JP2019500352A5 - - Google Patents

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JP2019500352A5
JP2019500352A5 JP2018529967A JP2018529967A JP2019500352A5 JP 2019500352 A5 JP2019500352 A5 JP 2019500352A5 JP 2018529967 A JP2018529967 A JP 2018529967A JP 2018529967 A JP2018529967 A JP 2018529967A JP 2019500352 A5 JP2019500352 A5 JP 2019500352A5
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alkyl
amino
alkoxy
carbonyl
heteroaryl
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JP2018529967A
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JP2019500352A (ja
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Priority claimed from PCT/US2016/066042 external-priority patent/WO2017100726A1/en
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JP2018529967A 2015-12-10 2016-12-11 ハンチントン病の治療方法 Ceased JP2019500352A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562265652P 2015-12-10 2015-12-10
US62/265,652 2015-12-10
PCT/US2016/066042 WO2017100726A1 (en) 2015-12-10 2016-12-11 Methods for treatng huntington's disease

Related Child Applications (2)

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JP2020181114A Division JP7025508B2 (ja) 2015-12-10 2020-10-29 ハンチントン病の治療方法
JP2020181113A Division JP7053760B2 (ja) 2015-12-10 2020-10-29 ハンチントン病の治療方法

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JP2019500352A JP2019500352A (ja) 2019-01-10
JP2019500352A5 true JP2019500352A5 (enExample) 2020-01-30

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JP2018529967A Ceased JP2019500352A (ja) 2015-12-10 2016-12-11 ハンチントン病の治療方法
JP2020181114A Active JP7025508B2 (ja) 2015-12-10 2020-10-29 ハンチントン病の治療方法
JP2020181113A Active JP7053760B2 (ja) 2015-12-10 2020-10-29 ハンチントン病の治療方法
JP2022014271A Active JP7394893B2 (ja) 2015-12-10 2022-02-01 ハンチントン病の治療方法

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JP2020181114A Active JP7025508B2 (ja) 2015-12-10 2020-10-29 ハンチントン病の治療方法
JP2020181113A Active JP7053760B2 (ja) 2015-12-10 2020-10-29 ハンチントン病の治療方法
JP2022014271A Active JP7394893B2 (ja) 2015-12-10 2022-02-01 ハンチントン病の治療方法

Country Status (20)

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US (4) US10874672B2 (enExample)
EP (2) EP3848035B1 (enExample)
JP (4) JP2019500352A (enExample)
KR (2) KR20220100719A (enExample)
CN (4) CN113750101A (enExample)
AU (2) AU2016366694C1 (enExample)
CA (2) CA3119019C (enExample)
DK (1) DK3386511T3 (enExample)
EA (1) EA201800367A1 (enExample)
ES (1) ES2879995T3 (enExample)
HU (1) HUE055031T2 (enExample)
IL (3) IL294124B2 (enExample)
LT (1) LT3386511T (enExample)
MX (2) MX391850B (enExample)
PH (1) PH12018501226A1 (enExample)
PL (1) PL3386511T3 (enExample)
PT (1) PT3386511T (enExample)
SG (1) SG11201804915RA (enExample)
SI (1) SI3386511T1 (enExample)
WO (1) WO2017100726A1 (enExample)

