JP2019535789A - Ｒｎａスプライシングを調節する方法 - Google Patents

Ｒｎａスプライシングを調節する方法 Download PDF

Info

Publication number
JP2019535789A
JP2019535789A JP2019528527A JP2019528527A JP2019535789A JP 2019535789 A JP2019535789 A JP 2019535789A JP 2019528527 A JP2019528527 A JP 2019528527A JP 2019528527 A JP2019528527 A JP 2019528527A JP 2019535789 A JP2019535789 A JP 2019535789A
Authority
JP
Japan
Prior art keywords
alkyl
amino
alkoxy
hydroxy
heterocyclyl
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2019528527A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019535789A5 (cg-RX-API-DMAC7.html
Inventor
ブハッタチャリイヤ アヌラドハ
ブハッタチャリイヤ アヌラドハ
ダッカ アマル
ダッカ アマル
エッフェンバーガー カースティン
エッフェンバーガー カースティン
ガッベタ ビジャヤラクシュミ
ガッベタ ビジャヤラクシュミ
リ ウェンチェング
リ ウェンチェング
ナリシュキン ニコライ
ナリシュキン ニコライ
トロッタ クリストファー
トロッタ クリストファー
ウィエディンガー カリ
ウィエディンガー カリ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
PTC Therapeutics Inc
Original Assignee
PTC 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 PTC Therapeutics Inc filed Critical PTC Therapeutics Inc
Publication of JP2019535789A publication Critical patent/JP2019535789A/ja
Publication of JP2019535789A5 publication Critical patent/JP2019535789A5/ja
Priority to JP2022108509A priority Critical patent/JP2022153413A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • 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
    • A61P11/02—Nasal agents, e.g. decongestants
    • 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
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • 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
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • 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
    • A61P13/08—Drugs for disorders of the urinary system of the prostate
    • 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
    • A61P13/10—Drugs for disorders of the urinary system of the bladder
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/24—Antidepressants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/04—Artificial tears; Irrigation solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/12—Ophthalmic agents for cataracts
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/02—Nutrients, e.g. vitamins, minerals
    • 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
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • A61P31/04—Antibacterial agents
    • 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
    • A61P31/10—Antimycotics
    • 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
    • A61P31/12—Antivirals
    • 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
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • 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
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A61P31/22—Antivirals for DNA viruses for herpes viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00—Antiparasitic agents
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • A61P37/02—Immunomodulators
    • 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
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • A61P37/08—Antiallergic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C255/00—Carboxylic acid nitriles
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C291/00—Compounds containing carbon and nitrogen and having functional groups not covered by groups C07C201/00 - C07C281/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Ophthalmology & Optometry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Hematology (AREA)
  • Pulmonology (AREA)
  • Urology & Nephrology (AREA)
  • Diabetes (AREA)
  • Pain & Pain Management (AREA)
JP2019528527A 2016-11-28 2017-11-27 Ｒｎａスプライシングを調節する方法 Pending JP2019535789A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022108509A JP2022153413A (ja) 2016-11-28 2022-07-05 Ｒｎａスプライシングを調節する方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662426619P 2016-11-28 2016-11-28
US62/426,619 2016-11-28
PCT/US2017/063323 WO2018098446A1 (en) 2016-11-28 2017-11-27 Methods for modulating rna splicing

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022108509A Division JP2022153413A (ja) 2016-11-28 2022-07-05 Ｒｎａスプライシングを調節する方法

Publications (2)

Publication Number Publication Date
JP2019535789A true JP2019535789A (ja) 2019-12-12
JP2019535789A5 JP2019535789A5 (cg-RX-API-DMAC7.html) 2021-06-17

Family

ID=62195368

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019528527A Pending JP2019535789A (ja) 2016-11-28 2017-11-27 Ｒｎａスプライシングを調節する方法
JP2022108509A Pending JP2022153413A (ja) 2016-11-28 2022-07-05 Ｒｎａスプライシングを調節する方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022108509A Pending JP2022153413A (ja) 2016-11-28 2022-07-05 Ｒｎａスプライシングを調節する方法

