CA2865957C - Compounds for treating spinal muscular atrophy - Google Patents

Compounds for treating spinal muscular atrophy Download PDF

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Publication number
CA2865957C
CA2865957C CA2865957A CA2865957A CA2865957C CA 2865957 C CA2865957 C CA 2865957C CA 2865957 A CA2865957 A CA 2865957A CA 2865957 A CA2865957 A CA 2865957A CA 2865957 C CA2865957 C CA 2865957C
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CA
Canada
Prior art keywords
pyridin
pyrano
amino
formula
compound
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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Application number
CA2865957A
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English (en)
French (fr)
Other versions
CA2865957A1 (en
Inventor
Chang-Sun Lee
Soongyu Choi
Gary Mitchell Karp
Hiroo Koyama
Hasane Ratni
Original Assignee
F Hoffmann La Roche AG
PTC Therapeutics Inc
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Publication date
Application filed by F Hoffmann La Roche AG, PTC Therapeutics Inc filed Critical F Hoffmann La Roche AG
Publication of CA2865957A1 publication Critical patent/CA2865957A1/en
Application granted granted Critical
Publication of CA2865957C publication Critical patent/CA2865957C/en
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Classifications

    • 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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  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Cosmetics (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CA2865957A 2012-03-01 2013-02-28 Compounds for treating spinal muscular atrophy Active CA2865957C (en)

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
CA2865957A1 CA2865957A1 (en) 2013-09-06
CA2865957C true CA2865957C (en) 2020-02-11

Family

ID=49083256

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2865957A Active CA2865957C (en) 2012-03-01 2013-02-28 Compounds for treating spinal muscular atrophy

Country Status (10)

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

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CA2862084C (en) 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
ES2697174T3 (es) 2012-02-10 2019-01-22 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014102778A2 (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
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WO2015105657A1 (en) 2013-12-19 2015-07-16 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
KR20220100719A (ko) 2015-12-10 2022-07-15 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
CA3043755A1 (en) 2016-11-28 2018-05-31 Ptc Therapeutics, Inc. Methods for modulating rna splicing
EP3363793A1 (en) 2017-02-15 2018-08-22 Merck Patent GmbH Hydrophobic compounds for optically active devices
EP3363786A1 (en) 2017-02-15 2018-08-22 Merck Patent GmbH Compounds for optically active devices
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
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AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
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BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
CN119320388A (zh) 2019-02-04 2025-01-17 斯基霍克疗法公司 用于调节剪接的方法和组合物
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
KR102913697B1 (ko) 2019-02-05 2026-01-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
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
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN114245794B (zh) 2019-05-13 2024-09-13 Ptc医疗公司 用于治疗亨廷顿氏病的化合物
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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US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy

Also Published As

Publication number Publication date
HK1200056A1 (en) 2015-07-31
JP6092264B2 (ja) 2017-03-08
EA029155B1 (ru) 2018-02-28
JP2015509956A (ja) 2015-04-02
CA2865957A1 (en) 2013-09-06
BR112014021531A2 (pt) 2017-06-20
US9371336B2 (en) 2016-06-21
BR112014021531B1 (pt) 2022-10-04
US20150166575A1 (en) 2015-06-18
MX2014010406A (es) 2015-03-10
BR112014021531A8 (pt) 2021-06-15
WO2013130689A1 (en) 2013-09-06
KR20140131384A (ko) 2014-11-12
EP2819519A1 (en) 2015-01-07
EA201491617A1 (ru) 2015-01-30
EP2819519B1 (en) 2019-10-23
MX352962B (es) 2017-12-15
CN104302181B (zh) 2017-09-15
KR102099997B1 (ko) 2020-04-13
CN104302181A (zh) 2015-01-21
EP2819519A4 (en) 2015-09-02

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