B (zh) | 2017-06-28 | 2023-03-28 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| RU2020105929A (ru) | 2017-08-04 | 2021-09-06 | Скайхоук Терапьютикс, Инк. | Способы и композиции для модулирования сплайсинга |
| JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス, インコーポレイテッド | ハンチントン病を処置するための化合物 |
| US11530207B2 (en) | 2018-04-10 | 2022-12-20 | Skyhawk Therapeutics, Inc. | Compounds for the treatment of cancer |
| KR20210038845A (ko) | 2018-06-27 | 2021-04-08 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료를 위한 헤테로아릴 화합물 |
| PL3814357T3 (pl) | 2018-06-27 | 2024-09-16 | Ptc Therapeutics, Inc. | Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona |
| EP3814360B8 (en) | 2018-06-27 | 2024-11-06 | PTC Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| EA202190581A1 (ru) | 2018-08-20 | 2021-07-13 | Рогкон, Инк. | Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a |
| WO2020154462A1 (en) * | 2019-01-23 | 2020-07-30 | Rogcon U.R., Inc. | Antisense oligonucleotides targeting scn2a retained introns |
| JP2022519637A (ja) * | 2019-02-04 | 2022-03-24 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| EP3920926A4 (en) * | 2019-02-05 | 2022-10-19 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| EP3920919A4 (en) * | 2019-02-05 | 2023-03-29 | Skyhawk Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODULATING SPLICE |
| KR20210135239A (ko) * | 2019-02-05 | 2021-11-12 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| JP2022519311A (ja) * | 2019-02-05 | 2022-03-22 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| JP7603592B2 (ja) * | 2019-02-05 | 2024-12-20 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| WO2020163544A1 (en) * | 2019-02-06 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| CN119638696A (zh) * | 2019-02-06 | 2025-03-18 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| EP3937942A4 (en) * | 2019-03-15 | 2022-11-16 | Skyhawk Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR CORRECTING ABERRANT SPLICING |
| AU2020271530B2 (en) | 2019-04-09 | 2026-02-05 | Envisagenics, Inc. | Cancer-specific molecules and methods of use thereof |
| CN109988163A (zh) * | 2019-04-15 | 2019-07-09 | 宁夏医科大学 | 一种异丙基托品醇异构体的制备分离方法 |
| CN114245794B (zh) | 2019-05-13 | 2024-09-13 | Ptc医疗公司 | 用于治疗亨廷顿氏病的化合物 |
| UY38687A (es) | 2019-05-17 | 2023-05-15 | Novartis Ag | Inhibidores del inflamasoma nlrp3, composiciones, combinaciones de los mismos y métodos para su uso |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| CN120137971A (zh) | 2019-07-25 | 2025-06-13 | 诺华股份有限公司 | 可调节的表达系统 |
| WO2021071982A1 (en) * | 2019-10-08 | 2021-04-15 | Skyhawk Therapeutics, Inc. | Compounds for modulating splicing |
| US20240216369A1 (en) | 2019-11-01 | 2024-07-04 | Novartis Ag | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
| CA3169667A1 (en) * | 2020-02-28 | 2021-09-02 | Dominic Reynolds | Pyridazine derivatives for modulating nucleic acid splicing |
| IL295955A (en) | 2020-02-28 | 2022-10-01 | Remix Therapeutics Inc | Compounds and methods for modulating splicing |
| JP2023515620A (ja) | 2020-02-28 | 2023-04-13 | リミックス セラピューティクス インコーポレイテッド | スプライシングを調節するための化合物及び方法 |
| EP4110775A1 (en) | 2020-02-28 | 2023-01-04 | Remix Therapeutics Inc. | Thiophenyl derivatives useful for modulating nucleic acid splicing |
| WO2021207554A1 (en) | 2020-04-08 | 2021-10-14 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| WO2021207530A1 (en) | 2020-04-08 | 2021-10-14 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| CN116997548A (zh) | 2020-05-13 | 2023-11-03 | Chdi基金会股份有限公司 | 用于治疗亨廷顿病的htt调节剂 |
