AR119651A1 - Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington - Google Patents
Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntingtonInfo
- Publication number
- AR119651A1 AR119651A1 ARP190101808A ARP190101808A AR119651A1 AR 119651 A1 AR119651 A1 AR 119651A1 AR P190101808 A ARP190101808 A AR P190101808A AR P190101808 A ARP190101808 A AR P190101808A AR 119651 A1 AR119651 A1 AR 119651A1
- Authority
- AR
- Argentina
- Prior art keywords
- alkyl
- alkoxy
- amino
- membered
- heterocyclyl
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 4
- 208000023105 Huntington disease Diseases 0.000 title abstract 3
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 abstract 22
- 125000000623 heterocyclic group Chemical group 0.000 abstract 10
- 125000001072 heteroaryl group Chemical group 0.000 abstract 9
- 229910052760 oxygen Inorganic materials 0.000 abstract 8
- 229910052717 sulfur Inorganic materials 0.000 abstract 8
- 125000006376 (C3-C10) cycloalkyl group Chemical group 0.000 abstract 7
- 125000001424 substituent group Chemical group 0.000 abstract 7
- 125000005842 heteroatom Chemical group 0.000 abstract 6
- 125000002950 monocyclic group Chemical group 0.000 abstract 6
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 abstract 6
- 125000004178 (C1-C4) alkyl group Chemical group 0.000 abstract 5
- 125000004191 (C1-C6) alkoxy group Chemical group 0.000 abstract 5
- 125000004890 (C1-C6) alkylamino group Chemical group 0.000 abstract 5
- GOJUJUVQIVIZAV-UHFFFAOYSA-N 2-amino-4,6-dichloropyrimidine-5-carbaldehyde Chemical group NC1=NC(Cl)=C(C=O)C(Cl)=N1 GOJUJUVQIVIZAV-UHFFFAOYSA-N 0.000 abstract 5
- 125000004093 cyano group Chemical group *C#N 0.000 abstract 5
- 229910052736 halogen Inorganic materials 0.000 abstract 5
- 150000002367 halogens Chemical class 0.000 abstract 5
- 125000002887 hydroxy group Chemical group [H]O* 0.000 abstract 5
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 abstract 5
- 125000006620 amino-(C1-C6) alkyl group Chemical group 0.000 abstract 4
- 125000002619 bicyclic group Chemical group 0.000 abstract 3
- 125000004122 cyclic group Chemical group 0.000 abstract 3
- 125000003367 polycyclic group Chemical group 0.000 abstract 3
- 229920006395 saturated elastomer Polymers 0.000 abstract 3
- 125000003545 alkoxy group Chemical group 0.000 abstract 2
- 229910052739 hydrogen Inorganic materials 0.000 abstract 2
- 239000001257 hydrogen Substances 0.000 abstract 2
- 125000004435 hydrogen atom Chemical class [H]* 0.000 abstract 2
- 229910052757 nitrogen Inorganic materials 0.000 abstract 2
- -1 pyrazine derivative heterocyclic compounds Chemical class 0.000 abstract 2
- 125000006700 (C1-C6) alkylthio group Chemical group 0.000 abstract 1
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 abstract 1
- 150000002923 oximes Chemical class 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- PBMFSQRYOILNGV-UHFFFAOYSA-N pyridazine Chemical compound C1=CC=NN=C1 PBMFSQRYOILNGV-UHFFFAOYSA-N 0.000 abstract 1
- 229910052701 rubidium Inorganic materials 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
- 239000012453 solvate Substances 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/50—Pyridazines; Hydrogenated pyridazines
- A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
- C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
- C07D491/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
- C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
- C07D491/04—Ortho-condensed systems
- C07D491/044—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
- C07D491/048—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
- C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
- C07D491/08—Bridged systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
- C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
- C07D495/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Neurosurgery (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Epidemiology (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
- Plural Heterocyclic Compounds (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
Abstract
