CL2020003386A1 - Agente profiláctico o terapéutico para atrofia muscular espinal - Google Patents

Agente profiláctico o terapéutico para atrofia muscular espinal

Info

Publication number
CL2020003386A1
CL2020003386A1 CL2020003386A CL2020003386A CL2020003386A1 CL 2020003386 A1 CL2020003386 A1 CL 2020003386A1 CL 2020003386 A CL2020003386 A CL 2020003386A CL 2020003386 A CL2020003386 A CL 2020003386A CL 2020003386 A1 CL2020003386 A1 CL 2020003386A1
Authority
CL
Chile
Prior art keywords
aliphatic heterocycle
optionally substituted
prophylactic
therapeutic agent
aromatic substituent
Prior art date
Application number
CL2020003386A
Other languages
English (en)
Inventor
Koji Fuji
Takeshi Yamasaki
Shunya Suzuki
Koji Ono
Hiroki TAKAHAGI
Original Assignee
Reborna Biosciences 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 Reborna Biosciences Inc filed Critical Reborna Biosciences Inc
Publication of CL2020003386A1 publication Critical patent/CL2020003386A1/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—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
    • 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
    • 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
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07D401/04—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 directly linked by a ring-member-to-ring-member bond
    • 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
    • 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
    • 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
    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/10—Spiro-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/502—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects
    • G01N33/5023—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects on expression patterns
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61C—DENTISTRY; APPARATUS OR METHODS FOR ORAL OR DENTAL HYGIENE
    • A61C8/00—Means to be fixed to the jaw-bone for consolidating natural teeth or for fixing dental prostheses thereon; Dental implants; Implanting tools
    • A61C8/0093—Features of implants not otherwise provided for
    • A61C8/0096—Implants for use in orthodontic treatment
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/136—Screening for pharmacological compounds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value
    • G01N2500/10—Screening for compounds of potential therapeutic value involving cells

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Pathology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)

Abstract

Un agente profiláctico o terapéutico para atrofia muscular espinal de acuerdo con la presente invención incluye un compuesto representado por la fórmula (I) o una sal de este: [Fórmula química 1] (I) en donde: W1, W2 yW3 se seleccionan cada uno independientemente del grupo que consiste en C R2, C R3, C Rc y C Rd, y se definen mediante uno de los siguientes (i) a (iv): (i) cuando W3 es C R2, entonces W1 es C R3, W2 es C Rc o N, y R1 es un átomo de hidrógeno; (ii) cuando W3 es C R3, entonces W1 es C R2, W2 es C Rc o N, y R1 es un átomo de hidrógeno, alquilo C1–8 o alcoxi C1–8; (iii) cuando W1 es C R2, entonces W2 es C Rc o N, W3 es C Rd, y R1 es un heterociclo alifático que contiene uno o más átomos de nitrógeno, donde el heterociclo alifático se sustituye opcionalmente con un sustituyente no aromático; y (iv) cuando W2 es C R2, W1 es C Rc, W3 es C Rd, y R1 es un heterociclo alifático que contiene uno o más átomos de nitrógeno, donde el heterociclo alifático se sustituye opcionalmente con un sustituyente no aromático; R2 es un anillo aromático de 6 o más miembros opcionalmente sustituido con un sustituyente no aromático; R3 es un heterociclo alifático que contiene uno o más átomos de nitrógeno, donde el heterociclo alifático se sustituye opcionalmente con un sustituyente no aromático; Q1 se selecciona de C Ra y N; Q2 se selecciona de C Rb y N; y Ra, Rb, Rc y Rd se seleccionan cada uno independientemente del grupo que consiste en un átomo de hidrógeno, halógeno, alquilo C1–8, alcoxi C1–8 y un grupo ciano.
CL2020003386A 2018-06-27 2020-12-24 Agente profiláctico o terapéutico para atrofia muscular espinal CL2020003386A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2018122551 2018-06-27

Publications (1)

Publication Number Publication Date
CL2020003386A1 true CL2020003386A1 (es) 2021-05-14

Family

ID=68986733

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020003386A CL2020003386A1 (es) 2018-06-27 2020-12-24 Agente profiláctico o terapéutico para atrofia muscular espinal

Country Status (22)

Country Link
US (1) US12012410B2 (es)
EP (1) EP3816160A4 (es)
JP (1) JP7427252B2 (es)
KR (1) KR102693282B1 (es)
CN (1) CN112469707B (es)
AU (2) AU2019293757B2 (es)
BR (1) BR112020026637A2 (es)
CA (1) CA3105174A1 (es)
CL (1) CL2020003386A1 (es)
CO (1) CO2021000808A2 (es)
CR (1) CR20210050A (es)
EA (1) EA202190116A1 (es)
IL (1) IL279786B1 (es)
JO (1) JOP20200338A1 (es)
MX (1) MX2020014116A (es)
NZ (1) NZ772352A (es)
PE (1) PE20211585A1 (es)
PH (1) PH12020552263A1 (es)
SG (1) SG11202013088YA (es)
UA (1) UA128696C2 (es)
WO (1) WO2020004594A1 (es)
ZA (1) ZA202100556B (es)