Families Citing this family (77)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1260897A1 (zh) 2015-08-21 2019-12-27 Portola Pharmaceuticals, Inc. 使用四氢异喹啉小分子来结合并调节pcsk9的蛋白活性的组合物和方法
WO2017034994A1 (en) 2015-08-21 2017-03-02 Portola Pharmaceuticals, Inc. Composition and methods of use of novel phenylalanine small organic compounds to directly modulate pcsk9 protein activity
EP3337788A4 (en) 2015-08-21 2019-03-27 Portola Pharmaceuticals, Inc. PHENYLPIPERAZINE PROPROTEIN CONVERTASE SUBTILISIN / -KEXIN TYPE 9 P (CSK9) MODULATORS AND THEIR USE
CA3119019C (en) 2015-12-10 2024-06-11 Ptc Therapeutics, Inc. Compounds and methods for treating huntington's disease
WO2017147328A1 (en) 2016-02-23 2017-08-31 Portola Pharmaceuticals, Inc. Compounds for binding proprotein convertase subtilisin/kexin type 9 (pcsk9)
EP4151627A1 (en) 2017-06-05 2023-03-22 PTC Therapeutics, Inc. Compounds for treating huntington's disease
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
CN111163838B (zh) * 2017-06-28 2023-03-28 Ptc医疗公司 用于治疗亨廷顿氏病的方法
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
WO2019126731A1 (en) 2017-12-22 2019-06-27 Petra Pharma Corporation Aminopyridine derivatives as phosphatidylinositol phosphate kinase inhibitors
CA3090130A1 (en) * 2018-02-02 2019-08-08 Vanderbilt University Antagonists of the muscarinic acetylcholine receptor m4
BR112020019373A2 (pt) * 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. Compostos para o tratamento da doença de hutington
JP2021521200A (ja) * 2018-04-10 2021-08-26 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 癌の処置のための化合物
WO2020005882A1 (en) * 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
IL279688B2 (en) * 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
KR20210038845A (ko) 2018-06-27 2021-04-08 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로아릴 화합물
US12168012B2 (en) 2018-07-25 2024-12-17 Novartis Ag NLRP3 inflammasome inhibitors
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2022519637A (ja) * 2019-02-04 2022-03-24 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
JP2022519296A (ja) * 2019-02-04 2022-03-22 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
CN113660936A (zh) * 2019-02-04 2021-11-16 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2020163406A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163541A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3920918A4 (en) * 2019-02-05 2022-11-16 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
KR102913697B1 (ko) 2019-02-05 2026-01-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2020163401A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7603594B2 (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
KR102925385B1 (ko) * 2019-02-06 2026-02-11 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2020167624A1 (en) 2019-02-13 2020-08-20 Ptc Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
AU2020222881C1 (en) 2019-02-13 2026-01-22 Ptc Therapeutics, Inc. Thioeno(3,2-b) pyridin-7-amine compounds for treating familial dysautonomia
JP2022525417A (ja) * 2019-03-15 2022-05-13 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 異常スプライシングを修正するための組成物および方法
CA3139821A1 (en) * 2019-05-13 2020-11-19 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
AR119731A1 (es) 2019-05-17 2022-01-05 Novartis Ag Inhibidores del inflamasoma nlrp3
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
CN114174514A (zh) 2019-07-25 2022-03-11 诺华股份有限公司 可调节的表达系统
WO2021071981A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
WO2021071984A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
