JP7421507B2 - ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物 - Google Patents

ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物 Download PDF

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JP7421507B2
JP7421507B2 JP2020572895A JP2020572895A JP7421507B2 JP 7421507 B2 JP7421507 B2 JP 7421507B2 JP 2020572895 A JP2020572895 A JP 2020572895A JP 2020572895 A JP2020572895 A JP 2020572895A JP 7421507 B2 JP7421507 B2 JP 7421507B2
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pyridazin
tetramethylpiperidin
triazolo
phenol
pyrazol
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JPWO2020005873A5 (enExample
JP2021528467A (ja
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ナディヤ シドレンコ
エムディー ラウフル アラム
マイケル エー アーノルド
スレシュ バブ
アヌラダ バタチャリア
グアンミン チェン
アレクセイ アイ ゲラシュト
ギャリー ミッチェル カープ
アンドリュー ジェイ カシック
アンソニー アール マゾッティ
ヨン－チョン ムーン
ジャナ ナラシムハン
ジガール パテル
アンソニー ターポフ
マシュー ジー ヴォル
ウーミン ヤン
ナンジン ジャン
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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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C07D491/048—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
    • 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
    • C07D491/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04—Ortho-condensed systems
    • 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

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Plural Heterocyclic Compounds (AREA)
JP2020572895A 2018-06-27 2019-06-25 ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物 Active JP7421507B2 (ja)

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US201862690653P 2018-06-27 2018-06-27
US62/690,653 2018-06-27
PCT/US2019/038889 WO2020005873A1 (en) 2018-06-27 2019-06-25 Heterocyclic and heteroaryl compounds for treating huntington's disease

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JP2021528467A JP2021528467A (ja) 2021-10-21
JPWO2020005873A5 JPWO2020005873A5 (enExample) 2022-06-21
JP7421507B2 true JP7421507B2 (ja) 2024-01-24

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EP (2) EP4434990A1 (enExample)
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KR (1) KR102823158B1 (enExample)
CN (1) CN112654625B (enExample)
AR (1) AR119651A1 (enExample)
AU (2) AU2019294478B2 (enExample)
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CA (1) CA3103976A1 (enExample)
CL (3) CL2020003378A1 (enExample)
CO (1) CO2020016285A2 (enExample)
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EA (1) EA202092896A1 (enExample)
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Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
KR20200033249A (ko) 2017-06-05 2020-03-27 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
US11395822B2 (en) 2017-06-28 2022-07-26 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
JP7569688B2 (ja) 2017-12-22 2024-10-18 ハイバーセル，インコーポレイテッド ホスファチジルイノシトールリン酸キナーゼ阻害剤としてのアミノピリジン誘導体
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
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
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
WO2020190793A1 (en) * 2019-03-15 2020-09-24 Skyhawk Therapeutics, Inc. Compositions and methods for correction of aberrant splicing
US20220204478A1 (en) * 2019-05-13 2022-06-30 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
TW202112767A (zh) 2019-06-17 2021-04-01 美商佩特拉製藥公司 作為磷脂酸肌醇磷酸激酶抑制劑之胺基吡啶衍生物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
AU2020377204A1 (en) * 2019-11-01 2022-06-02 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
JP7736700B2 (ja) * 2020-02-28 2025-09-09 リミックス セラピューティクス インコーポレイテッド 複素環アミド及びスプライシングを調節するためのその使用
EP4132923A1 (en) * 2020-04-09 2023-02-15 PTC Therapeutics, Inc. Compounds for treating huntington's disease
MX2022013856A (es) 2020-05-13 2023-04-05 Chdi Foundation Inc Moduladores htt para tratar la enfermedad de huntington.
