JP2019535789A5 - - Google Patents
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- JP2019535789A5 JP2019535789A5 JP2019528527A JP2019528527A JP2019535789A5 JP 2019535789 A5 JP2019535789 A5 JP 2019535789A5 JP 2019528527 A JP2019528527 A JP 2019528527A JP 2019528527 A JP2019528527 A JP 2019528527A JP 2019535789 A5 JP2019535789 A5 JP 2019535789A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- amino
- alkoxy
- hydroxy
- heterocyclyl
- 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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- 125000000217 alkyl group Chemical group 0.000 claims description 1170
- 125000003545 alkoxy group Chemical group 0.000 claims description 402
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 214
- 125000003282 alkyl amino group Chemical group 0.000 claims description 208
- 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 204
- 125000000623 heterocyclic group Chemical group 0.000 claims description 90
- 239000002773 nucleotide Substances 0.000 claims description 79
- 125000003729 nucleotide group Chemical group 0.000 claims description 79
- 125000003118 aryl group Chemical group 0.000 claims description 60
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 60
- 229910052736 halogen Inorganic materials 0.000 claims description 60
- 150000002367 halogens Chemical class 0.000 claims description 60
- 125000001072 heteroaryl group Chemical group 0.000 claims description 60
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical group O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 58
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 54
- 125000001424 substituent group Chemical group 0.000 claims description 50
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 48
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 40
- 108020004999 messenger RNA Proteins 0.000 claims description 35
- 238000000034 method Methods 0.000 claims description 35
- YZCKVEUIGOORGS-OUBTZVSYSA-N Deuterium Chemical compound [2H] YZCKVEUIGOORGS-OUBTZVSYSA-N 0.000 claims description 30
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 30
- 229910052805 deuterium Inorganic materials 0.000 claims description 30
- 150000002431 hydrogen Chemical class 0.000 claims description 30
- 229910052739 hydrogen Inorganic materials 0.000 claims description 30
- 239000001257 hydrogen Substances 0.000 claims description 30
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical group NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 29
- 229930024421 Adenine Natural products 0.000 claims description 29
- 108700005078 Synthetic Genes Proteins 0.000 claims description 25
- 238000011144 upstream manufacturing Methods 0.000 claims description 22
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims description 20
- 125000005241 heteroarylamino group Chemical group 0.000 claims description 20
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims description 20
- 125000001475 halogen functional group Chemical group 0.000 claims description 16
- 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 description 15
- 229960000643 adenine Drugs 0.000 claims description 15
- 108700024394 Exon Proteins 0.000 claims description 12
- 108091027974 Mature messenger RNA Proteins 0.000 claims description 12
- 150000001875 compounds Chemical class 0.000 claims description 12
- 125000004466 alkoxycarbonylamino group Chemical group 0.000 claims description 10
- 125000003806 alkyl carbonyl amino group Chemical group 0.000 claims description 10
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 10
- 125000004414 alkyl thio group Chemical group 0.000 claims description 10
- 125000005100 aryl amino carbonyl group Chemical group 0.000 claims description 10
- 125000001769 aryl amino group Chemical group 0.000 claims description 10
- 125000000000 cycloalkoxy group Chemical group 0.000 claims description 10
- 125000006310 cycloalkyl amino group Chemical group 0.000 claims description 10
- 125000006517 heterocyclyl carbonyl group Chemical group 0.000 claims description 10
- 125000005844 heterocyclyloxy group Chemical group 0.000 claims description 10
- 125000004043 oxo group Chemical group O=* 0.000 claims description 10
- 125000000446 sulfanediyl group Chemical group *S* 0.000 claims description 10
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 9
- 108091092195 Intron Proteins 0.000 claims description 9
- 108090000623 proteins and genes Proteins 0.000 claims description 9
- 239000003607 modifier Substances 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 5
- 239000006166 lysate Substances 0.000 claims description 4
- 238000004519 manufacturing process Methods 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 239000013592 cell lysate Substances 0.000 claims description 2
- 125000000033 alkoxyamino group Chemical group 0.000 claims 36
- -1 amino, amino Chemical group 0.000 claims 12
- 125000006193 alkinyl group Chemical group 0.000 claims 6
- 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 6
- 239000013598 vector Substances 0.000 claims 3
- 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
- 230000001276 controlling effect Effects 0.000 claims 1
- 238000001415 gene therapy Methods 0.000 claims 1
- 230000035897 transcription Effects 0.000 claims 1
- 238000013518 transcription Methods 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
