BR112012019515A2 - indutor de continuação de leitura, e agente terapêutico para doenças genéticas do tipo de mutações anti-senso - Google Patents

indutor de continuação de leitura, e agente terapêutico para doenças genéticas do tipo de mutações anti-senso

Info

Publication number
BR112012019515A2
BR112012019515A2 BR112012019515-8A BR112012019515A BR112012019515A2 BR 112012019515 A2 BR112012019515 A2 BR 112012019515A2 BR 112012019515 A BR112012019515 A BR 112012019515A BR 112012019515 A2 BR112012019515 A2 BR 112012019515A2
Authority
BR
Brazil
Prior art keywords
continuation
inducer
type
therapeutic agent
antisense mutations
Prior art date
Application number
BR112012019515-8A
Other languages
English (en)
Japanese (ja)
Inventor
Matsuda Ryoichi
Shiozuka Masataka
Wagatsuma Akira
Takahashi Yoshikazu
Ikeda Daishiro
Matsuo Masafumi
Nishida Atsushi
Nonomura Yoshiaki
Original Assignee
The University Of Tokyo
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 The University Of Tokyo filed Critical The University Of Tokyo
Publication of BR112012019515A2 publication Critical patent/BR112012019515A2/pt

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    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
    • 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
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • 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
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • A61P25/14—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
    • A61P25/28—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
    • 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
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • A61P9/02—Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
    • 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
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/20—Carbocyclic rings
    • C07H15/22—Cyclohexane rings, substituted by nitrogen atoms
    • C07H15/222—Cyclohexane rings substituted by at least two nitrogen atoms
    • C07H15/226—Cyclohexane rings substituted by at least two nitrogen atoms with at least two saccharide radicals directly attached to the cyclohexane rings
    • C07H15/234—Cyclohexane rings substituted by at least two nitrogen atoms with at least two saccharide radicals directly attached to the cyclohexane rings attached to non-adjacent ring carbon atoms of the cyclohexane rings, e.g. kanamycins, tobramycin, nebramycin, gentamicin A2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • Cardiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Obesity (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
  • Rheumatology (AREA)
  • Dermatology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Psychology (AREA)
  • Vascular Medicine (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)

Abstract

indutor de continuação de leitura, e agente terapêutico para doenças genéticas do tipo de mutações anti-senso. agente de indução de continuação da leitura para induzir continuação da leitura de um códon de terminação pré-maturo gerado por mutações anti-senso, o agente de indução de continuação da leitura compreendendo um composto tendo uma estrutura expressa pela seguinte fórmula estrutural (a), e uma droga para tratar uma doença genética causada por mutações anti-senso, a droga incluindo o agente de indução de continuação da leitura.
BR112012019515-8A 2010-02-03 2011-02-03 indutor de continuação de leitura, e agente terapêutico para doenças genéticas do tipo de mutações anti-senso BR112012019515A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2010021817 2010-02-03
JP2010-021817 2010-02-03
PCT/JP2011/052263 WO2011096484A1 (ja) 2010-02-03 2011-02-03 リードスルー誘導剤、及びナンセンス変異型遺伝性疾患治療薬

Publications (1)

Publication Number Publication Date
BR112012019515A2 true BR112012019515A2 (pt) 2018-03-13

Family

ID=44355477

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112012019515-8A BR112012019515A2 (pt) 2010-02-03 2011-02-03 indutor de continuação de leitura, e agente terapêutico para doenças genéticas do tipo de mutações anti-senso

Country Status (12)

