JP2023520925A - スプライシングを調節するための化合物及び方法 - Google Patents

スプライシングを調節するための化合物及び方法 Download PDF

Info

Publication number
JP2023520925A
JP2023520925A JP2022561469A JP2022561469A JP2023520925A JP 2023520925 A JP2023520925 A JP 2023520925A JP 2022561469 A JP2022561469 A JP 2022561469A JP 2022561469 A JP2022561469 A JP 2022561469A JP 2023520925 A JP2023520925 A JP 2023520925A
Authority
JP
Japan
Prior art keywords
compound
heterocyclyl
heteroaryl
alkyl
aryl
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2022561469A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023520925A5 (https=
Inventor
ドミニク レイノルズ,
マイケル ダブリュー. サイラー,
アナント エー. アグラワル,
フレデリック ヴァイランコート,
ピーター スミス,
セルジュ レジェ,
Original Assignee
リミックス セラピューティクス インコーポレイテッド
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 リミックス セラピューティクス インコーポレイテッド filed Critical リミックス セラピューティクス インコーポレイテッド
Publication of JP2023520925A publication Critical patent/JP2023520925A/ja
Publication of JP2023520925A5 publication Critical patent/JP2023520925A5/ja
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D513/00Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D495/00Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02Heterocyclic 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
    • C07D495/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Replacement Of Web Rolls (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Saccharide Compounds (AREA)
JP2022561469A 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法 Pending JP2023520925A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063007327P 2020-04-08 2020-04-08
US63/007,327 2020-04-08
US202063043920P 2020-06-25 2020-06-25
US63/043,920 2020-06-25
US202063072873P 2020-08-31 2020-08-31
US63/072,873 2020-08-31
US202063126493P 2020-12-16 2020-12-16
US63/126,493 2020-12-16
PCT/US2021/026435 WO2021207530A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (2)

Publication Number Publication Date
JP2023520925A true JP2023520925A (ja) 2023-05-22
JP2023520925A5 JP2023520925A5 (https=) 2024-04-18

Family

ID=75747105

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022561469A Pending JP2023520925A (ja) 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法
JP2022561468A Active JP7797407B2 (ja) 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022561468A Active JP7797407B2 (ja) 2020-04-08 2021-04-08 スプライシングを調節するための化合物及び方法

Country Status (14)

Country Link
US (2) US20240287097A1 (https=)
EP (2) EP4132935A1 (https=)
JP (2) JP2023520925A (https=)
KR (2) KR20230005210A (https=)
CN (2) CN116134036A (https=)
AU (2) AU2021253571A1 (https=)
BR (2) BR112022020337A2 (https=)
CA (2) CA3175205A1 (https=)
CL (1) CL2022002779A1 (https=)
CO (1) CO2022015926A2 (https=)
CR (1) CR20220567A (https=)
IL (1) IL297149A (https=)
MX (2) MX2022012676A (https=)
WO (2) WO2021207550A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN114126613A (zh) 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
CN113661162A (zh) 2019-02-06 2021-11-16 斯基霍克疗法公司 用于调节剪接的方法和组合物
BR112022023025A2 (pt) 2020-05-13 2023-03-28 Chdi Foundation Inc Moduladores de htt para tratar doença de huntington
WO2022194802A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thiadiazolopyrimidone derivatives
BR112023018466A2 (pt) * 2021-03-17 2023-10-10 Hoffmann La Roche Derivados de tiazolopirimidinona
CN116997555A (zh) * 2021-03-17 2023-11-03 豪夫迈·罗氏有限公司 新颖噻吩并嘧啶酮衍生物
WO2023064879A1 (en) * 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
US12281116B2 (en) 2021-11-17 2025-04-22 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
AU2022398247A1 (en) * 2021-11-24 2024-06-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN115112899B (zh) * 2022-06-15 2024-08-16 四川大学华西医院 检测羧肽酶a4的试剂和/或系统在制备恶性胸腔积液筛查产品中的用途

