CN113661241A - 用于降低kcnt1表达的化合物和方法 - Google Patents

用于降低kcnt1表达的化合物和方法 Download PDF

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Publication number
CN113661241A
CN113661241A CN202080020884.0A CN202080020884A CN113661241A CN 113661241 A CN113661241 A CN 113661241A CN 202080020884 A CN202080020884 A CN 202080020884A CN 113661241 A CN113661241 A CN 113661241A
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Prior art keywords
nucleobase
seq
oligomeric compound
modified
equivalent length
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CN202080020884.0A
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Chinese (zh)
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H-H·布维
苏珊·M·弗赖尔
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Priority to CN202311150264.0A priority Critical patent/CN117106778A/zh
Priority to CN202511762645.3A priority patent/CN121674396A/zh
Publication of CN113661241A publication Critical patent/CN113661241A/zh
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    • 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
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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
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  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CN202080020884.0A 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法 Pending CN113661241A (zh)

Priority Applications (2)

Application Number Priority Date Filing Date Title
CN202311150264.0A CN117106778A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法
CN202511762645.3A CN121674396A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法

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

Related Child Applications (2)

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CN202511762645.3A Division CN121674396A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法
CN202311150264.0A Division CN117106778A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法

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CN113661241A true CN113661241A (zh) 2021-11-16

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CN202080020884.0A Pending CN113661241A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法
CN202511762645.3A Pending CN121674396A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法
CN202311150264.0A Pending CN117106778A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法

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CN202511762645.3A Pending CN121674396A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法
CN202311150264.0A Pending CN117106778A (zh) 2019-03-15 2020-03-13 用于降低kcnt1表达的化合物和方法

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US (2) US20220177893A1 (https=)
EP (1) EP3938514A4 (https=)
JP (2) JP7564817B2 (https=)
KR (1) KR20210141983A (https=)
CN (3) CN113661241A (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)

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Publication number Priority date Publication date Assignee Title
SG11202001685TA (en) 2017-08-28 2020-03-30 Enanta Pharm Inc Hepatitis b antiviral agents
IL283190B2 (en) 2018-11-21 2025-08-01 Enanta Pharm Inc Functionalized heterocycles as antiviral agents
WO2021188414A1 (en) 2020-03-16 2021-09-23 Enanta Pharmaceuticals, Inc. Functionalized heterocyclic compounds as antiviral agents
EP4441229A1 (en) * 2021-12-01 2024-10-09 Atalanta Therapeutics, Inc. Compositions and methods for treatment of epilepsies
IL326478A (en) * 2023-08-16 2026-04-01 Servier Lab Oligonucleotides for modulation of KCNT1 expression

Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2018227247A1 (en) * 2017-06-13 2018-12-20 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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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
CA2817256A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2018227247A1 (en) * 2017-06-13 2018-12-20 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1

Non-Patent Citations (2)

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Title
FEN HUANG ET AL: "TMEM16C facilitates Na+-activatedd K+ currents in rat sensory neurons and regulates pain processing", NATURE NEUROSCIENCE, vol. 16, no. 9, 21 July 2013 (2013-07-21), pages 1284 - 1290, XP055567842, DOI: 10.1038/nn.3468 *
ZHANG Y. ET AL: "Regulation of neuronal excitablitiy by interaction of fraile x mental retardation protein with slack potassium channels", THE JOURNAL OF NEUROSCIENCE, vol. 32, no. 44, 31 December 2012 (2012-12-31), pages 15318 - 15327 *

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Publication number Publication date
WO2020190740A1 (en) 2020-09-24
CR20210519A (es) 2021-11-24
CO2021013371A2 (es) 2021-10-20
CN117106778A (zh) 2023-11-24
SG11202108625WA (en) 2021-09-29
CN121674396A (zh) 2026-03-17
TW202102675A (zh) 2021-01-16
JP7564817B2 (ja) 2024-10-09
JP2022526267A (ja) 2022-05-24
AU2020241693B2 (en) 2024-01-04
BR112021015494A2 (pt) 2021-10-05
US20250320505A1 (en) 2025-10-16
CL2021002398A1 (es) 2022-06-03
JP2025000805A (ja) 2025-01-07
CA3133247A1 (en) 2020-09-24
PH12021552220A1 (en) 2022-05-30
MX2021011132A (es) 2021-10-14
US20220177893A1 (en) 2022-06-09
AU2020241693A1 (en) 2021-09-02
EP3938514A4 (en) 2023-05-03
EP3938514A1 (en) 2022-01-19
PE20220168A1 (es) 2022-01-28
IL285546A (en) 2021-09-30
KR20210141983A (ko) 2021-11-23
JOP20210254A1 (ar) 2023-01-30

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