PL2809322T3 - Związki do leczenia zaniku mięśni pochodzenia rdzeniowego - Google Patents
Związki do leczenia zaniku mięśni pochodzenia rdzeniowegoInfo
- 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
Links
- 150000001875 compounds Chemical class 0.000 title 1
- 208000002320 spinal muscular atrophy Diseases 0.000 title 1
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)
- Cosmetics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261591102P | 2012-01-26 | 2012-01-26 | |
| EP13740919.9A EP2809322B9 (en) | 2012-01-26 | 2013-01-25 | Compounds for treating spinal muscular atrophy |
| PCT/US2013/023067 WO2013112788A1 (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 (12)
| 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) |
| MX (1) | MX357834B (pl) |
| PL (1) | PL2809322T3 (pl) |
| WO (1) | WO2013112788A1 (pl) |
Families Citing this family (32)
| 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 |
| CA2862084C (en) * | 2012-01-26 | 2021-05-11 | Ptc Therapeutics, Inc. | 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy |
| EA037123B1 (ru) | 2012-02-10 | 2021-02-09 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения спинальной мышечной атрофии |
| WO2013130689A1 (en) | 2012-03-01 | 2013-09-06 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| 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. |
| JP6689197B2 (ja) | 2013-08-19 | 2020-04-28 | エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト | スクリーニング方法 |
| WO2015095446A1 (en) * | 2013-12-19 | 2015-06-25 | Ptc Therapeutics, Inc. | Methods for modulating the amount of rna transcripts |
| EP3082820B1 (en) | 2013-12-19 | 2022-07-20 | PTC Therapeutics, Inc. | Methods for modulating the amount of rna transcripts |
| RS59718B1 (sr) * | 2014-05-15 | 2020-01-31 | Hoffmann La Roche | Jedinjenja za lečenje spinalne mišićne atrofije |
| US10882868B2 (en) | 2014-05-15 | 2021-01-05 | Hoffmann-La Roche Inc. | Compounds for treating spinal muscular atrophy |
| EP3310169B1 (en) | 2015-05-30 | 2023-05-17 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| BR112018009281B1 (pt) | 2015-11-12 | 2023-10-31 | F. Hoffmann-La Roche Ag | Composições para tratar atrofia muscular espinhal e seu uso |
| US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
| MX382671B (es) | 2015-12-10 | 2025-03-13 | Ptc Therapeutics Inc | Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington |
| JP6872550B2 (ja) | 2015-12-10 | 2021-05-19 | エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ | 架橋されたピペリジン誘導体 |
| 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 |
| 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 |
| JP7376471B2 (ja) | 2017-06-05 | 2023-11-08 | 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 |
| CN111372611A (zh) | 2017-06-14 | 2020-07-03 | Ptc医疗公司 | 修饰rna剪接的方法 |
| MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| MY201938A (en) | 2017-08-04 | 2024-03-25 | 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 |
| LT3814357T (lt) | 2018-06-27 | 2024-06-25 | Ptc Therapeutics, Inc. | Heterocikliniai ir heteroarilų junginiai, skirti hantingtono ligos gydymui |
| EA202092899A1 (ru) | 2018-06-27 | 2021-05-14 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Гетероарильные соединения для лечения болезни гентингтона |
| US11685746B2 (en) | 2018-06-27 | 2023-06-27 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| WO2020004594A1 (ja) * | 2018-06-27 | 2020-01-02 | 株式会社リボルナバイオサイエンス | 脊髄性筋萎縮症の予防または治療剤 |
| 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 |
| WO2024251925A1 (en) * | 2023-06-09 | 2024-12-12 | F. Hoffmann-La Roche Ag | Inducible gene expression system |
Family Cites Families (23)
| 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 |
| WO2003104216A1 (en) * | 2002-06-10 | 2003-12-18 | Acadia Pharmaceuticals Inc. | Urotensin ii receptor modulators |
| BR0312232A (pt) * | 2002-06-28 | 2005-05-10 | Vertex Pharma | Inibidores de caspases e seus usos |
| 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 |
| CA2595477A1 (en) | 2005-01-21 | 2006-07-27 | Janssen Pharmaceutica N.V. | Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors |
| 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 |
| US8337941B2 (en) | 2006-07-27 | 2012-12-25 | 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 |
| CA2862084C (en) * | 2012-01-26 | 2021-05-11 | Ptc Therapeutics, Inc. | 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy |
| EA037123B1 (ru) | 2012-02-10 | 2021-02-09 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения спинальной мышечной атрофии |
| WO2013130689A1 (en) | 2012-03-01 | 2013-09-06 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| EP2828247B1 (en) | 2012-03-23 | 2019-01-16 | PTC Therapeutics, Inc. | 4h-chromen-4-one derivatives for treating spinal muscular atrophy |
-
2013
- 2013-01-25 CA CA2862084A patent/CA2862084C/en active Active
- 2013-01-25 ES ES13740919T patent/ES2733644T3/es active Active
- 2013-01-25 KR KR1020147023617A patent/KR102064624B1/ko active Active
- 2013-01-25 CN CN201380016999.2A patent/CN104203239B/zh active Active
- 2013-01-25 MX MX2014008875A patent/MX357834B/es active IP Right Grant
- 2013-01-25 BR BR112014018027-0A patent/BR112014018027B1/pt active IP Right Grant
- 2013-01-25 JP JP2014554840A patent/JP6193888B2/ja active Active
- 2013-01-25 EP EP13740919.9A patent/EP2809322B9/en active Active
- 2013-01-25 WO PCT/US2013/023067 patent/WO2013112788A1/en not_active Ceased
- 2013-01-25 EA EA201491412A patent/EA028344B1/ru not_active IP Right Cessation
- 2013-01-25 US US14/373,937 patent/US9399649B2/en active Active
- 2013-01-25 PL PL13740919T patent/PL2809322T3/pl unknown
-
2017
- 2017-04-20 JP JP2017083760A patent/JP2017171667A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP2809322A1 (en) | 2014-12-10 |
| BR112014018027B1 (pt) | 2020-10-27 |
| KR102064624B1 (ko) | 2020-01-09 |
| JP6193888B2 (ja) | 2017-09-06 |
| EP2809322B1 (en) | 2019-05-15 |
| EP2809322A4 (en) | 2015-12-23 |
| US9399649B2 (en) | 2016-07-26 |
| US20150126515A1 (en) | 2015-05-07 |
| MX357834B (es) | 2018-07-26 |
| CA2862084A1 (en) | 2013-08-01 |
| BR112014018027A8 (pt) | 2017-07-11 |
| CA2862084C (en) | 2021-05-11 |
| KR20140127271A (ko) | 2014-11-03 |
| HK1202069A1 (en) | 2015-09-18 |
| BR112014018027A2 (pl) | 2017-06-20 |
| JP2015511224A (ja) | 2015-04-16 |
| EA201491412A1 (ru) | 2014-12-30 |
| EA028344B1 (ru) | 2017-11-30 |
| CN104203239A (zh) | 2014-12-10 |
| CN104203239B (zh) | 2017-09-08 |
| JP2017171667A (ja) | 2017-09-28 |
| EP2809322B9 (en) | 2019-10-30 |
| MX2014008875A (es) | 2014-10-14 |
| WO2013112788A1 (en) | 2013-08-01 |
| ES2733644T3 (es) | 2019-12-02 |
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