KR20220157407A - 핵산 스플라이싱을 조절하기 위한 피리다진 유도체 - Google Patents

핵산 스플라이싱을 조절하기 위한 피리다진 유도체 Download PDF

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KR20220157407A
KR20220157407A KR1020227033652A KR20227033652A KR20220157407A KR 20220157407 A KR20220157407 A KR 20220157407A KR 1020227033652 A KR1020227033652 A KR 1020227033652A KR 20227033652 A KR20227033652 A KR 20227033652A KR 20220157407 A KR20220157407 A KR 20220157407A
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compound
alkyl
heterocyclyl
heteroaryl
aryl
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Korean (ko)
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도미닉 레이놀즈
마이클 워커 실러
아난트 에이. 아그라왈
프레데릭 베일란코트
피터 스미스
앨런 티. 호퍼
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레믹스 테라퓨틱스 인크.
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/10—Spiro-condensed systems
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms 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
    • C07D417/04—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 directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C07D451/06—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07B—GENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR
    • C07B2200/00—Indexing scheme relating to specific properties of organic compounds
    • C07B2200/05—Isotopically modified compounds, e.g. labelled

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
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  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Cosmetics (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
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KR1020227033652A 2020-02-28 2021-02-28 핵산 스플라이싱을 조절하기 위한 피리다진 유도체 Pending KR20220157407A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202062983537P 2020-02-28 2020-02-28
US62/983,537 2020-02-28
US202063007134P 2020-04-08 2020-04-08
US63/007,134 2020-04-08
US202063040474P 2020-06-17 2020-06-17
US63/040,474 2020-06-17
US202063072781P 2020-08-31 2020-08-31
US63/072,781 2020-08-31
US202063126491P 2020-12-16 2020-12-16
US63/126,491 2020-12-16
PCT/US2021/020160 WO2021174170A1 (en) 2020-02-28 2021-02-28 Pyridazine dervatives for modulating nucleic acid splicing

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Publication Number Publication Date
KR20220157407A true KR20220157407A (ko) 2022-11-29

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KR1020227033652A Pending KR20220157407A (ko) 2020-02-28 2021-02-28 핵산 스플라이싱을 조절하기 위한 피리다진 유도체
KR1020227033654A Pending KR20220158236A (ko) 2020-02-28 2021-02-28 핵산 스플라이싱을 조절하기 위한 피리다진 유도체

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KR1020227033654A Pending KR20220158236A (ko) 2020-02-28 2021-02-28 핵산 스플라이싱을 조절하기 위한 피리다진 유도체

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US (2) US20230365526A1 (enExample)
EP (2) EP4110774A1 (enExample)
JP (2) JP2023515617A (enExample)
KR (2) KR20220157407A (enExample)
CN (2) CN115551847A (enExample)
AU (3) AU2021228286A1 (enExample)
BR (2) BR112022017089A2 (enExample)
CA (2) CA3169667A1 (enExample)
IL (2) IL295954A (enExample)
MX (2) MX2022010637A (enExample)
WO (2) WO2021174176A1 (enExample)

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MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
KR102913697B1 (ko) * 2019-02-05 2026-01-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2020163541A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
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
KR102925385B1 (ko) 2019-02-06 2026-02-11 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2021231571A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
US20250333397A1 (en) * 2021-08-30 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4426702A1 (en) * 2021-11-04 2024-09-11 Skyhawk Therapeutics, Inc. Condensed-pyrazine amine derivatives for treating sca3
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
EP4472969A4 (en) * 2022-01-31 2025-12-24 Novartis Ag PROCESS FOR THE SYNTHESIS OF PYRAZOLYL DERIVATIVES USEFUL AS ANTICANCER AGENTS
WO2023185468A1 (zh) * 2022-04-01 2023-10-05 浙江同源康医药股份有限公司 一类用作激酶抑制剂的化合物及其应用
AU2023327925A1 (en) * 2022-08-22 2025-02-20 Redx Pharma Limited Pyrido-pyrazoles as inhibitors of ddr's for the treatment of fibrotic disorders and cancer
WO2024182747A2 (en) * 2023-03-01 2024-09-06 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2025211457A1 (ja) * 2024-04-05 2025-10-09 日本新薬株式会社 Cugリピートrnaとmbnlタンパクとの結合阻害化合物および医薬

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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
WO2018140578A1 (en) * 2017-01-25 2018-08-02 President And Fellows Of Harvard College Non-fullerene solar panel acceptor compounds
CN111372611A (zh) * 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
MX2020001425A (es) * 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
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JP7270989B2 (ja) * 2017-10-20 2023-05-11 ヴァンダービルト ユニバーシティー ムスカリン性アセチルコリン受容体m4のアンタゴニスト
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TWI823932B (zh) * 2018-05-11 2023-12-01 中國大陸商迪哲(江蘇)醫藥有限公司 三唑并嘧啶化合物及其在治療癌症中之用途

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US20230365526A1 (en) 2023-11-16
CN115551847A (zh) 2022-12-30
AU2021228288A1 (en) 2022-09-22
WO2021174170A9 (en) 2022-09-15
MX2022010637A (es) 2023-01-19
AU2024201568A1 (en) 2024-03-28
WO2021174176A1 (en) 2021-09-02
KR20220158236A (ko) 2022-11-30
JP7901883B2 (ja) 2026-08-07
AU2021228286A1 (en) 2022-10-06
IL295953A (en) 2022-10-01
WO2021174170A1 (en) 2021-09-02
JP2023515617A (ja) 2023-04-13
CN115551843A (zh) 2022-12-30
BR112022017089A2 (pt) 2022-11-16
WO2021174176A9 (en) 2022-09-15
US20230140983A1 (en) 2023-05-11
JP2023515618A (ja) 2023-04-13
EP4110774A1 (en) 2023-01-04
IL295954A (en) 2022-10-01
CA3169667A1 (en) 2021-09-02
MX2022010634A (es) 2023-01-19
BR112022017107A2 (pt) 2022-11-16
CA3169643A1 (en) 2021-09-02
EP4110771A1 (en) 2023-01-04

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