EA202092001A1 - Соединения для лечения болезни гентингтона - Google Patents

Соединения для лечения болезни гентингтона

Info

Publication number
EA202092001A1
EA202092001A1 EA202092001A EA202092001A EA202092001A1 EA 202092001 A1 EA202092001 A1 EA 202092001A1 EA 202092001 A EA202092001 A EA 202092001A EA 202092001 A EA202092001 A EA 202092001A EA 202092001 A1 EA202092001 A1 EA 202092001A1
Authority
EA
Eurasian Patent Office
Prior art keywords
compounds
disease
treatment
forms
gentington
Prior art date
Application number
EA202092001A
Other languages
English (en)
Russian (ru)
Inventor
Надя Сидоренко
Суреш Бабу
Анурадха Бхаттачария
Йоунг-Чоон Моон
Яна Нарасимхан
Джигар С. Пател
Original Assignee
ПиТиСи ТЕРАПЬЮТИКС, ИНК.
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 ПиТиСи ТЕРАПЬЮТИКС, ИНК. filed Critical ПиТиСи ТЕРАПЬЮТИКС, ИНК.
Publication of EA202092001A1 publication Critical patent/EA202092001A1/ru

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic 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
    • C07D401/14—Heterocyclic 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
    • 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
    • A61K31/53—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
    • 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
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C07D401/00—Heterocyclic 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
    • C07D401/02—Heterocyclic 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 two hetero rings
    • C07D401/12—Heterocyclic 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 two hetero rings linked by a chain containing hetero atoms as chain links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/14—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D409/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D417/00—Heterocyclic 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
    • C07D417/14—Heterocyclic 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C07D451/06—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/10—Spiro-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • 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
    • C07D487/10—Spiro-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
    • C07D491/10—Spiro-condensed systems
    • C07D491/107—Spiro-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D493/00—Heterocyclic compounds containing oxygen atoms as the only ring hetero atoms in the condensed system
    • C07D493/02—Heterocyclic compounds containing oxygen atoms as the only ring hetero atoms in the condensed system in which the condensed system contains two hetero rings
    • C07D493/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02—Heterocyclic 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
    • C07D498/04—Ortho-condensed systems

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
EA202092001A 2018-03-27 2019-03-26 Соединения для лечения болезни гентингтона EA202092001A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862648699P 2018-03-27 2018-03-27
PCT/US2019/024068 WO2019191092A1 (en) 2018-03-27 2019-03-26 Compounds for treating huntington's disease

Publications (1)

Publication Number Publication Date
EA202092001A1 true EA202092001A1 (ru) 2021-01-29

Family

ID=68060393

Family Applications (2)

Application Number Title Priority Date Filing Date
EA202092001A EA202092001A1 (ru) 2018-03-27 2019-03-26 Соединения для лечения болезни гентингтона
EA202092003A EA202092003A1 (ru) 2018-03-27 2019-03-27 Соединения для лечения болезни гентингтона

Family Applications After (1)

Application Number Title Priority Date Filing Date
EA202092003A EA202092003A1 (ru) 2018-03-27 2019-03-27 Соединения для лечения болезни гентингтона

Country Status (13)

