AU2020241693B2 - Compounds and methods for reducing KCNT1 expression - Google Patents

Compounds and methods for reducing KCNT1 expression Download PDF

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Publication number
AU2020241693B2
AU2020241693B2 AU2020241693A AU2020241693A AU2020241693B2 AU 2020241693 B2 AU2020241693 B2 AU 2020241693B2 AU 2020241693 A AU2020241693 A AU 2020241693A AU 2020241693 A AU2020241693 A AU 2020241693A AU 2020241693 B2 AU2020241693 B2 AU 2020241693B2
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Australia
Prior art keywords
certain embodiments
modified
modified oligonucleotide
seq
kcnt1
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Ceased
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AU2020241693A
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AU2020241693A1 (en
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Huynh-Hoa Bui
Susan M. Freier
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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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    • C12N15/11—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
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  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Biochemistry (AREA)
  • Microbiology (AREA)
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  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2020241693A 2019-03-15 2020-03-13 Compounds and methods for reducing KCNT1 expression Ceased AU2020241693B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962819344P 2019-03-15 2019-03-15
US62/819,344 2019-03-15
US201962884501P 2019-08-08 2019-08-08
US62/884,501 2019-08-08
PCT/US2020/022680 WO2020190740A1 (en) 2019-03-15 2020-03-13 Compounds and methods for reducing kcnt1 expression

Publications (2)

Publication Number Publication Date
AU2020241693A1 AU2020241693A1 (en) 2021-09-02
AU2020241693B2 true AU2020241693B2 (en) 2024-01-04

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AU2020241693A Ceased AU2020241693B2 (en) 2019-03-15 2020-03-13 Compounds and methods for reducing KCNT1 expression

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US (2) US20220177893A1 (https=)
EP (1) EP3938514A4 (https=)
JP (2) JP7564817B2 (https=)
KR (1) KR20210141983A (https=)
CN (3) CN121674396A (https=)
AU (1) AU2020241693B2 (https=)
BR (1) BR112021015494A2 (https=)
CA (1) CA3133247A1 (https=)
CL (1) CL2021002398A1 (https=)
CO (1) CO2021013371A2 (https=)
CR (1) CR20210519A (https=)
IL (1) IL285546A (https=)
JO (1) JOP20210254A1 (https=)
MX (1) MX2021011132A (https=)
PE (1) PE20220168A1 (https=)
PH (1) PH12021552220A1 (https=)
SG (1) SG11202108625WA (https=)
TW (1) TW202102675A (https=)
WO (1) WO2020190740A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10952978B2 (en) 2017-08-28 2021-03-23 Enanta Pharmaceuticals, Inc. Hepatitis B antiviral agents
US11198693B2 (en) 2018-11-21 2021-12-14 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
MX2024006709A (es) * 2021-12-01 2024-08-19 Atalanta Therapeutics Inc Composiciones y metodos para el tratamiento de epilepsias.
WO2025036916A1 (en) * 2023-08-16 2025-02-20 Les Laboratoires Servier Oligonucleotides for modulating kcnt1 expression

Citations (2)

* 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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3260540A1 (en) * 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
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

Patent Citations (2)

* 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

Also Published As

Publication number Publication date
CL2021002398A1 (es) 2022-06-03
MX2021011132A (es) 2021-10-14
PH12021552220A1 (en) 2022-05-30
WO2020190740A1 (en) 2020-09-24
EP3938514A4 (en) 2023-05-03
CA3133247A1 (en) 2020-09-24
IL285546A (en) 2021-09-30
CR20210519A (es) 2021-11-24
EP3938514A1 (en) 2022-01-19
CN113661241A (zh) 2021-11-16
KR20210141983A (ko) 2021-11-23
US20250320505A1 (en) 2025-10-16
CN121674396A (zh) 2026-03-17
AU2020241693A1 (en) 2021-09-02
US20220177893A1 (en) 2022-06-09
JP7564817B2 (ja) 2024-10-09
SG11202108625WA (en) 2021-09-29
PE20220168A1 (es) 2022-01-28
JOP20210254A1 (ar) 2023-01-30
TW202102675A (zh) 2021-01-16
CO2021013371A2 (es) 2021-10-20
BR112021015494A2 (pt) 2021-10-05
JP2025000805A (ja) 2025-01-07
JP2022526267A (ja) 2022-05-24
CN117106778A (zh) 2023-11-24

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