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) IL279786B2 (es)
JO (1) JOP20200338A1 (es)
MX (1) MX2020014116A (es)
MY (1) MY203060A (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 (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2020105929A (ru) 2017-08-04 2021-09-06 Скайхоук Терапьютикс, Инк. Способы и композиции для модулирования сплайсинга
HRP20231253T1 (hr) 2018-06-27 2024-02-02 Bristol-Myers Squibb Company Supstituirani spojevi naftiridina korisni kao aktivatori t stanica
US20220089609A1 (en) * 2018-07-26 2022-03-24 Domain Therapeutics Substituted quinazolinone derivatives and their use as positive allosteric modulators of mglur4
EP3920926A4 (en) 2019-02-05 2022-10-19 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN119638696A (zh) 2019-02-06 2025-03-18 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP2023515620A (ja) * 2020-02-28 2023-04-13 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
WO2021207530A1 (en) * 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2021207554A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN117015536A (zh) * 2020-07-02 2023-11-07 雷密克斯医疗公司 作为核酸剪接和增殖性疾病治疗的调节剂的2-(吲唑-5-基)-6-(哌啶-4-基)-1,7-萘啶衍生物及相关化合物
KR20230051168A (ko) * 2020-07-02 2023-04-17 레믹스 테라퓨틱스 인크. 2-(인다졸-5-일)-6-(피페리딘-4-일)-1,7-나프티리딘 유도체, 및 핵산 스플라이싱의 조절제로서의 및 증식성 질환의 치료를 위한 관련 화합물
EP4178963A1 (en) 2020-07-02 2023-05-17 Remix Therapeutics Inc. 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative 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
WO2023064880A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
WO2023064879A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
WO2023133225A1 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20250109140A1 (en) 2022-01-05 2025-04-03 Remix Theraputics Inc. 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
US20250092065A1 (en) 2022-01-05 2025-03-20 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

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
US6566367B2 (en) * 2000-12-12 2003-05-20 Pfizer Inc. Spiro[isobenzofuran-1,4′-piperidin]-3-ones and 3H-spiroisobenzofuran-1,4′-piperidines
WO2010019243A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc. Methods for treating viral infections
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
GEP201706639B (en) 2009-08-17 2017-03-27 Intellikine Llc Heterocyclic compounds and uses thereof
AU2010305825A1 (en) 2009-10-13 2012-04-19 Merck Sharp & Dohme B.V. Condensed azine - derivatives for the treatment of diseases related to the acetylcholine receptor
JP6130305B2 (ja) 2011-02-23 2017-05-17 インテリカイン， エルエルシー キナーゼ阻害剤の組み合わせおよびそれらの使用
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
USRE47689E1 (en) 2011-12-30 2019-11-05 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
ES2733644T3 (es) 2012-01-26 2019-12-02 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
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
EA029155B1 (ru) 2012-03-01 2018-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
CA2868026C (en) 2012-03-23 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014093869A1 (en) 2012-12-13 2014-06-19 University Of Kansas 6-substituted quinazolinone inhibitors
WO2014151147A1 (en) 2013-03-15 2014-09-25 Intellikine, Llc Combination of kinase inhibitors and uses thereof
US20160089371A1 (en) 2013-03-15 2016-03-31 Intellikine, Llc Combination of Kinase Inhibitors and Uses Thereof
RU2673542C2 (ru) 2013-06-25 2018-11-28 Ф. Хоффманн-Ля Рош Аг Соединения для лечения спинальной мышечной атрофии
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
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP3082820B1 (en) 2013-12-19 2022-07-20 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
DK3143025T3 (da) 2014-05-15 2019-12-09 Hoffmann La Roche Forbindelser til behandling af spinal muskelatrofi
CA2952895C (en) 2014-06-25 2023-09-26 F. Hoffmann-La Roche Ag Imidazo[1,2-a]pyrazin-1yl-benzamide compounds for treating spinal muscular atrophy
JP6749343B2 (ja) 2015-05-20 2020-09-02 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化合物
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
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
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
US11136308B2 (en) * 2017-01-26 2021-10-05 Araxes Pharma Llc Substituted quinazoline and quinazolinone compounds and methods of use thereof
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
PH12020552263A1 (en) 2021-06-28
IL279786B2 (en) 2025-02-01
JPWO2020004594A1 (ja) 2021-07-15
IL279786B1 (en) 2024-10-01
KR20210025625A (ko) 2021-03-09
EP3816160A4 (en) 2022-03-16
ZA202100556B (en) 2024-09-25
EA202190116A1 (ru) 2021-08-30
KR102693282B1 (ko) 2024-08-12
WO2020004594A1 (ja) 2020-01-02
CR20210050A (es) 2021-06-10
CN112469707A (zh) 2021-03-09
IL279786A (en) 2021-03-01
CA3105174A1 (en) 2020-01-02
SG11202013088YA (en) 2021-02-25
BR112020026637A2 (pt) 2021-03-30
JP7427252B2 (ja) 2024-02-05
US20220287748A1 (en) 2022-09-15
MY203060A (en) 2024-06-06
PE20211585A1 (es) 2021-08-18
AU2022283732A1 (en) 2023-02-02
NZ772352A (en) 2024-05-31
MX2020014116A (es) 2021-06-15
UA128696C2 (uk) 2024-10-02
CN112469707B (zh) 2024-06-21
US12012410B2 (en) 2024-06-18
CO2021000808A2 (es) 2021-04-30
JOP20200338A1 (ar) 2020-12-24
AU2019293757B2 (en) 2022-09-15
AU2019293757A1 (en) 2021-02-04
EP3816160A1 (en) 2021-05-05

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
AR082886A1 (es) Compuestos y composiciones farmaceuticas que los contienen
AR038703A1 (es) Derivados de 5-feniltiazol y uso como inhibidor de quinasa p i 3
AR068538A1 (es) Compuesto heterociclico de 5 miembros con actividad supresora de la secrecion de acido
EA201101089A1 (ru) Производные оксадиазола в качестве агонистов рецептора s1p1
DOP2020000222A (es) Derivado policiclico de piridona
BRPI0718966B8 (pt) composto, composição farmacêutica, inibidor plk1 e agente antitumoral
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
ES2639757T3 (es) Síntesis de 2-(1H-indazol-6-il)espiro[ciclopropan-1,3'-indolin]-2'-onas quirales
PE20212253A1 (es) Compuestos de piperidina urea 4-metilsulfonil-sustituidos para el tratamiento de miocardiopatia dilatada (mcd)
CO2020003114A2 (es) Compuesto que tiene estructura ciclica
AR067665A1 (es) Derivados de trans-3- aza-biciclo ( 3.1.0) hexano
AR063805A1 (es) Derivados de triazol sustituidos como inhibidores de ksp. composiciones farmaceuticas.
AR103742A1 (es) Derivados de trifluorometilpropanamida
CO2018010787A2 (es) Compuesto de griseofulvina
AR117037A1 (es) Compuestos pirazol para el tratamiento de enfermedades autoinmunitarias
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.
CL2020002550A1 (es) Compuestos de pirazol sustituidos con heteroarilo y uso farmacéutico de los mismos.
AR119198A1 (es) Proceso para la preparación de inhibidores del canal de cloruro cic-1
AR116906A1 (es) Derivados de heteroarilo bicíclico
AR041605A1 (es) Compuesto 1- sustituido-4-nitroimidazol y metodo para preparar el mismo
AR122677A1 (es) Nuevos compuestos de dicianuro de heterociclil-carbonohidrazonoílo fusionado y su uso