WO2021071983A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
TW202131920A (zh) * 2019-11-01 2021-09-01 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
US11618751B1 (en) 2022-03-25 2023-04-04 Ventus Therapeutics U.S., Inc. Pyrido-[3,4-d]pyridazine amine derivatives useful as NLRP3 derivatives
AU2021228285A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3169667A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Pyridazine derivatives for modulating nucleic acid splicing
AU2021228284A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
CA3174353A1 (en) 2020-04-08 2021-10-14 Dominic Reynolds Compounds and methods for modulating splicing
MX2022012677A (es) 2020-04-08 2023-01-11 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
MX2022012575A (es) * 2020-04-09 2023-01-24 Ptc Therapeutics Inc Compuestos para usarse para tratar la enfermedad de huntington.
WO2021231571A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
TW202216671A (zh) * 2020-06-25 2022-05-01 瑞士商諾華公司 1,4—二取代的嗒𠯤化合物之製造方法
EP4178963A1 (en) 2020-07-02 2023-05-17 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
MX2023000168A (es) 2020-07-02 2023-04-20 Remix Therapeutics Inc Derivados de 2-(indazol-5-il)-6-(piperidin-4-il)-1,7-naftiridina y compuestos relacionados como moduladores para el corte y empalme de ácidos nucleicos y para el tratamiento de enfermedades proliferativas.
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
EP4244362A1 (en) 2020-11-12 2023-09-20 PTC Therapeutics, Inc. Novel rna transcript
US12115154B2 (en) 2020-12-16 2024-10-15 Srx Cardio, Llc Compounds for the modulation of proprotein convertase subtilisin/kexin type 9 (PCSK9)
TW202231281A (zh) * 2020-12-25 2022-08-16 大陸商上海拓界生物醫藥科技有限公司 一類含噠嗪的化合物及其醫藥用途
EP4289823A4 (en) * 2021-02-08 2025-02-26 Medshine Discovery Inc. SUBSTITUTED PYRIDAZINE PHENOL DERIVATIVES
KR102793998B1 (ko) 2021-02-25 2025-04-09 미츠비시 파워 가부시키가이샤 연소기용 통, 연소기, 및 가스 터빈
US11319319B1 (en) 2021-04-07 2022-05-03 Ventus Therapeutics U.S., Inc. Compounds for inhibiting NLRP3 and uses thereof
IL311135A (en) 2021-08-30 2024-04-01 Remix Therapeutics Inc Compounds and methods for modulating splicing
US20250333397A1 (en) 2021-08-30 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240400584A1 (en) 2021-08-30 2024-12-05 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2024002554A (es) 2021-08-30 2024-07-02 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
TW202330552A (zh) 2021-10-13 2023-08-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
US20250332173A1 (en) 2021-10-13 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
JP2024540608A (ja) * 2021-11-22 2024-10-31 アールジェンタ セラピューティクス, インコーポレイテッド 複素環式置換1,3,4-チアジアゾール及びピリダジン化合物ならびにそれらの使用方法
US20250326748A1 (en) 2022-01-05 2025-10-23 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023133217A1 (en) 2022-01-05 2023-07-13 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
WO2024012551A1 (zh) * 2022-07-14 2024-01-18 南京明德新药研发有限公司 氘取代的哒嗪苯并噻吩化合物及其应用
UY40374A (es) 2022-08-03 2024-02-15 Novartis Ag Inhibidores de inflamasoma nlrp3
US12331048B2 (en) 2022-10-31 2025-06-17 Ventus Therapeutics U.S., Inc. Pyrido-[3,4-d]pyridazine amine derivatives useful as NLRP3 inhibitors
AU2024402685A1 (en) 2023-12-21 2026-07-23 Dbv Technologies Immunotherapeutic methods, compounds, and compositions for treating or preventing celiac disease
WO2026101897A1 (en) * 2024-11-05 2026-05-15 Skyhawk Therapeutics, Inc. 6-(6-{[(1r,2r,3s,5s)-2-fluoro-8-azabicyclo[3.2.1 ]octan-3-yl] (methyl)amino }pyridazin-3-yl)-2-methyl-l,3-benzoxazol-5-ol to treat huntington's disease