Se proveen compuestos heterocíclicos derivados de piridazina y pirazina condensadas y composiciones farmacéuticas que los comprenden todo lo cual es utilizado para el tratamiento de la enfermedad de Huntington (EH). Reivindicación 1: Un compuesto que comprende, un compuesto de fórmula (1) o una forma del mismo, en la que: las líneas discontinuas representan uno o más dobles enlaces opcionalmente presentes donde esté permitido por las valencias disponibles; W¹ es independientemente C-Rᵃ, CH-Rᵃ, N, N-Rᵇ, O ó S donde esté permitido por las valencias disponibles; W² es independientemente C-Rᵃ, CH-Rᵃ, N o N-Rᵇ donde esté permitido por las valencias disponibles, y; W³ es independientemente C, CH o N donde esté permitido por las valencias disponibles; en donde al menos uno de W¹, W² o W³ es N o N-Rᵇ; W⁴ y W⁵ son independientemente C-Rᵃ o N, en donde cuando W¹ es S u O, W² es C-Rᵃ, y W³ es C; Rᵃ se selecciona, en cada caso, independientemente entre hidrógeno, ciano, halógeno, hidroxi, alquilo C₁₋₆, deutero-alquilo C₁₋₄, halo-alquilo C₁₋₆, alcoxi C₁₋₆, halo-alcoxi C₁₋₆, alcoxi C₁₋₆-alquilo C₁₋₆, amino, alquil C₁₋₆-amino, (alquil C₁₋₆)₂-amino, amino-alquilo C₁₋₆ e hidroxi-alquilo C₁₋₆; Rᵇ se selecciona entre hidrógeno y alquilo C₁₋₆; R¹ se selecciona entre cicloalquilo C₃₋₁₀ y heterociclilo, en donde heterociclilo es un sistema de anillos monocíclico de 3 - 7 miembros, bicíclico de 6 - 10 miembros o policíclico de 13 - 16 miembros, saturado o parcialmente insaturado, que tiene 1, 2 ó 3 miembros heteroátomos en el anillo seleccionados independientemente entre N, O ó S, y en la que, cada caso de cicloalquilo C₃₋₁₀ y heterociclilo está opcionalmente sustituido con uno, dos o tres sustituyentes R³ y opcionalmente, con un sustituyente R⁴ adicional, o, en la que, como alternativa, cada caso de cicloalquilo C₃₋₁₀ y heterociclilo está opcionalmente sustituido con uno, dos, tres o cuatro sustituyentes R³; R² se selecciona entre fenilo, heterociclilo y heteroarilo, en donde heterociclilo es un sistema de anillos monocíclico de 3 - 7 miembros, bicíclico de 6 - 10 miembros o policíclico de 13 - 16 miembros, saturado o parcialmente insaturado, que tiene 1, 2 ó 3 miembros heteroátomos en el anillo seleccionados independientemente entre N, O ó S, en donde heteroarilo es un sistema de anillos monocíclico de 3 - 7 miembros o bicíclico de 6 - 10 miembros que tiene 1, 2, 3 ó 4 miembros heteroátomos en el anillo seleccionados independientemente entre N, O ó S, y en la que, cada caso de fenilo, heterociclilo y heteroarilo está opcionalmente sustituido con uno, dos o tres sustituyentes R⁵, y opcionalmente, con un sustituyente R⁶ adicional; R³ se selecciona, en cada caso, independientemente entre ciano, halógeno, hidroxi, alquilo C₁₋₆, deutero-alquilo C₁₋₄, halo-alquilo C₁₋₆, alcoxi C₁₋₆, halo-alcoxi C₁₋₆, alcoxi C₁₋₆-alquilo C₁₋₆, amino, alquil C₁₋₆-amino, (alquil C₁₋₆)₂-amino, amino-alquilo C₁₋₆ e hidroxi-alquilo C₁₋₆; R⁴ se selecciona entre cicloalquilo C₃₋₁₀, fenilo, heteroarilo y heterociclilo, en donde heterociclilo es un sistema de anillos monocíclico de 3 - 7 miembros, bicíclico de 6 - 10 miembros o policíclico de 13 - 16 miembros, saturado o parcialmente insaturado, que tiene 1, 2 ó 3 miembros heteroátomos en el anillo seleccionados independientemente entre N, O ó S, en donde heteroarilo es un sistema de anillos monocíclico de 3 - 7 miembros o bicíclico de 6 - 10 miembros que tiene 1, 2, 3 ó 4 miembros heteroátomos en el anillo seleccionados independientemente entre N, O ó S, y en la que, cada caso de cicloalquilo C₃₋₁₀, fenilo, heterociclilo y heteroarilo está opcionalmente sustituido con uno, dos o tres sustituyentes R⁷; R⁵ se selecciona, en cada caso, independientemente entre halógeno, hidroxi, ciano, nitro, alquilo C₁₋₆, deutero-alquilo C₁₋₄, halo-alquilo C₁₋₆, alcoxi C₁₋₆, halo-alcoxi C₁₋₆, oxima, amino, alquil C₁₋₆-amino, (alquil C₁₋₆)₂-amino y alquil C₁₋₆-tio; R⁶ se selecciona entre fenilo y heteroarilo, en donde heteroarilo es un sistema de anillos monocíclico de 3 - 7 miembros o bicíclico de 6 - 10 miembros que tiene 1, 2, 3 ó 4 miembros heteroátomos en el anillo seleccionados independientemente entre N, O ó S, y en la que, cada caso de fenilo y heteroarilo está opcionalmente sustituido con uno, dos, tres o cuatro sustituyentes R⁸; R⁷ se selecciona, en cada caso, independientemente entre ciano, halógeno, hidroxi, alquilo C₁₋₆, deutero-alquilo C₁₋₄, halo-alquilo C₁₋₆, alcoxi C₁₋₆, halo-alcoxi C₁₋₆, alcoxi C₁₋₆-alquilo C₁₋₆, amino, alquil C₁₋₆-amino, (alquil C₁₋₆)₂-amino, amino-alquilo C₁₋₆ y cicloalquilo C₃₋₁₀; y R⁸ se selecciona, en cada caso, independientemente entre ciano, halógeno, hidroxi, alquilo C₁₋₆, deutero-alquilo C₁₋₄, halo-alquilo C₁₋₆, alcoxi C₁₋₆, halo-alcoxi C₁₋₆, alcoxi C₁₋₆-alquilo C₁₋₆, amino, alquil C₁₋₆-amino, (alquil C₁₋₆)₂-amino, amino-alquilo C₁₋₆ y cicloalquilo C₃₋₁₀; en donde una forma del compuesto se selecciona entre el grupo que consiste en sal, hidrato, solvato, racemato, enantiómero, diastereómero, estereoisómero y forma de tautómero del mismo.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862690653P | 2018-06-27 | 2018-06-27 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR119651A1 true AR119651A1 (es) | 2022-01-05 |