| KR20230051168A (ko) | 2020-07-02 | 2023-04-17 | 레믹스 테라퓨틱스 인크. | 2-(인다졸-5-일)-6-(피페리딘-4-일)-1,7-나프티리딘 유도체, 및 핵산 스플라이싱의 조절제로서의 및 증식성 질환의 치료를 위한 관련 화합물 |
| EP4178963A1 (en) | 2020-07-02 | 2023-05-17 | Remix Therapeutics Inc. | 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases |
| WO2022031998A2 (en) * | 2020-08-05 | 2022-02-10 | Skyhawk Therapeutics, Inc. | Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing |
| IL300352A (en) * | 2020-08-05 | 2023-04-01 | Skyhawk Therapeutics Inc | The compositions for cutting and fusion modulation |
| WO2022032007A2 (en) * | 2020-08-05 | 2022-02-10 | Skyhawk Therapeutics, Inc. | Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing |
| CN114113621A (zh) * | 2020-08-25 | 2022-03-01 | 张曼 | 尿液鸟嘌呤核苷酸结合蛋白亚基α-13及其多肽片段在烧伤中的应用 |
| WO2022060951A1 (en) * | 2020-09-16 | 2022-03-24 | Skyhawk Therapeutics, Inc. | Compositions for modulating splicing |
| WO2022060944A1 (en) * | 2020-09-16 | 2022-03-24 | Skyhawk Therapeutics, Inc. | Compositions for modulating splicing |
| WO2022060943A1 (en) * | 2020-09-16 | 2022-03-24 | Skyhawk Therapeutics, Inc. | Compositions for modulating splicing |
| US20240018522A1 (en) * | 2020-10-26 | 2024-01-18 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| WO2022109022A1 (en) * | 2020-11-17 | 2022-05-27 | Skyhawk Therapeutics, Inc. | Rna-targeting splicing modifiers for treatment of mda5-associated conditions and diseases |
| KR20240019083A (ko) * | 2021-06-04 | 2024-02-14 | 에프. 호프만-라 로슈 아게 | 트리아진 유도체 및 암 치료에서 이의 용도 |
| IL311135A (en) | 2021-08-30 | 2024-04-01 | Remix Therapeutics Inc | Compounds and methods for modulating splicing |
| WO2023034833A1 (en) | 2021-08-30 | 2023-03-09 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| US20240368163A1 (en) | 2021-08-30 | 2024-11-07 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| WO2023034836A1 (en) | 2021-08-30 | 2023-03-09 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| EP4395891A1 (en) | 2021-08-30 | 2024-07-10 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| MX2024003519A (es) | 2021-09-24 | 2024-04-01 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso. |
| WO2023064880A1 (en) | 2021-10-13 | 2023-04-20 | Remix Therapeutics Inc. | Compounds and methods for modulating nucleic acid splicing |
| WO2023064879A1 (en) | 2021-10-13 | 2023-04-20 | Remix Therapeutics Inc. | Compounds and methods for modulating nucleic acid splicing |
| US12281116B2 (en) | 2021-11-17 | 2025-04-22 | Chdi Foundation, Inc. | HTT modulators for treating Huntington's disease |
| MX2024006065A (es) * | 2021-11-18 | 2024-08-14 | Rgenta Therapeutics Inc | Amidas heterociclicas y metodos de utilizacion de las mismas. |
| CN114225051B (zh) * | 2021-12-16 | 2024-05-10 | 中国人民解放军空军军医大学 | 一种用于治疗银屑病的药物及其应用 |
| WO2023133225A1 (en) | 2022-01-05 | 2023-07-13 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| US20250092065A1 (en) | 2022-01-05 | 2025-03-20 | Remix Therapeutics Inc. | 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases |
| US20250109140A1 (en) | 2022-01-05 | 2025-04-03 | Remix Theraputics Inc. | 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases |
| CN114350813B (zh) * | 2022-03-17 | 2022-08-02 | 广州达安临床检验中心有限公司 | Tmem236基因在制备用于诊断直肠癌新辅助放疗抵抗标记物中的用途 |
| CN114592006A (zh) * | 2022-04-29 | 2022-06-07 | 昆明理工大学 | Memo1基因的新用途 |
