CN110352007A - 用于调节rna剪接的方法 - Google Patents

用于调节rna剪接的方法 Download PDF

Info

Publication number
CN110352007A
CN110352007A CN201780084649.8A CN201780084649A CN110352007A CN 110352007 A CN110352007 A CN 110352007A CN 201780084649 A CN201780084649 A CN 201780084649A CN 110352007 A CN110352007 A CN 110352007A
Authority
CN
China
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
CN201780084649.8A
Other languages
English (en)
Chinese (zh)
Inventor
阿努拉哈·巴特查里亚
阿迈尔·达卡
克斯廷·埃芬贝格尔
维加亚拉克什米·加贝塔
李文成
尼柯莱·纳雷什金
克里斯多佛·特罗塔
卡利·韦丁格
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 CN110352007A publication Critical patent/CN110352007A/zh
Pending legal-status Critical Current

Links

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
    • 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
    • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Communicable Diseases (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Pain & Pain Management (AREA)
  • Pulmonology (AREA)
CN201780084649.8A 2016-11-28 2017-11-27 用于调节rna剪接的方法 Pending CN110352007A (zh)

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

Publications (1)

Publication Number Publication Date
CN110352007A true CN110352007A (zh) 2019-10-18

Family

ID=62195368

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780084649.8A Pending CN110352007A (zh) 2016-11-28 2017-11-27 用于调节rna剪接的方法

Country Status (9)

Country Link
US (2) US11702646B2 (enExample)
EP (1) EP3544435A4 (enExample)
JP (2) JP2019535789A (enExample)
CN (1) CN110352007A (enExample)
AU (1) AU2017363369A1 (enExample)
CA (1) CA3043755A1 (enExample)
EA (1) EA201991309A1 (enExample)
MX (1) MX2019005588A (enExample)
WO (1) WO2018098446A1 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113604566A (zh) * 2021-07-13 2021-11-05 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
CN115362255A (zh) * 2020-02-04 2022-11-18 雷杰纳荣制药公司 用无七之子2(sos2)抑制剂治疗眼科疾患
WO2023143425A1 (zh) * 2022-01-25 2023-08-03 上海魁特迪生物科技有限公司 改善认知障碍的方法
CN122104811A (zh) * 2026-04-13 2026-05-29 四川大学华西第二医院 一种基于hapln1下调诱导胎盘细胞衰老异常的子痫前期动物模型构建方法及应用

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3310169B1 (en) 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
JP2019535789A (ja) 2016-11-28 2019-12-12 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを調節する方法
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods
AU2019359276A1 (en) * 2018-10-09 2021-04-29 The University Of North Carolina At Chapel Hill Regulated gene editing system
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163541A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
KR102913697B1 (ko) 2019-02-05 2026-01-15 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
JP7603594B2 (ja) 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
KR102925385B1 (ko) 2019-02-06 2026-02-11 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN114174514A (zh) * 2019-07-25 2022-03-11 诺华股份有限公司 可调节的表达系统
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
US12195458B2 (en) 2019-12-02 2025-01-14 Storm Therapeutics Limited Polyheterocyclic compounds as METTL3 inhibitors
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法
AU2021228285A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Compounds and methods for modulating splicing
AU2021228284A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
CA3169667A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Pyridazine derivatives for modulating nucleic acid splicing
CA3174353A1 (en) 2020-04-08 2021-10-14 Dominic Reynolds Compounds and methods for modulating splicing
MX2022012677A (es) 2020-04-08 2023-01-11 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
WO2021231571A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
MX2023000168A (es) 2020-07-02 2023-04-20 Remix Therapeutics Inc Derivados de 2-(indazol-5-il)-6-(piperidin-4-il)-1,7-naftiridina y compuestos relacionados como moduladores para el corte y empalme de ácidos nucleicos y para el tratamiento de enfermedades proliferativas.
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
EP4185568A1 (en) 2020-07-24 2023-05-31 The University of Rochester Inhibitors of interleukin-1 receptor-associated kinases 1 and 4
WO2022214520A1 (en) * 2021-04-07 2022-10-13 Janssen Sciences Ireland Unlimited Company Antibacterial compounds
CN116087519B (zh) * 2021-05-17 2025-03-14 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标志物及其应用
MX2024002554A (es) 2021-08-30 2024-07-02 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
IL311135A (en) 2021-08-30 2024-04-01 Remix Therapeutics Inc Compounds and methods for modulating splicing
US20250333397A1 (en) 2021-08-30 2025-10-30 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
US20250332173A1 (en) 2021-10-13 2025-10-30 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
TW202330552A (zh) 2021-10-13 2023-08-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
US20250326748A1 (en) 2022-01-05 2025-10-23 Remix Therapeutics Inc. Compounds and methods for modulating 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
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4568962A2 (en) * 2022-08-09 2025-06-18 PTC Therapeutics, Inc. Methods for modulating rna splicing