WO2022006543A1 (en) 2020-07-02 2022-01-06 Remix Therapeutics Inc. 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2022006550A1 (en) 2020-07-02 2022-01-06 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2022060943A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060944A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060951A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
AU2021380758A1 (en) * 2020-11-12 2023-06-15 Ptc Therapeutics Inc. Novel rna transcript
IL302718A (en) 2020-11-13 2023-07-01 Ptc Therapeutics Inc A tablet for use in the treatment of Huntington's disease and a method for its preparation
WO2022194800A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thienopyrimidinone derivatives
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
JP2024528066A (ja) 2021-07-30 2024-07-26 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ヘテロアリール化合物
US20250129090A1 (en) * 2021-11-04 2025-04-24 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
JP2024540477A (ja) 2021-11-17 2024-10-31 シーエイチディーアイ ファウンデーション，インコーポレーテッド ハンチントン病を治療するためのｈｔｔモジュレータとしてのｎ－（２ｈ－インダゾール－５－イル）ピラジン－２－カルボキサミド誘導体及び類似化合物
JP2025516584A (ja) * 2022-05-10 2025-05-30 フォグホーン セラピューティクス インコーポレイテッド 化合物及びその使用
CA3249778A1 (en) * 2022-06-15 2023-12-21 Ptc Therapeutics, Inc. HETEROCYCLIC AND HETEROARYL COMPOUNDS FOR THE TREATMENT OF HUNTINGTON'S DISEASE
TW202432551A (zh) 2022-10-26 2024-08-16 日商安斯泰來製藥股份有限公司 稠環嗒嗪衍生物
AU2024211840A1 (en) 2023-01-24 2025-07-03 Daiichi Sankyo Company, Limited Substituted benzene compound
WO2024233306A1 (en) * 2023-05-09 2024-11-14 Ptc Therapeutics, Inc. Crystalline forms of 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3h-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2h-1,2,3-triazol-2-yl)phenol
WO2024249539A1 (en) * 2023-06-02 2024-12-05 Merck Sharp & Dohme Llc 5,6 unsaturated bicyclic heterocyles useful as inhibitors of nod-like receptor protein 3
WO2024249389A2 (en) * 2023-06-02 2024-12-05 Merck Sharp & Dohme Llc 5,6 saturated bicyclic heterocyles useful as inhibitors of nod-like receptor protein 3
WO2025040014A1 (zh) * 2023-08-18 2025-02-27 英矽智能科技知识产权有限公司 并环化合物及其制备方法和用途

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004517862A (ja) 2001-01-10 2004-06-17 バーナリス リサーチ リミテッド トリアゾロ［４，５−ｄ］ピリミジン誘導体およびプリン作用性レセプターアンタゴニストとしてのこれらの使用
WO2012103806A1 (en) 2011-01-31 2012-08-09 Centaurus Biopharma Co., Ltd. Bicyclic heteroaryl compounds as gpr119 receptor agonists
JP2015514804A (ja) 2012-04-24 2015-05-21 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｄｎａ−ｐｋ阻害剤
JP2015535257A (ja) 2012-10-25 2015-12-10 アッシャー・サード・イニシアティブ・インコーポレイテッド ピラゾロピリダジンならびに網膜変性疾患およびアッシャー症候群に伴う聴力損失を処置するための方法
JP2017502957A (ja) 2013-12-26 2017-01-26 武田薬品工業株式会社 モノアシルグリセロールリパーゼ（ｍａｇｌ）阻害剤としての４−（ピペラジン−１−イル）−ピロリジン−２−オン化合物
WO2017023987A1 (en) 2015-08-03 2017-02-09 Samumed, Llc. 3-(1h-pyrrolo[3,2-c]pyridin-2-yl)-1h-pyrazolo[3,4-b]pyridines and therapeutic uses thereof
WO2017100726A1 (en) 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
WO2017210134A1 (en) 2016-05-31 2017-12-07 Board Of Regents, University Of Texas System Heterocyclic inhibitors of ptpn11
WO2018081091A1 (en) 2016-10-24 2018-05-03 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine derivatives as shp2 phosphatase inhibitors

Family Cites Families (185)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BE637271A (enExample) 1963-04-04 1900-01-01
US3558618A (en) 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
GB1383409A (en) 1972-09-09 1974-02-12 Pfizer Ltd Derivatives of 2-amino- and 4-amino-quinazoline and pharmaceutical compositions containing them
US4122274A (en) 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