| KR102470669B1 (ko) * | 2022-06-03 | 2022-11-25 | 충남대학교 산학협력단 | 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물 |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| UY40374A (es) | 2022-08-03 | 2024-02-15 | Novartis Ag | Inhibidores de inflamasoma nlrp3 |
| TW202432551A (zh) * | 2022-10-26 | 2024-08-16 | 日商安斯泰來製藥股份有限公司 | 稠環嗒嗪衍生物 |
| US20240150759A1 (en) * | 2022-11-04 | 2024-05-09 | The Board Of Trustees Of The Leland Stanford Junior University | Splicing Modulators for the Treatment of Timothy Syndrome |
| CR20250371A (es) * | 2023-02-15 | 2025-10-03 | Ascidian Therapeutics Inc | Moléculas de tran-sempalme de htt |
| WO2025247338A1 (zh) * | 2024-05-30 | 2025-12-04 | 纽欧申医药(上海)有限公司 | 哒嗪环类化合物、其药物组合物及其应用 |
| CN119569728B (zh) * | 2024-12-02 | 2025-09-30 | 重庆飞因科生物科技有限公司 | 吡啶并吡咯类衍生物及其在制备富亮氨酸重复激酶2抑制剂中的应用 |
Family Cites Families (133)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| NZ227684A (en) | 1988-01-30 | 1991-07-26 | Merck Sharp & Dohme | Pyrazine, pyridazine, and pyrimidine derivatives and pharmaceutical compositions |
| GB9005737D0 (en) | 1990-03-14 | 1990-05-09 | Beecham Group Plc | Novel compounds |
| FR2676444B1 (fr) * | 1991-05-16 | 1995-03-10 | Sanofi Elf | Nouveaux derives d'amino-3 pyridazines actifs sur le systeme nerveux central, procede de preparation et compositions pharmaceutiques en contenant. |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| CA2457527A1 (en) | 2001-08-31 | 2003-03-13 | Gen-Probe Incorporated | Affinity-shifted probes for quantifying analyte polynucleotides |
| US7135484B2 (en) | 2002-08-14 | 2006-11-14 | Abbott Laboratories | Azabicyclic compounds are central nervous system active agents |
| DE60326894D1 (de) | 2002-09-30 | 2009-05-07 | Neurosearch As | 1,4-Diazabicycloalkanderivate, deren Herstellung und Verwendung |
| US8014953B2 (en) | 2003-08-08 | 2011-09-06 | The Regents Of The University Of California | RNA surveillance among curated proteins |
| US7399765B2 (en) | 2003-09-19 | 2008-07-15 | Abbott Laboratories | Substituted diazabicycloalkane derivatives |
| US7160876B2 (en) | 2003-12-22 | 2007-01-09 | Abbott Laboratories | Fused bicycloheterocycle substituted quinuclidine derivatives |
| US7309699B2 (en) | 2003-12-22 | 2007-12-18 | Abbott Laboratories | 3-Quinuclidinyl amino-substituted biaryl derivatives |
| US7655657B2 (en) | 2003-12-22 | 2010-02-02 | Abbott Laboratories | Fused bicycloheterocycle substituted quinuclidine derivatives |
| US7309487B2 (en) | 2004-02-09 | 2007-12-18 | George Inana | Methods and compositions for detecting and treating retinal diseases |
| PE20060437A1 (es) | 2004-06-18 | 2006-06-08 | Novartis Ag | COMPUESTOS AZA-BICICLONONANOS COMO LIGANDOS COLINERGICOS DE nAChR |
| GB0415746D0 (en) | 2004-07-14 | 2004-08-18 | Novartis Ag | Organic compounds |
| JP5635727B2 (ja) | 2004-10-07 | 2014-12-03 | 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. | チアゾリルmglur5アンタゴニスト及びそれらの使用のための方法 |
| EP1809282B1 (en) | 2004-10-18 | 2013-01-09 | Amgen, Inc | Thiadiazole compounds and methods of use |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| CN101146796A (zh) | 2005-01-26 | 2008-03-19 | 先灵公司 | 作为蛋白激酶抑制剂用于治疗癌症的3-(吲唑-5-基)-(1,2,4)三嗪衍生物及相关化合物 |
| EP1871762A2 (en) | 2005-04-18 | 2008-01-02 | Neurogen Corporation | Subtituted heteroaryl cb1 antagonists |
| US7598052B2 (en) | 2005-10-11 | 2009-10-06 | The Regents Of The University Of Michigan | Expression profile of thyroid cancer |