Country Status (9)

Country Link
US (2) US11702646B2 (cg-RX-API-DMAC7.html)
EP (1) EP3544435A4 (cg-RX-API-DMAC7.html)
JP (2) JP2019535789A (cg-RX-API-DMAC7.html)
CN (1) CN110352007A (cg-RX-API-DMAC7.html)
AU (1) AU2017363369A1 (cg-RX-API-DMAC7.html)
CA (1) CA3043755A1 (cg-RX-API-DMAC7.html)
EA (1) EA201991309A1 (cg-RX-API-DMAC7.html)
MX (1) MX2019005588A (cg-RX-API-DMAC7.html)
WO (1) WO2018098446A1 (cg-RX-API-DMAC7.html)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
CN110352007A (zh) 2016-11-28 2019-10-18 Ptc医疗公司 用于调节rna剪接的方法
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
IL272413B (en) 2017-08-04 2022-09-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
EP3833357A4 (en) * 2018-08-07 2022-06-08 The Children's Hospital of Philadelphia REGULATION OF GENE EXPRESSION BY ALTERNATIVE SPLICING, AND THERAPEUTIC METHODS
WO2020076892A1 (en) * 2018-10-09 2020-04-16 The University Of North Carolina At Chapel Hill Regulated gene editing system
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920928A4 (en) 2019-02-06 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
US20220307053A1 (en) 2019-07-25 2022-09-29 Novartis Ag Regulatable expression systems
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression
IL293340A (en) 2019-12-02 2022-07-01 Storm Therapeutics Ltd Polyheterocyclic compounds as mettl3 inhibitors
AU2021218807A1 (en) * 2020-02-12 2022-09-15 The Children's Hospital Of Philadelphia Compositions and methods for inducible alternative splicing regulation of gene expression
IL295955A (en) 2020-02-28 2022-10-01 Remix Therapeutics Inc Compounds and methods for modulating splicing
EP4110775A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
KR20220157407A (ko) 2020-02-28 2022-11-29 레믹스 테라퓨틱스 인크. 핵산 스플라이싱을 조절하기 위한 피리다진 유도체
WO2021174164A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN116134036A (zh) 2020-04-08 2023-05-16 雷密克斯医疗公司 用于调节剪接的化合物和方法
JP2023520916A (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整するための化合物及び方法
AU2021272972A1 (en) 2020-05-13 2022-12-08 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
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
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
WO2022214520A1 (en) * 2021-04-07 2022-10-13 Janssen Sciences Ireland Unlimited Company Antibacterial compounds
CN113267626B (zh) * 2021-05-17 2023-01-13 郑州大学第一附属医院 一种用于高危人群贲门腺癌早期筛查的试纸条
CN113604566B (zh) * 2021-07-13 2023-05-16 中山大学孙逸仙纪念医院 lncRNA BCYRN1在膀胱癌预后、治疗中的应用
US20240409536A1 (en) 2021-08-30 2024-12-12 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4395892A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240400584A1 (en) 2021-08-30 2024-12-05 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240368163A1 (en) 2021-08-30 2024-11-07 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023064879A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
US20250332173A1 (en) 2021-10-13 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
JP2024540477A (ja) 2021-11-17 2024-10-31 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 ハンチントン病を治療するためのｈｔｔモジュレータとしてのｎ－（２ｈ－インダゾール－５－イル）ピラジン－２－カルボキサミド誘導体及び類似化合物
WO2023133229A2 (en) 2022-01-05 2023-07-13 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
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
CN118647405A (zh) * 2022-01-25 2024-09-13 上海魁特迪生物科技有限公司 改善认知障碍的方法
EP4568962A2 (en) * 2022-08-09 2025-06-18 PTC Therapeutics, Inc. Methods for modulating rna splicing

