JP7236707B2 - リピート不安定性と関連する疾患を治療する方法 - Google Patents

リピート不安定性と関連する疾患を治療する方法 Download PDF

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JP7236707B2
JP7236707B2 JP2019529315A JP2019529315A JP7236707B2 JP 7236707 B2 JP7236707 B2 JP 7236707B2 JP 2019529315 A JP2019529315 A JP 2019529315A JP 2019529315 A JP2019529315 A JP 2019529315A JP 7236707 B2 JP7236707 B2 JP 7236707B2
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cag
pharmaceutical composition
repeat
dna
repeats
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JP2019524160A5 (enExample
JP2019524160A (ja
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クリストファー イー ピアソン
雅之 中森
和彦 中谷
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Hospital for Sick Children HSC
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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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4375—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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
    • 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
    • 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
    • A61P25/28—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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Dermatology (AREA)
  • Dispersion Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2019529315A 2016-08-12 2017-08-12 リピート不安定性と関連する疾患を治療する方法 Active JP7236707B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662374072P 2016-08-12 2016-08-12
US62/374,072 2016-08-12
PCT/IB2017/054932 WO2018029660A1 (en) 2016-08-12 2017-08-12 Methods of treating diseases associated with repeat instability

Publications (3)

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JP2019524160A JP2019524160A (ja) 2019-09-05
JP2019524160A5 JP2019524160A5 (enExample) 2020-09-24
JP7236707B2 true JP7236707B2 (ja) 2023-03-10

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JP2019529315A Active JP7236707B2 (ja) 2016-08-12 2017-08-12 リピート不安定性と関連する疾患を治療する方法

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US (1) US11672786B2 (enExample)
EP (1) EP3496713B1 (enExample)
JP (1) JP7236707B2 (enExample)
CN (1) CN110381943A (enExample)
CA (1) CA3033590A1 (enExample)
WO (1) WO2018029660A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7084611B2 (ja) * 2018-04-06 2022-06-15 国立大学法人大阪大学 脊髄小脳変性症３１型抑制剤

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2049664T3 (da) 2006-08-11 2012-01-02 Prosensa Technologies Bv Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser
JP5866283B2 (ja) * 2009-07-28 2016-02-17 サンガモ バイオサイエンシーズ， インコーポレイテッド トリヌクレオチド反復疾患を治療するための方法および組成物

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
HAGIHARA M; ET AL，SMALL MOLECULE MODULATES HAIRPIN STRUCTURES IN CAG TRINUCLEOTIDE REPEATS，CHEMBIOCHEM，2011年，VOL:12，PAGE(S):1686 - 1689，http://dx.doi.org/10.1002/cbic.201100260
JINXING LI; ET AL，NAPHTHYRIDINE-BENZOAZAQUINOLONE: EVALUATION OF A TRICYCLIC SYSTEM FOR THE BINDING 以下備考，CHEMISTRY AN ASIAN JOURNAL，2016年07月05日，VOL:11, NR:13，PAGE(S):1971 - 1981，https://www.ncbi.nlm.nih.gov/pubmed/27146450，TO (CAG)N REPEAT DNA AND RNA
中谷和彦, 外３名, 「CAG リピート結合リガンドの発見に基づいたリピート伸長抑制」, ゲノム特定領域最終報告書 2005年度
阪田彬裕, 外７名, 「ＣＡＧリピートＤＮＡおよびＲＮＡを標的としたナフチリジン－アザキノロン誘導体の開発と転写および翻訳への阻害効果」, 日本薬学会第１３６年会要旨集(2016 March), 28S-am01S

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Publication number Publication date
CA3033590A1 (en) 2018-02-15
CN110381943A (zh) 2019-10-25
US20210283114A1 (en) 2021-09-16
JP2019524160A (ja) 2019-09-05
US11672786B2 (en) 2023-06-13
EP3496713A1 (en) 2019-06-19
WO2018029660A1 (en) 2018-02-15
EP3496713B1 (en) 2024-03-13

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