| US8603457B2 (en) | 2005-12-02 | 2013-12-10 | University Of Rochester | Nonsense suppression and genetic codon alteration by targeted modification |
| US7834178B2 (en) | 2006-03-01 | 2010-11-16 | Bristol-Myers Squibb Company | Triazine 11-beta hydroxysteroid dehydrogenase type 1 inhibitors |
| US7872017B2 (en) * | 2006-05-19 | 2011-01-18 | Abbott Laboratories | Fused bicycloheterocycle substituted azabicyclic alkane derivatives |
| WO2007138038A1 (en) | 2006-05-30 | 2007-12-06 | Neurosearch A/S | Novel 1,4-diaza-bicyclo[3.2.2]nonyl oxadiazolyl derivatives and their medical use |
| US8314119B2 (en) | 2006-11-06 | 2012-11-20 | Abbvie Inc. | Azaadamantane derivatives and methods of use |
| US8343941B2 (en) | 2007-03-30 | 2013-01-01 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
| US8283116B1 (en) | 2007-06-22 | 2012-10-09 | Ptc Therapeutics, Inc. | Methods of screening for compounds for treating spinal muscular atrophy using SMN mRNA translation regulation |
| DE102007032507A1 (de) * | 2007-07-12 | 2009-04-02 | Merck Patent Gmbh | Pyridazinonderivate |
| DK2203173T3 (en) | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
| WO2009067579A1 (en) | 2007-11-21 | 2009-05-28 | Abbott Laboratories | Biaryl substituted azabicyclic alkane derivatives as nicotinic acetylcholine receptor activity modulators |
| US8437067B2 (en) | 2008-03-18 | 2013-05-07 | Konica Minolta Holdings, Inc. | Electrochemical display element |
| US8633019B2 (en) | 2008-05-27 | 2014-01-21 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| US8133724B2 (en) | 2008-09-17 | 2012-03-13 | University Of Maryland, Baltimore | Human androgen receptor alternative splice variants as biomarkers and therapeutic targets |
| CN102264737A (zh) | 2008-12-23 | 2011-11-30 | 雅培制药有限公司 | 抗病毒化合物 |
| MX2011009751A (es) | 2009-03-16 | 2011-09-29 | Opko Curna Llc | Tratamiento de enfermedades relacionadas con factor nuclear (eritroide derivado 2) tipo 2 (nrf2) por inhibicion del transcrito antisentido natural a nrf2. |
| CN102625840A (zh) | 2009-04-10 | 2012-08-01 | 肌肉学研究协会 | 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法 |
| PT3449926T (pt) | 2009-06-17 | 2019-11-12 | Cold Spring Harbor Laboratory | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| JO3250B1 (ar) | 2009-09-22 | 2018-09-16 | Novartis Ag | إستعمال منشطات مستقبل نيكوتينيك أسيتيل كولين ألفا 7 |
| EP2305679A1 (en) | 2009-09-28 | 2011-04-06 | GenKyoTex SA | Pyrazoline dione derivatives as nadph oxidase inhibitors |
| WO2011090669A1 (en) | 2010-01-20 | 2011-07-28 | Abbott Laboratories | Methods for treating pain |
| RU2625791C2 (ru) | 2010-02-05 | 2017-07-19 | Хептейрес Терапьютикс Лимитед | Производные 1,2,4-триазин-4-амина |
| WO2011133920A1 (en) | 2010-04-23 | 2011-10-27 | Cytokinetics, Inc. | Certain amino-pyridines and amino-triazines, compositions thereof, and methods for their use |
| AR081331A1 (es) | 2010-04-23 | 2012-08-08 | Cytokinetics Inc | Amino- pirimidinas composiciones de las mismas y metodos para el uso de los mismos |
| AR081626A1 (es) | 2010-04-23 | 2012-10-10 | Cytokinetics Inc | Compuestos amino-piridazinicos, composiciones farmaceuticas que los contienen y uso de los mismos para tratar trastornos musculares cardiacos y esqueleticos |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| EP2585455B1 (en) | 2010-06-24 | 2019-08-07 | The Regents of the University of California | Compounds and uses thereof in modulating levels of various amyloid beta peptide alloforms |