Families Citing this family (14)

* 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
SG11202012972YA (en) 2018-06-27 2021-01-28 Bristol Myers Squibb Co Substituted naphthyridinone compounds useful as t cell activators
IL280213B2 (en) * 2018-07-26 2024-06-01 Domain Therapeutics Altered quinazolinone derivatives and their use as positive allosteric modulators of MGLUR4
CN115485025A (zh) * 2020-02-28 2022-12-16 雷密克斯医疗公司 用于调节剪接的化合物和方法
JP2023520924A (ja) 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調節するための化合物及び方法
WO2021207554A1 (en) * 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2023000167A (es) 2020-07-02 2023-05-03 Remix Therapeutics Inc Derivados de 5-[5-(piperidin-4-il)tieno[3,2-c]pirazol-2-il]indazol y compuestos relacionados como moduladores para el corte y empalme de ácidos nucleicos y para el tratamiento de enfermedades proliferativas.
IL299543A (en) 2020-07-02 2023-02-01 Remix Therapeutics Inc 2-(Indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as nucleic acid splicing modulators and for the treatment of multiple diseases
CN112028896A (zh) * 2020-10-14 2020-12-04 奥锐特药业(天津)有限公司 阿卡替尼的新晶型及其制备方法
TW202330552A (zh) * 2021-10-13 2023-08-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
IL312078A (en) 2021-10-13 2024-06-01 Remix Therapeutics Inc Compounds and Methods for Modulating Nucleic Acid Splicing
WO2023133217A1 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
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
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT1050750B (it) * 1975-12-05 1981-03-20 Erba Carlo Spa Derivati della 3.4 di idro chinazolina
EP1347982B1 (en) 2000-12-12 2005-11-16 Neurogen Corporation Spiro isobenzofuran-1,4'-piperidin]-3-ones and 3h-spiroisobenzofuran-1,4'-piperidines
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
MX361732B (es) 2009-06-17 2018-12-14 Cold Spring Harbor Laboratory Composiciones y metodos para la modulacion de division de smn2 en un sujeto.
NZ598220A (en) 2009-08-17 2014-02-28 Intellikine Llc Heterocyclic compounds and uses thereof
MX2012004289A (es) 2009-10-13 2012-06-12 Msd Oss Bv Derivados de azina condensada para el tratamiento de enfermedades relacionadas con el receptor de acetilcolina.
TWI592411B (zh) 2011-02-23 2017-07-21 英特爾立秦有限責任公司 激酶抑制劑之組合及其用途
US8710064B2 (en) * 2011-10-20 2014-04-29 China Medical University 2-aryl-4-quinazolinones and their pharmaceutical compositions
US9682141B2 (en) 2011-11-11 2017-06-20 Intellikine Llc Combination of kinase inhibitors and uses thereof
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
PL2809322T3 (pl) 2012-01-26 2019-11-29 Ptc Therapeutics Inc Związki do leczenia zaniku mięśni pochodzenia rdzeniowego
WO2013119916A2 (en) 2012-02-10 2013-08-15 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
MX352962B (es) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal.
EP2828247B1 (en) 2012-03-23 2019-01-16 PTC Therapeutics, Inc. 4h-chromen-4-one derivatives for treating spinal muscular atrophy
US9580393B2 (en) 2012-12-13 2017-02-28 University Of Kansas 6-substituted quinazolinone inhibitors
EP2968340A4 (en) 2013-03-15 2016-08-10 Intellikine Llc COMBINING KINASE INHIBITORS AND USES THEREOF
US20160089371A1 (en) 2013-03-15 2016-03-31 Intellikine, Llc Combination of Kinase Inhibitors and Uses Thereof
MX363456B (es) 2013-06-25 2019-03-25 Hoffmann La Roche Compuestos para tratar atrofia muscular espinal.
CN105392790B (zh) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途
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
AU2015261046C1 (en) 2014-05-15 2019-07-25 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
EP3160967B1 (en) 2014-06-25 2019-05-08 F. Hoffmann-La Roche AG N-(imidazo[1,2-a]pyrazin-6-yl)-benzamide compounds for treating spinal muscular atrophy
CN107635999B (zh) 2015-05-20 2020-09-25 豪夫迈·罗氏有限公司 用于治疗脊髓性肌萎缩的化合物
WO2017015562A1 (en) * 2015-07-22 2017-01-26 Araxes Pharma Llc Substituted quinazoline compounds and their use as inhibitors of g12c mutant kras, hras and/or nras proteins
CN108026046B (zh) * 2015-07-22 2021-12-21 亚瑞克西斯制药公司 取代的喹唑啉化合物及其作为g12c突变体kras、hras和/或nras蛋白质的抑制剂的用途
WO2017080967A1 (en) 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
EP3573970A1 (en) * 2017-01-26 2019-12-04 Araxes Pharma LLC 1-(6-(3-hydroxynaphthalen-1-yl)quinazolin-2-yl)azetidin-1-yl)prop-2-en-1-one derivatives and similar compounds as kras g12c inhibitors for the treatment of cancer
JP6868410B2 (ja) 2017-02-03 2021-05-12 エスアイアイ・プリンテック株式会社 液体噴射ヘッドチップ、液体噴射ヘッドおよび液体噴射装置