JPS56150091A (en) 1980-03-28 1981-11-20 Janssen Pharmaceutica Nv 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture
US4342870A (en) 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
FR2567518B1 (fr) 1984-07-11 1987-11-13 Sanofi Sa Nouveaux composes a noyau heterocyclique azote, leur preparation et les medicaments qui en contiennent
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5726182A (en) 1990-05-02 1998-03-10 Abbott Laboratories Quinolizinone type compounds
AU4231293A (en) 1992-05-13 1993-12-13 E.I. Du Pont De Nemours And Company Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides
CA2162361C (en) 1993-05-11 2008-10-21 Ryszard Kole Antisense oligonucleotides which combat aberrant splicing and methods of using the same
WO1996039407A1 (en) 1995-06-06 1996-12-12 Abbott Laboratories Quinolizinone type compounds
US5916916A (en) 1996-10-10 1999-06-29 Eli Lilly And Company 1-aryloxy-2-arylnaphthyl compounds, intermediates, compositions, and methods
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
CA2269561C (en) 1998-04-22 2007-06-05 Dainippon Ink And Chemicals, Inc. Naphthalene derivative and liquid crystal composition comprising the same
US6630488B1 (en) 1998-09-21 2003-10-07 Biochem Pharma, Inc. Quinolizinones as integrin inhibitors
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
DE60024861T2 (de) 1999-10-28 2006-07-06 Trine Pharmaceuticals, Inc., Waltham Pumpeninhibitoren zur freisetzung von medikamenten
CN1891699A (zh) 2000-01-24 2007-01-10 基纳西亚股份有限公司 用于治疗的吗啉代基取代的化合物
CA2437942C (en) 2000-11-09 2013-06-11 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US6774134B2 (en) 2000-12-20 2004-08-10 Bristol-Myers Squibb Company Heterocyclic substituted 2-methyl-benzimidazole antiviral agents
ATE354573T1 (de) 2000-12-21 2007-03-15 Vertex Pharma ßPYRAZOLVERBINDUNGEN, DIE SICH ALS PROTEINKINASEINHIBITOREN EIGNENß
CA2445697A1 (en) 2001-04-26 2002-11-07 Daiichi Pharmaceutical Co., Ltd. Drug efflux pump inhibitor
DE60236322D1 (de) 2001-12-07 2010-06-17 Vertex Pharma Verbindungen auf pyrimidin-basis als gsk-3-hemmer
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
ATE548354T1 (de) 2002-07-24 2012-03-15 Ptc Therapeutics Inc Ureido-substituierte benzoesäureverbindungen und ihre verwendung für die nonsense-suppression und behandlung von erkrankungen
AU2003262833A1 (en) 2002-08-23 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Fluorescent glycosides and methods for their use
NZ538512A (en) 2002-09-30 2006-12-22 Neurosearch As 1,4-diazabicycloalkane derivatives, their preparation and use in treatment of disorders responsive to modulation of cholmergic and/or monoamine receptors
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
ATE458735T1 (de) 2003-06-20 2010-03-15 Novartis Vaccines & Diagnostic Pyridino[1,2-a pyrimidin-4-onverbindungen als mittel gegen krebs
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
TW200517381A (en) 2003-08-01 2005-06-01 Genelabs Tech Inc Bicyclic heteroaryl derivatives
US7160888B2 (en) 2003-08-22 2007-01-09 Warner Lambert Company Llc [1,8]naphthyridin-2-ones and related compounds for the treatment of schizophrenia
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US7655657B2 (en) 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US20050137203A1 (en) 2003-12-22 2005-06-23 Jianguo Ji 3-quinuclidinyl amino-substituted biaryl derivatives
US20050245531A1 (en) 2003-12-22 2005-11-03 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
CN100582109C (zh) 2003-12-24 2010-01-20 生物区科学管理控股有限公司 用于治疗呼吸道合胞体病毒感染的多环试剂
SE0400184D0 (sv) 2004-01-30 2004-01-30 Fyrkloevern Scandinavia Ab New therapeutical use
TW200536830A (en) 2004-02-06 2005-11-16 Chugai Pharmaceutical Co Ltd 1-(2H)-isoquinolone derivative
