JP2022153413A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2022153413A5 JP2022153413A5 JP2022108509A JP2022108509A JP2022153413A5 JP 2022153413 A5 JP2022153413 A5 JP 2022153413A5 JP 2022108509 A JP2022108509 A JP 2022108509A JP 2022108509 A JP2022108509 A JP 2022108509A JP 2022153413 A5 JP2022153413 A5 JP 2022153413A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- amino
- alkoxy
- hydroxy
- intron
- 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.)
- Pending
Links
- 125000000217 alkyl group Chemical group 0.000 claims description 738
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 124
- 125000003282 alkyl amino group Chemical group 0.000 claims description 118
- 125000003545 alkoxy group Chemical group 0.000 claims description 106
- GOJUJUVQIVIZAV-UHFFFAOYSA-N 2-amino-4,6-dichloropyrimidine-5-carbaldehyde Chemical group NC1=NC(Cl)=C(C=O)C(Cl)=N1 GOJUJUVQIVIZAV-UHFFFAOYSA-N 0.000 claims description 102
- 239000002773 nucleotide Substances 0.000 claims description 81
- 125000003729 nucleotide group Chemical group 0.000 claims description 81
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical group O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 59
- 125000000623 heterocyclic group Chemical group 0.000 claims description 46
- 108700005078 Synthetic Genes Proteins 0.000 claims description 45
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 43
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 40
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 40
- 125000003118 aryl group Chemical group 0.000 claims description 38
- 238000000034 method Methods 0.000 claims description 37
- 229910052736 halogen Inorganic materials 0.000 claims description 36
- 150000002367 halogens Chemical class 0.000 claims description 36
- 125000001072 heteroaryl group Chemical group 0.000 claims description 36
- 125000001424 substituent group Chemical group 0.000 claims description 36
- 108020004999 messenger RNA Proteins 0.000 claims description 33
- 229930024421 Adenine Natural products 0.000 claims description 29
- 229960000643 adenine Drugs 0.000 claims description 29
- GFFGJBXGBJISGV-UHFFFAOYSA-N adenyl group Chemical group N1=CN=C2N=CNC2=C1N GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 29
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 26
- 238000011144 upstream manufacturing Methods 0.000 claims description 25
- 125000003342 alkenyl group Chemical group 0.000 claims description 24
- 150000001875 compounds Chemical class 0.000 claims description 19
- YZCKVEUIGOORGS-OUBTZVSYSA-N Deuterium Chemical compound [2H] YZCKVEUIGOORGS-OUBTZVSYSA-N 0.000 claims description 18
- 125000000304 alkynyl group Chemical group 0.000 claims description 18
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 18
- 229910052805 deuterium Inorganic materials 0.000 claims description 18
- 150000002431 hydrogen Chemical class 0.000 claims description 18
- 229910052739 hydrogen Inorganic materials 0.000 claims description 18
- 239000001257 hydrogen Substances 0.000 claims description 18
- 108700024394 Exon Proteins 0.000 claims description 17
- ZZUFCTLCJUWOSV-UHFFFAOYSA-N furosemide Chemical compound C1=C(Cl)C(S(=O)(=O)N)=CC(C(O)=O)=C1NCC1=CC=CO1 ZZUFCTLCJUWOSV-UHFFFAOYSA-N 0.000 claims description 14
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims description 12
- 125000005241 heteroarylamino group Chemical group 0.000 claims description 12