Family
ID=67180896
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190101808A AR119651A1 (es) | 2018-06-27 | 2019-06-28 | Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington |
Country Status (25)
| Country | Link |
|---|---|
| US (1) | US11858941B2 (es) |
| EP (2) | EP4434990A1 (es) |
| JP (1) | JP7421507B2 (es) |
| KR (1) | KR20210042265A (es) |
| CN (1) | CN112654625B (es) |
| AR (1) | AR119651A1 (es) |
| AU (2) | AU2019294478B2 (es) |
| BR (1) | BR112020026545A2 (es) |
| CA (1) | CA3103976A1 (es) |
| CL (3) | CL2020003378A1 (es) |
| CO (1) | CO2020016285A2 (es) |
| DK (1) | DK3814357T3 (es) |
| EA (1) | EA202092896A1 (es) |
| ES (1) | ES2983003T3 (es) |
| FI (1) | FI3814357T3 (es) |
| IL (1) | IL279688B1 (es) |
| LT (1) | LT3814357T (es) |
| MX (1) | MX2020014098A (es) |
| PE (1) | PE20211378A1 (es) |
| PL (1) | PL3814357T3 (es) |
| PT (1) | PT3814357T (es) |
| RS (1) | RS65727B1 (es) |
| SG (1) | SG11202012674PA (es) |
| SI (1) | SI3814357T1 (es) |
| WO (1) | WO2020005873A1 (es) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2021001091A (es) | 2015-12-10 | 2022-04-26 | Ptc Therapeutics Inc | Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington. |
| US11407753B2 (en) | 2017-06-05 | 2022-08-09 | 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 |
| BR122020005073A2 (pt) | 2017-08-04 | 2020-10-13 | Skyhawk Therapeutics, Inc. | Composto, composição farmacêutica e uso do composto |
| CN112020500A (zh) | 2017-12-22 | 2020-12-01 | 拉文纳制药公司 | 作为磷脂酰肌醇磷酸激酶抑制剂的氨基吡啶衍生物 |
| BR112020019373A2 (pt) | 2018-03-27 | 2020-12-29 | Ptc Therapeutics, Inc. | Compostos para o tratamento da doença de hutington |
| US11858941B2 (en) | 2018-06-27 | 2024-01-02 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating Huntington's disease |
| US11685746B2 (en) | 2018-06-27 | 2023-06-27 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| KR20210135241A (ko) | 2019-02-05 | 2021-11-12 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| KR20210135507A (ko) | 2019-02-06 | 2021-11-15 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| 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 |
| BR112021022624A2 (pt) * | 2019-05-13 | 2022-01-04 | Ptc Therapeutics Inc | Compostos para o tratamento da doença de huntington |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| 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 |
| MX2022005254A (es) | 2019-11-01 | 2022-06-29 | Novartis Ag | El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington. |
| MX2022013856A (es) | 2020-05-13 | 2023-04-05 | Chdi Foundation Inc | Moduladores htt para tratar la enfermedad de huntington. |
| TW202216710A (zh) | 2020-07-02 | 2022-05-01 | 美商雷密克斯醫療公司 | 調節剪接之化合物及方法 |
| KR20230118067A (ko) | 2020-07-02 | 2023-08-10 | 레믹스 테라퓨틱스 인크. | 5-[5-(피페리딘-4-일)티에노[3,2-c]피라졸-2-일]인다졸 유도체, 및 핵산 스플라이싱의 조절제로서의 및 증식성 질환의 치료를 위한 관련 화합물 |
| WO2022060944A1 (en) * | 2020-09-16 | 2022-03-24 | Skyhawk Therapeutics, Inc. | Compositions for modulating splicing |
| WO2022060951A1 (en) * | 2020-09-16 | 2022-03-24 | Skyhawk Therapeutics, Inc. | Compositions for modulating splicing |
| WO2022060943A1 (en) * | 2020-09-16 | 2022-03-24 | Skyhawk Therapeutics, Inc. | Compositions for modulating splicing |
| AU2021380758A1 (en) * | 2020-11-12 | 2023-06-15 | Ptc Therapeutics Inc. | Novel rna transcript |
| WO2022104058A1 (en) | 2020-11-13 | 2022-05-19 | Ptc Therapeutics Inc. | Tablet for use in treating huntington's disease and method of making the same |
| EP4308572A1 (en) * | 2021-03-17 | 2024-01-24 | F. Hoffmann-La Roche AG | New thienopyrimidinone derivatives |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| KR20240054264A (ko) | 2021-07-30 | 2024-04-25 | 피티씨 테라퓨틱스, 인크. | Heteroaryl compounds for treating huntington's disease |
| WO2023081859A1 (en) * | 2021-11-04 | 2023-05-11 | Skyhawk Therapeutics, Inc. | Triazine amino derivatives for treating sca3 |
| WO2023220134A1 (en) * | 2022-05-10 | 2023-11-16 | Foghorn Therapeutics Inc. | Pyrazine derivatives and uses thereof |