EP1732560A4 (en) 2004-04-08 2010-08-18 Neurogen Corp SUBSTITUTED CINNOLINE-4-YLAMINE
BRPI0510560A (pt) 2004-05-04 2007-11-20 Warner Lambert Co pirido[2,3-d] pirimidin-7-onas pirrolil substituìdas e seus derivados como agentes terapêuticos
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
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
GB0501999D0 (en) 2005-02-01 2005-03-09 Sentinel Oncology Ltd Pharmaceutical compounds
JP2006219453A (ja) 2005-02-14 2006-08-24 Tokyo Univ Of Pharmacy & Life Science キノリン環を母核とする金属識別型二波長性蛍光分子
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
WO2006138418A2 (en) 2005-06-14 2006-12-28 President And Fellows Of Harvard College Improvement of cognitive performance with sirtuin activators
KR101286569B1 (ko) 2005-07-04 2013-07-23 하이 포인트 파마슈티칼스, 엘엘씨 신규 약제
US7473784B2 (en) 2005-08-01 2009-01-06 Bristol-Myers Squibb Company Benzothiazole and azabenzothiazole compounds useful as kinase inhibitors
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
EP2535413A3 (en) 2005-11-10 2016-04-13 Roche Innovation Center Copenhagen A/S Splice switching oligomers for the TNF superfamily receptors and their use in treatment of disease
CN101305004B (zh) 2005-12-06 2011-11-16 神经研究公司 新颖的二氮杂双环芳基衍生物和它们的医药用途
US20070197509A1 (en) 2005-12-21 2007-08-23 Painceptor Pharma Corporation Compositions and methods for modulating gated ion channels
WO2007089611A2 (en) 2006-01-26 2007-08-09 Isis Pharmaceuticals Inc. Compositions and their uses directed to huntingtin
JP5213723B2 (ja) 2006-01-27 2013-06-19 アイシス ファーマシューティカルズ， インコーポレーテッド マイクロｒｎａの調節に使用するためのオリゴマー化合物及び組成物
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
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
CN101410385B (zh) 2006-03-28 2011-08-24 高点制药有限责任公司 具有组胺h3受体活性的苯并噻唑类
WO2007130383A2 (en) 2006-04-28 2007-11-15 Northwestern University Compositions and treatments using pyridazine compounds and secretases
US20080247964A1 (en) 2006-05-08 2008-10-09 Yuelian Xu Substituted azaspiro derivatives
TW200812588A (en) * 2006-05-15 2008-03-16 Neurogen Corp CRF1 receptor ligands comprising heteroaryl fused bicycles
US7910578B2 (en) 2006-05-23 2011-03-22 Neurosearch A/S 8,10-diaza-bicyclo[4.3.1]decane derivatives and their medical use
WO2008011109A2 (en) 2006-07-20 2008-01-24 Amgen Inc. Substituted pyridone compounds and methods of use
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
ES2394971T3 (es) 2006-08-03 2013-02-07 Rottapharm S.P.A. Derivados de quinolinas y 6-1H-imidazo-quinazolina, agentes antiinflamatorios y analgésicos potentes nuevos
CA2667545A1 (en) 2006-10-25 2008-05-02 Neurosearch A/S Oxadiazole and thiadiazole compounds and their use as nicotinic acetylcholine receptor modulators
US8314119B2 (en) 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
DE602007012308D1 (de) 2006-12-22 2011-03-10 Avexa Ltd Bicyclische pyrimidinone und deren verwendung
WO2008083226A2 (en) 2006-12-28 2008-07-10 Navinta Llc Process for preparation of liquid dosage form containing sodium 4-phenylbutyrate
FR2914188B1 (fr) 2007-03-28 2012-06-22 Trophos Nouvelle composition a base d'oxime de cholest-4-en-3-one
EP2014656A3 (en) 2007-06-11 2011-08-24 High Point Pharmaceuticals, LLC New heteocyclic h3 antagonists
WO2009042907A1 (en) 2007-09-27 2009-04-02 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
PL2193133T3 (pl) 2007-09-27 2016-01-29 Fundacion Centro Nac De Investigaciones Oncologicas Carlos Iii Imidazolotiadiazole do stosowania jako inhibitory kinazy białkowej
US8153813B2 (en) 2007-12-20 2012-04-10 Abbott Laboratories Benzothiazole and benzooxazole derivatives and methods of use
US8993580B2 (en) 2008-03-14 2015-03-31 Intellikine Llc Benzothiazole kinase inhibitors and methods of use
