CA3065547A1 - Methods for modifying rna splicing - Google Patents
Methods for modifying rna splicing Download PDFInfo
- Publication number
- CA3065547A1 CA3065547A1 CA3065547A CA3065547A CA3065547A1 CA 3065547 A1 CA3065547 A1 CA 3065547A1 CA 3065547 A CA3065547 A CA 3065547A CA 3065547 A CA3065547 A CA 3065547A CA 3065547 A1 CA3065547 A1 CA 3065547A1
- Authority
- CA
- Canada
- Prior art keywords
- amino
- 4alkyl
- carbonyl
- seq
- 4alkoxy
- 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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Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/433—Thidiazoles
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/454—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/50—Pyridazines; Hydrogenated pyridazines
- A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
- A61K31/551—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having two nitrogen atoms, e.g. dilazep
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
- C07D401/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
- C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762519226P | 2017-06-14 | 2017-06-14 | |
| US62/519,226 | 2017-06-14 | ||
| PCT/US2018/037412 WO2018232039A1 (en) | 2017-06-14 | 2018-06-13 | Methods for modifying rna splicing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3065547A1 true CA3065547A1 (en) | 2018-12-20 |
Family
ID=64659912
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3065547A Pending CA3065547A1 (en) | 2017-06-14 | 2018-06-13 | Methods for modifying rna splicing |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US11608501B2 (https=) |
| EP (1) | EP3638318A4 (https=) |
| JP (1) | JP2020523365A (https=) |
| KR (1) | KR20200017476A (https=) |
| CN (1) | CN111372611A (https=) |
| AU (1) | AU2018284853A1 (https=) |
| BR (1) | BR112019026508A2 (https=) |
| CA (1) | CA3065547A1 (https=) |
| EA (1) | EA202090034A1 (https=) |
| IL (1) | IL271053A (https=) |
| WO (1) | WO2018232039A1 (https=) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4249472A3 (en) | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| EA201991309A1 (ru) | 2016-11-28 | 2019-11-29 | Способы модуляции сплайсинга рнк | |
| KR20200017476A (ko) | 2017-06-14 | 2020-02-18 | 피티씨 테라퓨틱스, 인크. | Rna 스플라이싱의 변경 방법 |
| WO2019028440A1 (en) | 2017-08-04 | 2019-02-07 | Skyhawk Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODULATING SPLICING |
| WO2019126731A1 (en) | 2017-12-22 | 2019-06-27 | Petra Pharma Corporation | Aminopyridine derivatives as phosphatidylinositol phosphate kinase inhibitors |
| WO2020163382A1 (en) * | 2019-02-04 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| CN113692402A (zh) * | 2019-02-04 | 2021-11-23 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| WO2020163248A1 (en) | 2019-02-04 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| JP2022519294A (ja) * | 2019-02-04 | 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 |
| EP3920918A4 (en) * | 2019-02-05 | 2022-11-16 | Skyhawk Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING |
| CN114126613A (zh) * | 2019-02-05 | 2022-03-01 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| CN113677342A (zh) * | 2019-02-05 | 2021-11-19 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| KR20210134657A (ko) * | 2019-02-05 | 2021-11-10 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| EP3920915A4 (en) | 2019-02-05 | 2022-10-05 | Skyhawk Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODULATING SPLICE |
| WO2020163544A1 (en) * | 2019-02-06 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| CN113661162A (zh) | 2019-02-06 | 2021-11-16 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| AR119731A1 (es) | 2019-05-17 | 2022-01-05 | Novartis Ag | Inhibidores del inflamasoma nlrp3 |
| EP3982977A4 (en) * | 2019-06-14 | 2023-07-05 | Children's Medical Research Institute | Methods of treating cancer with an inhibitor of znf827 |