Country Link
US (2) US20130005953A1 (pt)
EP (1) EP2535051B1 (pt)
JP (1) JP5705136B2 (pt)
KR (1) KR20120123509A (pt)
CN (1) CN102740860B (pt)
AU (1) AU2011211744B2 (pt)
BR (1) BR112012019515A2 (pt)
CA (1) CA2788730A1 (pt)
IL (1) IL221251A0 (pt)
MX (1) MX2012009028A (pt)
RU (1) RU2571060C2 (pt)
WO (1) WO2011096484A1 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101703137B1 (ko) 2014-05-30 2017-02-07 선문대학교 산학협력단 당 부가 아미노글리코사이드 화합물, 이의 제조방법 및 이를 유효성분으로 함유하는 약학적 조성물
CN108014110A (zh) * 2016-11-03 2018-05-11 武汉科技大学 灭瘟素的无义突变通读活性用途
US20180315708A1 (en) * 2017-05-01 2018-11-01 Globalfoundries Inc. Power rail and mol constructs for fdsoi
EP3688837A4 (en) * 2017-09-25 2021-06-02 Antwave Intellectual Property Limited SYSTEMS, DEVICES, AND METHODS FOR IMPROVING ANTENNA PERFORMANCE IN ELECTRONIC DEVICES
JP6953053B1 (ja) 2020-03-11 2021-10-27 隆光 矢野 −１フレームシフトを誘導するための１本鎖核酸分子及び組成物
KR102517522B1 (ko) 2020-12-28 2023-04-03 고려대학교 산학협력단 2-데옥시-스킬로이노사민을 포함하는 아미노사이클리톨 화합물, 이의 제조방법 및 이를 함유하는 약학 조성물

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US5488038A (en) * 1992-11-27 1996-01-30 Zaidan Hojin Biseibutsu Kagaku Kenkyu Kai Dibekacin derivatives and arbekacin derivatives active against resistant bacteria
CA2438750A1 (en) 2001-02-20 2002-08-29 Uab Research Foundation Aminoglycoside treatment for lysosomal storage diseases
EP1402889A1 (en) 2001-06-13 2004-03-31 Masayuki Arakawa Remedies for diseases caused by nonsense mutation
GB0600948D0 (en) 2006-01-18 2006-02-22 Univ Dundee Prevention/Treatment Of Ichthyosis Vulgaris, Atopy And Other Disorders
PT2390255T (pt) * 2006-04-03 2016-10-24 Technion Res & Dev Foundation Novos aminoglicosídeos e utilizações destes no tratamento de distúrbios genéticos
CN1843330A (zh) * 2006-05-24 2006-10-11 山东蓝金生物工程有限公司 一种含氨基糖苷类抗菌素的缓释剂及其应用
RU2316590C1 (ru) * 2006-06-16 2008-02-10 Институт биоорганической химии им. академиков М.М. Шемякина и Ю.А. Овчинникова Российской академии наук РЕКОМБИНАНТНАЯ ПЛАЗМИДНАЯ ДНК pE-His8-TrxL-Ar2, КОДИРУЮЩАЯ ГИБРИДНЫЙ БЕЛОК, СОДЕРЖАЩИЙ АНТИМИКРОБНЫЙ ПЕПТИД АРЕНИЦИН МОРСКОГО КОЛЬЧАТОГО ЧЕРВЯ Arenicola marina, И ШТАММ Escherichia coli BL21(DE3)/pE-His8-TrxL-Ar2 - ПРОДУЦЕНТ ГИБРИДНОГО БЕЛКА, СОДЕРЖАЩЕГО АРЕНИЦИН
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JP5061345B2 (ja) * 2006-09-26 2012-10-31 国立大学法人山口大学 薬剤耐性病原性微生物の薬剤感受性増強方法、および高度薬剤耐性菌の出現予防方法
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Also Published As

Publication number Publication date
MX2012009028A (es) 2012-10-15
AU2011211744B2 (en) 2015-06-18
CA2788730A1 (en) 2011-08-11
AU2011211744A1 (en) 2012-08-23
CN102740860A (zh) 2012-10-17
JPWO2011096484A1 (ja) 2013-06-13
IL221251A0 (en) 2012-10-31
US9358246B2 (en) 2016-06-07
RU2571060C2 (ru) 2015-12-20
CN102740860B (zh) 2014-11-19
RU2012137245A (ru) 2014-03-10
US20150005249A1 (en) 2015-01-01
US20130005953A1 (en) 2013-01-03
EP2535051A4 (en) 2013-09-11
EP2535051B1 (en) 2016-04-20
JP5705136B2 (ja) 2015-04-22
WO2011096484A1 (ja) 2011-08-11
EP2535051A1 (en) 2012-12-19
KR20120123509A (ko) 2012-11-08

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