| WO2023244996A2 (en) * | 2022-06-15 | 2023-12-21 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating huntington's disease |
Family Cites Families (194)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BE637271A (es) | 1963-04-04 | 1900-01-01 | ||
| US3558618A (en) | 1968-04-01 | 1971-01-26 | Dow Chemical Co | Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones |
| GB1383409A (en) | 1972-09-09 | 1974-02-12 | Pfizer Ltd | Derivatives of 2-amino- and 4-amino-quinazoline and pharmaceutical compositions containing them |
| US4122274A (en) | 1977-05-25 | 1978-10-24 | Bristol-Myers Company | 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones |
| JPS56150091A (en) | 1980-03-28 | 1981-11-20 | Janssen Pharmaceutica Nv | 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture |
| US4342870A (en) | 1980-03-28 | 1982-08-03 | Janssen Pharmaceutica N.V. | Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives |
| FR2567518B1 (fr) | 1984-07-11 | 1987-11-13 | Sanofi Sa | Nouveaux composes a noyau heterocyclique azote, leur preparation et les medicaments qui en contiennent |
| US5089633A (en) | 1987-04-28 | 1992-02-18 | Georgia Tech Research Corporation | Substituted isocoumarins |
| 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 (ja) | 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 | 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法 |
| AU6153096A (en) | 1995-06-06 | 1996-12-24 | Abbott Laboratories | Quinolizinone type compounds |
| US5916916A (en) | 1996-10-10 | 1999-06-29 | Eli Lilly And Company | 1-aryloxy-2-arylnaphthyl compounds, intermediates, compositions, and methods |
| US5869500A (en) | 1996-12-13 | 1999-02-09 | Hoffmann-La Roche Inc. | Pyridone compounds useful in treating Alzheimer's disease |
| CA2269561C (en) | 1998-04-22 | 2007-06-05 | Dainippon Ink And Chemicals, Inc. | Naphthalene derivative and liquid crystal composition comprising the same |
| US6630488B1 (en) | 1998-09-21 | 2003-10-07 | Biochem Pharma, Inc. | Quinolizinones as integrin inhibitors |
| US6214986B1 (en) | 1998-10-07 | 2001-04-10 | Isis Pharmaceuticals, Inc. | Antisense modulation of bcl-x expression |
| US6172216B1 (en) | 1998-10-07 | 2001-01-09 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL-X expression |
| US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
| DE60024861T2 (de) | 1999-10-28 | 2006-07-06 | Trine Pharmaceuticals, Inc., Waltham | Pumpeninhibitoren zur freisetzung von medikamenten |
| CN1263743C (zh) | 2000-01-24 | 2006-07-12 | 基纳西亚股份有限公司 | 用于治疗的吗啉代基取代的化合物 |
| AU2002232536A1 (en) | 2000-11-09 | 2002-05-21 | 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 |
| US6989385B2 (en) | 2000-12-21 | 2006-01-24 | Vertex Pharmaceuticals Incorporated | Pyrazole compounds useful as protein kinase inhibitors |
| GB0100624D0 (en) | 2001-01-10 | 2001-02-21 | Vernalis Res Ltd | Chemical compounds VII |
| EP1389463A4 (en) | 2001-04-26 | 2008-09-17 | Daiichi Seiyaku Co | MEDICAMENT FOR INHIBITING A DRUG REMOVAL PUMP |
| JP2005516005A (ja) | 2001-12-07 | 2005-06-02 | バーテクス ファーマスーティカルズ インコーポレイテッド | Ｇｓｋ−３阻害剤として有用なピリミジンベースの化合物 |
| 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 |
| WO2004019002A2 (en) | 2002-08-23 | 2004-03-04 | The Board Of Trustees Of The Leland Stanford Junior University | Fluorescent glycosides and methods for their use |
| EP1551835B1 (en) | 2002-09-30 | 2007-02-14 | NeuroSearch A/S | Novel 1,4-diazabicycloalkane derivatives, their preparation and use |
| US20070078144A1 (en) | 2003-01-29 | 2007-04-05 | Stockwell Brent R | Agents for treating neurodegenerative diseases |
| US7326711B2 (en) | 2003-06-20 | 2008-02-05 | Chiron Corporation | Pyridino[1,2-a]pyrimidin-4-one compounds as anticancer agents |
| GB0315494D0 (en) | 2003-07-02 | 2003-08-06 | Biofocus Plc | Compounds which bind to the active site of protein kinase enzymes |
| KR20060054410A (ko) | 2003-08-01 | 2006-05-22 | 제네랩스 테크놀로지스, 인코포레이티드 | 플라비비리다에에 대한 2고리 이미다졸 유도체 |
| 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 |
| 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 |
| US7655657B2 (en) | 2003-12-22 | 2010-02-02 | 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 |
| BRPI0510560A (pt) | 2004-05-04 | 2007-11-20 | Warner Lambert Co | pirido[2,3-d] pirimidin-7-onas pirrolil substituìdas e seus derivados como agentes terapêuticos |
| WO2006078834A1 (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 (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 |