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims description 12
- 108091092195 Intron Proteins 0.000 claims description 10
- 108091027974 Mature messenger RNA Proteins 0.000 claims description 10
- 125000004414 alkyl thio group Chemical group 0.000 claims description 10
- 125000001769 aryl amino group Chemical group 0.000 claims description 10
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 8
- 108090000623 proteins and genes Proteins 0.000 claims description 8
- 125000004466 alkoxycarbonylamino group Chemical group 0.000 claims description 6
- 125000003806 alkyl carbonyl amino group Chemical group 0.000 claims description 6
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 6
- 125000005100 aryl amino carbonyl group Chemical group 0.000 claims description 6
- 125000000000 cycloalkoxy group Chemical group 0.000 claims description 6
- 125000001475 halogen functional group Chemical group 0.000 claims description 6
- 125000006517 heterocyclyl carbonyl group Chemical group 0.000 claims description 6
- 125000005844 heterocyclyloxy group Chemical group 0.000 claims description 6
- 239000006166 lysate Substances 0.000 claims description 6
- 239000003607 modifier Substances 0.000 claims description 6
- 125000004043 oxo group Chemical group O=* 0.000 claims description 6
- 239000013592 cell lysate Substances 0.000 claims description 5
- 102000004169 proteins and genes Human genes 0.000 claims description 5
- 230000001105 regulatory effect Effects 0.000 claims description 3
- 230000002068 genetic effect Effects 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 230000035897 transcription Effects 0.000 claims description 2
- 238000013518 transcription Methods 0.000 claims description 2
- 239000013598 vector Substances 0.000 claims 14
- 150000003839 salts Chemical class 0.000 claims 10
- 238000004113 cell culture Methods 0.000 claims 5
- 125000004209 (C1-C8) alkyl group Chemical group 0.000 claims 4
- ZACWYYKZMLGOTG-UHFFFAOYSA-N 2-(4,6-dimethylpyrazolo[1,5-a]pyrazin-2-yl)-7-(1-ethylpiperidin-4-yl)-9-methylpyrido[1,2-a]pyrimidin-4-one Chemical compound C1CN(CC)CCC1C1=CN2C(=O)C=C(C3=NN4C=C(C)N=C(C)C4=C3)N=C2C(C)=C1 ZACWYYKZMLGOTG-UHFFFAOYSA-N 0.000 claims 4
- ZUYOOCCQYSTVKL-UHFFFAOYSA-N 2-(4,6-dimethylpyrazolo[1,5-a]pyrazin-2-yl)-7-(4-methylpiperazin-1-yl)pyrido[1,2-a]pyrimidin-4-one Chemical compound C1CN(C)CCN1C1=CN2C(=O)C=C(C3=NN4C=C(C)N=C(C)C4=C3)N=C2C=C1 ZUYOOCCQYSTVKL-UHFFFAOYSA-N 0.000 claims 4
- GYFRQCMDLBNZSF-UHFFFAOYSA-N 2-(4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methylpiperidin-4-yl)pyrido[1,2-a]pyrimidin-4-one Chemical class C1=C2C(CC)=NC(C)=CN2N=C1C(N=C1C(C)=C2)=CC(=O)N1C=C2C1CCN(C)CC1 GYFRQCMDLBNZSF-UHFFFAOYSA-N 0.000 claims 4
- 239000002253 acid Substances 0.000 claims 2
- 125000006310 cycloalkyl amino group Chemical group 0.000 claims 2
- 239000012458 free base Substances 0.000 claims 2
- 238000001415 gene therapy Methods 0.000 claims 2
- 238000010353 genetic engineering Methods 0.000 claims 2
- 125000000446 sulfanediyl group Chemical group *S* 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 241000710929 Alphavirus Species 0.000 claims 1
- 241000711404 Avian avulavirus 1 Species 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 108091034057 RNA (poly(A)) Proteins 0.000 claims 1
- 241000700618 Vaccinia virus Species 0.000 claims 1
- 230000001225 therapeutic effect Effects 0.000 claims 1
