BR112022020423A2 - Compostos e métodos para modulação de splicing - Google Patents

Compostos e métodos para modulação de splicing

Info

Publication number
BR112022020423A2
BR112022020423A2 BR112022020423A BR112022020423A BR112022020423A2 BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2 BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2
Authority
BR
Brazil
Prior art keywords
compounds
methods
splicing
splicing modulation
modulation
Prior art date
Application number
BR112022020423A
Other languages
English (en)
Inventor
Reynolds Dominic
Leger Serge
W Seiler Michael
A Agrawal Anant
Vaillancourt Frederic
Smith Peter
T Hopper Allen
Prajapati Sudeep
Soueidan Olivier
Original Assignee
Remix Therapeutics Inc
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 Remix Therapeutics Inc filed Critical Remix Therapeutics Inc
Publication of BR112022020423A2 publication Critical patent/BR112022020423A2/pt

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A61K31/502—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with carbocyclic ring systems, e.g. cinnoline, phthalazine
    • 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
    • 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
    • A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • 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
    • 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
    • C07D403/02—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 two hetero rings
    • C07D403/04—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 two hetero rings directly linked by a ring-member-to-ring-member bond
    • 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
    • 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
    • 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
    • C07D471/08—Bridged 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Communicable Diseases (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Oncology (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Replacement Of Web Rolls (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

COMPOSTOS E MÉTODOS PARA MODULAÇÃO DE SPLICING. A presente invenção refere-se a compostos e composições relacionadas que, entre outros, modulam o splicing de ácido nucleico, por exemplo, splicing de um pré-mRNA, assim como a métodos de uso dos mesmos.
BR112022020423A 2020-04-08 2021-04-08 Compostos e métodos para modulação de splicing BR112022020423A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063007331P 2020-04-08 2020-04-08
US202063044318P 2020-06-25 2020-06-25
US202063072922P 2020-08-31 2020-08-31
US202063126494P 2020-12-16 2020-12-16
PCT/US2021/026481 WO2021207554A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (1)

Publication Number Publication Date
BR112022020423A2 true BR112022020423A2 (pt) 2022-11-29

Family

ID=75690721

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022020423A BR112022020423A2 (pt) 2020-04-08 2021-04-08 Compostos e métodos para modulação de splicing

Country Status (14)

Country Link
US (2) US20230167093A1 (pt)
EP (1) EP4132646A1 (pt)
JP (1) JP2023520916A (pt)
KR (1) KR20220167298A (pt)
CN (1) CN115551593A (pt)
AU (1) AU2021253587A1 (pt)
BR (1) BR112022020423A2 (pt)
CA (1) CA3174353A1 (pt)
CL (1) CL2022002780A1 (pt)
CR (1) CR20220566A (pt)
IL (1) IL297137A (pt)
MX (1) MX2022012678A (pt)
TW (1) TW202208344A (pt)
WO (2) WO2021207532A1 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018309187C1 (en) 2017-08-04 2023-09-07 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2023532623A (ja) 2020-05-13 2023-07-31 シーエイチディーアイ ファウンデーション，インコーポレーテッド ハンチントン病を処置するためのｈｔｔモジュレータ
CA3233973A1 (en) * 2021-10-13 2023-04-20 Dominic Reynolds Compounds and methods for modulating nucleic acid splicing
WO2023097007A1 (en) * 2021-11-24 2023-06-01 Remix Therapeutics Inc. Compounds and methods for modulating splicing
TW202346305A (zh) * 2022-01-05 2023-12-01 美商雷密克斯醫療公司 用於調節剪切之化合物及方法
US11957759B1 (en) 2022-09-07 2024-04-16 Arvinas Operations, Inc. Rapidly accelerated fibrosarcoma (RAF) degrading compounds and associated methods of use

