EA202192527A1 - COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION - Google Patents

COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION

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Publication number
EA202192527A1
EA202192527A1 EA202192527A EA202192527A EA202192527A1 EA 202192527 A1 EA202192527 A1 EA 202192527A1 EA 202192527 A EA202192527 A EA 202192527A EA 202192527 A EA202192527 A EA 202192527A EA 202192527 A1 EA202192527 A1 EA 202192527A1
Authority
EA
Eurasian Patent Office
Prior art keywords
compounds
methods
kcnt1
reducing
pharmaceutical compositions
Prior art date
Application number
EA202192527A
Other languages
Russian (ru)
Inventor
Хуинх-Хоа Буи
Сьюзан М. Фрейер
Original Assignee
Ионис Фармасьютикалз, Инк.
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 Ионис Фармасьютикалз, Инк. filed Critical Ионис Фармасьютикалз, Инк.
Priority claimed from PCT/US2020/022680 external-priority patent/WO2020190740A1/en
Publication of EA202192527A1 publication Critical patent/EA202192527A1/en

Links

Abstract

Предложены соединения, способы и фармацевтические композиции для снижения количества или активности РНК KCNT1 в клетке или у субъекта и в некоторых случаях для снижения количества белка KCNT1 в клетке или у субъекта. Такие соединения, методы и фармацевтические композиции полезны для облегчения по меньшей мере одного симптома или признака неврологического заболевания. Такие симптомы и отличительные признаки включают судороги, энцефалопатию и поведенческие аномалии. Неограничивающими примерами неврологических заболеваний, при которых полезны эти соединения, способы и фармацевтические композиции, являются эпилепсия младенчества с мигрирующими фокальными припадками (EIMFS), аутосомно-доминантная ночная лобная эпилепсия (ADNFLE), синдром Веста и синдром Охтахара.Compounds, methods, and pharmaceutical compositions are provided for reducing the amount or activity of KCNT1 RNA in a cell or subject and, in some cases, for reducing the amount of KCNT1 protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful in alleviating at least one symptom or sign of a neurological disease. Such symptoms and hallmarks include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological diseases for which these compounds, methods, and pharmaceutical compositions are useful are epilepsy of infancy with migrating focal seizures (EIMFS), autosomal dominant nocturnal frontal epilepsy (ADNFLE), West's syndrome, and Ochtahar's syndrome.

EA202192527A 2019-08-08 2020-03-13 COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION EA202192527A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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)

Publication Number Publication Date
EA202192527A1 true EA202192527A1 (en) 2021-12-13

Family

ID=80631201

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192527A EA202192527A1 (en) 2019-08-08 2020-03-13 COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION

Country Status (1)

Country Link
EA (1) EA202192527A1 (en)

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