- 230000005030 transcription termination Effects 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241001529453 unidentified herpesvirus Species 0.000 claims 1
- 241001430294 unidentified retrovirus Species 0.000 claims 1
- 229910018540 Si C Inorganic materials 0.000 description 8
- 229910010271 silicon carbide Inorganic materials 0.000 description 8
- 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 description 6
- 241001024304 Mino Species 0.000 description 5
- TVEXGJYMHHTVKP-UHFFFAOYSA-N 6-oxabicyclo[3.2.1]oct-3-en-7-one Chemical compound C1C2C(=O)OC1C=CC2 TVEXGJYMHHTVKP-UHFFFAOYSA-N 0.000 description 4
- 150000001412 amines Chemical class 0.000 description 4
- 210000004556 brain Anatomy 0.000 description 4
- 239000000126 substance Substances 0.000 description 4
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Chemical group C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 description 1
- 240000007594 Oryza sativa Species 0.000 description 1
- 235000007164 Oryza sativa Nutrition 0.000 description 1
- 235000009566 rice Nutrition 0.000 description 1
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662426619P | 2016-11-28 | 2016-11-28 | |
| US62/426,619 | 2016-11-28 | ||
| JP2019528527A JP2019535789A (ja) | 2016-11-28 | 2017-11-27 | Ｒｎａスプライシングを調節する方法 |
| PCT/US2017/063323 WO2018098446A1 (en) | 2016-11-28 | 2017-11-27 | Methods for modulating rna splicing |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019528527A Division JP2019535789A (ja) | 2016-11-28 | 2017-11-27 | Ｒｎａスプライシングを調節する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022153413A JP2022153413A (ja) | 2022-10-12 |
| JP2022153413A5 true JP2022153413A5 (enExample) | 2023-01-31 |
Family
ID=62195368
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019528527A Pending JP2019535789A (ja) | 2016-11-28 | 2017-11-27 | Ｒｎａスプライシングを調節する方法 |
| JP2022108509A Pending JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Ｒｎａスプライシングを調節する方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019528527A Pending JP2019535789A (ja) | 2016-11-28 | 2017-11-27 | Ｒｎａスプライシングを調節する方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11702646B2 (enExample) |
| EP (1) | EP3544435A4 (enExample) |
| JP (2) | JP2019535789A (enExample) |
| CN (1) | CN110352007A (enExample) |
| AU (1) | AU2017363369A1 (enExample) |
| CA (1) | CA3043755A1 (enExample) |
| EA (1) | EA201991309A1 (enExample) |
| MX (1) | MX2019005588A (enExample) |
| WO (1) | WO2018098446A1 (enExample) |
Families Citing this family (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10668171B2 (en) | 2015-05-30 | 2020-06-02 | Ptc Therapeutics, Inc. | Methods for modulating RNA splicing |
| JP2019535789A (ja) | 2016-11-28 | 2019-12-12 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを調節する方法 |
| JP2020523365A (ja) | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを改変する方法 |
| CN111194215B (zh) | 2017-08-04 | 2024-03-01 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| CN112805009A (zh) * | 2018-08-07 | 2021-05-14 | 费城儿童医院 | 基因表达的选择性剪接调控及治疗方法 |
| JP2022504166A (ja) * | 2018-10-09 | 2022-01-13 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 調節された遺伝子編集システム |
| WO2020163405A1 (en) | 2019-02-05 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| JP7603595B2 (ja) | 2019-02-06 | 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 |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| WO2021014428A1 (en) | 2019-07-25 | 2021-01-28 | Novartis Ag | Regulatable expression systems |