Country Link
US (3) US12103926B2 (enExample)
EP (2) EP3774744B1 (enExample)
JP (2) JP7399870B2 (enExample)
KR (2) KR20210005559A (enExample)
CN (2) CN112135815A (enExample)
AU (2) AU2019243048A1 (enExample)
BR (2) BR112020019373A2 (enExample)
CA (2) CA3094703A1 (enExample)
EA (2) EA202092001A1 (enExample)
IL (2) IL277498A (enExample)
MX (2) MX2020009957A (enExample)
SG (2) SG11202009212WA (enExample)
WO (2) WO2019191092A1 (enExample)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3119019C (en) 2015-12-10 2024-06-11 Ptc Therapeutics, Inc. Compounds and methods for treating huntington's disease
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
BR112020019373A2 (pt) 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. Compostos para o tratamento da doença de hutington
JP7478142B2 (ja) 2018-06-07 2024-05-02 ディスアーム セラピューティクス, インコーポレイテッド Sarm1阻害剤
WO2020005882A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
KR20210038845A (ko) 2018-06-27 2021-04-08 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로아릴 화합물
IL279688B2 (en) * 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
CA3123215C (en) 2018-12-19 2024-04-02 Disarm Therapeutics, Inc. Inhibitors of sarm1 in combination with neuroprotective agents
WO2020163248A1 (en) * 2019-02-04 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
WO2020163541A1 (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
CA3139821A1 (en) 2019-05-13 2020-11-19 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
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
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
WO2022032007A2 (en) * 2020-08-05 2022-02-10 Skyhawk Therapeutics, Inc. Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing
CA3188104A1 (en) 2020-08-05 2022-02-10 Michael Luzzio Compositions for modulating splicing
WO2022031998A2 (en) * 2020-08-05 2022-02-10 Skyhawk Therapeutics, Inc. Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing
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
WO2022230912A1 (ja) 2021-04-28 2022-11-03 アステラス製薬株式会社 置換トリアジン化合物
EP4347571B1 (en) * 2021-06-04 2026-04-22 F. Hoffmann-La Roche AG Triazine derivatives and their use in the treatment of cancer.
WO2023028536A1 (en) * 2021-08-25 2023-03-02 Ptc Therapeutics, Inc. 1,2,4-triazine derivatives useful as inhibitors of nlrp3
US20250333397A1 (en) * 2021-08-30 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN116063307B (zh) * 2021-10-29 2025-08-19 中国药科大学 Shp2与cdk4/6双靶点抑制化合物合成及其制备方法与应用
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
MX2024008548A (es) * 2022-01-07 2024-07-19 Transthera Sciences Nanjing Inc Inhibidor del inflamosoma nlrp3 y uso del mismo.
JP2025523930A (ja) * 2022-07-21 2025-07-25 エフ. ホフマン-ラ ロシュ アーゲー Nlrp3阻害剤
UY40374A (es) 2022-08-03 2024-02-15 Novartis Ag Inhibidores de inflamasoma nlrp3
WO2024097598A1 (en) * 2022-11-02 2024-05-10 Merck Sharp & Dohme Llc Triazines useful as inhibitors of nod-like receptor protein 3
EP4683918A1 (en) * 2023-03-20 2026-01-28 Insilico Medicine IP Limited Nlrp3 inflammasome inhibitors and uses thereof
AU2024304006A1 (en) * 2023-06-16 2026-01-08 Plexium, Inc. Compounds and pharmaceutical compositions that degrade swi/snf-related matrix-associated actin-dependent regulator of chromatin subfamily a
EP4777504A1 (en) * 2023-09-12 2026-07-22 Zomagen Biosciences Ltd Nlrp3 modulators
WO2026067453A1 (en) * 2024-09-25 2026-04-02 Insilico Medicine Ip Limited Novel compounds as nlrp3 inflammasome inhibitors and uses thereof

Family Cites Families (209)

