BR112014018027B1 - composto, composição farmacêutica e usos dos mesmos e métodos para aumentar a inclusão de éxon 7 de ame2 em rnam que é transcrito a partir do gene ame2 e para aumentar a quantidade de proteína smn - Google Patents

composto, composição farmacêutica e usos dos mesmos e métodos para aumentar a inclusão de éxon 7 de ame2 em rnam que é transcrito a partir do gene ame2 e para aumentar a quantidade de proteína smn Download PDF

Info

Publication number
BR112014018027B1
BR112014018027B1 BR112014018027-0A BR112014018027A BR112014018027B1 BR 112014018027 B1 BR112014018027 B1 BR 112014018027B1 BR 112014018027 A BR112014018027 A BR 112014018027A BR 112014018027 B1 BR112014018027 B1 BR 112014018027B1
Authority
BR
Brazil
Prior art keywords
isochromen
pyrazin
amino
compound
formula
Prior art date
Application number
BR112014018027-0A
Other languages
English (en)
Portuguese (pt)
Other versions
BR112014018027A8 (pt
BR112014018027A2 (enExample
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 BR112014018027A2 publication Critical patent/BR112014018027A2/pt
Publication of BR112014018027A8 publication Critical patent/BR112014018027A8/pt
Publication of BR112014018027B1 publication Critical patent/BR112014018027B1/pt

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D513/00—Heterocyclic 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
    • C07D513/02—Heterocyclic 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
    • C07D513/04—Ortho-condensed systems
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous 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
    • C07D311/00—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
    • C07D311/02—Heterocyclic 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
    • C07D311/76—Benzo[c]pyrans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic 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
    • C07D405/02—Heterocyclic 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
    • C07D405/04—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic 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
    • C07D405/14—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D407/00—Heterocyclic 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
    • C07D407/02—Heterocyclic 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
    • C07D407/04—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings
    • C07D409/04—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/14—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic 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
    • C07D471/02—Heterocyclic 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
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic 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
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D497/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms
    • C07D497/02—Heterocyclic 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
    • C07D497/04—Ortho-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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Cosmetics (AREA)
BR112014018027-0A 2012-01-26 2013-01-25 composto, composição farmacêutica e usos dos mesmos e métodos para aumentar a inclusão de éxon 7 de ame2 em rnam que é transcrito a partir do gene ame2 e para aumentar a quantidade de proteína smn BR112014018027B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261591102P 2012-01-26 2012-01-26
US61/591,102 2012-01-26
PCT/US2013/023067 WO2013112788A1 (en) 2012-01-26 2013-01-25 Compounds for treating spinal muscular atrophy

Publications (3)

Publication Number Publication Date
BR112014018027A2 BR112014018027A2 (enExample) 2017-06-20
BR112014018027A8 BR112014018027A8 (pt) 2017-07-11
BR112014018027B1 true BR112014018027B1 (pt) 2020-10-27

Family

ID=48873933

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112014018027-0A BR112014018027B1 (pt) 2012-01-26 2013-01-25 composto, composição farmacêutica e usos dos mesmos e métodos para aumentar a inclusão de éxon 7 de ame2 em rnam que é transcrito a partir do gene ame2 e para aumentar a quantidade de proteína smn

Country Status (12)

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

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
USRE47689E1 (en) 2011-12-30 2019-11-05 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
CN119528903A (zh) 2012-02-10 2025-02-28 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
CA2865957C (en) 2012-03-01 2020-02-11 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
KR102109992B1 (ko) 2012-03-23 2020-05-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
US10987370B2 (en) * 2012-12-24 2021-04-27 Ramot At Tel-Aviv University Ltd. Methods of inducing read-through of a nonsense mutation associated with ataxia telangiectasia, Rett syndrome or spinal muscular atrophy by erythromycin or azithromycin
BR112015032718A2 (pt) 2013-08-19 2019-09-17 Hoffmann La Roche composto de modificação de splicing do gene foxm1, uso do composto, formulação farmacêutica e métodos de triagem de compostos
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
PL3663296T3 (pl) * 2014-05-15 2023-08-07 F. Hoffmann-La Roche Ag Sposób wytwarzania związków użytecznych w leczeniu rdzeniowego zaniku mięśni
US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
CN108289959B (zh) 2015-11-12 2021-07-16 豪夫迈·罗氏有限公司 用于治疗脊髓性肌萎缩的组合物
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
EP3386978B1 (en) 2015-12-10 2021-01-27 H. Hoffnabb-La Roche Ag Bridged piperidine derivatives
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
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
US11702646B2 (en) 2016-11-28 2023-07-18 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
KR20200033249A (ko) 2017-06-05 2020-03-27 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
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
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
BR112020026545A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos heterocíclicos e de heteroarila para o tratamento da doença de huntington
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
SG11202013088YA (en) 2018-06-27 2021-02-25 Reborna Biosciences Inc Prophylactic or therapeutic agent for spinal muscular atrophy
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
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
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
AU2024286618A1 (en) * 2023-06-09 2025-10-30 F. Hoffmann-La Roche Ag Inducible gene expression system