| BR112022002905A2 (pt) * | 2019-08-19 | 2022-07-12 | Stoke Therapeutics Inc | Composições e métodos para modular splicing e expressão de proteína |
| BR112022010561A2 (pt) | 2019-12-02 | 2022-11-16 | Storm Therapeutics Ltd | Compostos poli-heterocíclicos como inibidores de mettl3 |
| KR20220146501A (ko) * | 2020-02-12 | 2022-11-01 | 더 칠드런스 호스피탈 오브 필라델피아 | 유전자 발현의 유도성 대체 스플라이싱 조절을 위한 조성물 및 방법 |
| BR112022017089A2 (pt) | 2020-02-28 | 2022-11-16 | Remix Therapeutics Inc | Derivados de piridazina para modular o splicing de ácido nucleico |
| CA3169697A1 (en) | 2020-02-28 | 2021-09-02 | Dominic Reynolds | Thiophenyl derivatives useful for modulating nucleic acid splicing |
| EP4110459A1 (en) | 2020-02-28 | 2023-01-04 | Remix Therapeutics Inc. | Heterocyclic amides and their use for modulating splicing |
| WO2021174167A1 (en) | 2020-02-28 | 2021-09-02 | 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 |
| EP4132935A1 (en) | 2020-04-08 | 2023-02-15 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| MX2022013856A (es) | 2020-05-13 | 2023-04-05 | Chdi Foundation Inc | Moduladores htt para tratar la enfermedad de huntington. |
| JP2023532331A (ja) | 2020-07-02 | 2023-07-27 | リミックス セラピューティクス インコーポレイテッド | 核酸をスプライシングするため及び増殖性疾患を処置するための修飾因子としての５－［５－（ピペリジン－４－イル）チエノ［３，２－ｃ］ピラゾール－２－イル］インダゾール誘導体及び関連する化合物並びにスプライシングを調節するための化合物及び方法 |
| AU2021299515A1 (en) | 2020-07-02 | 2023-02-02 | 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 |
| WO2022214520A1 (en) * | 2021-04-07 | 2022-10-13 | Janssen Sciences Ireland Unlimited Company | Antibacterial compounds |
| CN116087519B (zh) * | 2021-05-17 | 2025-03-14 | 郑州大学第一附属医院 | 一种用于高危人群贲门腺癌早期筛查的标志物及其应用 |
| CN113604566B (zh) * | 2021-07-13 | 2023-05-16 | 中山大学孙逸仙纪念医院 | lncRNA BCYRN1在膀胱癌预后、治疗中的应用 |
| US20240400584A1 (en) | 2021-08-30 | 2024-12-05 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| EP4396177A1 (en) | 2021-08-30 | 2024-07-10 | 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 |
| PE20251687A1 (es) | 2021-08-30 | 2025-07-02 | Remix Therapeutics Inc | Compuestos y metodos para modular splicing |
| WO2023034836A1 (en) | 2021-08-30 | 2023-03-09 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| EP4416156A1 (en) | 2021-10-13 | 2024-08-21 | Remix Therapeutics Inc. | Compounds and methods for modulating nucleic acid splicing |
| EP4416141A1 (en) | 2021-10-13 | 2024-08-21 | Remix Therapeutics Inc. | Compounds and methods for modulating nucleic acid splicing |
| JP2024540477A (ja) | 2021-11-17 | 2024-10-31 | シーエイチディーアイ ファウンデーション，インコーポレーテッド | ハンチントン病を治療するためのｈｔｔモジュレータとしてのｎ－（２ｈ－インダゾール－５－イル）ピラジン－２－カルボキサミド誘導体及び類似化合物 |
| 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 |
| WO2023133229A2 (en) | 2022-01-05 | 2023-07-13 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| TW202346305A (zh) | 2022-01-05 | 2023-12-01 | 美商雷密克斯醫療公司 | 用於調節剪切之化合物及方法 |
| CN118647405A (zh) * | 2022-01-25 | 2024-09-13 | 上海魁特迪生物科技有限公司 | 改善认知障碍的方法 |
| EP4568962A2 (en) * | 2022-08-09 | 2025-06-18 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
Family Cites Families (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3558618A (en) | 1968-04-01 | 1971-01-26 | Dow Chemical Co | Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones |
| US4122274A (en) | 1977-05-25 | 1978-10-24 | Bristol-Myers Company | 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones |
