MX2020014116A - Agente profilactico o terapeutico para atrofia muscular espinal. - Google Patents

Agente profilactico o terapeutico para atrofia muscular espinal.

Info

Publication number
MX2020014116A
MX2020014116A MX2020014116A MX2020014116A MX2020014116A MX 2020014116 A MX2020014116 A MX 2020014116A MX 2020014116 A MX2020014116 A MX 2020014116A MX 2020014116 A MX2020014116 A MX 2020014116A MX 2020014116 A MX2020014116 A MX 2020014116A
Authority
MX
Mexico
Prior art keywords
sup
sub
optionally substituted
prophylactic
therapeutic agent
Prior art date
Application number
MX2020014116A
Other languages
English (en)
Inventor
Koji Ono
Takeshi Yamasaki
Koji Fuji
Shunya Suzuki
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 MX2020014116A publication Critical patent/MX2020014116A/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
    • 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
    • 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
    • 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] (ver fórmula) (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.
MX2020014116A 2018-06-27 2019-06-27 Agente profilactico o terapeutico para atrofia muscular espinal. MX2020014116A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2018122551 2018-06-27
PCT/JP2019/025732 WO2020004594A1 (ja) 2018-06-27 2019-06-27 脊髄性筋萎縮症の予防または治療剤

Publications (1)

Publication Number Publication Date
MX2020014116A true MX2020014116A (es) 2021-06-15

Family

ID=68986733

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020014116A MX2020014116A (es) 2018-06-27 2019-06-27 Agente profilactico o terapeutico para atrofia muscular espinal.

Country Status (21)

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)
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
BR122020005073A2 (pt) 2017-08-04 2020-10-13 Skyhawk Therapeutics, Inc. Composto, composição farmacêutica e uso do composto
AU2019291794B2 (en) 2018-06-27 2022-06-16 Bristol-Myers Squibb Company Substituted naphthyridinone compounds useful as T cell activators
CA3102326A1 (en) * 2018-07-26 2020-01-30 Domain Therapeutics Substituted quinazolinone derivatives and their use as positive allosteric modulators of mglur4
BR112022017188A2 (pt) * 2020-02-28 2022-11-08 Remix Therapeutics Inc Amidas heterocíclicas e seu uso para modulação de splicing
WO2021207530A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
AU2021253587A1 (en) 2020-04-08 2022-12-01 Remix Therapeutics Inc. Compounds and methods for modulating splicing
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
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
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
TW202333689A (zh) 2021-10-13 2023-09-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
EP4416156A1 (en) * 2021-10-13 2024-08-21 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
WO2023133225A1 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
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
BR0116113A (pt) 2000-12-12 2004-08-03 Neurogen Corp Espiro[isobenzofuran-1,4'-piperidin]-3-onas e 3h-espiroisoben-zofuran-1,4'-piperidinas
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
PE20170003A1 (es) 2009-08-17 2017-03-15 Intellikine Llc Compuestos heterociclicos y usos de los mismos
US8841289B2 (en) 2009-10-13 2014-09-23 Merck Sharp & Dohme B.V. Heterocyclic derivatives
AR085397A1 (es) 2011-02-23 2013-09-25 Intellikine Inc Combinacion de inhibidores de quinasa y sus usos
US8710064B2 (en) * 2011-10-20 2014-04-29 China Medical University 2-aryl-4-quinazolinones and their pharmaceutical compositions
EP2776837A4 (en) 2011-11-11 2015-05-20 Intellikine Llc COMBINATION OF KINASEINHIBITORS AND THEIR USE
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
US9580393B2 (en) 2012-12-13 2017-02-28 University Of Kansas 6-substituted quinazolinone inhibitors
US20160089371A1 (en) 2013-03-15 2016-03-31 Intellikine, Llc Combination of Kinase Inhibitors and Uses Thereof
EP2968340A4 (en) * 2013-03-15 2016-08-10 Intellikine Llc COMBINING KINASE INHIBITORS AND USES THEREOF
EP3013345B1 (en) 2013-06-25 2017-11-22 F. Hoffmann-La Roche AG Compounds for treating spinal muscular atrophy
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
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
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
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
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
WO2016184832A1 (en) 2015-05-20 2016-11-24 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
CA2993013A1 (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
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
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
EP3816160A4 (en) 2022-03-16
EP3816160A1 (en) 2021-05-05
IL279786B1 (en) 2024-10-01
SG11202013088YA (en) 2021-02-25
CL2020003386A1 (es) 2021-05-14
PE20211585A1 (es) 2021-08-18
BR112020026637A2 (pt) 2021-03-30
JOP20200338A1 (ar) 2020-12-24
US20220287748A1 (en) 2022-09-15
CN112469707A (zh) 2021-03-09
KR102693282B1 (ko) 2024-08-12
WO2020004594A1 (ja) 2020-01-02
KR20210025625A (ko) 2021-03-09
CO2021000808A2 (es) 2021-04-30
EA202190116A1 (ru) 2021-08-30
JP7427252B2 (ja) 2024-02-05
AU2022283732A1 (en) 2023-02-02
CR20210050A (es) 2021-06-10
AU2019293757B2 (en) 2022-09-15
PH12020552263A1 (en) 2021-06-28
JPWO2020004594A1 (ja) 2021-07-15
AU2019293757A1 (en) 2021-02-04
CN112469707B (zh) 2024-06-21
NZ772352A (en) 2024-05-31
UA128696C2 (uk) 2024-10-02
IL279786A (en) 2021-03-01
CA3105174A1 (en) 2020-01-02
ZA202100556B (en) 2024-09-25
US12012410B2 (en) 2024-06-18