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090305900A1 (en) * 2005-06-17 2009-12-10 Abdelmajid Belouchi Genemap of the human genes associated with longevity
US20140249210A1 (en) * 2009-01-14 2014-09-04 Philadelphia Health & Education Corporation D/B/A Drexel University College Of Medicine Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
US20150005289A1 (en) * 2012-02-10 2015-01-01 Ptc Therapeutics Inc. Compounds for treating spinal muscular atrophy
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
WO2016042015A1 (en) * 2014-09-16 2016-03-24 Centre National De La Recherche Scientifique (Cnrs) Method for evaluating developmental competence of an oocyte

Family Cites Families (54)

* 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
EP0640083A1 (en) 1992-05-13 1995-03-01 E.I. Du Pont De Nemours And Company Substituted pyrido 1,2-a]pyrimidinone derivatives as fungicides
KR19990022524A (ko) 1995-06-06 1999-03-25 웨인스톡 스티븐 에프. 퀴놀리지논형화합물
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
HK1040076A1 (zh) 1998-09-21 2002-05-24 希雷生物化学有限公司 作为整联蛋白抑制剂的喹嗪酮
EP1227084B1 (en) 1999-10-28 2005-12-14 Trine Pharmaceuticals, Inc. Drug discharge pump inhibitors
ATE363471T1 (de) 2000-01-24 2007-06-15 Astrazeneca Ab Durch einen morpholinrest substituierte therapeutische verbindungen
WO2002053576A1 (en) 2001-01-05 2002-07-11 The General Hospital Corporation Viral delivery system for infectious transfer of large genomic dna inserts
JPWO2002087589A1 (ja) 2001-04-26 2004-08-12 第一製薬株式会社 薬剤排出ポンプ阻害薬
ATE466581T1 (de) 2001-12-07 2010-05-15 Vertex Pharma Verbindungen auf pyrimidin-basis als gsk-3-hemmer
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.
WO2005105801A1 (en) 2004-05-04 2005-11-10 Warner-Lambert Company Llc Pyrrolyl substituted pyrido[2,3-d]pyrimidin-7-ones and derivatives thereof as therapeutic agents
US7399767B2 (en) 2005-01-21 2008-07-15 Ortho-Mcneil Pharmaceutical, Inc. Heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors
AU2006242371A1 (en) * 2005-04-29 2006-11-09 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of nucleic acid at post-transcriptional level
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
US7696335B2 (en) 2005-10-13 2010-04-13 Bc Cancer Agency Kits for multiple non-cross reacting recombination reactions utilizing loxP sequences
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
US8337941B2 (en) 2006-07-27 2012-12-25 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
AR064517A1 (es) 2006-12-22 2009-04-08 Avexa Ltd Pirimidinionas biciclicas y sus usos
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
US8318732B2 (en) 2008-07-02 2012-11-27 Avexa Limited Compounds having antiviral properties
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
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
EP2501231B1 (en) 2009-11-20 2016-12-21 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
KR20130088002A (ko) 2010-01-13 2013-08-07 재단법인 한국파스퇴르연구소 항-감염성 피리도(1,2-ａ)피리미딘
EP3034078A1 (en) 2010-09-27 2016-06-22 Emergent Product Development Gaithersburg Inc. 2-pyridone antimicrobial compositions
US9662314B2 (en) 2011-10-21 2017-05-30 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
MX352861B (es) 2011-12-30 2017-12-13 Ptc Therapeutics Inc Compuestos para tratar la atrofia muscular espinal.
MX357834B (es) 2012-01-26 2018-07-26 Ptc Therapeutics Inc Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal.
US9371336B2 (en) 2012-03-01 2016-06-21 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
EP2828247B1 (en) 2012-03-23 2019-01-16 PTC Therapeutics, Inc. 4h-chromen-4-one derivatives for treating spinal muscular atrophy
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
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP3256126B1 (en) 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
EP3298017B1 (en) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Compounds for treating spinal muscular atrophy
EP3310169B1 (en) 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
JP2019535789A (ja) 2016-11-28 2019-12-12 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを調節する方法
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
US20230152257A1 (en) 2017-09-25 2023-05-18 Skyhawk Therapeutics, Inc. Methods and compositions for screening and identification of splicing