| CA2806341C (en) | 2010-07-29 | 2020-03-24 | Rigel Pharmaceuticals, Inc. | Ampk-activating heterocyclic compounds and methods for using the same |
| US20130071369A1 (en) | 2011-03-14 | 2013-03-21 | Nse Products, Inc. | Oral Formulations For Promoting Cellular Purification |
| US9464065B2 (en) | 2011-03-24 | 2016-10-11 | The Scripps Research Institute | Compounds and methods for inducing chondrogenesis |
| US9447075B2 (en) | 2011-08-02 | 2016-09-20 | The Brigham And Women's Hospital, Inc. | Pyridazine derivatives as EAAT2 activators |
| US10252984B2 (en) | 2011-10-27 | 2019-04-09 | Mayo Foundation For Medical Education And Research | Inhibiting G protein coupled receptor 6 kinase polypeptides |
| USRE47689E1 (en) | 2011-12-30 | 2019-11-05 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| ES2733644T3 (es) | 2012-01-26 | 2019-12-02 | Ptc Therapeutics Inc | Compuestos para tratar atrofia muscular espinal |
| WO2013117693A1 (en) | 2012-02-10 | 2013-08-15 | Glaxosmithkline Intellectual Property Development Limited | Pde4 inhibitor for treating huntington's disease |
| JP6092897B2 (ja) | 2012-02-10 | 2017-03-08 | ピーティーシー セラピューティクス, インコーポレイテッド | 脊髄性筋萎縮症を治療するための化合物 |
| EA029155B1 (ru) | 2012-03-01 | 2018-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения спинальной мышечной атрофии |
| CA2868026C (en) | 2012-03-23 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| US8729263B2 (en) | 2012-08-13 | 2014-05-20 | Novartis Ag | 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions |
| AU2013204200B2 (en) | 2012-10-11 | 2016-10-20 | Brandeis University | Treatment of amyotrophic lateral sclerosis |
| ES2762326T5 (es) | 2012-10-15 | 2023-04-27 | Ionis Pharmaceuticals Inc | Métodos para modular la expresión de C9ORF72 |
| JP6338223B2 (ja) * | 2013-01-15 | 2018-06-06 | ノバルティス アーゲー | アルファ7ニコチン性アセチルコリン受容体アゴニストの使用 |
| KR101879921B1 (ko) * | 2013-01-15 | 2018-07-18 | 노파르티스 아게 | 기면증의 치료를 위한 알파 7 니코틴성 아세틸콜린 수용체 작용물질의 용도 |
| ES2865736T3 (es) * | 2013-01-15 | 2021-10-15 | Novartis Ag | Uso de agonistas del receptor de acetilcolina nicotínico alfa 7 |
| ITMI20130063A1 (it) * | 2013-01-17 | 2014-07-18 | Valier Alberto | Dispositivo manuale per la raccolta dal suolo di frutti o altri oggetti |
| US9040712B2 (en) | 2013-01-23 | 2015-05-26 | Novartis Ag | Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions |
| RU2673542C2 (ru) | 2013-06-25 | 2018-11-28 | Ф. Хоффманн-Ля Рош Аг | Соединения для лечения спинальной мышечной атрофии |
| EA030631B1 (ru) | 2013-07-31 | 2018-09-28 | Новартис Аг | 1,4-дизамещенные аналоги пиридазинхинолина и способы лечения состояний, связанных с smn-дефицитом |
| CN105392790B (zh) | 2013-08-19 | 2019-04-19 | 豪夫迈·罗氏有限公司 | 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途 |
| EP3039019B1 (en) | 2013-08-26 | 2020-04-22 | Purdue Pharma L.P. | Azaspiro[4.5]decane derivatives and use thereof |
| US9982260B2 (en) | 2013-09-30 | 2018-05-29 | The Regents Of The University Of California | Identification of structurally similar small molecules that enhance therapeutic exon skipping |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| WO2015136947A1 (en) | 2014-03-14 | 2015-09-17 | Raqualia Pharma Inc. | Azaspiro derivatives as trpm8 antagonists |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| AU2015272127A1 (en) | 2014-06-10 | 2017-01-19 | Erasmus University Medical Center Rotterdam | Methods for characterizing alternatively or aberrantly spliced mRNA isoforms |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| WO2016027168A2 (en) * | 2014-08-20 | 2016-02-25 | Lifesplice Pharma Llc | Splice modulating oligonucleotides and methods of use thereof |