| JP5121707B2 (ja) | 2005-07-04 | 2013-01-16 | ハイ ポイント ファーマシューティカルズ，エルエルシー | 新規医薬 |
| US7473784B2 (en) | 2005-08-01 | 2009-01-06 | Bristol-Myers Squibb Company | Benzothiazole and azabenzothiazole compounds useful as kinase inhibitors |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| CA2629323A1 (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 |
| CN101305004B (zh) | 2005-12-06 | 2011-11-16 | 神经研究公司 | 新颖的二氮杂双环芳基衍生物和它们的医药用途 |
| CN101360738A (zh) | 2005-12-21 | 2009-02-04 | 佩因赛普托药物公司 | 调节门控离子通道的组合物和方法 |
| EP2428227B1 (en) | 2006-01-26 | 2016-06-22 | Ionis Pharmaceuticals, Inc. | Compositions and their uses directed to Huntingtin |
| EP1984499B1 (en) | 2006-01-27 | 2015-05-27 | 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 |
| MX2008011123A (es) | 2006-03-28 | 2008-09-30 | High Point Pharmaceuticals Llc | Benzotiazoles que tienen actividad de receptor h3 de histamina. |
| 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 |
| EP2029607A1 (en) | 2006-05-23 | 2009-03-04 | NeuroSearch A/S | Novel 8,10-diaza-bicycloý4.3.1¨decane derivatives and their medical use |
| ATE469144T1 (de) | 2006-07-20 | 2010-06-15 | Amgen Inc | Substituierte pyridonverbindungen und anwendungsverfahren |
| WO2008013997A2 (en) | 2006-07-27 | 2008-01-31 | The Trustees Of Columbia University In The City Of New York | Fluorescent substrates for monoamine transporters as optical false neurotransmitters |
| WO2008014822A1 (en) | 2006-08-03 | 2008-02-07 | Rottapharm S.P.A. | 6-1h-imidazo-quinazoline and quinolines derivatives, new potent analgesics and anti-inflammatory agents |
| MX2009003892A (es) | 2006-10-25 | 2009-04-23 | Neurosearch As | Compuestos de oxadiazol y tiadiazol y su uso como moduladores del receptor de acetilcolina nicotinico. |
| US8314119B2 (en) | 2006-11-06 | 2012-11-20 | Abbvie Inc. | Azaadamantane derivatives and methods of use |
| DE602007012308D1 (de) | 2006-12-22 | 2011-03-10 | Avexa Ltd | Bicyclische pyrimidinone und deren verwendung |
| WO2008083226A2 (en) | 2006-12-28 | 2008-07-10 | Navinta Llc | Process for preparation of liquid dosage form containing sodium 4-phenylbutyrate |
| FR2914188B1 (fr) | 2007-03-28 | 2012-06-22 | Trophos | Nouvelle composition a base d'oxime de cholest-4-en-3-one |
| EP2014656A3 (en) | 2007-06-11 | 2011-08-24 | High Point Pharmaceuticals, LLC | New heteocyclic h3 antagonists |
| HUE025976T2 (en) | 2007-09-27 | 2016-05-30 | Fund Centro Nac De Investig Oncologicas Carlos Iii | Imidazolothiadiazoles for use as protein kinase inhibitors |
| AU2008304231A1 (en) | 2007-09-27 | 2009-04-02 | Albany Molecular Research, Inc. | 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 |
| WO2009151546A2 (en) | 2008-05-27 | 2009-12-17 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| JP2011524383A (ja) | 2008-06-20 | 2011-09-01 | ロッタファルム・ソシエタ・ペル・アチオニ | ６−１ｈ−イミダゾ−キナゾリンおよびキノリン誘導体、新規ｍａｏ阻害剤およびイミダゾリンレセプターリガンド |
| EP2138493A1 (en) | 2008-06-26 | 2009-12-30 | Sanofi-Aventis | Substituted pyrimidone derivatives |
| PL2212324T3 (pl) | 2008-07-02 | 2014-11-28 | Avexa Ltd | Związki posiadające właściwości przeciwwirusowe |
| US8932818B2 (en) | 2008-08-13 | 2015-01-13 | Ptc Therapeutics, Inc. | Screening assays for compounds that modulate programmed ribosomal frameshifting |
| WO2010024903A1 (en) | 2008-08-29 | 2010-03-04 | Yangbo Feng | BENZO[d]OXAZOLES AND BENZO[d]THIAZOLES AS KINASE INHIBITORS |
| CA2739490A1 (en) | 2008-10-16 | 2010-04-22 | Schering Corporation | Pyrrolidine, piperidine and piperazine derivatives and methods of use thereof |
| GB2465405A (en) | 2008-11-10 | 2010-05-19 | Univ Basel | Triazine, pyrimidine and pyridine analogues and their use in therapy |
| US8283360B2 (en) | 2008-12-19 | 2012-10-09 | Merck Sharp & Dohme Corp. | Bicyclic heterocyclic derivatives and methods of use thereof |
| BRPI1008020A2 (pt) | 2009-02-11 | 2016-03-15 | Sunovion Pharmaceuticals Inc | antagonistas e agonistas inversos h3 da histamina e métodos de uso dos mesmos |
| 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 |
| MX2011014019A (es) | 2009-06-19 | 2012-02-22 | Abbott Lab | Derivados de diazahomoadamantano y sus metodos de uso. |
| WO2011032045A1 (en) | 2009-09-11 | 2011-03-17 | Isis 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 |
| US20130012506A1 (en) | 2010-01-13 | 2013-01-10 | Zaesung No | Anti-infective pyrido (1,2-a) pyrimidines |
| ES2733708T3 (es) | 2010-02-08 | 2019-12-02 | Ionis Pharmaceuticals Inc | Reducción selectiva de variantes alélicas |