Similar Documents

Publication Publication Date Title
MX2020014116A (es) Agente profilactico o terapeutico para atrofia muscular espinal.
MX2021000795A (es) Compuestos heterociclicos para inhibir la actividad de shp2.
SG11201900869QA (en) Pharmaceutical compositions containing substituted polycyclic pyridone derivatives and prodrug thereof
MX2020001776A (es) Sulfonilureas y sulfoniltioureas como inhibidores de nlrp3.
JP2016536363A5 (es)
MX2023011675A (es) Beta-hidroxietilaminas heteroaril sustituidas para su uso en el tratamiento de hiperglucemia.
JP2016536364A5 (es)
MX2020012185A (es) Derivado policiclico de piridona.
EP2597088A4 (en) P2X4 Receptor Antagonists
RU2016122093A (ru) Замещенные бензамиды для борьбы с членистоногими
MX2009012283A (es) Derivado heterociclico que contiene nitrogeno que tiene actividad inhibitoria de 11beta-hidroxiesteroide-deshidrogenasa tipo 1.
MX2009004920A (es) Nuevos derivados de aminopirimidina como inhibidores de plk1.
WO2012003405A4 (en) Sgc stimulators
EA201290401A1 (ru) Азотсодержащее гетероциклическое соединение и сельскохозяйственный фунгицид
MX2020009530A (es) Inhibidores de la o-glicoproteína-2-acetamido-2-desoxi-3-dglicopir anosidasa.
TW200612920A (en) Novel imidazolidine derivatives
MX2010001170A (es) Derivados de imidazolona, metodo de preparacion de los mismos y uso biologico de los mismos.
MX344276B (es) Compuesto de piperidina novedoso o sal del mismo.
MX2020011317A (es) Compuestos heterociclicos triciclicos utiles como inhibidores de la integrasa de vih.
WO2008136444A1 (ja) 縮合複素環誘導体
MX2021011286A (es) Compuestos de heterociclil(fenil)metanol útiles en el tratamiento de la hiperglucemia.
MX2020010433A (es) Compuestos de pirazol sustituidos con heteroarilo y uso farmaceutico de los mismos.
MX2018011974A (es) Composicion para controlar microsporidia en peces y metodo para controlar microsporidia en peces utilizando la misma.
PH12020552283A1 (en) Pyrrolo[1,2-b] pyridazine derivatives
PH12020552186A1 (en) Cyanotriazole compounds and uses thereof