US20090258907A1 (en) 2008-04-09 2009-10-15 Abbott Laboratories Compounds useful as inhibitors of rock kinases
US20090264433A1 (en) 2008-04-21 2009-10-22 Institute For Oneworld Health Compounds, Compositions and Methods Comprising Triazine Derivatives
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
KR20110036583A (ko) 2008-06-20 2011-04-07 로타팜 에스.피.에이. 6-1ｈ-이미다조-퀴나졸린 및 퀴놀린 유도체, 신규 ｍａｏ 억제제 및 이미다졸린 수용체 리간드
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
US8318732B2 (en) 2008-07-02 2012-11-27 Avexa Limited Compounds having antiviral properties
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
WO2010024903A1 (en) 2008-08-29 2010-03-04 Yangbo Feng BENZO[d]OXAZOLES AND BENZO[d]THIAZOLES AS KINASE INHIBITORS
WO2010045303A2 (en) 2008-10-16 2010-04-22 Schering Corporation Pyrrolidine, piperidine and piperazine derivatives and methods of use thereof
GB2465405A (en) 2008-11-10 2010-05-19 Univ Basel Triazine, pyrimidine and pyridine analogues and their use in therapy
US8283360B2 (en) 2008-12-19 2012-10-09 Merck Sharp & Dohme Corp. Bicyclic heterocyclic derivatives and methods of use thereof
WO2010093425A1 (en) 2009-02-11 2010-08-19 Sepracor Inc. Histamine h3 inverse agonists and antagonists and methods of use thereof
FR2945289A1 (fr) 2009-05-11 2010-11-12 Sanofi Aventis Derives de 2-cycloamino-5-(pyridin-4-yl)imidazo°2,1-b! °1,3,4!thiadiazole, leur preparation et leur application en therapeutique
KR20120044978A (ko) 2009-06-19 2012-05-08 아보트 러보러터리즈 디아자호모아다만탄 유도체 및 이들의 사용 방법
EP3626823A1 (en) 2009-09-11 2020-03-25 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression
WO2011050245A1 (en) 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors
WO2011057204A2 (en) 2009-11-06 2011-05-12 The Johns Hopkins University Lrrk2-mediated neuronal toxicity
US8754220B2 (en) 2009-11-20 2014-06-17 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide M1 receptor positive allosteric modulators
CA2786329A1 (en) 2010-01-13 2011-07-21 Institut National De La Sante Et De La Recherche Medicale (Inserm) Anti - infective pyrido (1,2-a) pyrimidines
JP2013518603A (ja) 2010-02-08 2013-05-23 アイシス ファーマシューティカルズ， インコーポレーテッド 反復伸張に関連する疾患または病態の治療に有用な方法および組成物
EP3208347B1 (en) 2010-02-08 2019-08-14 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP3321361B1 (en) 2010-02-08 2019-03-27 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP2575866A4 (en) 2010-05-24 2013-10-16 Presidio Pharmaceuticals Inc HCV NS5A INHIBITORS
AU2011282217B2 (en) 2010-07-19 2015-12-03 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (DMPK) expression
WO2012019106A2 (en) 2010-08-06 2012-02-09 Board Of Regents Of The University Of Nebraska Positive and negative modulators of nmda receptors
WO2012075393A2 (en) 2010-12-02 2012-06-07 President And Fellows Of Harvard College Activators of proteasomal degradation and uses thereof
WO2012104823A2 (en) 2011-02-04 2012-08-09 Novartis Ag Pyridopyrimidinone compounds in the treatment of neurodegenerative diseases
WO2012109395A1 (en) 2011-02-08 2012-08-16 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US8703763B2 (en) 2011-03-02 2014-04-22 Hoffmann-La Roche Inc. Bridged piperidine derivatives
WO2013019938A1 (en) 2011-08-02 2013-02-07 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as eaat2 activators
EP3205725B1 (en) 2011-08-11 2019-03-27 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US8871756B2 (en) 2011-08-11 2014-10-28 Hoffmann-La Roche Inc. Compounds for the treatment and prophylaxis of Respiratory Syncytial Virus disease
EP2560008B1 (en) 2011-08-18 2016-11-30 Korea Institute of Science and Technology Pharmaceutical compositions for preventing or treating degenerative brain disease and method of screening the same