| CA2789038A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| JP2013518603A (ja) | 2010-02-08 | 2013-05-23 | アイシス ファーマシューティカルズ， インコーポレーテッド | 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 |
| EP2575866A4 (en) | 2010-05-24 | 2013-10-16 | Presidio Pharmaceuticals Inc | HCV NS5A INHIBITORS |
| US20130237585A1 (en) | 2010-07-19 | 2013-09-12 | University Of Rochester | Modulation of dystrophia myotonica-protein kinase (dmpk) expression |
| WO2012019106A2 (en) | 2010-08-06 | 2012-02-09 | Board Of Regents Of The University Of Nebraska | Positive and negative modulators of nmda receptors |
| WO2012075393A2 (en) | 2010-12-02 | 2012-06-07 | President And Fellows Of Harvard College | Activators of proteasomal degradation and uses thereof |
| 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 |
| 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 |
| WO2013019938A1 (en) | 2011-08-02 | 2013-02-07 | 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 |
| US20150051389A1 (en) | 2011-08-11 | 2015-02-19 | Isis Pharmaceuticals, Inc. | Selective antisense 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 |
| US9976138B2 (en) | 2011-08-29 | 2018-05-22 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| US9662314B2 (en) | 2011-10-21 | 2017-05-30 | Tufts Medical Center, Inc. | Compounds and methods for the treatment of muscular disease, and related screening methods |
| GB201119538D0 (en) | 2011-11-10 | 2011-12-21 | Viral Ltd | Pharmaceutical compounds |
| EP2785713B1 (en) | 2011-11-28 | 2016-09-07 | Novartis AG | Novel trifluoromethyl-oxadiazole derivatives and their use in the treatment of disease |
| JP6193881B2 (ja) | 2011-12-30 | 2017-09-06 | 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 | 脊髄性筋萎縮症を治療するための化合物 |
| US9399649B2 (en) | 2012-01-26 | 2016-07-26 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| JP6092897B2 (ja) | 2012-02-10 | 2017-03-08 | ピーティーシー セラピューティクス， インコーポレイテッド | 脊髄性筋萎縮症を治療するための化合物 |
| WO2013130689A1 (en) | 2012-03-01 | 2013-09-06 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CN104470909B (zh) | 2012-03-23 | 2018-04-24 | Ptc医疗公司 | 用于治疗脊髓性肌萎缩的化合物 |
| BR112014026703B1 (pt) * | 2012-04-24 | 2022-10-25 | Vertex Pharmaceuticals Incorporated | Inibidores de dna-pk e seus usos, composição farmacêutica e seus usos, e método de sensibilização de uma célula |
| AU2013289938A1 (en) | 2012-07-13 | 2015-01-29 | Indiana University Research & Technology Corporation | Compounds for treatment of 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 |
| WO2014059341A2 (en) | 2012-10-12 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Antisense compounds and uses thereof |
| EP2906256B1 (en) | 2012-10-12 | 2018-08-22 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| JP6291502B2 (ja) | 2012-10-25 | 2018-03-14 | アッシャー・サード・イニシアティブ・インコーポレイテッド | ピラゾロピリダジンならびに網膜変性疾患およびアッシャー症候群に伴う聴力損失を処置するための方法 |
| US9227976B2 (en) * | 2012-10-25 | 2016-01-05 | Usher Iii Initiative, Inc. | Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome |
| JP6255711B2 (ja) | 2012-11-01 | 2018-01-10 | 株式会社リコー | エレクトロクロミック化合物、エレクトロクロミック組成物及び表示素子 |
| US9040712B2 (en) | 2013-01-23 | 2015-05-26 | Novartis Ag | Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions |
| WO2014121287A2 (en) | 2013-02-04 | 2014-08-07 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| AU2014224975B2 (en) | 2013-03-05 | 2017-09-14 | Merck Patent Gmbh | Triazolo(4,5-d)pyrimidine derivatives for the treatment of diseases such as cancer |
| BR112015028475A2 (pt) | 2013-05-14 | 2017-07-25 | Hoffmann La Roche | novos aza-oxo-indois para o tratamento ou profilaxia de infecção virótica respiratória sincitial |
| EP3013345B1 (en) | 2013-06-25 | 2017-11-22 | F. Hoffmann-La Roche AG | Compounds for treating spinal muscular atrophy |
| EA030631B1 (ru) | 2013-07-31 | 2018-09-28 | Новартис Аг | 1,4-дизамещенные аналоги пиридазинхинолина и способы лечения состояний, связанных с smn-дефицитом |
| WO2015024876A2 (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 |
| US9624170B2 (en) | 2013-12-26 | 2017-04-18 | Takeda Pharmaceutical Company Limited | 4-(piperrazin-1-yl)-pyrrolidin-2-one compounds as monoacylglycerol lipase (MAGL) inhibitors |
| PL3094728T3 (pl) | 2014-01-16 | 2022-07-04 | Wave Life Sciences Ltd. | Projekt chiralny |
| ES2699354T3 (es) | 2014-01-17 | 2019-02-08 | Novartis Ag | Derivados de 1-(triazin-3-il/piridazin-3-il)-piper(-azin)idina y composiciones de las mismas para inhibir la actividad de SHP2 |
