KR102099997B1 - 척수성 근위축증을 치료하기 위한 화합물 - Google Patents

척수성 근위축증을 치료하기 위한 화합물 Download PDF

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KR102099997B1
KR102099997B1 KR1020147027577A KR20147027577A KR102099997B1 KR 102099997 B1 KR102099997 B1 KR 102099997B1 KR 1020147027577 A KR1020147027577 A KR 1020147027577A KR 20147027577 A KR20147027577 A KR 20147027577A KR 102099997 B1 KR102099997 B1 KR 102099997B1
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pyridin
alkyl
pyrano
formula
amino
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KR20140131384A (ko
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창-순 이
순규 최
개리 미첼 카르프
히로오 고야마
하산 라트니
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피티씨 테라퓨틱스, 인크.
에프. 호프만-라 로슈 아게
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/04—Ortho-condensed systems
    • C07D491/044—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
    • C07D491/052—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being six-membered

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Cosmetics (AREA)
KR1020147027577A 2012-03-01 2013-02-28 척수성 근위축증을 치료하기 위한 화합물 Active KR102099997B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261605487P 2012-03-01 2012-03-01
US61/605,487 2012-03-01
PCT/US2013/028131 WO2013130689A1 (en) 2012-03-01 2013-02-28 Compounds for treating spinal muscular atrophy

Publications (2)

Publication Number Publication Date
KR20140131384A KR20140131384A (ko) 2014-11-12
KR102099997B1 true KR102099997B1 (ko) 2020-04-13

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US (1) US9371336B2 (https=)
EP (1) EP2819519B1 (https=)
JP (1) JP6092264B2 (https=)
KR (1) KR102099997B1 (https=)
CN (1) CN104302181B (https=)
BR (1) BR112014021531B1 (https=)
CA (1) CA2865957C (https=)
EA (1) EA029155B1 (https=)
MX (1) MX352962B (https=)
WO (1) WO2013130689A1 (https=)

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EP2812004B1 (en) 2012-02-10 2018-06-27 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CA2894992A1 (en) * 2012-12-24 2014-07-03 Ramot At Tel-Aviv University Ltd. Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same
EP3035935B1 (en) 2013-08-19 2020-03-11 F. Hoffmann-La Roche AG Compounds for use in the prophylaxis and treatment of cancer
US10195202B2 (en) 2013-12-19 2019-02-05 Ptc Therapeutics, Inc. Methods for modulating the amount of RNA transcripts
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
EP3133065A1 (en) 2015-08-21 2017-02-22 Merck Patent GmbH Compounds for optically active devices
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
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
EP3363786A1 (en) 2017-02-15 2018-08-22 Merck Patent GmbH Compounds for optically active devices
EP3363793A1 (en) 2017-02-15 2018-08-22 Merck Patent GmbH Hydrophobic compounds for optically active devices
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
JP2020523365A (ja) 2017-06-14 2020-08-06 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スプライシングを改変する方法
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
JP7312749B2 (ja) 2017-08-04 2023-07-21 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 スプライシングをモジュレートする方法および組成物
EA202092001A1 (ru) 2018-03-27 2021-01-29 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни гентингтона
CN112469707B (zh) 2018-06-27 2024-06-21 利伯纳生物科学株式会社 脊髓性肌萎缩症的预防剂或治疗剂
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
HRP20240935T1 (hr) 2018-06-27 2024-11-22 Ptc Therapeutics, Inc. Heterociklični i heteroaril spojevi za liječenje huntingtonove bolesti
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
WO2020163405A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7551629B2 (ja) 2019-02-05 2024-09-17 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
JP7603595B2 (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
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
JP7649257B2 (ja) 2019-05-13 2025-03-19 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN111116460A (zh) * 2019-12-31 2020-05-08 阿里生物新材料（常州）有限公司 一种2-氯-4-羟基吡啶-3-甲醛氢溴酸盐的合成方法
JP7763397B2 (ja) 2020-05-20 2025-11-04 エイエムオー・アイルランド 光学活性装置で使用するためのアザクマリン及びアザチオクマリン誘導体
WO2023114539A1 (en) * 2021-12-17 2023-06-22 Pretzel Therapeutics, Inc. Amino chromen-2-one modulators of polrmt
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Also Published As

Publication number Publication date
BR112014021531A8 (pt) 2021-06-15
BR112014021531A2 (pt) 2017-06-20
WO2013130689A1 (en) 2013-09-06
JP2015509956A (ja) 2015-04-02
EP2819519A4 (en) 2015-09-02
US20150166575A1 (en) 2015-06-18
HK1200056A1 (en) 2015-07-31
MX2014010406A (es) 2015-03-10
EP2819519B1 (en) 2019-10-23
CA2865957C (en) 2020-02-11
KR20140131384A (ko) 2014-11-12
EP2819519A1 (en) 2015-01-07
JP6092264B2 (ja) 2017-03-08
CN104302181A (zh) 2015-01-21
US9371336B2 (en) 2016-06-21
EA201491617A1 (ru) 2015-01-30
CN104302181B (zh) 2017-09-15
MX352962B (es) 2017-12-15
CA2865957A1 (en) 2013-09-06
BR112014021531B1 (pt) 2022-10-04
EA029155B1 (ru) 2018-02-28

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