CN116096725A - 用于调节剪接的化合物和方法 - Google Patents

用于调节剪接的化合物和方法 Download PDF

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Publication number
CN116096725A
CN116096725A CN202180040958.1A CN202180040958A CN116096725A CN 116096725 A CN116096725 A CN 116096725A CN 202180040958 A CN202180040958 A CN 202180040958A CN 116096725 A CN116096725 A CN 116096725A
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China
Prior art keywords
compound
heterocyclyl
heteroaryl
aryl
alkyl
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CN202180040958.1A
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English (en)
Chinese (zh)
Inventor
D.雷诺兹
M.W.塞勒
A.A.阿格拉瓦尔
F.维尔兰科特
P.史密斯
S.莱格
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Remix Medical
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Remix Medical
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Publication of CN116096725A publication Critical patent/CN116096725A/zh
Pending legal-status Critical Current

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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)
CN202180040958.1A 2020-04-08 2021-04-08 用于调节剪接的化合物和方法 Pending CN116096725A (zh)

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/US2021/026435 WO2021207530A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (1)

Publication Number Publication Date
CN116096725A true CN116096725A (zh) 2023-05-09

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CN202180040958.1A Pending CN116096725A (zh) 2020-04-08 2021-04-08 用于调节剪接的化合物和方法
CN202180040768.XA Pending CN116134036A (zh) 2020-04-08 2021-04-08 用于调节剪接的化合物和方法

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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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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
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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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Publication number Publication date
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
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
JP7797407B2 (ja) 2026-01-13
KR20230004575A (ko) 2023-01-06

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