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
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Active
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
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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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  • 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)
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 (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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MX357834B (es) 2012-01-26 2018-07-26 Ptc Therapeutics Inc Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal.
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
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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
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
KR102099997B1 (ko) 2020-04-13
CA2865957A1 (en) 2013-09-06
BR112014021531B1 (pt) 2022-10-04
EA029155B1 (ru) 2018-02-28

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