| US20170355956A1 (en) | 2014-09-04 | 2017-12-14 | Rutgers, The State University Of New Jersey | Compositions for increasing survival of motor neuron protein (smn) levels in target cells and methods of use thereof for the treatment of spinal muscular atrophy |
| US20170334977A1 (en) | 2014-10-20 | 2017-11-23 | The Brigham And Women's Hospital, Inc. | Targeting Apolipoprotein E (APOE) in Neurologic Disease |
| US10053697B1 (en) | 2015-01-09 | 2018-08-21 | The Board Of Trustees Of The Leland Stanford Junior University | Programmable alternative splicing devices and uses thereof |
| ES2907461T3 (es) | 2015-01-20 | 2022-04-25 | Boehringer Ingelheim Animal Health Usa Inc | Compuestos antihelmínticos, composiciones y procedimientos de uso de los mismos |
| EP3053577A1 (en) | 2015-02-09 | 2016-08-10 | F. Hoffmann-La Roche AG | Compounds for the treatment of cancer |
| WO2016128343A1 (en) * | 2015-02-09 | 2016-08-18 | F. Hoffmann-La Roche Ag | Compounds for the treatment of cancer |
| EP4249472A3 (en) * | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| SG10201912699RA (en) | 2015-07-20 | 2020-02-27 | Genzyme Corp | Colony stimulating factor-1 receptor (csf-1r) inhibitors |
| WO2017041036A1 (en) | 2015-09-02 | 2017-03-09 | Pathways Bioscience, Llc | Compositions and methods for treatment or prevention of oral mucositis |
| CN108260355A (zh) | 2015-09-24 | 2018-07-06 | 葛兰素史克知识产权开发有限公司 | 吲哚胺2,3-双加氧酶的调节剂 |
| WO2017062751A1 (en) | 2015-10-08 | 2017-04-13 | The Regents Of The University Of California | Compounds and methods for promoting stress resistance |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| JP6659841B2 (ja) | 2015-11-12 | 2020-03-04 | エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft | 脊髄性筋萎縮症を処置するための組成物 |
| WO2017100276A1 (en) | 2015-12-08 | 2017-06-15 | The Arizona Board Of Regents On Behalf Of The University Of Arizona | Compositions and methods for treatment, amelioration, and prevention of diabetes-related skin ulcers |
| CN110946865B (zh) * | 2015-12-10 | 2024-01-26 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2017112954A1 (en) | 2015-12-23 | 2017-06-29 | Moonshot Pharma Llc | Methods for inducing an immune response by promoting premature termination codon read-through |
| EP3402793A4 (en) | 2016-01-15 | 2019-10-09 | The Brigham and Women's Hospital, Inc. | Pyridazine derivatives as EAAT2 activators |
| CN108778345B (zh) | 2016-02-01 | 2022-11-29 | 阿拉基斯医疗公司 | 治疗rna介导的疾病的化合物和方法 |
| BR112018017077A2 (pt) | 2016-02-23 | 2018-12-26 | Univ Indiana Res & Tech Corp | terapias de combinação para tratamento de atrofia muscular espinhal |
| CN109069870B (zh) | 2016-02-24 | 2022-04-29 | 洛克菲勒大学 | 基于胚胎细胞的用于亨廷顿氏病的治疗候选物筛选系统、模型及它们的应用 |
| US11066456B2 (en) | 2016-02-25 | 2021-07-20 | Washington University | Compositions comprising TREM2 and methods of use thereof |
| US20170268066A1 (en) | 2016-03-15 | 2017-09-21 | Chalmers Ventures Ab | Cancer biomarkers |
| ES2797301T3 (es) | 2016-03-29 | 2020-12-01 | Merck Patent Gmbh | Derivados de piperidinil-propanona |
| EP3440100A4 (en) | 2016-04-04 | 2020-01-22 | University of Florida Research Foundation, Incorporated | HANDLING EIF3 TO MODULATE A NON-ATG TRANSLATION ASSOCIATED WITH A REPETITION (RAN) |
| US10577360B2 (en) | 2016-04-12 | 2020-03-03 | Esteve Pharmaceuticals, S.A. | Arylamide derivatives having multimodal activity against pain |