US9976138B2 (en) 2011-08-29 2018-05-22 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
US9662314B2 (en) 2011-10-21 2017-05-30 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
GB201119538D0 (en) 2011-11-10 2011-12-21 Viral Ltd Pharmaceutical compounds
US9670193B2 (en) 2011-11-28 2017-06-06 Novartis Ag Trifluoromethyl-oxadiazole derivatives and their use in the treatment of disease
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
US9212209B2 (en) 2012-07-13 2015-12-15 Indiana University Research And Technology Corporation Screening methods for spinal muscular atrophy
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
US20150275208A1 (en) 2012-10-12 2015-10-01 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US9227976B2 (en) * 2012-10-25 2016-01-05 Usher Iii Initiative, Inc. Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome
JP6255711B2 (ja) 2012-11-01 2018-01-10 株式会社リコー エレクトロクロミック化合物、エレクトロクロミック組成物及び表示素子
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
EP3778618A1 (en) 2013-02-04 2021-02-17 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
ES2636936T3 (es) 2013-03-05 2017-10-10 Merck Patent Gmbh Derivados de triazolo[4,5-d]pirimidina para el tratamiento de enfermedades tales como cáncer
MX2015015691A (es) 2013-05-14 2016-03-04 Hoffmann La Roche Aza-oxo-indoles novedosos para el tratamiento y profilaxis de la infeccion del virus sincital respiratorio.
CN105358151B (zh) 2013-06-25 2019-04-12 豪夫迈·罗氏有限公司 用于治疗脊髓性肌萎缩的化合物
PL3027600T3 (pl) 2013-07-31 2022-08-08 Novartis Ag 1,4-dwupodstawione pochodne pirydazyny i ich zastosowanie do leczenia stanów związanych z niedoborem smn
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
WO2015105657A1 (en) 2013-12-19 2015-07-16 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
ES2699354T3 (es) 2014-01-17 2019-02-08 Novartis Ag Derivados de 1-(triazin-3-il/piridazin-3-il)-piper(-azin)idina y composiciones de las mismas para inhibir la actividad de SHP2
AR099134A1 (es) 2014-01-24 2016-06-29 Hoffmann La Roche Procedimiento para la preparación de n-[(3-aminooxetán-3-il)metil]-2-(1,1-dioxo-3,5-dihidro-1,4-benzotiazepín-4-il)-6-metil-quinazolín-4-amina
CA2940621C (en) 2014-03-14 2022-03-15 Raqualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
EP3119761B1 (en) 2014-03-19 2023-06-07 Amydis Diagnostics, Inc. Amyloid targeting agents and methods of using the same
PL3663296T3 (pl) 2014-05-15 2023-08-07 F. Hoffmann-La Roche Ag Sposób wytwarzania związków użytecznych w leczeniu rdzeniowego zaniku mięśni
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN106573811A (zh) 2014-06-17 2017-04-19 诺维信公司 从废水中去除生物磷
JP6675334B2 (ja) 2014-06-25 2020-04-01 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するためのイミダゾ［１，２−ａ］ピラジン−１−イルベンズアミド化合物
EP3227301A1 (de) 2014-12-02 2017-10-11 Bayer CropScience AG Bicyclische verbindungen als schädlingsbekämpfungsmittel
EA037696B1 (ru) 2014-12-24 2021-05-12 ЮНИКЕР АйПи Б.В. Супрессия гена гентингтина, индуцированная рнк-интерференцией
EP3244891B9 (en) 2015-01-16 2022-11-16 The General Hospital Corporation Compounds for improving mrna splicing
EP3256126B1 (en) 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
GB201502567D0 (en) 2015-02-16 2015-04-01 Sentinel Oncology Ltd Pharmaceutical compounds
CN112679467B (zh) 2015-03-11 2024-11-01 Fmc公司 杂环取代的二环唑杀有害生物剂
GB201506933D0 (en) 2015-04-23 2015-06-10 Sentinel Oncology Ltd Pharmaceutical compounds
JP6749343B2 (ja) 2015-05-20 2020-09-02 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための化合物
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017059251A1 (en) 2015-10-02 2017-04-06 Incyte Corporation Heterocyclic compounds useful as pim kinase inhibitors
KR20180081520A (ko) 2015-11-12 2018-07-16 에프. 호프만-라 로슈 아게 근위축성 측삭 경화증의 치료를 위한 화합물