| AR099134A1 (es) | 2014-01-24 | 2016-06-29 | Hoffmann La Roche | Procedimiento para la preparación de n-[(3-aminooxetán-3-il)metil]-2-(1,1-dioxo-3,5-dihidro-1,4-benzotiazepín-4-il)-6-metil-quinazolín-4-amina |
| MX2016011632A (es) | 2014-03-14 | 2016-12-12 | Raqualia Pharma Inc | Derivados de azaspiro como antagonistas de trpm8. |
| EP3119761B1 (en) | 2014-03-19 | 2023-06-07 | Amydis Diagnostics, Inc. | Amyloid targeting agents and methods of using the same |
| EP3663296B1 (en) | 2014-05-15 | 2023-05-17 | 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 |
| CN106573811A (zh) | 2014-06-17 | 2017-04-19 | 诺维信公司 | 从废水中去除生物磷 |
| BR112016026951B1 (pt) | 2014-06-25 | 2022-09-20 | F. Hoffmann-La Roche Ag | Imidazo[1,2-a]pirazin-1-il-benzamida, seu uso, e composições farmacêuticas |
| US20180282347A1 (en) | 2014-12-02 | 2018-10-04 | Bayer Cropscience Aktiengesellschaft | Bicyclic compounds as pest control agents |
| PL3237618T3 (pl) | 2014-12-24 | 2019-09-30 | Uniqure Ip B.V. | Supresja genu huntingtyny indukowana rnai |
| MX2017009295A (es) | 2015-01-16 | 2018-03-07 | Massachusetts Gen Hospital | Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). |
| 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 |
| CN112608300B (zh) | 2015-03-11 | 2024-04-26 | 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 |
| WO2016184832A1 (en) | 2015-05-20 | 2016-11-24 | F. Hoffmann-La Roche Ag | Compounds for treating spinal muscular atrophy |
| EP4249472A3 (en) | 2015-05-30 | 2023-12-13 | PTC Therapeutics, Inc. | Methods for modulating rna splicing |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| 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 |
| US9920032B2 (en) | 2015-10-02 | 2018-03-20 | Incyte Corporation | Heterocyclic compounds useful as pim kinase inhibitors |
| JP6659841B2 (ja) | 2015-11-12 | 2020-03-04 | エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ | 脊髄性筋萎縮症を処置するための組成物 |
| MX2018005041A (es) | 2015-11-12 | 2018-08-01 | Hoffmann La Roche | Compuestos para tratar la esclerosis lateral amiotrofica. |
| US10383867B2 (en) | 2015-11-28 | 2019-08-20 | Russell Dahl | Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders |
| MX2021001091A (es) * | 2015-12-10 | 2022-04-26 | Ptc Therapeutics Inc | Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington. |
| JP6872550B2 (ja) | 2015-12-10 | 2021-05-19 | エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ | 架橋されたピペリジン誘導体 |
| US10526345B2 (en) | 2016-04-08 | 2020-01-07 | Mankind Pharma Ltd. | Compounds as GPR119 agonists |
| AR108325A1 (es) | 2016-04-27 | 2018-08-08 | Samumed Llc | Isoquinolin-3-il carboxamidas y preparación y uso de las mismas |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| CA3024706A1 (en) | 2016-05-31 | 2017-12-07 | Board Of Regents, The University Of Texas System | Heterocyclic inhibitors of ptpn11 |
| US20190264267A1 (en) | 2016-07-25 | 2019-08-29 | Wave Life Sciences Ltd. | Phasing |
| TWI848901B (zh) * | 2016-10-24 | 2024-07-21 | 美商傳達治療有限公司 | Shp2磷酸酶抑制劑及其使用方法 |
| WO2018187209A1 (en) | 2017-04-03 | 2018-10-11 | 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 |
| US11407753B2 (en) | 2017-06-05 | 2022-08-09 | 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 |
| BR122020005073A2 (pt) | 2017-08-04 | 2020-10-13 | Skyhawk Therapeutics, Inc. | Composto, composição farmacêutica e uso do composto |
| WO2019165073A1 (en) | 2018-02-21 | 2019-08-29 | Relay Therapeutics, Inc. | Shp2 phosphatase inhibitors and methods of use thereof |
| CN112166110B (zh) * | 2018-03-21 | 2023-08-11 | 传达治疗有限公司 | Shp2磷酸酶抑制剂及其使用方法 |
| WO2019183364A1 (en) | 2018-03-21 | 2019-09-26 | 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 |
| US11858941B2 (en) | 2018-06-27 | 2024-01-02 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating Huntington's disease |
| JP7515415B2 (ja) | 2018-06-27 | 2024-07-12 | ピーティーシー セラピューティクス， インコーポレイテッド | ハンチントン病を処置するためのヘテロアリール化合物 |
| US11685746B2 (en) | 2018-06-27 | 2023-06-27 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| 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 |
| BR112021022624A2 (pt) | 2019-05-13 | 2022-01-04 | Ptc Therapeutics Inc | Compostos para o tratamento da doença de huntington |
| WO2021007378A1 (en) | 2019-07-11 | 2021-01-14 | Ptc Therapeutics, Inc. | Compounds for use in treating huntington's disease |
| MX2022005254A (es) | 2019-11-01 | 2022-06-29 | Novartis Ag | El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington. |
