JOP20210254A1 - مركبات وطرق لتقليل التعبير الوراثي عن kcnt1 - Google Patents

مركبات وطرق لتقليل التعبير الوراثي عن kcnt1

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Publication number
JOP20210254A1
JOP20210254A1 JOP/2021/0254A JOP20210254A JOP20210254A1 JO P20210254 A1 JOP20210254 A1 JO P20210254A1 JO P20210254 A JOP20210254 A JO P20210254A JO P20210254 A1 JOP20210254 A1 JO P20210254A1
Authority
JO
Jordan
Prior art keywords
compounds
methods
kcnt1
reducing
pharmaceutical compositions
Prior art date
Application number
JOP/2021/0254A
Other languages
English (en)
Inventor
Huynh-Hoa Bui
Susan M Freier
Original Assignee
Ionis Pharmaceuticals 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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JOP20210254A1 publication Critical patent/JOP20210254A1/ar

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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
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    • C12N2320/30—Special therapeutic applications
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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Pain & Pain Management (AREA)
  • Neurology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Biophysics (AREA)
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  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

يتم توفير مركبات، طرق، وتركيبات صيدلية لتقليل مقدار أو نشاط KCNT1 RNA في خلية أو خاضع للعلاج، وفي حالات معينة تقليل مقدار بروتين KCNT1 في خلية أو خاضع للعلاج. تعتبر هذه المركبات، الطرق، والتركيبات الصيدلية مفيدة لتحسين عرض واحد على الأقل أو سمة مميزة واحدة على الأقل لحالة عصبية. وتتضمن هذه الأعراض والسمات المميزة نوبات، اعتلال الدماغ، وحالات الشذوذ السلوكي. وتتمثل الأمثلة غير الحصرية على الحالات العصبية التي تستفيد من هذه المركبات، الطرق، والتركيبات الصيدلية في الصرع لدى الرضع مع هجرة النوبات البؤرية (EIMFS)، صرع الفص الجبهي الليلي ذي الصبغة الجسدية السائدة (ADNFLE)، والبداية المبكرة لحالات اعتلال الدماغ الناتجة عن الصرع بما في ذلك متلازمة West ومتلازمة Ohtahara.
JOP/2021/0254A 2019-03-15 2020-03-13 مركبات وطرق لتقليل التعبير الوراثي عن kcnt1 JOP20210254A1 (ar)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962819344P 2019-03-15 2019-03-15
US201962884501P 2019-08-08 2019-08-08
PCT/US2020/022680 WO2020190740A1 (en) 2019-03-15 2020-03-13 Compounds and methods for reducing kcnt1 expression

Publications (1)

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JOP20210254A1 true JOP20210254A1 (ar) 2023-01-30

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US (1) US20220177893A1 (ar)
EP (1) EP3938514A4 (ar)
JP (1) JP2022526267A (ar)
KR (1) KR20210141983A (ar)
CN (2) CN113661241A (ar)
AU (1) AU2020241693B2 (ar)
BR (1) BR112021015494A2 (ar)
CA (1) CA3133247A1 (ar)
CL (1) CL2021002398A1 (ar)
CO (1) CO2021013371A2 (ar)
CR (1) CR20210519A (ar)
IL (1) IL285546A (ar)
JO (1) JOP20210254A1 (ar)
MX (1) MX2021011132A (ar)
PE (1) PE20220168A1 (ar)
SG (1) SG11202108625WA (ar)
TW (1) TW202102675A (ar)
WO (1) WO2020190740A1 (ar)

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WO2020106816A1 (en) 2018-11-21 2020-05-28 Enanta Pharmaceuticals, Inc. Functionalized heterocycles as antiviral agents
WO2021188414A1 (en) 2020-03-16 2021-09-23 Enanta Pharmaceuticals, Inc. Functionalized heterocyclic compounds as antiviral agents
WO2023102490A1 (en) * 2021-12-01 2023-06-08 Atalanta Therapeutics, Inc. Compositions and methods for treatment of epilepsies

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050288242A1 (en) * 2001-05-18 2005-12-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of RAS gene expression using short interfering nucleic acid (siNA)
US7601501B2 (en) * 2006-08-11 2009-10-13 The Scripps Research Institute Controlling osteogenesis by inhibition of osteogenic suppressors
JP6336755B2 (ja) * 2010-11-12 2018-06-06 ザ ジェネラル ホスピタル コーポレイション ポリコームに関連する非コードｒｎａ
US20200129538A1 (en) * 2017-06-13 2020-04-30 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1

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Publication number Publication date
MX2021011132A (es) 2021-10-14
JP2022526267A (ja) 2022-05-24
BR112021015494A2 (pt) 2021-10-05
EP3938514A1 (en) 2022-01-19
CO2021013371A2 (es) 2021-10-20
CL2021002398A1 (es) 2022-06-03
SG11202108625WA (en) 2021-09-29
PE20220168A1 (es) 2022-01-28
AU2020241693A1 (en) 2021-09-02
US20220177893A1 (en) 2022-06-09
EP3938514A4 (en) 2023-05-03
CN113661241A (zh) 2021-11-16
IL285546A (en) 2021-09-30
CR20210519A (es) 2021-11-24
CN117106778A (zh) 2023-11-24
TW202102675A (zh) 2021-01-16
KR20210141983A (ko) 2021-11-23
AU2020241693B2 (en) 2024-01-04
WO2020190740A1 (en) 2020-09-24
CA3133247A1 (en) 2020-09-24

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