SG11201911615WA - Compounds for treating huntington's disease - Google Patents

Compounds for treating huntington's disease

Info

Publication number
SG11201911615WA
SG11201911615WA SG11201911615WA SG11201911615WA SG11201911615WA SG 11201911615W A SG11201911615W A SG 11201911615WA SG 11201911615W A SG11201911615W A SG 11201911615WA SG 11201911615W A SG11201911615W A SG 11201911615WA SG 11201911615W A SG11201911615W A SG 11201911615WA
Authority
SG
Singapore
Prior art keywords
disease
compounds
treating huntington
huntington
treating
Prior art date
Application number
SG11201911615WA
Inventor
Matthew Woll
Lukiana Amedzo
Suresh Babu
Scott Barraza
Anuradha Bhattacharyya
Gary Karp
Anthony Mazzotti
Jana Narasimhan
Jigar Patel
Anthony Turpoff
Zhenrong Xu
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 SG11201911615WA publication Critical patent/SG11201911615WA/en

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/14Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47Quinolines; Isoquinolines
    • A61K31/4709Non-condensed quinolines and containing further heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/50Pyridazines; Hydrogenated pyridazines
    • A61K31/5025Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D413/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D417/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/14Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 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
    • 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
    • C07D471/08Bridged 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
    • C07D491/00Heterocyclic 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
    • C07D491/02Heterocyclic 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
    • C07D491/04Ortho-condensed systems
    • C07D491/044Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
    • C07D491/048Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D498/00Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/04Ortho-condensed systems
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic 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

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (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)
SG11201911615WA 2017-06-05 2018-06-05 Compounds for treating huntington's disease SG11201911615WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762514999P 2017-06-05 2017-06-05
PCT/US2018/035954 WO2018226622A1 (en) 2017-06-05 2018-06-05 Compounds for treating huntington's disease

Publications (1)

Publication Number Publication Date
SG11201911615WA true SG11201911615WA (en) 2020-01-30

Family

ID=64566872

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201911615WA SG11201911615WA (en) 2017-06-05 2018-06-05 Compounds for treating huntington's disease

Country Status (13)

Country Link
US (2) US11407753B2 (en)
EP (2) EP4151627A1 (en)
JP (1) JP7376471B2 (en)
KR (1) KR20200033249A (en)
CN (1) CN111065626A (en)
AU (2) AU2018282154B2 (en)
BR (1) BR112019025740A2 (en)
CA (1) CA3066224A1 (en)
EA (1) EA201992878A1 (en)
IL (2) IL300875A (en)
MX (1) MX2019014514A (en)
SG (1) SG11201911615WA (en)
WO (1) WO2018226622A1 (en)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2021001091A (en) 2015-12-10 2022-04-26 Ptc Therapeutics Inc Methods for treatng huntington's disease.
US11407753B2 (en) 2017-06-05 2022-08-09 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
BR112019027719A2 (en) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. methods to treat huntington's disease
JP7399870B2 (en) 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 Compounds to treat Huntington's disease
EP3814360A1 (en) 2018-06-27 2021-05-05 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
JP7421507B2 (en) 2018-06-27 2024-01-24 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 Heterocyclic and heteroaryl compounds for treating Huntington's disease
CN114174514A (en) 2019-07-25 2022-03-11 诺华股份有限公司 Adjustable expression system
CA3156848A1 (en) 2019-11-01 2021-05-06 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
TW202208358A (en) 2020-05-13 2022-03-01 美商Chdi基金會股份有限公司 Htt modulators for treating huntington’s disease
JP2023532331A (en) 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modifiers for splicing nucleic acids and for treating proliferative diseases and compounds and methods for modulating splicing
TW202216710A (en) 2020-07-02 2022-05-01 美商雷密克斯醫療公司 Compounds and methods for modulating splicing
CA3199442A1 (en) 2020-11-12 2022-05-19 Ptc Therapeutics Inc. Novel rna transcript
TW202237119A (en) * 2020-12-10 2022-10-01 美商住友製藥腫瘤公司 Alk-5 inhibitors and uses thereof
KR20230157367A (en) * 2021-03-17 2023-11-16 에프. 호프만-라 로슈 아게 New thiazolopyrimidinone derivatives
JP2024510487A (en) * 2021-03-17 2024-03-07 エフ. ホフマン-ラ ロシュ アーゲー Novel thiadiazolopyrimidone derivatives
TW202346305A (en) * 2022-01-05 2023-12-01 美商雷密克斯醫療公司 Compounds and methods for modulating splicing
US20220151938A1 (en) * 2022-01-28 2022-05-19 Ptc Therapeutics, Inc. Tablet for use in treating huntington's disease and method of making the same
WO2023170115A1 (en) * 2022-03-10 2023-09-14 F. Hoffmann-La Roche Ag Pyrido[1,2-a]pyrimidin-4-one derivatives
WO2023225244A1 (en) * 2022-05-20 2023-11-23 Biogen Ma Inc. Heterocyclic compounds for treating huntington's disease