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090305900A1 (en) * 2005-06-17 2009-12-10 Abdelmajid Belouchi Genemap of the human genes associated with longevity
US20140249210A1 (en) * 2009-01-14 2014-09-04 Philadelphia Health & Education Corporation D/B/A Drexel University College Of Medicine Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
US20150005289A1 (en) * 2012-02-10 2015-01-01 Ptc Therapeutics Inc. Compounds for treating spinal muscular atrophy
CN104349777A (zh) * 2012-02-10 2015-02-11 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
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
WO2016042015A1 (en) * 2014-09-16 2016-03-24 Centre National De La Recherche Scientifique (Cnrs) Method for evaluating developmental competence of an oocyte

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JAMES PALACINO等: "SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice", 《NAT CHEM BIOL》 *
NIKOLAI A. NARYSHKIN等: "SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy", 《SCIENCE》 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115362255A (zh) * 2020-02-04 2022-11-18 雷杰纳荣制药公司 用无七之子2(sos2)抑制剂治疗眼科疾患
CN113604566A (zh) * 2021-07-13 2021-11-05 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
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
WO2023143425A1 (zh) * 2022-01-25 2023-08-03 上海魁特迪生物科技有限公司 改善认知障碍的方法
CN122104811A (zh) * 2026-04-13 2026-05-29 四川大学华西第二医院 一种基于hapln1下调诱导胎盘细胞衰老异常的子痫前期动物模型构建方法及应用

Also Published As

Publication number Publication date
CA3043755A1 (en) 2018-05-31
WO2018098446A1 (en) 2018-05-31
US11702646B2 (en) 2023-07-18
EP3544435A4 (en) 2020-11-04
US20190330615A1 (en) 2019-10-31
EP3544435A1 (en) 2019-10-02
MX2019005588A (es) 2019-10-15
JP2022153413A (ja) 2022-10-12
EA201991309A1 (ru) 2019-11-29
JP2019535789A (ja) 2019-12-12
AU2017363369A1 (en) 2019-05-30
US20230272367A1 (en) 2023-08-31

Similar Documents

Publication Publication Date Title
US20230310651A1 (en) Methods for modulating rna splicing
JP2022153413A (ja) Ｒｎａスプライシングを調節する方法
US10688099B2 (en) Methods for modulating the amount of RNA transcripts
US10851101B2 (en) Compounds for treating spinal muscular atrophy
CN104302181B (zh) 用于治疗脊髓性肌萎缩的化合物
CN104470909B (zh) 用于治疗脊髓性肌萎缩的化合物
WO2015095449A1 (en) Methods for modulating the amount rna transcripts
WO2015095446A1 (en) Methods for modulating the amount of rna transcripts
BR112014016443B1 (pt) Mistura de pó e método de fabricação de um componente sinterizado
HK40016425A (en) Methods for modulating rna splicing

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40016425

Country of ref document: HK

AD01 Patent right deemed abandoned

Effective date of abandoning: 20231103

AD01 Patent right deemed abandoned
REG Reference to a national code

Ref country code: HK

Ref legal event code: WD

Ref document number: 40016425

Country of ref document: HK