Family Cites Families (173)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL297170A (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
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
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
JPS5852307A (ja) 1981-09-22 1983-03-28 Hitachi Chem Co Ltd 水分散樹脂組成物
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
ATE368107T1 (de) 1993-05-11 2007-08-15 Univ North Carolina Antisense oligonukleotide die anomales splicing verhindern und deren verwendung
KR19990022524A (ko) 1995-06-06 1999-03-25 웨인스톡 스티븐 에프. 퀴놀리지논형화합물
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
HK1040076A1 (zh) 1998-09-21 2002-05-24 希雷生物化学有限公司 作为整联蛋白抑制剂的喹嗪酮
US6214986B1 (en) 1998-10-07 2001-04-10 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
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
EP1227084B1 (en) 1999-10-28 2005-12-14 Trine Pharmaceuticals, Inc. Drug discharge pump inhibitors
ATE363471T1 (de) 2000-01-24 2007-06-15 Astrazeneca Ab Durch einen morpholinrest substituierte therapeutische verbindungen
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
BR0116493A (pt) * 2000-12-21 2003-09-30 Vertex Pharma Compostos de pirazol úteis como inibidores de proteìna cinase
JPWO2002087589A1 (ja) 2001-04-26 2004-08-12 第一製薬株式会社 薬剤排出ポンプ阻害薬
ATE466581T1 (de) 2001-12-07 2010-05-15 Vertex Pharma Verbindungen auf pyrimidin-basis als gsk-3-hemmer
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
EP1542667B1 (en) 2002-07-24 2012-03-07 PTC Therapeutics, Inc. Ureido substituted benzoic acid compounds and their use for nonsense suppression and the treatment of disease
DE60311853T2 (de) 2002-09-30 2007-06-21 Neurosearch A/S Neue 1,4-diazabicycloalkanderivate, deren herstellung und verwendung
PT1551835E (pt) * 2002-09-30 2007-03-30 Neurosearch As Novos derivados de 1, 4-diazabicicloalcano, sua preparação e utilização
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
ES2339862T3 (es) 2003-06-20 2010-05-26 Novartis Vaccines And Diagnostics, Inc. Compuestos de piridino 1,2-a-pirimidin-4-ona como agentes anticancerosos.
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
BRPI0413234A (pt) 2003-08-01 2006-10-03 Genelabs Tech Inc derivados de imidazola bicìclica contra flaviviridae
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
US20050137203A1 (en) 2003-12-22 2005-06-23 Jianguo Ji 3-quinuclidinyl amino-substituted biaryl derivatives
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US20050245531A1 (en) * 2003-12-22 2005-11-03 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
CA2551178C (en) 2003-12-24 2012-11-06 Biota Scientific Management Pty Ltd Polycyclic agents for the treatment of respiratory syncytial virus infections
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
CN1964717A (zh) 2004-04-08 2007-05-16 神经能质公司 经取代∴啉-4-基胺
WO2005105801A1 (en) 2004-05-04 2005-11-10 Warner-Lambert Company Llc Pyrrolyl substituted pyrido[2,3-d]pyrimidin-7-ones and derivatives thereof as therapeutic agents
US7399767B2 (en) 2005-01-21 2008-07-15 Ortho-Mcneil Pharmaceutical, Inc. 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
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
CN103110635A (zh) * 2005-07-04 2013-05-22 海波因特制药有限责任公司 组胺h3受体拮抗剂
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
WO2007058894A2 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
EP2422819B1 (en) 2006-01-26 2017-03-01 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
EP2388328A1 (en) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
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
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
WO2007133756A2 (en) 2006-05-15 2007-11-22 Neurogen Corporation Crf1 receptor ligands comprising heteroaryl fused bicycles
JP2009537599A (ja) * 2006-05-23 2009-10-29 ノイロサーチ アクティーゼルスカブ 新規8,10−ジアザ−ビシクロ[4.3.1]デカン誘導体及びそれらの医学的使用
EP2079724B1 (en) 2006-07-20 2010-05-26 Amgen Inc. Substituted pyridone compounds and methods of use
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
EP2079734A1 (en) * 2006-10-25 2009-07-22 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
AR064517A1 (es) 2006-12-22 2009-04-08 Avexa Ltd Pirimidinionas biciclicas y sus usos
US20080171792A1 (en) 2006-12-28 2008-07-17 Jobdevairakkam Christopher New Use of highly concentrated formulations of 4-phenylbutyrate for treatment of certain disorders
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
DK2193133T3 (en) 2007-09-27 2015-10-05 Fundacion Ct Nac Investigaciones Oncologicas Carlos Iii IMIDAZOLOTHIADIAZOLES USED AS PROTEINKINASE INHIBITORS
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
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
WO2009131958A2 (en) 2008-04-21 2009-10-29 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 (pt) 2008-06-20 2015-10-13 Rottapharm Spa derivados de 6-1h-imidazo-quinazolina e de quinolinas, novos inibidores de mao e ligantes a receptor de imidazolina.
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
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
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
AU2010214101A1 (en) 2009-02-11 2011-09-01 Sunovion Pharmaceuticals 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
WO2010145208A1 (en) * 2009-06-19 2010-12-23 Abbott Laboratories Diazahomoadamantane derivatives and methods of use thereof
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
US20150018301A1 (en) 2009-11-06 2015-01-15 The Johns Hopkins University LRRK-2-Mediated Neuronal Toxicity
EP2501231B1 (en) 2009-11-20 2016-12-21 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
KR20130088002A (ko) 2010-01-13 2013-08-07 재단법인 한국파스퇴르연구소 항-감염성 피리도(1,2-a)피리미딘
JP6018506B2 (ja) 2010-02-08 2016-11-02 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 対立遺伝子多様体の選択的低減
ES2733708T3 (es) 2010-02-08 2019-12-02 Ionis Pharmaceuticals Inc Reducción selectiva de variantes alélicas