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008539698A (ja) * 2005-04-29 2008-11-20 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 転写後レベルでの核酸発現調節のための方法および組成物
JP2015508075A (ja) * 2012-02-10 2015-03-16 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
WO2015105657A1 (en) * 2013-12-19 2015-07-16 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015173181A1 (en) * 2014-05-15 2015-11-19 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy

Family Cites Families (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3558618A (en) 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
US4122274A (en) 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
JPS56150091A (en) 1980-03-28 1981-11-20 Janssen Pharmaceutica Nv 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture
US4342870A (en) 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5599816A (en) 1990-05-02 1997-02-04 Abbott Laboratories Quinolizinone type compounds
AU4231293A (en) 1992-05-13 1993-12-13 E.I. Du Pont De Nemours And Company Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides
JPH11510478A (ja) 1995-06-06 1999-09-14 アボツト・ラボラトリーズ キノリジノン型化合物
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
EP1115724A1 (en) 1998-09-21 2001-07-18 Shire Biochem Inc. Quinolizinones as integrin inhibitors
DE60024861T2 (de) 1999-10-28 2006-07-06 Trine Pharmaceuticals, Inc., Waltham Pumpeninhibitoren zur freisetzung von medikamenten
CN1891699A (zh) 2000-01-24 2007-01-10 基纳西亚股份有限公司 用于治疗的吗啉代基取代的化合物
WO2002053576A1 (en) 2001-01-05 2002-07-11 The General Hospital Corporation Viral delivery system for infectious transfer of large genomic dna inserts
US20050009843A1 (en) 2001-04-26 2005-01-13 Kiyoshi Nakayama Medicine for inhibiting drug elimination pump
JP2005516005A (ja) 2001-12-07 2005-06-02 バーテクス ファーマスーティカルズ インコーポレイテッド Ｇｓｋ−３阻害剤として有用なピリミジンベースの化合物
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
EP1542667B1 (en) 2002-07-24 2012-03-07 PTC Therapeutics, Inc. Ureido substituted benzoic acid compounds and their use for nonsense suppression and the treatment of disease
US9068234B2 (en) 2003-01-21 2015-06-30 Ptc Therapeutics, Inc. Methods and agents for screening for compounds capable of modulating gene expression
ES2339862T3 (es) 2003-06-20 2010-05-26 Novartis Vaccines And Diagnostics, Inc. Compuestos de piridino 1,2-a-pirimidin-4-ona como agentes anticancerosos.
US20080255162A1 (en) 2004-05-04 2008-10-16 Warner-Lambert Company Llc Pyrrolyl Substituted Pyrido[2,3-D]Pyrimidin-7-Ones and Derivatives Thereof as Therapeutic Agents
CA2595477A1 (en) 2005-01-21 2006-07-27 Janssen Pharmaceutica N.V. Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
CA2612389A1 (en) * 2005-06-17 2006-12-28 Genizon Biosciences, Inc. Genemap of the human genes associated with longevity
EP1948803A4 (en) 2005-10-13 2009-11-25 Bc Cancer Agency MODULAR GENOME FOR SYNTHETIC BIOLOGY AND METABOLISM MANIPULATION
AR059339A1 (es) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
WO2008013997A2 (en) 2006-07-27 2008-01-31 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
EP2114903B1 (en) 2006-12-22 2011-01-26 Avexa Limited Bicyclic pyrimidinones and uses thereof
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
PL2212324T3 (pl) 2008-07-02 2014-11-28 Avexa Ltd Związki posiadające właściwości przeciwwirusowe
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
WO2010083338A2 (en) * 2009-01-14 2010-07-22 Philadelphia Health And Education Corporation Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
US20100303776A1 (en) 2009-04-16 2010-12-02 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of multiple nucleic acids
WO2011050245A1 (en) 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors
WO2011062853A1 (en) 2009-11-20 2011-05-26 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
BR112012017473A2 (pt) 2010-01-13 2019-09-24 Institut National De La Sante Et De La Rech Medicale Inserm Epst pirido(1,2-a) pirimidinas, seu uso, bem como composição farmacêutica compreendendo tais compostos
WO2012047487A1 (en) 2010-09-27 2012-04-12 Evolva Sa 2-pyridone antimicrobial compositions
WO2013059606A1 (en) 2011-10-21 2013-04-25 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CN104203239B (zh) 2012-01-26 2017-09-08 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
US9371336B2 (en) 2012-03-01 2016-06-21 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
MX358514B (es) 2012-03-23 2018-08-24 Ptc Therapeutics Inc Compuestos para tratar la atrofia muscular espinal.
US9212209B2 (en) 2012-07-13 2015-12-15 Indiana University Research And Technology Corporation Screening methods for spinal muscular atrophy
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
CA2915764A1 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
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
WO2016042015A1 (en) * 2014-09-16 2016-03-24 Centre National De La Recherche Scientifique (Cnrs) Method for evaluating developmental competence of an oocyte
EP3256126B1 (en) * 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
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
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
CN110352007A (zh) 2016-11-28 2019-10-18 Ptc医疗公司 用于调节rna剪接的方法
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
IL272413B (en) 2017-08-04 2022-09-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
WO2019060917A2 (en) 2017-09-25 2019-03-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR SCREENING AND IDENTIFYING SPREADING MODULATORS