| IL297044A (en) | 2020-04-09 | 2022-12-01 | Ptc Therapeutics Inc | Compounds for the treatment of Huntington's disease |
| AU2021380758A1 (en) | 2020-11-12 | 2023-06-15 | Ptc Therapeutics Inc. | Novel rna transcript |
| KR20240054264A (ko) | 2021-07-30 | 2024-04-25 | 피티씨 테라퓨틱스, 인크. | Heteroaryl compounds for treating huntington's disease |
-
2019
- 2019-06-25 US US17/254,660 patent/US11858941B2/en active Active
- 2019-06-25 PL PL19736566.1T patent/PL3814357T3/pl unknown
- 2019-06-25 BR BR112020026545-4A patent/BR112020026545A2/pt unknown
- 2019-06-25 EA EA202092896A patent/EA202092896A1/ru unknown
- 2019-06-25 MX MX2020014098A patent/MX2020014098A/es unknown
- 2019-06-25 LT LTEPPCT/US2019/038889T patent/LT3814357T/lt unknown
- 2019-06-25 IL IL279688A patent/IL279688B1/en unknown
- 2019-06-25 PT PT197365661T patent/PT3814357T/pt unknown
- 2019-06-25 PE PE2020002185A patent/PE20211378A1/es unknown
- 2019-06-25 SG SG11202012674PA patent/SG11202012674PA/en unknown
- 2019-06-25 EP EP24168248.3A patent/EP4434990A1/en active Pending
- 2019-06-25 CN CN201980051041.4A patent/CN112654625B/zh active Active
- 2019-06-25 RS RS20240779A patent/RS65727B1/sr unknown
- 2019-06-25 EP EP19736566.1A patent/EP3814357B1/en active Active
- 2019-06-25 JP JP2020572895A patent/JP7421507B2/ja active Active
- 2019-06-25 ES ES19736566T patent/ES2983003T3/es active Active
- 2019-06-25 DK DK19736566.1T patent/DK3814357T3/da active
- 2019-06-25 FI FIEP19736566.1T patent/FI3814357T3/fi active
- 2019-06-25 KR KR1020207036440A patent/KR20210042265A/ko not_active Application Discontinuation
- 2019-06-25 AU AU2019294478A patent/AU2019294478B2/en active Active
- 2019-06-25 WO PCT/US2019/038889 patent/WO2020005873A1/en active Application Filing
- 2019-06-25 CA CA3103976A patent/CA3103976A1/en active Pending
- 2019-06-25 SI SI201930785T patent/SI3814357T1/sl unknown
- 2019-06-28 AR ARP190101808A patent/AR119651A1/es unknown
-
2020
- 2020-12-23 CL CL2020003378A patent/CL2020003378A1/es unknown
- 2020-12-24 CO CONC2020/0016285A patent/CO2020016285A2/es unknown
-
2021
- 2021-11-16 CL CL2021003022A patent/CL2021003022A1/es unknown
-
2022
- 2022-12-02 CL CL2022003434A patent/CL2022003434A1/es unknown
-
2023
- 2023-05-23 AU AU2023203241A patent/AU2023203241A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AR119651A1 (es) | Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington | |
| CO2019000686A2 (es) | Compuestos de indol sustituidos con [1,2,4]triazolo[1,5-a]piridinilo | |
| AR121661A2 (es) | Esteroides neuroactivos, composiciones y usos de los mismos | |
| PE20190656A1 (es) | Compuestos de tiazolo-piridina sustituida como inhibidores de malt1 | |
| DOP2018000187A (es) | Derivados de pirazolo[1,5-a]pirazin-4-ilo | |
| AR108709A1 (es) | Compuestos moduladores de fxr (nr1h4) | |
| PE20190395A1 (es) | Pirimidin-2-ilamino-1h-pirazoles como inhibidores de lrrk2 para el uso en el tratamiento de trastornos neurodegenerativos | |
| AR105648A1 (es) | Métodos para la preparación de ácidos biliares y derivados de los mismos | |
| AR053120A1 (es) | Aminopiridinas como inhibidores de beta secretasa | |
| PE20180232A1 (es) | Derivados de indol mono o disustituidos como inhibidores de la replicacion viral del dengue | |
| PE20181024A1 (es) | Derivados de ciclohexano sustituido con amido | |
| AR089143A1 (es) | Triazolopiridinas sustituidas con actividad inhibidora de ttk | |
| AR088175A1 (es) | 3-pirimidin-4-il-oxazolidin-2-onas utiles para tratar cancer y composiciones farmaceuticas que las contienen | |
| AR100810A1 (es) | Inhibidores de fosfatidilinositol 3-quinasa | |
| AR099379A1 (es) | Compuestos tricíclicos como agentes antineoplásicos | |
| AR099228A1 (es) | Inhibidores macrocíclicos de fxia que tienen grupos heterocíclicos | |
| AR094346A1 (es) | Derivados de azaindol como inhibidores de las proteína quinasas | |
| AR099823A1 (es) | Compuestos bicíclicos de 7 eslabones | |
| AR106865A1 (es) | Piridinas sustituidas y métodos de uso | |
| AR077695A1 (es) | Derivados de pirimidina como inhibidores del factor ixa | |
| AR103170A1 (es) | Compuestos y composiciones farmacéuticas de los mismos para el tratamiento de desordenes inflamatorios y osteoartritis | |
| AR095885A1 (es) | Compuestos derivados de arilpirrolopiridina como inhibidores de lrrk2 | |
| AR103412A1 (es) | Derivados de indol mono o disustituido como inhibidores de la replicación del virus del dengue | |
| AR060401A1 (es) | Derivados de cromen-2-ona | |
| AR094550A1 (es) | Inhibidores de btk |