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5854142B2 (ja) * 1975-04-16 1983-12-02 株式会社興人 イソカルボスチリル誘導体の製造方法
US5089633A (en) * 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US6180625B1 (en) 1998-03-24 2001-01-30 Novo Nordisk A/S Heterocyclic compounds regulating clotting
WO2003005025A1 (en) * 2001-07-03 2003-01-16 Biovitrum Ab Methods for identifying compounds modulating the activity of ppar-gamma
GB0205281D0 (en) * 2002-03-06 2002-04-17 Novartis Ag Organic compounds
US20040077529A1 (en) 2002-06-10 2004-04-22 Per-Fredrik Lehmann Urotensin II receptor agents
PL374598A1 (en) * 2002-06-28 2005-10-31 Vertex Pharmaceuticals Incorporated Caspase inhibitors and uses thereof
JP2004168707A (ja) * 2002-11-20 2004-06-17 Bayer Cropscience Ag イソチアゾリルベンゾオキサジン誘導体および病害防除剤
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
ATE440825T1 (de) 2003-06-06 2009-09-15 Vertex Pharma Pyrimidin-derivate zur verwendung als modulatoren von atp-bindende kassette transportern
CN101146798A (zh) 2005-01-21 2008-03-19 詹森药业有限公司 用作雌激素受体调节剂的新的杂环苯并[c]色烯衍生物
KR100781704B1 (ko) * 2005-04-20 2007-12-03 에스케이케미칼주식회사 피리딘 유도체와 이의 제조방법, 및 이를 포함하는약제조성물
AR059339A1 (es) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
KR20080091949A (ko) 2007-04-10 2008-10-15 에스케이케미칼주식회사 락톤형 피리딘 화합물을 포함하는 허혈성 질환의 예방 및치료용 약학조성물
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
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
USRE47689E1 (en) 2011-12-30 2019-11-05 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
CN119528903A (zh) 2012-02-10 2025-02-28 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
CA2865957C (en) 2012-03-01 2020-02-11 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
KR102109992B1 (ko) 2012-03-23 2020-05-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물

Also Published As

Publication number Publication date
JP2015511224A (ja) 2015-04-16
CA2862084A1 (en) 2013-08-01
KR20140127271A (ko) 2014-11-03
EA028344B1 (ru) 2017-11-30
EA201491412A1 (ru) 2014-12-30
CN104203239A (zh) 2014-12-10
CA2862084C (en) 2021-05-11
EP2809322B9 (en) 2019-10-30
MX2014008875A (es) 2014-10-14
WO2013112788A1 (en) 2013-08-01
BR112014018027A8 (pt) 2017-07-11
JP6193888B2 (ja) 2017-09-06
PL2809322T3 (pl) 2019-11-29
US20150126515A1 (en) 2015-05-07
HK1202069A1 (en) 2015-09-18
JP2017171667A (ja) 2017-09-28
BR112014018027A2 (enExample) 2017-06-20
ES2733644T3 (es) 2019-12-02
EP2809322B1 (en) 2019-05-15
KR102064624B1 (ko) 2020-01-09
EP2809322A4 (en) 2015-12-23
MX357834B (es) 2018-07-26
CN104203239B (zh) 2017-09-08
US9399649B2 (en) 2016-07-26
EP2809322A1 (en) 2014-12-10

Similar Documents

Publication Publication Date Title
BR112014018027B1 (pt) composto, composição farmacêutica e usos dos mesmos e métodos para aumentar a inclusão de éxon 7 de ame2 em rnam que é transcrito a partir do gene ame2 e para aumentar a quantidade de proteína smn
CN104470909B (zh) 用于治疗脊髓性肌萎缩的化合物
CN104302181B (zh) 用于治疗脊髓性肌萎缩的化合物
BR112014016287B1 (pt) Composto e composição farmacêutica
HK1202069B (en) Compounds for treating spinal muscular atrophy
HK1200056B (en) Compounds for treating spinal muscular atrophy
HK1202527B (en) 4h-chromen-4-one derivatives for treating spinal muscular atrophy

Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06T Formal requirements before examination [chapter 6.20 patent gazette]
B06A Patent application procedure suspended [chapter 6.1 patent gazette]
B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 25/01/2013, OBSERVADAS AS CONDICOES LEGAIS.