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)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (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)
  • General Health & Medical Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (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

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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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KR102109992B1 (ko) 2012-03-23 2020-05-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
KR20150098675A (ko) * 2012-12-24 2015-08-28 라모트 앳 텔-아비브 유니버시티 리미티드 넌센스 돌연변이로부터 생긴 유전적 질환을 치료하기 위한 물질 및 이를 확인하는 방법
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
EP3082820B1 (en) 2013-12-19 2022-07-20 PTC Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
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IL294124B2 (en) 2015-12-10 2024-02-01 Ptc Therapeutics Inc Methods for treating huntington's disease
EA201991309A1 (ru) 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
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US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
MX2020009957A (es) 2018-03-27 2021-01-15 Ptc Therapeutics Inc Compuestos para el tratamiento de enfermedad de hungtinton.
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
PL3814357T3 (pl) 2018-06-27 2024-09-16 Ptc Therapeutics, Inc. Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona
EP3814345B8 (en) 2018-06-27 2024-10-30 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
BR112020026637A2 (pt) 2018-06-27 2021-03-30 Reborna Biosciences, Inc. Agente profilático ou terapêutico para atrofia muscular espinhal
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
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
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
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US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
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Also Published As

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

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