Family Cites Families (188)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BE637271A (en) 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
JPS5852307A (en) 1981-09-22 1983-03-28 Hitachi Chem Co Ltd Water-dispersed resin composition
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5599816A (en) 1990-05-02 1997-02-04 Abbott Laboratories Quinolizinone type compounds
EP0640083A1 (en) 1992-05-13 1995-03-01 E.I. Du Pont De Nemours And Company Substituted pyrido 1,2-a]pyrimidinone derivatives as fungicides
JPH08510130A (en) 1993-05-11 1996-10-29 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Antisense oligonucleotides that inhibit 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
EP1115724A1 (en) 1998-09-21 2001-07-18 Shire Biochem 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
EP1227084B1 (en) 1999-10-28 2005-12-14 Trine Pharmaceuticals, Inc. Drug discharge pump inhibitors
CA2398163C (en) 2000-01-24 2011-02-22 Kinacia Pty Ltd. Therapeutic morpholino-substituted compounds
US20050054836A1 (en) 2000-11-09 2005-03-10 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
ATE528303T1 (en) 2000-12-21 2011-10-15 Vertex Pharma PYRAZOLE COMPOUNDS AS PROTEIN KINASE INHIBITORS
CA2445697A1 (en) 2001-04-26 2002-11-07 Daiichi Pharmaceutical Co., Ltd. Drug efflux pump inhibitor
EP1474147B1 (en) 2001-12-07 2010-05-05 Vertex Pharmaceuticals Incorporated Pyrimidine-based compounds useful as gsk-3 inhibitors
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
CA2493458A1 (en) 2002-07-24 2004-01-29 Ptc Therapeutics, Inc. Ureido substituted benzoic acid compounds, their use for nonsense suppression and the treatment of diseases caused by such mutations
ATE426602T1 (en) 2002-09-30 2009-04-15 Neurosearch As 1,4-DIAZABICYCLOACANDERIVATES, THEIR PREPARATION AND USE
MXPA05005116A (en) * 2002-11-12 2005-07-01 Abbott Lab Bicyclic-substituted amines as histamine-3 receptor ligands.
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
ES2339862T3 (en) 2003-06-20 2010-05-26 Novartis Vaccines And Diagnostics, Inc. PIRIDINE COMPOUNDS 1,2-A-PIRIMIDIN-4-ONA AS ANTI-TARGET AGENTS.
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
US7511145B2 (en) * 2003-08-01 2009-03-31 Genelabs Technologies, 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
KR20060127909A (en) 2003-12-24 2006-12-13 비오타 사이언티픽 매니지먼트 피티와이 엘티디 Polycyclic agents for the treatment of respiratory syncytial virus infections
SE0400184D0 (en) 2004-01-30 2004-01-30 Fyrkloevern Scandinavia Ab New therapeutic use
TW200536830A (en) 2004-02-06 2005-11-16 Chugai Pharmaceutical Co Ltd 1-(2H)-isoquinolone derivative
CN101426772B (en) * 2004-04-08 2014-01-08 塔尔基公司 Benzotriazine inhibitors of kinases
AU2005232672A1 (en) * 2004-04-08 2005-10-27 Neurogen Corporation Substituted cinnolin-4-ylamines
JP2007536370A (en) 2004-05-04 2007-12-13 ワーナー−ランバート カンパニー リミテッド ライアビリティー カンパニー Pyrrolyl-substituted pyrido [2,3-d] pyrimidin-7-one and its derivatives as therapeutic agents
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
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 (en) * 2005-02-14 2006-08-24 Tokyo Univ Of Pharmacy & Life Science Metal-discrimination-type two-color fluorescent molecule containing quinoline ring as parent nucleus
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
US8501739B2 (en) 2005-07-04 2013-08-06 High Point Pharmaceuticals, Llc Medicaments