- 125000003342 alkenyl group Chemical group 0.000 description 16
- 125000000304 alkynyl group Chemical group 0.000 description 12
- 239000000126 substance Substances 0.000 description 8
- RODSCGMZJVUSFO-UHFFFAOYSA-N CC(C)N(C)C=C Chemical compound CC(C)N(C)C=C RODSCGMZJVUSFO-UHFFFAOYSA-N 0.000 description 1
- 0 CC1(C)*CCC1 Chemical compound CC1(C)*CCC1 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022108509A JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Ｒｎａスプライシングを調節する方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662426619P | 2016-11-28 | 2016-11-28 | |
| US62/426,619 | 2016-11-28 | ||
| PCT/US2017/063323 WO2018098446A1 (en) | 2016-11-28 | 2017-11-27 | Methods for modulating rna splicing |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022108509A Division JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Ｒｎａスプライシングを調節する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019535789A JP2019535789A (ja) | 2019-12-12 |
| JP2019535789A5 true JP2019535789A5 (cg-RX-API-DMAC7.html) | 2021-06-17 |
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 After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11702646B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3544435A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP2019535789A (cg-RX-API-DMAC7.html) |
| CN (1) | CN110352007A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017363369A1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3043755A1 (cg-RX-API-DMAC7.html) |
| EA (1) | EA201991309A1 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2019005588A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018098446A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (38)
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| CN111372611A (zh) | 2017-06-14 | 2020-07-03 | Ptc医疗公司 | 修饰rna剪接的方法 |
| IL272413B (en) | 2017-08-04 | 2022-09-01 | Skyhawk Therapeutics Inc | Methods and compositions for modulating splicing |
| EP3833357A4 (en) * | 2018-08-07 | 2022-06-08 | The Children's Hospital of Philadelphia | REGULATION OF GENE EXPRESSION BY ALTERNATIVE SPLICING, AND THERAPEUTIC METHODS |
| WO2020076892A1 (en) * | 2018-10-09 | 2020-04-16 | The University Of North Carolina At Chapel Hill | Regulated gene editing system |
| CN114007613A (zh) | 2019-02-05 | 2022-02-01 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| EP3920928A4 (en) | 2019-02-06 | 2022-09-28 | Skyhawk Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| US20220307053A1 (en) | 2019-07-25 | 2022-09-29 | Novartis Ag | Regulatable expression systems |
| WO2021034985A1 (en) * | 2019-08-19 | 2021-02-25 | Stoke Therapeutics, Inc. | Compositions and methods for modulating splicing and protein expression |
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| IL295955A (en) | 2020-02-28 | 2022-10-01 | Remix Therapeutics Inc | Compounds and methods for modulating splicing |
| EP4110775A1 (en) | 2020-02-28 | 2023-01-04 | Remix Therapeutics Inc. | Thiophenyl derivatives useful for modulating nucleic acid splicing |
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| CN116134036A (zh) | 2020-04-08 | 2023-05-16 | 雷密克斯医疗公司 | 用于调节剪接的化合物和方法 |
| JP2023520916A (ja) | 2020-04-08 | 2023-05-22 | 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整するための化合物及び方法 |
| AU2021272972A1 (en) | 2020-05-13 | 2022-12-08 | Chdi Foundation, Inc. | HTT modulators for treating Huntington's disease |
| 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 |
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| 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 |
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| 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 |
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| WO2016042015A1 (en) * | 2014-09-16 | 2016-03-24 | Centre National De La Recherche Scientifique (Cnrs) | Method for evaluating developmental competence of an oocyte |
| EP3256126B1 (en) * | 2015-02-09 | 2024-03-27 | F. Hoffmann-La Roche AG | Compounds for the treatment of cancer |
| 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 |
| EP4249472A3 (en) | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| CN110352007A (zh) | 2016-11-28 | 2019-10-18 | Ptc医疗公司 | 用于调节rna剪接的方法 |
| CN111372611A (zh) | 2017-06-14 | 2020-07-03 | Ptc医疗公司 | 修饰rna剪接的方法 |
| IL272413B (en) | 2017-08-04 | 2022-09-01 | Skyhawk Therapeutics Inc | Methods and compositions for modulating splicing |
| WO2019060917A2 (en) | 2017-09-25 | 2019-03-28 | Skyhawk Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR SCREENING AND IDENTIFYING SPREADING MODULATORS |
-
2017
- 2017-11-27 CN CN201780084649.8A patent/CN110352007A/zh active Pending
- 2017-11-27 MX MX2019005588A patent/MX2019005588A/es unknown
- 2017-11-27 WO PCT/US2017/063323 patent/WO2018098446A1/en not_active Ceased
- 2017-11-27 JP JP2019528527A patent/JP2019535789A/ja active Pending
- 2017-11-27 EA EA201991309A patent/EA201991309A1/ru unknown
- 2017-11-27 EP EP17873550.2A patent/EP3544435A4/en not_active Withdrawn
- 2017-11-27 CA CA3043755A patent/CA3043755A1/en active Pending
- 2017-11-27 US US16/463,775 patent/US11702646B2/en active Active
- 2017-11-27 AU AU2017363369A patent/AU2017363369A1/en not_active Abandoned
-
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
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