| US4342870A (en) | 1980-03-28 | 1982-08-03 | Janssen Pharmaceutica N.V. | Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives |
| JPS56150091A (en) | 1980-03-28 | 1981-11-20 | Janssen Pharmaceutica Nv | 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture |
| US5089633A (en) | 1987-04-28 | 1992-02-18 | Georgia Tech Research Corporation | Substituted isocoumarins |
| US5726182A (en) | 1990-05-02 | 1998-03-10 | Abbott Laboratories | Quinolizinone type compounds |
| AU4231293A (en) | 1992-05-13 | 1993-12-13 | E.I. Du Pont De Nemours And Company | Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides |
| EP0871628A1 (en) | 1995-06-06 | 1998-10-21 | Abbott Laboratories | Quinolizinone type compounds |
| US5869500A (en) | 1996-12-13 | 1999-02-09 | Hoffmann-La Roche Inc. | Pyridone compounds useful in treating Alzheimer's disease |
| HK1040076A1 (zh) | 1998-09-21 | 2002-05-24 | 希雷生物化学有限公司 | 作为整联蛋白抑制剂的喹嗪酮 |
| CN1414951A (zh) | 1999-10-28 | 2003-04-30 | 基本治疗公司 | 药剂排出泵抑制药 |
| CN1263743C (zh) | 2000-01-24 | 2006-07-12 | 基纳西亚股份有限公司 | 用于治疗的吗啉代基取代的化合物 |
| US7214515B2 (en) | 2001-01-05 | 2007-05-08 | The General Hospital Corporation | Viral delivery system for infectious transfer of large genomic DNA inserts |
| US20050009843A1 (en) | 2001-04-26 | 2005-01-13 | Kiyoshi Nakayama | Medicine for inhibiting drug elimination pump |
| AU2002364536B2 (en) | 2001-12-07 | 2008-10-23 | Vertex Pharmaceuticals, Inc. | Pyrimidine-based compounds useful as GSK-3 inhibitors |
| GB0205281D0 (en) | 2002-03-06 | 2002-04-17 | Novartis Ag | Organic compounds |
| CA2493458A1 (en) | 2002-07-24 | 2004-01-29 | Ptc Therapeutics, Inc. | Ureido substituted benzoic acid compounds, their use for nonsense suppression and the treatment of diseases caused by such mutations |
| US9068234B2 (en) | 2003-01-21 | 2015-06-30 | Ptc Therapeutics, Inc. | Methods and agents for screening for compounds capable of modulating gene expression |
| AU2004249730A1 (en) | 2003-06-20 | 2004-12-29 | Novartis Vaccines And Diagnostics, Inc. | Pyridino(1,2-A)pyrimidin-4-one compounds as anticancer agents |
| ES2292130T3 (es) | 2004-05-04 | 2008-03-01 | Warner-Lambert Company Llc | Pirido(2,3-d)pirimidin-7-onas pirrolil-sustituidas y derivados de las mismas como agentes terapeuticos. |
| EP1846397A1 (en) | 2005-01-21 | 2007-10-24 | Janssen Pharmaceutica N.V. | Novel heterocyclic benzoy[c]chromene derivatives useful as modulators of the estrogen receptors |
| JP2008539698A (ja) * | 2005-04-29 | 2008-11-20 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 転写後レベルでの核酸発現調節のための方法および組成物 |
| US7563601B1 (en) | 2005-06-01 | 2009-07-21 | City Of Hope | Artificial riboswitch for controlling pre-mRNA splicing |
| EP1910569A4 (en) | 2005-06-17 | 2009-08-05 | Genizon Biosciences Inc | GENEMAP GENE CARD OF HUMAN GENES ASSOCIATED WITH LONGEVITY |
| US20070243617A1 (en) | 2005-10-13 | 2007-10-18 | Holt Robert A | Modular genomes for synthetic biology and metabolic engineering |
| AR059339A1 (es) | 2006-02-09 | 2008-03-26 | Chugai Pharmaceutical Co Ltd | Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene |
| US8110681B2 (en) | 2006-03-17 | 2012-02-07 | The United States Of America As Represented By The Secretary, Department Of Health And Human Services | Compounds for the treatment of spinal muscular atrophy and other uses |
| US8337941B2 (en) | 2006-07-27 | 2012-12-25 | The Trustees Of Columbia University In The City Of New York | Fluorescent substrates for monoamine transporters as optical false neurotransmitters |