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS57142989A (en) * 1981-02-27 1982-09-03 Dai Ichi Seiyaku Co Ltd Thiazolopyrimidine derivative
JPS58177997A (ja) * 1982-04-12 1983-10-18 Dai Ichi Seiyaku Co Ltd チアジアゾロピリミジノン誘導体
WO1995028405A1 (en) * 1994-04-19 1995-10-26 Takeda Chemical Industries, Ltd. Bicyclic thiophene derivatives and use as gonadotropin releasing hormone antagonists
WO2015163339A1 (ja) * 2014-04-23 2015-10-29 田辺三菱製薬株式会社 新規二環性または三環性複素環化合物

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB242502A (en) * 1925-03-10 1925-11-12 Hoe & Co R Improvements in stapling and folding mechanism
DE3763282D1 (de) * 1986-03-19 1990-07-26 Kumiai Chemical Industry Co Fungizide zur verwendung in der landwirtschaft und im gartenbau.
US6153614A (en) * 1998-07-16 2000-11-28 Abbott Laboratories Piperazinyl pyrimidine dione compounds selective for adrenoceptors
AR038118A1 (es) * 2002-01-14 2004-12-29 Upjohn Co Compuestos derivados de la bencinamida del acido 7-oxo-4,7-dihidrotien[2,3-b[piridin-6-carboxilico 3-sustituido que son utiles como antivirales
US20060194871A1 (en) * 2003-04-11 2006-08-31 Barvian Kevin K Heterocyclic mchr1 antagoists
US20050176738A1 (en) * 2003-11-07 2005-08-11 Neurocrine Biosciences, Inc. Melanin-concentrating hormone receptor antagonists and compositions and methods related thereto
US7745447B2 (en) * 2005-10-26 2010-06-29 Bristol-Myers Squibb Company Substituted thieno[3,2-D]pyrimidines as non-basic melanin concentrating hormone receptor-1 antagonists
AR056155A1 (es) * 2005-10-26 2007-09-19 Bristol Myers Squibb Co Antagonistas del receptor 1 de la hormona de concentracion de melanina no basica
KR100846988B1 (ko) * 2006-03-06 2008-07-16 제일약품주식회사 신규한 티에노피리미딘 유도체 또는 이의 약학적으로허용가능한 염, 이의 제조방법 및 이를 함유하는 약학조성물
WO2008057601A2 (en) * 2006-11-08 2008-05-15 The Rockefeller University Organic compounds
CA2805724A1 (en) * 2010-07-16 2012-01-19 Rockefeller University Oxadiazolo[3,2-a]pyrimidines and thiadiazolo[3,2-a]pyrimidines
EP2721036B1 (en) * 2011-06-15 2015-07-22 Takeda GmbH Novel 3,4,4a,10b-tetrahydro-1h-thiopyrano[4,3-c]isoquinoline compounds
US20130102601A1 (en) * 2011-10-21 2013-04-25 F. Hoffmann-La Roche Ltd Pyrimidin-4-one derivatives and their use in the treatment, amelioration or prevention of a viral disease
EP2804610B1 (en) * 2012-01-16 2018-10-31 The Rockefeller University Organic compounds
DK2812004T3 (en) 2012-02-10 2018-10-15 Ptc Therapeutics Inc COMPOUNDS FOR TREATMENT OF SPINAL MUSCLE DROPHY
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
SI3143025T1 (sl) * 2014-05-15 2020-01-31 F. Hoffmann-La Roche Ag Spojine za zdravljenje spinalne mišične atrofije
US10294243B2 (en) * 2014-06-05 2019-05-21 Bayer Cropscience Aktiengesellschaft Bicyclic compounds as pesticides
WO2016128343A1 (en) 2015-02-09 2016-08-18 F. Hoffmann-La Roche Ag Compounds for the treatment of cancer
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
CN106279211B (zh) * 2015-06-03 2020-09-15 北京大学 一种噻唑并嘧啶酮化合物及其制备方法和应用
JP6791717B2 (ja) * 2015-10-22 2020-11-25 田辺三菱製薬株式会社 医薬組成物
AR106472A1 (es) * 2015-10-26 2018-01-17 Gilead Apollo Llc Inhibidores de acc y usos de los mismos
IL294124B2 (en) 2015-12-10 2024-02-01 Ptc Therapeutics Inc Methods for treating huntington's disease
CA3037642A1 (en) * 2016-09-23 2018-03-29 Bayer Aktiengesellschaft N 3 -cyclically substituted thienouraciles and use thereof
EA201991309A1 (ru) 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
EA201992780A1 (ru) * 2017-06-21 2020-06-02 ШАЙ ТЕРАПЬЮТИКС ЭлЭлСи Соединения, которые взаимодействуют с суперсемейством ras, для лечения рака, воспалительных заболеваний, ras-опатий и фиброзного заболевания
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
KR20200057071A (ko) 2017-09-25 2020-05-25 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱 조절제의 스크리닝 및 확인을 위한 방법 및 조성물
CA3094988A1 (en) * 2018-03-30 2019-10-03 Biotheryx, Inc. Thienopyrimidinone compounds
CN112272666A (zh) 2018-04-10 2021-01-26 斯基霍克疗法公司 用于治疗癌症的化合物
BR112020026637A2 (pt) * 2018-06-27 2021-03-30 Reborna Biosciences, Inc. Agente profilático ou terapêutico para atrofia muscular espinhal
BR112023018466A2 (pt) * 2021-03-17 2023-10-10 Hoffmann La Roche Derivados de tiazolopirimidinona
WO2022194802A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thiadiazolopyrimidone derivatives