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE60010448T2 (de) * 1999-03-17 2005-04-07 Astrazeneca Ab Amid-derivate
GB0324790D0 (en) * 2003-10-24 2003-11-26 Astrazeneca Ab Amide derivatives
TWI320783B (en) * 2005-04-14 2010-02-21 Otsuka Pharma Co Ltd Heterocyclic compound
CA2699384A1 (en) * 2007-09-12 2009-03-19 Wyeth Llc Isoquinolinyl and isoindolinyl derivatives as histamine-3 antagonists
BR112014019750B1 (pt) 2012-02-10 2020-03-03 F. Hoffmann-La Roche Ag. Composto, composição farmacêutica e seus usos
MY174339A (en) 2012-08-13 2020-04-09 Novartis Ag 1,4-disubstituted pyridazine analogs and methods for treating smn-deficiency-related conditions
WO2014151147A1 (en) * 2013-03-15 2014-09-25 Intellikine, Llc Combination of kinase inhibitors and uses thereof
CN107428729B (zh) 2015-02-09 2021-07-16 豪夫迈·罗氏有限公司 用于治疗癌症的化合物
MA41898A (fr) * 2015-04-10 2018-02-13 Hoffmann La Roche Dérivés de quinazolinone bicyclique
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
ES2904513T3 (es) * 2015-09-23 2022-04-05 Janssen Pharmaceutica Nv 1,4-Benzodiazepinas biheteroarilo sustituidas y usos de las mismas para el tratamiento del cáncer
WO2017100726A1 (en) 2015-12-10 2017-06-15 Ptc Therapeutics, Inc. Methods for treatng huntington's disease
US10913730B2 (en) * 2016-10-10 2021-02-09 Dong-A Socio Holdings Co., Ltd. Heteroaryl compounds and their use as Mer inhibitors
EP3544435A4 (en) 2016-11-28 2020-11-04 PTC Therapeutics, Inc. RNA SPLICE MODULATION PROCESSES
US10787435B2 (en) * 2017-01-22 2020-09-29 Fuijan Cosunter Pharmaceutical Co. Ltd. ASK1 inhibitor and preparation method and use thereof
WO2018232039A1 (en) 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
AU2018309187C1 (en) 2017-08-04 2023-09-07 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN111373057A (zh) 2017-09-25 2020-07-03 斯基霍克疗法公司 用于筛选和鉴定剪接调节剂的方法和组合物
CN109721597B (zh) * 2017-10-30 2021-05-14 上海迪诺医药科技有限公司 吡啶并氮杂卓衍生物、其药物组合物及应用
EP3706747A4 (en) * 2017-11-08 2021-08-04 Merck Sharp & Dohme Corp. PRMT5 INHIBITORS
PT3717471T (pt) * 2017-12-02 2022-03-08 Galapagos Nv Novos compostos e composições farmacêuticas dos mesmos para o tratamento de doenças
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

Also Published As

Publication number Publication date
US20230159496A1 (en) 2023-05-25
WO2021207532A1 (en) 2021-10-14
CR20220566A (es) 2023-01-23
EP4132646A1 (en) 2023-02-15
CN115551593A (zh) 2022-12-30
KR20220167298A (ko) 2022-12-20
WO2021207554A1 (en) 2021-10-14
AU2021253587A1 (en) 2022-12-01
TW202208344A (zh) 2022-03-01
MX2022012678A (es) 2023-01-11
US20230167093A1 (en) 2023-06-01
IL297137A (en) 2022-12-01
CL2022002780A1 (es) 2023-04-28
CA3174353A1 (en) 2021-10-14
JP2023520916A (ja) 2023-05-22

Similar Documents

Publication Publication Date Title
BR112022017210A2 (pt) Compostos e métodos para modulação de splicing
BR112022017107A2 (pt) Derivados de piridazina para modulação de splicing de ácido nucleico
BR112022020418A2 (pt) Compostos e métodos para modulação de splicing
BR112022020423A2 (pt) Compostos e métodos para modulação de splicing
BR112023022592A2 (pt) Cartucho de grampos compreendendo acionadores de grampos e suportes de estabilidade
BR112021024108A2 (pt) Inibidores de tead e usos dos mesmos
BR112021021736A2 (pt) Batente de tecido para um instrumento cirúrgico
CL2020000937A1 (es) Anticuerpo multiespecífico.
BR112016014020A2 (pt) Compostos moduladores de tetra-hidropiridopirazinas de gpr6, seus sais e os usos dos mesmos
BRPI0811926A8 (pt) Composições herbicidas, processo para a preparação de uma composição herbicida, processo para o controle de vegetação indesejável e uso de um ácido graxo para estabilizar uma composição herbicida
MX2022010681A (es) Compuestos y metodos para modular el empalme.
BR112023021068A2 (pt) Compostos, composições e métodos para tratar câncer
BR112018002904A2 (pt) sistemas de solvente de triamida alquiltiofosfórica e/ou dcd estabilizados por amina em altas temperaturas e uso em aplicações agrícolas
CR20190150A (es) Formulaciones de anticuerpos inhibidores de masp-2 altamente concentrados, de baja viscosidad, kits, y métodos
BR112018075201A2 (pt) moduladores do receptor beta-3 adrenérgico úteis para o tratamento ou prevenção de distúrbios relacionados com os mesmos
BR112022009723A2 (pt) Anticorpos específicos para cd47, pd-l1, e usos dos mesmos
BR112022008061A2 (pt) Processo eficaz para síntese de 2-amino-5-cloro-n,3-dimetilbenzamida
BR112023023223A2 (pt) Degradadores de cdk2 e usos dos mesmos
BR112022019284A2 (pt) Aptâmeros que se ligam a análogos e derivados de tiamina
CL2017001433A1 (es) Compuestos a base de cumarina y métodos asociados
BR112022014331A2 (pt) Composição de asfalto, processo para preparar uma composição de asfalto e uso da composição
CO2024002647A2 (es) Compuestos y métodos para modular el empalme
CR20220483A (es) Compuestos y métodos para modular el empalme
DOP2020000226A (es) Método para marcar un hidrocarburo de petróleo
CO2024004482A2 (es) Composiciones moduladoras de la subunidad beta e de inhibina (inhbe) y métodos de uso de las mismas