| TW202112767A (zh) | 2019-06-17 | 2021-04-01 | 美商佩特拉製藥公司 | 作為磷脂酸肌醇磷酸激酶抑制劑之胺基吡啶衍生物 |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| EP4004213A1 (en) * | 2019-07-25 | 2022-06-01 | Novartis AG | Regulatable expression systems |
| AU2020377204A1 (en) | 2019-11-01 | 2022-06-02 | Novartis Ag | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
| CN120025336A (zh) * | 2019-12-18 | 2025-05-23 | 加利福尼亚大学董事会 | Lin28抑制剂和其使用方法 |
| EP4084796A1 (en) * | 2020-01-02 | 2022-11-09 | The General Hospital Corporation | Rna splicing modulation |
| JP7736700B2 (ja) | 2020-02-28 | 2025-09-09 | リミックス セラピューティクス インコーポレイテッド | 複素環アミド及びスプライシングを調節するためのその使用 |
| WO2021174167A1 (en) | 2020-02-28 | 2021-09-02 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| CA3169697A1 (en) | 2020-02-28 | 2021-09-02 | Dominic Reynolds | Thiophenyl derivatives useful for modulating nucleic acid splicing |
| MX2022010634A (es) * | 2020-02-28 | 2023-01-19 | Remix Therapeutics Inc | Derivados de piridazina para modular el empalme de acidos nucleicos. |
| 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 |
| KR20230005210A (ko) | 2020-04-08 | 2023-01-09 | 레믹스 테라퓨틱스 인크. | 스플라이싱을 조절하기 위한 화합물 및 방법 |
| BR112022023025A2 (pt) | 2020-05-13 | 2023-03-28 | Chdi Foundation Inc | Moduladores de htt para tratar doença de huntington |
| CN116157135A (zh) * | 2020-06-03 | 2023-05-23 | 纽约市哥伦比亚大学理事会 | 使用基于crispr的碱基编辑器系统抑制谱系特异性抗原的组合物和方法 |
| 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 |
| EP4175956A1 (en) | 2020-07-02 | 2023-05-10 | 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 |
| WO2022093835A1 (en) * | 2020-10-26 | 2022-05-05 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| WO2022103980A1 (en) * | 2020-11-12 | 2022-05-19 | Ptc Therapeutics Inc. | Novel rna transcript |
| WO2022204471A1 (en) * | 2021-03-26 | 2022-09-29 | Ptc Therapeutics Inc. | Regulation of transgene expression using a small molecule inducible splicing switch |
| 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 |
| WO2023034812A1 (en) | 2021-08-30 | 2023-03-09 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| IL311132A (en) | 2021-08-30 | 2024-04-01 | Remix Therapeutics Inc | Splicing Modulation Compounds and Methods |
| US20240400584A1 (en) | 2021-08-30 | 2024-12-05 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| WO2023064879A1 (en) | 2021-10-13 | 2023-04-20 | Remix Therapeutics Inc. | Compounds and methods for modulating nucleic acid splicing |
| CA3233973A1 (en) | 2021-10-13 | 2023-04-20 | Dominic Reynolds | 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 |
| WO2023133217A1 (en) | 2022-01-05 | 2023-07-13 | 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 |
| US20250326748A1 (en) | 2022-01-05 | 2025-10-23 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| 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 |
| UY40374A (es) | 2022-08-03 | 2024-02-15 | Novartis Ag | Inhibidores de inflamasoma nlrp3 |
| AU2024305544A1 (en) * | 2023-06-16 | 2026-01-08 | Plexium, Inc. | Compounds and pharmaceutical compositions that degrade swi/snf-related matrix-associated actin-dependent regulator of chromatin subfamily a |
| 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 |
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2018
- 2018-06-13 KR KR1020207000911A patent/KR20200017476A/ko not_active Ceased
- 2018-06-13 AU AU2018284853A patent/AU2018284853A1/en not_active Abandoned
- 2018-06-13 US US16/622,223 patent/US11608501B2/en active Active
- 2018-06-13 EP EP18817883.4A patent/EP3638318A4/en not_active Withdrawn
- 2018-06-13 CN CN201880052678.0A patent/CN111372611A/zh active Pending
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| AU2018284853A1 (en) | 2019-12-19 |
| WO2018232039A1 (en) | 2018-12-20 |
| EA202090034A1 (ru) | 2020-04-16 |
| BR112019026508A2 (pt) | 2020-07-14 |
| CN111372611A (zh) | 2020-07-03 |
| US20200370043A1 (en) | 2020-11-26 |
| US11608501B2 (en) | 2023-03-21 |
| IL271053A (en) | 2020-01-30 |
| JP2020523365A (ja) | 2020-08-06 |
| EP3638318A1 (en) | 2020-04-22 |
| EP3638318A4 (en) | 2021-03-17 |
| KR20200017476A (ko) | 2020-02-18 |
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