* 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
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
US4902695A (en) * 1989-02-13 1990-02-20 Eli Lilly And Company Excitatory amino acid receptor antagonists
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
US7423133B2 (en) 2002-08-23 2008-09-09 The Board Of Trustees Of The Leland Stanford Junior University Fluorescent glycosides and methods for their use
DE60311853T2 (de) 2002-09-30 2007-06-21 Neurosearch A/S Neue 1,4-diazabicycloalkanderivate, deren herstellung und verwendung
CA2765093A1 (en) 2002-11-12 2004-05-27 Abbott Laboratories Bicyclic-substituted amines as histamine-3 receptor ligands
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
US20040224952A1 (en) 2003-05-07 2004-11-11 Cowart Marlon D. Fused bicyclic-substituted amines as histamine-3 receptor ligands
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
US20050245531A1 (en) 2003-12-22 2005-11-03 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
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
ZA200609267B (en) 2004-04-08 2009-02-25 Targegen Inc Benzotriazine inhibitors of kinases
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
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
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
US7473784B2 (en) 2005-08-01 2009-01-06 Bristol-Myers Squibb Company Benzothiazole and azabenzothiazole compounds useful as kinase inhibitors
US7576110B2 (en) 2005-09-22 2009-08-18 Abbott Laboratories Benzothiazole cyclobutyl amine derivatives
US8258109B2 (en) 2005-10-20 2012-09-04 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
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
CA2632642A1 (en) 2005-12-06 2007-06-14 Neurosearch A/S Novel diazabicyclic aryl derivatives and their medical use
JP2009520700A (ja) 2005-12-21 2009-05-28 ペインセプター ファーマ コーポレーション 依存性イオンチャネルを調節するための組成物および方法
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
NZ570524A (en) 2006-03-28 2011-08-26 High Point Pharmaceuticals Llc Benzothiazoles having histamine H3 receptor activity
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
US7994181B2 (en) 2006-08-03 2011-08-09 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
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
WO2010024903A1 (en) 2008-08-29 2010-03-04 Yangbo Feng BENZO[d]OXAZOLES AND BENZO[d]THIAZOLES AS KINASE INHIBITORS
EP2350067A2 (en) 2008-10-16 2011-08-03 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
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
US8765747B2 (en) 2009-06-12 2014-07-01 Dana-Farber Cancer Institute, Inc. Fused 2-aminothiazole compounds
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
ES2733708T3 (es) 2010-02-08 2019-12-02 Ionis Pharmaceuticals Inc Reducción selectiva de variantes alélicas
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
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
CN102971311B (zh) 2010-05-06 2015-07-08 百时美施贵宝公司 作为gpr119调节剂的二环杂芳基化合物
CA2800509A1 (en) 2010-05-24 2011-12-01 Presidio Pharmaceuticals, Inc. Inhibitors of hcv ns5a
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
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
CN102617548A (zh) 2011-01-31 2012-08-01 北京赛林泰医药技术有限公司 作为gpr受体激动剂的双环杂芳基化合物及其组合物和应用
WO2012104823A2 (en) 2011-02-04 2012-08-09 Novartis Ag Pyridopyrimidinone compounds in the treatment of neurodegenerative diseases
CN106278999B (zh) 2011-02-07 2019-10-25 比奥根Ma公司 S1p调节剂
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
US20140303235A1 (en) 2011-08-11 2014-10-09 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
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
US9199946B2 (en) 2012-04-03 2015-12-01 Bristol-Myers Squibb Company Pyrimidinone carboxamide inhibitors of endothelial lipase
SG11201406584TA (en) 2012-04-24 2014-11-27 Vertex Pharma Dna-pk inhibitors
KR20150008406A (ko) 2012-05-15 2015-01-22 노파르티스 아게 Abl1, abl2 및 bcr-abl1의 활성을 억제하기 위한 벤즈아미드 유도체
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
EP4086347A3 (en) 2012-10-12 2023-01-11 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP2906255B1 (en) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. 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
CA2889494C (en) 2012-10-25 2018-12-18 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
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
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
WO2015105657A1 (en) 2013-12-19 2015-07-16 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
CN106573880B (zh) 2014-03-19 2019-07-30 阿米达斯公司 淀粉样蛋白靶向剂及其使用方法
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.
MX381224B (es) 2014-10-31 2025-03-12 Massachusetts Gen Hospital Potentes moduladores de gamma-secretasa.
GB201419579D0 (en) 2014-11-03 2014-12-17 Iomet Pharma Ltd Pharmaceutical compound
CN107207526A (zh) 2014-12-02 2017-09-26 拜耳作物科学股份公司 作为害虫防治剂的双环化合物
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
CN107635984B (zh) 2015-03-11 2021-04-13 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
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
US10383867B2 (en) 2015-11-28 2019-08-20 Russell Dahl Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders
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
EP3426653B1 (en) 2016-03-11 2021-12-15 AC Immune SA Bicyclic compounds for diagnosis and therapy
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
US10730868B2 (en) 2016-07-14 2020-08-04 Bristol-Myers Squibb Company Bicyclic heteroaryl substituted compounds
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
GB201616627D0 (en) * 2016-09-30 2016-11-16 Mission Therapeutics Limited Novel compounds
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
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.
WO2019165073A1 (en) 2018-02-21 2019-08-29 Relay Therapeutics, Inc. 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
US12138263B2 (en) 2018-03-21 2024-11-12 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine SHP2 phosphatase inhibitors and methods of use thereof
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
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
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
MX2022012575A (es) 2020-04-09 2023-01-24 Ptc Therapeutics Inc Compuestos para usarse para tratar la enfermedad de huntington.
EP4244362A1 (en) 2020-11-12 2023-09-20 PTC Therapeutics, Inc. Novel rna transcript
CN118159539A (zh) 2021-07-30 2024-06-07 Ptc医疗公司 用于治疗亨廷顿氏病的杂芳基化合物