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
US10383867B2 (en) 2015-11-28 2019-08-20 Russell Dahl Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders
EP3386978B1 (en) 2015-12-10 2021-01-27 H. Hoffnabb-La Roche Ag Bridged piperidine derivatives
US10526345B2 (en) 2016-04-08 2020-01-07 Mankind Pharma Ltd. Compounds as GPR119 agonists
AR108325A1 (es) 2016-04-27 2018-08-08 Samumed Llc Isoquinolin-3-il carboxamidas y preparación y uso de las mismas
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
WO2018187209A1 (en) 2017-04-03 2018-10-11 Acceleron Pharma Inc. Compositions and methods for treating spinal muscular atrophy
WO2018218133A1 (en) * 2017-05-26 2018-11-29 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine derivatives as shp2 phosphatase inhibitors
KR20200033249A (ko) 2017-06-05 2020-03-27 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
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
US20200392161A1 (en) 2018-02-21 2020-12-17 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
WO2019183364A1 (en) 2018-03-21 2019-09-26 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine shp2 phosphatase inhibitors and methods of use thereof
SG11202009245TA (en) * 2018-03-21 2020-10-29 Relay Therapeutics Inc Shp2 phosphatase inhibitors and methods of use thereof
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
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
WO2020190793A1 (en) 2019-03-15 2020-09-24 Skyhawk Therapeutics, Inc. Compositions and methods for correction of aberrant splicing
US20220204478A1 (en) 2019-05-13 2022-06-30 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2021007378A1 (en) 2019-07-11 2021-01-14 Ptc Therapeutics, Inc. Compounds for use in treating huntington's disease
AU2020377204A1 (en) 2019-11-01 2022-06-02 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
EP4132923A1 (en) 2020-04-09 2023-02-15 PTC Therapeutics, Inc. Compounds for treating huntington's disease
AU2021380758A1 (en) 2020-11-12 2023-06-15 Ptc Therapeutics Inc. Novel rna transcript
JP2024528066A (ja) 2021-07-30 2024-07-26 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ヘテロアリール化合物

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004517862A (ja) 2001-01-10 2004-06-17 バーナリス リサーチ リミテッド トリアゾロ［４，５−ｄ］ピリミジン誘導体およびプリン作用性レセプターアンタゴニストとしてのこれらの使用
WO2012103806A1 (en) 2011-01-31 2012-08-09 Centaurus Biopharma Co., Ltd. Bicyclic heteroaryl compounds as gpr119 receptor agonists
JP2015514804A (ja) 2012-04-24 2015-05-21 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｄｎａ−ｐｋ阻害剤
JP2015535257A (ja) 2012-10-25 2015-12-10 アッシャー・サード・イニシアティブ・インコーポレイテッド ピラゾロピリダジンならびに網膜変性疾患およびアッシャー症候群に伴う聴力損失を処置するための方法
JP2017502957A (ja) 2013-12-26 2017-01-26 武田薬品工業株式会社 モノアシルグリセロールリパーゼ（ｍａｇｌ）阻害剤としての４−（ピペラジン−１−イル）−ピロリジン−２−オン化合物
WO2017023987A1 (en) 2015-08-03 2017-02-09 Samumed, Llc. 3-(1h-pyrrolo[3,2-c]pyridin-2-yl)-1h-pyrazolo[3,4-b]pyridines and therapeutic uses thereof
WO2017100726A1 (en) 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
WO2017210134A1 (en) 2016-05-31 2017-12-07 Board Of Regents, University Of Texas System Heterocyclic inhibitors of ptpn11
WO2018081091A1 (en) 2016-10-24 2018-05-03 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine derivatives as shp2 phosphatase inhibitors

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Bioorganic & Medicinal Chemistry，2014年，Vol.22(23)，p.6616-6624
RN:1349790-59-5、他全８化合物，DATABASE REGISTRY [ONLINE]，2011年12月06日，Retrieved from STN
RN:2224380-48-5、他全３化合物，DATABASE REGISTRY [ONLINE]，2018年05月20日，Retrieved from STN
Tetrahedron Letters，2014年，Vol.55(10)，p.1778-1783
Tetrahedron，2011年，Vol.67(29)，p.5293-5303

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