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
WO2007056580A2 (en) * 2005-11-08 2007-05-18 Pharmadyn, Inc. Methods and compositions for treating diseases associated with pathogenic proteins
AU2006315758A1 (en) 2005-11-10 2007-05-24 Ercole Biotech, Inc. Splice switching oligomers for TNF superfamily receptors and their use in treatment of disease
BRPI0620113A2 (en) * 2005-12-21 2017-11-21 Painceptor Pharma Corp methods of modulating the activity of an introduced ion channel, and of treating a disorder, and, composed
US7951934B2 (en) 2006-01-26 2011-05-31 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
JP5213723B2 (en) 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 Oligomer compounds and compositions for use in modulating microRNA
AR059339A1 (en) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd CUMARINE DERIVATIVES FOR PROLIFERATIVE DISORDERS OF CELLS, PHARMACEUTICAL COMPOSITION AND THERAPEUTIC AGENT CONTAINING THEM
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
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
JP2009537599A (en) 2006-05-23 2009-10-29 ノイロサーチ アクティーゼルスカブ Novel 8,10-diaza-bicyclo [4.3.1] decane derivatives and their medical use
DE602007006835D1 (en) * 2006-07-20 2010-07-08 Amgen Inc SUBSTITUTED PYRIDONE COMPOUNDS AND APPLICATION METHODS
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
CA2659696C (en) 2006-08-03 2014-04-22 Rottapharm S.P.A. 6-1h-imidazo-quinazoline and quinolines derivatives, new potent analgesics and anti-inflammatory agents
WO2008020302A2 (en) * 2006-08-17 2008-02-21 Pfizer Products Inc. Heteroaromatic quinoline-based compounds as phosphodiesterase (pde) inhibitors
WO2008049864A1 (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 (en) 2006-12-22 2011-03-10 Avexa Ltd BICYCLIC PYRIMIDINONE AND ITS USE
WO2008083226A2 (en) 2006-12-28 2008-07-10 Navinta Llc Process for preparation of liquid dosage form containing sodium 4-phenylbutyrate
FR2914188B1 (en) 2007-03-28 2012-06-22 Trophos NEW CHOLEST-4-EN-3-ONE OXIME COMPOSITION
EP2014656A3 (en) 2007-06-11 2011-08-24 High Point Pharmaceuticals, LLC New heteocyclic h3 antagonists
CA2700841A1 (en) 2007-09-27 2009-04-02 The United States Of America, As Represented By The Secretary, Departmen T Of Health And Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
ES2548768T3 (en) 2007-09-27 2015-10-20 Fundación Centro Nacional De Investigaciones Oncológicas Carlos Iii Imidazolothiadiazoles for use as protein kinase inhibitors
US8153813B2 (en) 2007-12-20 2012-04-10 Abbott Laboratories Benzothiazole and benzooxazole derivatives and methods of use
WO2009114874A2 (en) 2008-03-14 2009-09-17 Intellikine, Inc. Benzothiazole kinase inhibitors and methods of use
WO2009126635A1 (en) 2008-04-09 2009-10-15 Abbott Laboratories 2-amino-benzothiazole derivates 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
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
BRPI0822462A2 (en) * 2008-06-20 2015-10-13 Rottapharm Spa 6-1h-imidazo-quinazoline and quinoline derivatives, novel hand inhibitors and imidazoline receptor binders.
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
EP2212324B1 (en) 2008-07-02 2014-03-26 Avexa Limited Compounds having antiviral properties
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
