JP2022153413A5 - - Google Patents
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- 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
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- 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.)
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- 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 (https=) | 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 (https=) |
| EP (1) | EP3544435A4 (https=) |
| JP (2) | JP2019535789A (https=) |
| CN (1) | CN110352007A (https=) |
| AU (1) | AU2017363369A1 (https=) |
| CA (1) | CA3043755A1 (https=) |
| EA (1) | EA201991309A1 (https=) |
| MX (1) | MX2019005588A (https=) |
| WO (1) | WO2018098446A1 (https=) |
Families Citing this family (41)
| 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 |
| JP7312749B2 (ja) | 2017-08-04 | 2023-07-21 | 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 | スプライシングをモジュレートする方法および組成物 |
| US20210355509A1 (en) * | 2018-08-07 | 2021-11-18 | The Children's Hospital Of Philadelphia | Alternative splicing regulation of gene expression and therapeutic methods |
| CN113166779A (zh) * | 2018-10-09 | 2021-07-23 | 北卡罗来纳大学查佩尔希尔分校 | 调控的基因编辑系统 |
| JP7551629B2 (ja) | 2019-02-05 | 2024-09-17 | 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 |
| 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 |
| JP7603594B2 (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 |
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| EP4103717A4 (en) * | 2020-02-12 | 2024-02-28 | The Children's Hospital of Philadelphia | COMPOSITIONS AND METHODS FOR INDUCIBLE ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION |
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-
2017
- 2017-11-27 JP JP2019528527A patent/JP2019535789A/ja active Pending
- 2017-11-27 WO PCT/US2017/063323 patent/WO2018098446A1/en not_active Ceased
- 2017-11-27 MX MX2019005588A patent/MX2019005588A/es unknown
- 2017-11-27 CN CN201780084649.8A patent/CN110352007A/zh active Pending
- 2017-11-27 EP EP17873550.2A patent/EP3544435A4/en not_active Withdrawn
- 2017-11-27 AU AU2017363369A patent/AU2017363369A1/en not_active Abandoned
- 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 EA EA201991309A patent/EA201991309A1/ru unknown
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2022
- 2022-07-05 JP JP2022108509A patent/JP2022153413A/ja active Pending
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2023
- 2023-05-12 US US18/316,871 patent/US20230272367A1/en not_active Abandoned
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