PL2809322T3 - Związki do leczenia zaniku mięśni pochodzenia rdzeniowego - Google Patents

Związki do leczenia zaniku mięśni pochodzenia rdzeniowego

Info

Publication number
PL2809322T3
PL2809322T3 PL13740919T PL13740919T PL2809322T3 PL 2809322 T3 PL2809322 T3 PL 2809322T3 PL 13740919 T PL13740919 T PL 13740919T PL 13740919 T PL13740919 T PL 13740919T PL 2809322 T3 PL2809322 T3 PL 2809322T3
Authority
PL
Poland
Prior art keywords
compounds
muscular atrophy
spinal muscular
treating spinal
treating
Prior art date
Application number
PL13740919T
Other languages
English (en)
Inventor
Guangming Chen
Amal Dakka
Gary Mitchell Karp
Chunshi Li
Jana Narasimhan
Nikolai Naryshkin
Marla L Weetall
Ellen Welch
Xin Zhao
Original Assignee
Ptc Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ptc Therapeutics Inc filed Critical Ptc Therapeutics Inc
Publication of PL2809322T3 publication Critical patent/PL2809322T3/pl

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D513/00Heterocyclic 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
    • C07D513/02Heterocyclic 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
    • C07D513/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D311/00Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
    • C07D311/02Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings ortho- or peri-condensed with carbocyclic rings or ring systems
    • C07D311/76Benzo[c]pyrans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/04Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/14Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D407/00Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00
    • C07D407/02Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00 containing two hetero rings
    • C07D407/04Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D409/00Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/02Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings
    • C07D409/04Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D409/00Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/14Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D497/00Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms
    • C07D497/02Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D497/04Ortho-condensed systems

Landscapes

  • 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)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Pyrane Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Cosmetics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL13740919T 2012-01-26 2013-01-25 Związki do leczenia zaniku mięśni pochodzenia rdzeniowego PL2809322T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261591102P 2012-01-26 2012-01-26
PCT/US2013/023067 WO2013112788A1 (en) 2012-01-26 2013-01-25 Compounds for treating spinal muscular atrophy
EP13740919.9A EP2809322B9 (en) 2012-01-26 2013-01-25 Compounds for treating spinal muscular atrophy

Publications (1)

Publication Number Publication Date
PL2809322T3 true PL2809322T3 (pl) 2019-11-29

Family

ID=48873933

Family Applications (1)

Application Number Title Priority Date Filing Date
PL13740919T PL2809322T3 (pl) 2012-01-26 2013-01-25 Związki do leczenia zaniku mięśni pochodzenia rdzeniowego

Country Status (13)

Country Link
US (1) US9399649B2 (pl)
EP (1) EP2809322B9 (pl)
JP (2) JP6193888B2 (pl)
KR (1) KR102064624B1 (pl)
CN (1) CN104203239B (pl)
BR (1) BR112014018027B1 (pl)
CA (1) CA2862084C (pl)
EA (1) EA028344B1 (pl)
ES (1) ES2733644T3 (pl)
HK (1) HK1202069A1 (pl)
MX (1) MX357834B (pl)
PL (1) PL2809322T3 (pl)
WO (1) WO2013112788A1 (pl)

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WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
PL2809322T3 (pl) * 2012-01-26 2019-11-29 Ptc Therapeutics Inc Związki do leczenia zaniku mięśni pochodzenia rdzeniowego
WO2013119916A2 (en) 2012-02-10 2013-08-15 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
MX352962B (es) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal.
EP2828247B1 (en) 2012-03-23 2019-01-16 PTC Therapeutics, Inc. 4h-chromen-4-one derivatives for treating spinal muscular atrophy
BR112015015075A2 (pt) * 2012-12-24 2019-01-15 Univ Ramot agentes para tratar doenças genéticas resultantes de mutações sem sentido e métodos para identificar as mesmas.
CN105392790B (zh) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途
WO2015095446A1 (en) * 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015105657A1 (en) 2013-12-19 2015-07-16 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
WO2017080967A1 (en) * 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
WO2017087486A1 (en) 2015-11-16 2017-05-26 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein
ES2879995T3 (es) 2015-12-10 2021-11-23 Ptc Therapeutics Inc Métodos para el tratamiento de la enfermedad de Huntington
CN108137579B (zh) 2015-12-10 2022-01-07 豪夫迈·罗氏有限公司 桥连哌啶衍生物
JP6738044B2 (ja) * 2016-07-22 2020-08-12 Jnc株式会社 ベンゾピラン骨格を有する液晶性化合物、液晶組成物、および液晶表示素子
CN110352007A (zh) 2016-11-28 2019-10-18 Ptc医疗公司 用于调节rna剪接的方法
WO2018226622A1 (en) 2017-06-05 2018-12-13 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
BR112019026508A2 (pt) 2017-06-14 2020-07-14 Ptc Therapeutics, Inc. métodos para modificar o splicing do rna
EP3644996B1 (en) 2017-06-28 2023-07-26 PTC Therapeutics, Inc. Methods for treating huntington's disease
CA3067592A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
JP7399870B2 (ja) 2018-03-27 2023-12-18 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
WO2020005873A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
CR20210050A (es) * 2018-06-27 2021-06-10 Reborna Biosciences Inc Agente profiláctico o terapéutico para atrofia muscular espinal
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
JP2022521467A (ja) 2019-02-05 2022-04-08 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
JP2022519323A (ja) 2019-02-06 2022-03-22 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
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing

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JP5276878B2 (ja) * 2007-09-27 2013-08-28 富士フイルム株式会社 ベンゾオキサジノン系化合物の製造方法
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
PL2809322T3 (pl) * 2012-01-26 2019-11-29 Ptc Therapeutics Inc Związki do leczenia zaniku mięśni pochodzenia rdzeniowego
WO2013119916A2 (en) 2012-02-10 2013-08-15 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
MX352962B (es) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal.
EP2828247B1 (en) 2012-03-23 2019-01-16 PTC Therapeutics, Inc. 4h-chromen-4-one derivatives for treating spinal muscular atrophy

Also Published As

Publication number Publication date
CN104203239A (zh) 2014-12-10
BR112014018027A2 (pl) 2017-06-20
BR112014018027A8 (pt) 2017-07-11
MX2014008875A (es) 2014-10-14
KR102064624B1 (ko) 2020-01-09
EA201491412A1 (ru) 2014-12-30
EA028344B1 (ru) 2017-11-30
WO2013112788A1 (en) 2013-08-01
ES2733644T3 (es) 2019-12-02
BR112014018027B1 (pt) 2020-10-27
JP2017171667A (ja) 2017-09-28
US9399649B2 (en) 2016-07-26
EP2809322B9 (en) 2019-10-30
EP2809322A4 (en) 2015-12-23
US20150126515A1 (en) 2015-05-07
JP6193888B2 (ja) 2017-09-06
CA2862084C (en) 2021-05-11
JP2015511224A (ja) 2015-04-16
CA2862084A1 (en) 2013-08-01
MX357834B (es) 2018-07-26
EP2809322A1 (en) 2014-12-10
EP2809322B1 (en) 2019-05-15
CN104203239B (zh) 2017-09-08
KR20140127271A (ko) 2014-11-03
HK1202069A1 (en) 2015-09-18

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