Also Published As

Publication number Publication date
CR20210050A (es) 2021-06-10
EA202190116A1 (ru) 2021-08-30
MX2020014116A (es) 2021-06-15
ZA202100556B (en) 2024-09-25
US20220287748A1 (en) 2022-09-15
IL279786A (en) 2021-03-01
BR112020026637A2 (pt) 2021-03-30
EP3816160A4 (en) 2022-03-16
CN112469707B (zh) 2024-06-21
CA3105174A1 (en) 2020-01-02
JP7427252B2 (ja) 2024-02-05
PH12020552263A1 (en) 2021-06-28
NZ772352A (en) 2024-05-31
KR20210025625A (ko) 2021-03-09
WO2020004594A1 (ja) 2020-01-02
SG11202013088YA (en) 2021-02-25
AU2019293757A1 (en) 2021-02-04
AU2019293757B2 (en) 2022-09-15
CO2021000808A2 (es) 2021-04-30
PE20211585A1 (es) 2021-08-18
US12012410B2 (en) 2024-06-18
CN112469707A (zh) 2021-03-09
EP3816160A1 (en) 2021-05-05
KR102693282B1 (ko) 2024-08-12
UA128696C2 (uk) 2024-10-02
JOP20200338A1 (ar) 2020-12-24
IL279786B1 (en) 2024-10-01
AU2022283732A1 (en) 2023-02-02
JPWO2020004594A1 (ja) 2021-07-15

Similar Documents

Publication Publication Date Title
CO2021000808A2 (es) Agente profiláctico o terapéutico para atrofia muscular espinal
PE20220905A1 (es) Pirroles triciclicos condensados como moduladores de alfa-1 antitripsina
DOP2018000187A (es) Derivados de pirazolo[1,5-a]pirazin-4-ilo
AR109709A1 (es) Inhibidores de la fosfatidilinositol 3-quinasa
AR038703A1 (es) Derivados de 5-feniltiazol y uso como inhibidor de quinasa p i 3
AR082886A1 (es) Compuestos y composiciones farmaceuticas que los contienen
AR076415A1 (es) Compuestos heterociclicos nitrogenados fusionados y su uso en el control de artropodos
BR112019007108A2 (pt) misturas herbicidas
AR068538A1 (es) Compuesto heterociclico de 5 miembros con actividad supresora de la secrecion de acido
PE20200608A1 (es) Nuevos derivados de azaquinolina
AR078163A1 (es) Utilizacion de aminodihidrotiazinas para el tratamiento o prevencion de la diabetes
PE20040373A1 (es) Composiciones herbicidas que comprenden derivados de isoxazolina
CO5550398A2 (es) 7-aminotriazolpirimidinas, proceso e intermedios para su preparacion, composiciones que las comprenden y su uso para controlar hongos dañinos
AR078463A1 (es) Composicion y metodo para controlar plagas de artropodos
PE20212253A1 (es) Compuestos de piperidina urea 4-metilsulfonil-sustituidos para el tratamiento de miocardiopatia dilatada (mcd)
DOP2020000222A (es) Derivado policiclico de piridona
AR063805A1 (es) Derivados de triazol sustituidos como inhibidores de ksp. composiciones farmaceuticas.
AR103742A1 (es) Derivados de trifluorometilpropanamida
CO2018010787A2 (es) Compuesto de griseofulvina
AR115007A1 (es) Derivados de triazolona o sales de los mismos y composiciones farmacéuticas que comprenden los mismos
AR117037A1 (es) Compuestos pirazol para el tratamiento de enfermedades autoinmunitarias
CL2020002550A1 (es) Compuestos de pirazol sustituidos con heteroarilo y uso farmacéutico de los mismos.
AR073043A1 (es) Compuestos de azetidinas polisustituidos, composiciones farmaceuticas que los contienen, metodo de preparacion y uso de los mismos en el tratamiento de enfermedades respiratorias, metabolicas y del sistema nervioso central, entre otras.
BR112018008885A8 (pt) composto heterocíclico condensado
AR119198A1 (es) Proceso para la preparación de inhibidores del canal de cloruro cic-1