JP2011529722A (en) 2008-08-14 2011-12-15 カーディアック ペースメイカーズ, インコーポレイテッド Performance evaluation and adaptation of acoustic communication links
GB2465405A (en) 2008-11-10 2010-05-19 Univ Basel Triazine, pyrimidine and pyridine analogues and their use in therapy
WO2010071819A1 (en) 2008-12-19 2010-06-24 Schering Corporation Bicyclic heterocyclic derivatives and methods of use thereof
RU2011137419A (en) 2009-02-11 2013-03-20 Суновион Фармасьютикалз Инк. REVERSE AGONISTS AND HISTAMINE H3 ANTAGONISTS AND WAYS OF THEIR APPLICATION
FR2945289A1 (en) 2009-05-11 2010-11-12 Sanofi Aventis 2-CYCLOAMINO-5- (PYRIDIN-4-YL) IMIDAZO-2,1-B1 DERIVATIVES 1,3,4! THIADIAZOLE, THEIR PREPARATION AND THEIR THERAPEUTIC APPLICATION
RU2549551C2 (en) 2009-06-19 2015-04-27 Эббви Инк. Diazahomoadamantane derivatives and methods of using them
SI2475675T1 (en) 2009-09-11 2017-03-31 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
WO2011062853A1 (en) 2009-11-20 2011-05-26 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
EP2523956A1 (en) 2010-01-13 2012-11-21 Institut Pasteur Korea Anti - infective pyrido (1,2 -a) pyrimidines
EP3628750A1 (en) 2010-02-08 2020-04-01 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
AU2011213562B2 (en) 2010-02-08 2016-07-07 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP2575866A4 (en) * 2010-05-24 2013-10-16 Presidio Pharmaceuticals Inc Inhibitors of hcv ns5a
EP2595664B1 (en) 2010-07-19 2018-10-17 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
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
PT2672823T (en) * 2011-02-07 2016-11-21 Biogen Ma Inc S1p modulating agents
US10017764B2 (en) 2011-02-08 2018-07-10 Ionis 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
US9447075B2 (en) 2011-08-02 2016-09-20 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as EAAT2 activators
US8871756B2 (en) 2011-08-11 2014-10-28 Hoffmann-La Roche Inc. Compounds for the treatment and prophylaxis of Respiratory Syncytial Virus disease
EP2742135B2 (en) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
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
EP2751269B1 (en) 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
WO2013059606A1 (en) 2011-10-21 2013-04-25 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
AU2012345557A1 (en) 2011-11-28 2014-06-26 Novartis Ag Novel trifluoromethyl-oxadiazole derivatives and their use in the treatment of disease
EP2797592B1 (en) 2011-12-30 2019-08-28 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
KR102064624B1 (en) 2012-01-26 2020-01-09 피티씨 테라퓨틱스, 인크. Compounds for treating spinal muscular atrophy
KR102137087B1 (en) 2012-02-10 2020-07-24 피티씨 테라퓨틱스, 인크. Compounds for treating spinal muscular atrophy
CA2865957C (en) 2012-03-01 2020-02-11 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CN104470909B (en) 2012-03-23 2018-04-24 Ptc医疗公司 For treating the compound of Duchenne-Arandisease
HUE054031T2 (en) 2012-04-24 2021-08-30 Vertex Pharma Dna-pk inhibitors
EP2872493B1 (en) 2012-07-13 2018-11-14 Indiana University Research and Technology Corporation 5,6,7-trimethoxy 4-phenyl quinolin-2-one derivatives for treatment of spinal muscular atrophy
MY174339A (en) 2012-08-13 2020-04-09 Novartis Ag 1,4-disubstituted pyridazine analogs and methods for treating smn-deficiency-related conditions
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