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS57142989A (en) * 1981-02-27 1982-09-03 Dai Ichi Seiyaku Co Ltd Thiazolopyrimidine derivative
JPS58177997A (ja) * 1982-04-12 1983-10-18 Dai Ichi Seiyaku Co Ltd チアジアゾロピリミジノン誘導体
WO1995028405A1 (en) * 1994-04-19 1995-10-26 Takeda Chemical Industries, Ltd. Bicyclic thiophene derivatives and use as gonadotropin releasing hormone antagonists
WO2015163339A1 (ja) * 2014-04-23 2015-10-29 田辺三菱製薬株式会社 新規二環性または三環性複素環化合物

Also Published As

Publication number Publication date
EP4132936A1 (en) 2023-02-15
CR20220567A (es) 2023-01-13
EP4132935A1 (en) 2023-02-15
CA3175193A1 (en) 2021-10-14
KR20230005210A (ko) 2023-01-09
JP2023520924A (ja) 2023-05-22
CL2022002779A1 (es) 2023-04-28
AU2021251220A1 (en) 2022-11-03
BR112022020418A2 (pt) 2023-05-02
BR112022020337A2 (pt) 2022-12-13
CO2022015926A2 (es) 2022-11-29
JP7797407B2 (ja) 2026-01-13
WO2021207550A1 (en) 2021-10-14
IL297149A (en) 2022-12-01
WO2021207530A8 (en) 2021-12-02
WO2021207530A1 (en) 2021-10-14
MX2022012676A (es) 2023-01-11
US20240287097A1 (en) 2024-08-29
AU2021253571A1 (en) 2022-11-10
MX2022012677A (es) 2023-01-11
KR20230004575A (ko) 2023-01-06
US20240279239A1 (en) 2024-08-22
CA3175205A1 (en) 2021-10-14
CN116134036A (zh) 2023-05-16
CN116096725A (zh) 2023-05-09

Similar Documents

Publication Publication Date Title
JP7736700B2 (ja) 複素環アミド及びスプライシングを調節するためのその使用
JP7797407B2 (ja) スプライシングを調節するための化合物及び方法
TWI912305B (zh) 調節剪接之化合物及方法
JP2023515617A (ja) 核酸スプライシングを調節するためのピリダジン誘導体
KR20220158237A (ko) 스플라이싱을 조절하기 위한 화합물 및 방법
EP4110775A1 (en) Thiophenyl derivatives useful for modulating nucleic acid splicing
IL311132A (en) Splicing Modulation Compounds and Methods
JP2023532331A (ja) 核酸をスプライシングするため及び増殖性疾患を処置するための修飾因子としての5-[5-(ピペリジン-4-イル)チエノ[3,2-c]ピラゾール-2-イル]インダゾール誘導体及び関連する化合物並びにスプライシングを調節するための化合物及び方法
KR20260047325A (ko) 스플라이싱을 조절하기 위한 화합물 및 방법

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240408

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240408

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250225

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20250227

RD03 Notification of appointment of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7423

Effective date: 20250417

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20250423

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250526

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250825

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20251104

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260302

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260421