| EP3785728A3 (en) | 2016-06-13 | 2021-04-28 | Scholar Rock, Inc. | Use of myostatin inhibitors and combination therapies |
| CN109563515A (zh) * | 2016-07-01 | 2019-04-02 | 阿拉基斯医疗公司 | 化合物和调节rna功能的方法 |
| WO2018098446A1 (en) | 2016-11-28 | 2018-05-31 | Ptc Therapeutics, Inc | Methods for modulating rna splicing |
| CN114432318A (zh) | 2017-02-20 | 2022-05-06 | 国立大学法人京都大学 | 用于起因于剪接异常的遗传性疾病的药物组合物及治疗方法 |
| US11715549B2 (en) | 2017-05-01 | 2023-08-01 | Thomas Jefferson University | Systems-level analysis of 32 TCGA cancers reveals disease-dependent tRNA fragmentation patterns and very selective associations with messenger RNAs and repeat elements |
| BR112019026508A2 (pt) | 2017-06-14 | 2020-07-14 | Ptc Therapeutics, Inc. | métodos para modificar o splicing do rna |
| CN111163838B (zh) | 2017-06-28 | 2023-03-28 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| RU2020105929A (ru) * | 2017-08-04 | 2021-09-06 | Скайхоук Терапьютикс, Инк. | Способы и композиции для модулирования сплайсинга |
| WO2019057123A1 (zh) | 2017-09-20 | 2019-03-28 | 杭州英创医药科技有限公司 | 作为ido抑制剂和/或ido-hdac双重抑制剂的多环化合物 |
| MX2020003579A (es) | 2017-10-12 | 2020-07-22 | Revolution Medicines Inc | Compuestos de piridina, pirazina, y triazina como inhibidores de shp2 alostericos. |
| CA3079727A1 (en) | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of rare diseases |
| EP3479845A1 (en) | 2017-11-06 | 2019-05-08 | Stalicla S.A. | Challenge test for diagnosing subtype of autism spectrum disease |
| WO2019094694A1 (en) | 2017-11-10 | 2019-05-16 | University Of Massachusetts | Compositions and methods for the treatment of expanded repeat-associated disorders |
| JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス, インコーポレイテッド | ハンチントン病を処置するための化合物 |
| US11530207B2 (en) | 2018-04-10 | 2022-12-20 | Skyhawk Therapeutics, Inc. | Compounds for the treatment of cancer |
| EP3814360B8 (en) | 2018-06-27 | 2024-11-06 | PTC Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| CA3105174A1 (en) | 2018-06-27 | 2020-01-02 | Reborna Biosciences, Inc. | Prophylactic or therapeutic agent for spinal muscular atrophy |
| KR20210038845A (ko) | 2018-06-27 | 2021-04-08 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료를 위한 헤테로아릴 화합물 |
| PL3814357T3 (pl) | 2018-06-27 | 2024-09-16 | Ptc Therapeutics, Inc. | Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona |
| US11987568B2 (en) | 2018-08-03 | 2024-05-21 | H. Lee Moffitt Cancer Center And Research Institute, Inc. | Allosteric inhibitor of WEE1 kinase |
| TW202028183A (zh) | 2018-10-10 | 2020-08-01 | 大陸商江蘇豪森藥業集團有限公司 | 含氮雜芳類衍生物調節劑、其製備方法和應用 |
| US11129829B2 (en) * | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| CA3169667A1 (en) | 2020-02-28 | 2021-09-02 | Dominic Reynolds | Pyridazine derivatives for modulating nucleic acid splicing |
| JP2023515620A (ja) | 2020-02-28 | 2023-04-13 | リミックス セラピューティクス インコーポレイテッド | スプライシングを調節するための化合物及び方法 |
| EP4110775A1 (en) | 2020-02-28 | 2023-01-04 | Remix Therapeutics Inc. | Thiophenyl derivatives useful for modulating nucleic acid splicing |
| IL295955A (en) | 2020-02-28 | 2022-10-01 | Remix Therapeutics Inc | Compounds and methods for modulating splicing |
| WO2021207554A1 (en) | 2020-04-08 | 2021-10-14 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| WO2021207530A1 (en) | 2020-04-08 | 2021-10-14 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
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