CN116134036A - 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
CN116134036A
CN116134036A CN202180040768.XA CN202180040768A CN116134036A CN 116134036 A CN116134036 A CN 116134036A CN 202180040768 A CN202180040768 A CN 202180040768A CN 116134036 A CN116134036 A CN 116134036A
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CN
China
Prior art keywords
compound
heterocyclyl
heteroaryl
formula
aryl
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CN202180040768.XA
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English (en)
Chinese (zh)
Inventor
D.雷诺兹
S.莱格
M.W.塞勒
A.A.阿格拉瓦尔
F.维尔兰科特
P.史密斯
S.普拉贾帕蒂
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Remix Medical
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Remix Medical
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Publication of CN116134036A publication Critical patent/CN116134036A/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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Replacement Of Web Rolls (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Saccharide Compounds (AREA)
CN202180040768.XA 2020-04-08 2021-04-08 用于调节剪接的化合物和方法 Pending CN116134036A (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/US2021/026477 WO2021207550A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (1)

Publication Number Publication Date
CN116134036A true CN116134036A (zh) 2023-05-16

Family

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Family Applications (2)

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

Family Applications After (1)

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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

Country Status (14)

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US (2) US20240287097A1 (https=)
EP (2) EP4132935A1 (https=)
JP (2) JP2023520925A (https=)
KR (2) KR20230005210A (https=)
CN (2) CN116134036A (https=)
AU (2) AU2021253571A1 (https=)
BR (2) BR112022020337A2 (https=)
CA (2) CA3175205A1 (https=)
CL (1) CL2022002779A1 (https=)
CO (1) CO2022015926A2 (https=)
CR (1) CR20220567A (https=)
IL (1) IL297149A (https=)
MX (2) MX2022012676A (https=)
WO (2) WO2021207550A1 (https=)

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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
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
BR112022023025A2 (pt) 2020-05-13 2023-03-28 Chdi Foundation Inc Moduladores de htt para tratar doença de huntington
WO2022194802A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thiadiazolopyrimidone derivatives
BR112023018466A2 (pt) * 2021-03-17 2023-10-10 Hoffmann La Roche Derivados de tiazolopirimidinona
CN116997555A (zh) * 2021-03-17 2023-11-03 豪夫迈·罗氏有限公司 新颖噻吩并嘧啶酮衍生物
WO2023064879A1 (en) * 2021-10-13 2023-04-20 Remix Therapeutics Inc. 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
AU2022398247A1 (en) * 2021-11-24 2024-06-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
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

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JP2023520925A (ja) 2023-05-22
EP4132936A1 (en) 2023-02-15
CR20220567A (es) 2023-01-13
EP4132935A1 (en) 2023-02-15
CA3175193A1 (en) 2021-10-14
KR20230005210A (ko) 2023-01-09
JP2023520924A (ja) 2023-05-22
CL2022002779A1 (es) 2023-04-28
AU2021251220A1 (en) 2022-11-03
BR112022020418A2 (pt) 2023-05-02
BR112022020337A2 (pt) 2022-12-13
CO2022015926A2 (es) 2022-11-29
JP7797407B2 (ja) 2026-01-13
WO2021207550A1 (en) 2021-10-14
IL297149A (en) 2022-12-01
WO2021207530A8 (en) 2021-12-02
WO2021207530A1 (en) 2021-10-14
MX2022012676A (es) 2023-01-11
US20240287097A1 (en) 2024-08-29
AU2021253571A1 (en) 2022-11-10
MX2022012677A (es) 2023-01-11
KR20230004575A (ko) 2023-01-06
US20240279239A1 (en) 2024-08-22
CA3175205A1 (en) 2021-10-14
CN116096725A (zh) 2023-05-09

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