CA2887884A1 (en) 2012-10-12 2014-04-17 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 (en) * 2012-11-01 2018-01-10 株式会社リコー Electrochromic compound, electrochromic composition, and display element
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
ES2817050T3 (en) 2013-02-04 2021-04-06 Ionis Pharmaceuticals Inc Selective antisense compounds and uses thereof
EP2964649B1 (en) 2013-03-05 2017-05-10 Merck Patent GmbH Triazolo[4,5-d]pyrimidine derivatives for the treatment of diseases such as cancer
CA2910508A1 (en) 2013-05-14 2014-11-20 Song Feng Aza-oxo-indoles for the treatment and prophylaxis of respiratory syncytial virus infection
MX363456B (en) 2013-06-25 2019-03-25 Hoffmann La Roche Compounds for treating spinal muscular atrophy.
AU2014296255B2 (en) 2013-07-31 2017-08-03 Novartis Ag 1,4-disubstituted pyridazine derivatives and their use for treating SMN-deficiency-related conditions
MX2016001963A (en) 2013-08-19 2016-05-26 Hoffmann La Roche Screening method.
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
US10195202B2 (en) 2013-12-19 2019-02-05 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
KR102423317B1 (en) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 Chiral design
CN105899493B (en) 2014-01-17 2019-03-29 诺华股份有限公司 For inhibiting the active 1- of SHP2 (triazine -3- base/pyridazine -3- base)-piperazine (- piperazine) piperidine derivatives and combinations thereof
AR099134A1 (en) 2014-01-24 2016-06-29 Hoffmann La Roche PROCEDURE FOR THE PREPARATION OF N - [(3-AMINOOXETAN-3-IL) METHYL] -2- (1,1-DIOXO-3,5-DIHIDRO-1,4-BENZOTIAZEPÍN-4-IL) -6-METIL- QUINAZOLÍN-4-AMINA
WO2015136947A1 (en) 2014-03-14 2015-09-17 Raqualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
CA2942975C (en) 2014-03-19 2023-08-29 Amydis Diagnostics, Inc. Substituted acrylamide and substituted ethenesulfonamide derivatives and pharmaceutical compositions thereof useful as amyloid targeting agents
PL3663296T3 (en) 2014-05-15 2023-08-07 F. Hoffmann-La Roche Ag Process for the preparation of compounds useful for treating spinal muscular atrophy
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US20170121197A1 (en) 2014-06-17 2017-05-04 Novozymes A/S Biological Phosphorus Removal from Wastewater
EP3160967B1 (en) 2014-06-25 2019-05-08 F. Hoffmann-La Roche AG N-(imidazo[1,2-a]pyrazin-6-yl)-benzamide compounds for treating spinal muscular atrophy
GB201419579D0 (en) * 2014-11-03 2014-12-17 Iomet Pharma Ltd Pharmaceutical compound
JP6839094B2 (en) 2014-12-24 2021-03-03 ユニクア・アイピー・ベーフェー RNAi-induced huntingtin gene suppression
DK3244891T3 (en) 2015-01-16 2022-10-24 Massachusetts Gen Hospital COMPOUNDS TO ENHANCE MRNA SPLICING
JP6884102B2 (en) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト Compounds for the treatment of cancer
GB201502567D0 (en) 2015-02-16 2015-04-01 Sentinel Oncology Ltd Pharmaceutical compounds
ES2914224T3 (en) 2015-04-07 2022-06-08 Fujikura Ltd Loose tube, loose tube type optical fiber cable, single fiber insulation method for loose tube optical fiber ribbon, loose tube manufacturing method and method for assembling a plurality of optical fibers
GB201506933D0 (en) 2015-04-23 2015-06-10 Sentinel Oncology Ltd Pharmaceutical compounds
CN104897232B (en) * 2015-04-30 2018-10-12 青岛海尔股份有限公司 The use volume measurement method of refrigerator and refrigerator