EP2536738A4 (en) 2010-02-08 2014-09-17 Isis Pharmaceuticals Inc METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES OR SUFFERING IN CONNECTION WITH REPEAT EXTENSIONS
CA2800509A1 (en) 2010-05-24 2011-12-01 Presidio Pharmaceuticals, Inc. Inhibitors of hcv ns5a
US20120008349A1 (en) * 2010-07-12 2012-01-12 Scharf Mesa P Power inverter systems with high-accuracy reference signal generation and associated methods of control
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
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
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
US20140303235A1 (en) 2011-08-11 2014-10-09 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric 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
US20130046093A1 (en) 2011-08-18 2013-02-21 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
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
CA2856334A1 (en) 2011-11-28 2013-06-06 Christina Hebach Novel trifluoromethyl-oxadiazole derivatives and their use in the treatment of disease
MX352861B (es) 2011-12-30 2017-12-13 Ptc Therapeutics Inc Compuestos para tratar la atrofia muscular espinal.
MX357834B (es) 2012-01-26 2018-07-26 Ptc Therapeutics Inc Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal.
EP3406252B1 (en) 2012-02-10 2020-05-13 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
US9371336B2 (en) 2012-03-01 2016-06-21 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
SG11201406584TA (en) 2012-04-24 2014-11-27 Vertex Pharma Dna-pk inhibitors
US8980934B2 (en) * 2012-06-22 2015-03-17 University Health Network Kinase inhibitors and method of treating cancer with same
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
EP4086347A3 (en) 2012-10-12 2023-01-11 Ionis 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
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
EP2951304B1 (en) 2013-02-04 2020-07-08 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CA2903903C (en) 2013-03-05 2021-05-04 Merck Patent Gmbh Triazolo[4,5-d]pyrimidine derivatives for the treatment of diseases such as cancer
JP2016518421A (ja) 2013-05-14 2016-06-23 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 呼吸器合胞体ウイルス感染症の処置および予防のためのアザ−オキソ−インドール
CA2913785C (en) 2013-06-25 2021-08-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
CN105636953B (zh) * 2013-07-31 2018-01-02 诺华股份有限公司 1,4‑二取代的哒嗪衍生物及其用于治疗与smn缺乏相关的病症的用途
CA2915764A1 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
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
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
WO2015107494A1 (en) 2014-01-17 2015-07-23 Novartis Ag 1 -(triazin-3-yi_/pyridazin-3-yl)-piper(-azine)idine derivatives and compositions thereof for inhibiting the activity of 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
CN106103416B (zh) 2014-03-14 2021-03-02 拉夸里亚创药株式会社 作为trpm8拮抗剂的氮杂螺衍生物
AU2015261046C1 (en) 2014-05-15 2019-07-25 F. Hoffmann-La Roche Ag Compounds 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
MX375925B (es) 2014-06-25 2025-03-07 Hoffmann La Roche Compuestos imidazo[1,2-a]pirazin-1-il-benzamida para tratar atrofia muscular espinal.
AU2015370903B2 (en) 2014-12-24 2021-06-17 Uniqure Ip B.V. RNAi induced huntingtin gene suppression
CA3199430A1 (en) 2015-01-16 2016-07-21 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
GB201506933D0 (en) 2015-04-23 2015-06-10 Sentinel Oncology Ltd Pharmaceutical compounds
EP3298017B1 (en) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Compounds for treating spinal muscular atrophy
EP3310169B1 (en) 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
CN117965536A (zh) 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
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
WO2017059251A1 (en) 2015-10-02 2017-04-06 Incyte Corporation Heterocyclic compounds useful as pim kinase inhibitors
AR106652A1 (es) 2015-11-12 2018-02-07 Hoffmann La Roche Compuestos para tratar la esclerosis lateral amiotrófica
AU2016353961B2 (en) 2015-11-12 2019-08-29 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
CA3119019C (en) 2015-12-10 2024-06-11 Ptc Therapeutics, Inc. Compounds and methods for treating huntington's disease
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
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
WO2017210134A1 (en) 2016-05-31 2017-12-07 Board Of Regents, University Of Texas System Heterocyclic inhibitors of ptpn11
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
TW202500565A (zh) 2016-10-24 2025-01-01 美商傳達治療有限公司 Shp2磷酸酶抑制劑及其使用方法
US11857599B2 (en) 2017-04-03 2024-01-02 Acceleron Pharma Inc. Compositions and methods for treating spinal muscular atrophy
US11591336B2 (en) 2017-05-26 2023-02-28 D. E. Shaw Research, Llc Substituted pyrazolo[3,4-b]pyrazines as SHP2 phosphatase inhibitors
EP4151627A1 (en) 2017-06-05 2023-03-22 PTC Therapeutics, Inc. Compounds for treating huntington's disease
CN111163838B (zh) 2017-06-28 2023-03-28 Ptc医疗公司 用于治疗亨廷顿氏病的方法
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
WO2019165073A1 (en) 2018-02-21 2019-08-29 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
US12138263B2 (en) 2018-03-21 2024-11-12 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine SHP2 phosphatase inhibitors and methods of use thereof
CN117263942A (zh) 2018-03-21 2023-12-22 传达治疗有限公司 Shp2磷酸酶抑制剂及其使用方法
BR112020019373A2 (pt) 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. Compostos para o tratamento da doença de hutington
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
KR20210038845A (ko) 2018-06-27 2021-04-08 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로아릴 화합물
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
TW202131920A (zh) 2019-11-01 2021-09-01 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

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