CA3175205A1 - Compounds and methods for modulating splicing - Google Patents

Compounds and methods for modulating splicing

Info

Publication number
CA3175205A1
CA3175205A1 CA3175205A CA3175205A CA3175205A1 CA 3175205 A1 CA3175205 A1 CA 3175205A1 CA 3175205 A CA3175205 A CA 3175205A CA 3175205 A CA3175205 A CA 3175205A CA 3175205 A1 CA3175205 A1 CA 3175205A1
Authority
CA
Canada
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
CA3175205A
Other languages
English (en)
French (fr)
Inventor
Dominic Reynolds
Michael W. Seiler
Anant A. AGRAWAL
Frederic VAILLANCOURT
Peter Smith
Serge Leger
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Remix Therapeutics Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA3175205A1 publication Critical patent/CA3175205A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D513/00—Heterocyclic 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
    • C07D513/02—Heterocyclic 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
    • C07D513/04—Ortho-condensed 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
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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)
  • 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)
CA3175205A 2020-04-08 2021-04-08 Compounds and methods for modulating splicing Pending CA3175205A1 (en)

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 (1)

Publication Number Publication Date
CA3175205A1 true CA3175205A1 (en) 2021-10-14

Family

ID=75747105

Family Applications (2)

Application Number Title Priority Date Filing Date
CA3175205A Pending CA3175205A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing
CA3175193A Pending CA3175193A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3175193A Pending CA3175193A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

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

Family Cites Families (39)

* 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
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
DE3763282D1 (de) * 1986-03-19 1990-07-26 Kumiai Chemical Industry Co Fungizide zur verwendung in der landwirtschaft und im gartenbau.
TW449600B (en) * 1994-04-19 2001-08-11 Takeda Chemical Industries Ltd Condensed-ring thiophene derivatives, their production and use
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
CA2946702C (en) * 2014-04-23 2022-06-28 Mitsubishi Tanabe Pharma Corporation Bicyclic or tricyclic heterocyclic compound
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

Also Published As

Publication number Publication date
JP2023520925A (ja) 2023-05-22
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
CN116134036A (zh) 2023-05-16
CN116096725A (zh) 2023-05-09

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