EP3298017B1 (en) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Compounds for treating spinal muscular atrophy
WO2016196386A1 (en) 2015-05-30 2016-12-08 Ptc Therapeutics, Inc. Methods for modulating rna splicing
MA43072A (en) 2015-07-22 2018-05-30 Wave Life Sciences Ltd COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
US10226448B2 (en) 2015-08-03 2019-03-12 Samumed, Llc 3-(1H-pyrrolo[3,2-C]pyridin-2-yl)-1H-pyrazolo[3,4-B]pyridines and therapeutic uses thereof
WO2017059251A1 (en) * 2015-10-02 2017-04-06 Incyte Corporation Heterocyclic compounds useful as pim kinase inhibitors
CN108289959B (en) 2015-11-12 2021-07-16 豪夫迈·罗氏有限公司 Composition for treating spinal muscular atrophy
KR20180081520A (en) 2015-11-12 2018-07-16 에프. 호프만-라 로슈 아게 Compounds for the Treatment of Amyotrophic Lateral Sclerosis
US10383867B2 (en) * 2015-11-28 2019-08-20 Russell Dahl Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders
CN108137579B (en) 2015-12-10 2022-01-07 豪夫迈·罗氏有限公司 Bridged piperidine derivatives
MX2021001091A (en) 2015-12-10 2022-04-26 Ptc Therapeutics Inc Methods for treatng huntington's disease.
JP6549785B2 (en) 2016-04-01 2019-07-24 京セラ株式会社 User equipment and method
US10526345B2 (en) 2016-04-08 2020-01-07 Mankind Pharma Ltd. Compounds as GPR119 agonists
AR108325A1 (en) 2016-04-27 2018-08-08 Samumed Llc ISOQUINOLIN-3-IL CARBOXAMIDS AND PREPARATION AND USE OF THE SAME
MA45270A (en) 2016-05-04 2017-11-09 Wave Life Sciences Ltd COMPOSITIONS OF OLIGONUCLEOTIDES AND RELATED PROCESSES
CN109475531B (en) 2016-05-31 2021-08-17 得克萨斯州立大学董事会 Heterocyclic inhibitors of PTPN11
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
TW201819386A (en) 2016-10-24 2018-06-01 美商傳達治療有限公司 SHP2 phosphatase inhibitors and methods of use thereof
US11857599B2 (en) 2017-04-03 2024-01-02 Acceleron Pharma Inc. Compositions and methods for treating spinal muscular atrophy
EP3630770A1 (en) 2017-05-26 2020-04-08 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine derivatives as shp2 phosphatase inhibitors
US11407753B2 (en) 2017-06-05 2022-08-09 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
BR112019027719A2 (en) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. methods to treat huntington's disease
EP3661509A4 (en) 2017-08-04 2021-01-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3755699A1 (en) 2018-02-21 2020-12-30 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
EP3768680A1 (en) 2018-03-21 2021-01-27 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine shp2 phosphatase inhibitors and methods of use thereof
TW202003471A (en) 2018-03-21 2020-01-16 美商傳達治療有限公司 SHP2 phosphatase inhibitors and methods of use thereof
JP7399870B2 (en) 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 Compounds to treat Huntington's disease
CN112805280A (en) 2018-06-27 2021-05-14 Ptc医疗公司 Heteroaryl compounds for the treatment of huntington's disease
EP3814360A1 (en) 2018-06-27 2021-05-05 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
JP7421507B2 (en) 2018-06-27 2024-01-24 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 Heterocyclic and heteroaryl compounds for treating Huntington's disease
CA3139821A1 (en) 2019-05-13 2020-11-19 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
CA3156848A1 (en) 2019-11-01 2021-05-06 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease

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