JP7797407B2 - スプライシングを調節するための化合物及び方法 - Google Patents

スプライシングを調節するための化合物及び方法

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Publication number
JP7797407B2
JP7797407B2 JP2022561468A JP2022561468A JP7797407B2 JP 7797407 B2 JP7797407 B2 JP 7797407B2 JP 2022561468 A JP2022561468 A JP 2022561468A JP 2022561468 A JP2022561468 A JP 2022561468A JP 7797407 B2 JP7797407 B2 JP 7797407B2
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compound
heterocyclyl
formula
heteroaryl
absent
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JP2023520924A5 (enExample
JP2023520924A (ja
Inventor
ドミニク レイノルズ，
セルジュ レジェ，
マイケル ダブリュー． サイラー，
アナント エー． アグラワル，
フレデリック ヴァイランコート，
ピーター スミス，
スディープ プラジャパティ，
Original Assignee
リミックス セラピューティクス インコーポレイテッド
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D513/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04—Ortho-condensed systems
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with 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
    • 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
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Replacement Of Web Rolls (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Saccharide Compounds (AREA)
JP2022561468A 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法 Active JP7797407B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063007327P 2020-04-08 2020-04-08
US63/007,327 2020-04-08
US202063043920P 2020-06-25 2020-06-25
US63/043,920 2020-06-25
US202063072873P 2020-08-31 2020-08-31
US63/072,873 2020-08-31
US202063126493P 2020-12-16 2020-12-16
US63/126,493 2020-12-16
PCT/US2021/026477 WO2021207550A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (3)

Publication Number Publication Date
JP2023520924A JP2023520924A (ja) 2023-05-22
JP2023520924A5 JP2023520924A5 (enExample) 2024-04-18
JP7797407B2 true JP7797407B2 (ja) 2026-01-13

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JP2022561468A Active JP7797407B2 (ja) 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法
JP2022561469A Active JP7882782B2 (ja) 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法

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Country Status (14)

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US (2) US20240287097A1 (enExample)
EP (2) EP4132936A1 (enExample)
JP (2) JP7797407B2 (enExample)
KR (2) KR20230004575A (enExample)
CN (2) CN116096725A (enExample)
AU (2) AU2021251220A1 (enExample)
BR (2) BR112022020418A2 (enExample)
CA (2) CA3175205A1 (enExample)
CL (1) CL2022002779A1 (enExample)
CO (1) CO2022015926A2 (enExample)
CR (1) CR20220567A (enExample)
IL (1) IL297149A (enExample)
MX (2) MX2022012677A (enExample)
WO (2) WO2021207550A1 (enExample)

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WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
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
IL297963A (en) 2020-05-07 2023-01-01 Adorx Therapeutics Ltd Antagonists of the adenosine a2a receptor
WO2021231571A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
EP4308577B1 (en) * 2021-03-17 2024-12-25 F. Hoffmann-La Roche AG New thiadiazolopyrimidone derivatives
CR20230437A (es) * 2021-03-17 2023-10-16 Hoffmann La Roche Nuevos derivados de tiazolopirimidinona
EP4308572B1 (en) * 2021-03-17 2025-04-23 F. Hoffmann-La Roche AG New thienopyrimidinone derivatives
TW202330552A (zh) * 2021-10-13 2023-08-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
JP2024541481A (ja) * 2021-11-24 2024-11-08 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
CN115112899B (zh) * 2022-06-15 2024-08-16 四川大学华西医院 检测羧肽酶a4的试剂和/或系统在制备恶性胸腔积液筛查产品中的用途
CN121653249B (zh) * 2026-02-09 2026-05-26 中国医学科学院医学生物学研究所 Dnal1基因作为靶点在筛选防治寨卡病毒或2型登革病毒感染药物中的应用

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JP2002520417A (ja) 1998-07-16 2002-07-09 アボット・ラボラトリーズ アドレナリン受容体選択的ピペラジニルピリミジンジオン化合物
JP2005527489A (ja) 2002-01-14 2005-09-15 ファルマシア・アンド・アップジョン・カンパニー・エルエルシー 抗ウイルス化合物としてのチエノ（３，２−ｂ）ピリドン
JP2006522812A (ja) 2003-04-11 2006-10-05 スミスクライン ビーチャム コーポレーション 複素環ｍｃｈｒ１アンタゴニスト
JP2009513658A (ja) 2005-10-26 2009-04-02 ブリストル−マイヤーズ スクイブ カンパニー メラニン凝集ホルモン受容体−１拮抗薬としてのチエノピリミジノン誘導体
WO2007102679A1 (en) 2006-03-06 2007-09-13 Je Il Pharmaceutical Co., Ltd. Novel thienopyrimidine derivatives or pharmaceutically acceptable salts thereof, process for the preparation thereof and pharmaceutical composition comprising the same
WO2012009688A1 (en) 2010-07-16 2012-01-19 Rockefeller University Organic compounds
JP2014519519A (ja) 2011-06-15 2014-08-14 タケダ ゲゼルシャフト ミット ベシュレンクテル ハフツング 新規３，４，４ａ，１０ｂ−テトラヒドロ−１Ｈ−チオピラノ［４，３−ｃ］イソキノリン化合物
JP2014530838A (ja) 2011-10-21 2014-11-20 エフ． ホフマン−エルエーロシュ アーゲー ピリミジン−４−オン誘導体及びウイルス性疾患の治療、改善又は予防におけるその使用
WO2013109632A2 (en) 2012-01-16 2013-07-25 The Rockefeller University Organic compounds
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JP2017515863A (ja) 2014-05-15 2017-06-15 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための化合物
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IL297149A (en) 2022-12-01
EP4132936A1 (en) 2023-02-15
BR112022020337A2 (pt) 2022-12-13
US20240279239A1 (en) 2024-08-22
WO2021207530A8 (en) 2021-12-02
KR20230005210A (ko) 2023-01-09
EP4132935A1 (en) 2023-02-15
BR112022020418A2 (pt) 2023-05-02
MX2022012676A (es) 2023-01-11
CL2022002779A1 (es) 2023-04-28
US20240287097A1 (en) 2024-08-29
CA3175193A1 (en) 2021-10-14
CN116096725A (zh) 2023-05-09
AU2021251220A1 (en) 2022-11-03
WO2021207550A1 (en) 2021-10-14
JP7882782B2 (ja) 2026-06-30
AU2021253571A1 (en) 2022-11-10
WO2021207530A1 (en) 2021-10-14
JP2023520924A (ja) 2023-05-22
MX2022012677A (es) 2023-01-11
CO2022015926A2 (es) 2022-11-29
CR20220567A (es) 2023-01-13
JP2023520925A (ja) 2023-05-22
CA3175205A1 (en) 2021-10-14
CN116134036A (zh) 2023-05-16
KR20230004575A (ko) 2023-01-06

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