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008539698A (ja) * 2005-04-29 2008-11-20 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 転写後レベルでの核酸発現調節のための方法および組成物
JP2015508075A (ja) * 2012-02-10 2015-03-16 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
WO2015105657A1 (en) * 2013-12-19 2015-07-16 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015173181A1 (en) * 2014-05-15 2015-11-19 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
MERCER T. R. ET AL., GENOME RESEARCH, vol. 25, JPN6021051633, 2015, pages 290 - 303, ISSN: 0004897912 *
NARYSHKIN N. A. ET AL., SCIENCE, vol. 345, JPN6021051622, 2014, pages 688 - 693, ISSN: 0004676233 *
PALACINO J. ET AL., NATURE CHEMICAL BIOLOGY, vol. 11, JPN6021051629, 2015, pages 511 - 517, ISSN: 0004897911 *
RATNI H. ET AL., J. MED. CHEM., vol. 59, JPN6021051625, 14 June 2016 (2016-06-14), pages 6086 - 6100, ISSN: 0004897913 *

Also Published As

Publication number Publication date
AU2017363369A1 (en) 2019-05-30
JP2022153413A (ja) 2022-10-12
US11702646B2 (en) 2023-07-18
EA201991309A1 (ru) 2019-11-29
MX2019005588A (es) 2019-10-15
US20230272367A1 (en) 2023-08-31
EP3544435A4 (en) 2020-11-04
US20190330615A1 (en) 2019-10-31
EP3544435A1 (en) 2019-10-02
WO2018098446A1 (en) 2018-05-31
CA3043755A1 (en) 2018-05-31
CN110352007A (zh) 2019-10-18

Similar Documents

Publication Publication Date Title
JP2022153413A (ja) Ｒｎａスプライシングを調節する方法
US20230310651A1 (en) Methods for modulating rna splicing
US10688099B2 (en) Methods for modulating the amount of RNA transcripts
JP6092897B2 (ja) 脊髄性筋萎縮症を治療するための化合物
CN104302181B (zh) 用于治疗脊髓性肌萎缩的化合物
US9399649B2 (en) Compounds for treating spinal muscular atrophy
WO2015095449A1 (en) Methods for modulating the amount rna transcripts
EA028382B1 (ru) Соединения для лечения спинальной мышечной атрофии
BR112014016443B1 (pt) Mistura de pó e método de fabricação de um componente sinterizado
WO2015095446A1 (en) Methods for modulating the amount of rna transcripts
HK40016425A (en) Methods for modulating rna splicing

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191002

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201125

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20201125

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210428

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220105

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220405

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220705

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20221018