| EP2269992A1 (en) | 2006-12-22 | 2011-01-05 | Avexa Limited | Bicyclic pyrimidinones for the treatment of viral infections |
| WO2009151546A2 (en) * | 2008-05-27 | 2009-12-17 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| EP2138493A1 (en) | 2008-06-26 | 2009-12-30 | Sanofi-Aventis | Substituted pyrimidone derivatives |
| EP2759544A1 (en) | 2008-07-02 | 2014-07-30 | Avexa Limited | Compounds having antiviral properties |
| WO2010019236A1 (en) | 2008-08-13 | 2010-02-18 | Ptc Therapeutics, Inc | Methods for treating spinal muscular atrophy |
| WO2010083338A2 (en) | 2009-01-14 | 2010-07-22 | Philadelphia Health And Education Corporation | Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease |
| US20100303776A1 (en) | 2009-04-16 | 2010-12-02 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of multiple nucleic acids |
| WO2011050245A1 (en) | 2009-10-23 | 2011-04-28 | Yangbo Feng | Bicyclic heteroaryls as kinase inhibitors |
| US8754220B2 (en) | 2009-11-20 | 2014-06-17 | Merck Sharp & Dohme Corp. | Quinolizidinone carboxamide M1 receptor positive allosteric modulators |
| BR112012017473A2 (pt) | 2010-01-13 | 2019-09-24 | Institut National De La Sante Et De La Rech Medicale Inserm Epst | pirido(1,2-a) pirimidinas, seu uso, bem como composição farmacêutica compreendendo tais compostos |
| WO2012047487A1 (en) | 2010-09-27 | 2012-04-12 | Evolva Sa | 2-pyridone antimicrobial compositions |
| US9662314B2 (en) | 2011-10-21 | 2017-05-30 | Tufts Medical Center, Inc. | Compounds and methods for the treatment of muscular disease, and related screening methods |
| CA2861609C (en) | 2011-12-30 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2862084C (en) | 2012-01-26 | 2021-05-11 | Ptc Therapeutics, Inc. | 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy |
| PL2812004T3 (pl) * | 2012-02-10 | 2019-01-31 | Ptc Therapeutics, Inc. | Związki do leczenia rdzeniowego zaniku mięśni |
| EA029155B1 (ru) | 2012-03-01 | 2018-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения спинальной мышечной атрофии |
| MX358514B (es) | 2012-03-23 | 2018-08-24 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
| EP2872493B1 (en) | 2012-07-13 | 2018-11-14 | Indiana University Research and Technology Corporation | 5,6,7-trimethoxy 4-phenyl quinolin-2-one derivatives for treatment of spinal muscular atrophy |
| US9040712B2 (en) | 2013-01-23 | 2015-05-26 | Novartis Ag | Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions |
| RU2016109324A (ru) | 2013-08-19 | 2017-09-26 | Ф. Хоффманн-Ля Рош Аг | Способ скрининга |
| WO2015095449A1 (en) | 2013-12-19 | 2015-06-25 | Ptc Therapeutics, Inc. | Methods for modulating the amount rna transcripts |
| US10195202B2 (en) | 2013-12-19 | 2019-02-05 | Ptc Therapeutics, Inc. | Methods for modulating the amount of RNA transcripts |
| WO2015095446A1 (en) | 2013-12-19 | 2015-06-25 | Ptc Therapeutics, Inc. | Methods for modulating the amount of rna transcripts |
| PE20170128A1 (es) | 2014-05-15 | 2017-03-16 | Ptc Therapeutics Inc | Compuestos para tratar atrofia muscular espinal |
| WO2016042015A1 (en) * | 2014-09-16 | 2016-03-24 | Centre National De La Recherche Scientifique (Cnrs) | Method for evaluating developmental competence of an oocyte |
| JP6884102B2 (ja) * | 2015-02-09 | 2021-06-09 | エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト | がんの治療のための化合物 |