Also Published As

Publication number Publication date
US20210024525A1 (en) 2021-01-28
IL277497A (en) 2020-11-30
EP3774744A1 (en) 2021-02-17
SG11202009212WA (en) 2020-10-29
US20210009590A1 (en) 2021-01-14
BR112020019373A2 (pt) 2020-12-29
EA202092003A1 (ru) 2021-02-10
MX2020009957A (es) 2021-01-15
CA3094700A1 (en) 2019-10-03
AU2019243048A1 (en) 2020-10-15
JP7399870B2 (ja) 2023-12-18
WO2019191229A8 (en) 2019-12-19
US12103926B2 (en) 2024-10-01
EP3773552A4 (en) 2022-01-26
WO2019191229A1 (en) 2019-10-03
CA3094703A1 (en) 2019-10-03
US20230331725A1 (en) 2023-10-19
WO2019191092A1 (en) 2019-10-03
MX2020009956A (es) 2021-01-15
CN112203653A (zh) 2021-01-08
CN112203653B (zh) 2024-08-16
EP3773552A1 (en) 2021-02-17
AU2019243943A1 (en) 2020-10-15
KR20210005558A (ko) 2021-01-14
IL277498A (en) 2020-11-30
JP7399869B2 (ja) 2023-12-18
KR20210005559A (ko) 2021-01-14
US11780839B2 (en) 2023-10-10
JP2021519318A (ja) 2021-08-10
BR112020019264A2 (pt) 2021-01-12
CN112135815A (zh) 2020-12-25
EP3774744B1 (en) 2025-02-12
WO2019191092A8 (en) 2020-05-07
JP2021519291A (ja) 2021-08-10
SG11202009211VA (en) 2020-10-29

Similar Documents

Publication Publication Date Title
EA202092003A1 (ru) Соединения для лечения болезни гентингтона
EA202092899A1 (ru) Гетероарильные соединения для лечения болезни гентингтона
EA202092896A1 (ru) Гетероциклические и гетероарильные соединения для лечения болезни гентингтона
EA201800367A1 (ru) Способы лечения болезни хантингтона
MX2021013854A (es) Compuestos para tratar enfermedad de huntington.
EA201992878A1 (ru) Соединения для лечения болезни хантингтона
JOP20230318A1 (ar) مشتقات تترا هيدرو – إيميدازوبيريدين كمعدِّلات مناعية pd-l1
EA201891203A1 (ru) Гетероциклические соединения в качестве иммуномодуляторов
MX2023004923A (es) Compuestos heterociclicos como inmunomoduladores.
EA201891494A1 (ru) Гетероциклические соединения в качестве иммуномодуляторов
EA201991399A1 (ru) Аминотриазолопиридиновые соединения и их применение в лечении рака
EA201891191A1 (ru) 2-замещенные соединения хиназолина, содержащие замещенную гетероциклическую группу, и способы их применения
EA202091480A1 (ru) Аминоиндольные соединения, пригодные в качестве ингибиторов tlr
EA201890338A1 (ru) Оксадиазольные производные, пригодные в качестве ингибиторов hdac
EA201892207A1 (ru) Индолкарбоксамидные соединения
MX389965B (es) Compuestos de pirrolopiridina-anilina para el tratamiento de trastornos dermicos
EA202090900A1 (ru) Ингибиторы иммунопротеасом
MX2019013562A (es) Compuestos de anilina heteroaromatica o fusionados para el tratamiento de trastornos dermicos.
EA202092695A1 (ru) Композиции, содержащие глюкозу и гемицеллюлозу, и их применение
EA201891342A1 (ru) Изоиндольные соединения
EA202090164A1 (ru) Изотретиноиновые перорально-слизистые композиции и способы их применения
EA202190045A1 (ru) Составы/композиции, содержащие ибрутиниб
EA202090719A1 (ru) Модифицированные нуклеозидные амидофосфиты
EA201792170A1 (ru) Производные индола
EA202190766A1 (ru) Иммуномодуляторы, их композиции и способы применения