| CN107635999B (zh) | 2015-05-20 | 2020-09-25 | 豪夫迈·罗氏有限公司 | 用于治疗脊髓性肌萎缩的化合物 |
| US10668171B2 (en) | 2015-05-30 | 2020-06-02 | Ptc Therapeutics, Inc. | Methods for modulating RNA splicing |
| JP2019535789A (ja) | 2016-11-28 | 2019-12-12 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを調節する方法 |
| JP2020523365A (ja) | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを改変する方法 |
| CN111194215B (zh) | 2017-08-04 | 2024-03-01 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| JP7195328B2 (ja) | 2017-09-25 | 2022-12-23 | 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 | スプライシングモジュレーターのスクリーニングおよび特定を行うための方法および組成物 |
-
2017
- 2017-11-27 JP JP2019528527A patent/JP2019535789A/ja active Pending
- 2017-11-27 EP EP17873550.2A patent/EP3544435A4/en not_active Withdrawn
- 2017-11-27 EA EA201991309A patent/EA201991309A1/ru unknown
- 2017-11-27 AU AU2017363369A patent/AU2017363369A1/en not_active Abandoned
- 2017-11-27 CA CA3043755A patent/CA3043755A1/en active Pending
- 2017-11-27 WO PCT/US2017/063323 patent/WO2018098446A1/en not_active Ceased
- 2017-11-27 CN CN201780084649.8A patent/CN110352007A/zh active Pending
- 2017-11-27 MX MX2019005588A patent/MX2019005588A/es unknown
- 2017-11-27 US US16/463,775 patent/US11702646B2/en active Active
-
2022
- 2022-07-05 JP JP2022108509A patent/JP2022153413A/ja active Pending
-
2023
- 2023-05-12 US US18/316,871 patent/US20230272367A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022153413A5 (enExample) | ||
| JP2019535789A5 (enExample) | ||
| JP6906591B2 (ja) | 筋特異的核酸調節エレメント並びにその方法及び使用 | |
| JP2020523365A5 (enExample) | ||
| Babij et al. | Characterization of a mammalian smooth muscle myosin heavy-chain gene: complete nucleotide and protein coding sequence and analysis of the 5'end of the gene. | |
| EP3710009B1 (en) | Processes for the resolution of benzodiazepin-2-one and benzoazepin-2-one derivatives | |
| ES2643576T3 (es) | Dúplex oligonucleotídicos que comprenden nucleótidos de tipo ADN y de tipo ARN y usos de los mismos | |
| JP5823969B2 (ja) | 心臓特異的核酸調節因子ならびにこの方法および使用 | |
| JP2020523365A (ja) | Ｒｎａスプライシングを改変する方法 | |
| Nicolaides et al. | Analysis of the 5′ Region ofPMS2Reveals Heterogeneous Transcripts and a Novel Overlapping Gene | |
| TW201923077A (zh) | 用於基因組編輯之多核苷酸、組合物及方法 | |
| Järving et al. | On the evolutionary origin of cyclooxygenase (COX) isozymes: characterization of marine invertebrate COX genes points to independent duplication events in vertebrate and invertebrate lineages | |
| ES2623464T3 (es) | Nuevo factor de viabilidad neuronal y su uso para tratar la distrofia de conos | |
| CA2831342A1 (en) | Medicament for liver regeneration and for treatment of liver failure | |
| JP2022536951A (ja) | 環状ポリリボヌクレオチドを投与する方法 | |
| WO2020097049A1 (en) | Vector system for expressing regulatory rna | |
| US20170029786A1 (en) | Two plasmid mammalian expression system | |
| EP0852620B1 (en) | Human jtv1 gene overlaps pms2 gene | |
| Lu et al. | Characterization of small nontranslated polyadenylylated RNAs in vaccinia virus-infected cells. | |
| ES2256885T3 (es) | Nuevas quimiocinas del tipo cc. | |
| WO2016104906A1 (ko) | 형질전환동물 제작을 위한 형질주입 시스템 | |
| WO2019203317A1 (ja) | 増殖性疾患を処置するための医薬組成物 | |
| CA3132420A1 (en) | Expression constructs for the genetic modification of cells | |
| CN102807622A (zh) | 可靶向激活traf6信号通路的融合蛋白及其重组载体与应用 | |
| CN100427599C (zh) | Albsin/il－18mat杂交细胞因子 |