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EA202091125A1 - Соединения и способы для снижения экспрессии snca - Google Patents

Соединения и способы для снижения экспрессии snca

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Publication number
EA202091125A1
EA202091125A1 EA202091125A EA202091125A EA202091125A1 EA 202091125 A1 EA202091125 A1 EA 202091125A1 EA 202091125 A EA202091125 A EA 202091125A EA 202091125 A EA202091125 A EA 202091125A EA 202091125 A1 EA202091125 A1 EA 202091125A1
Authority
EA
Eurasian Patent Office
Prior art keywords
disease
methods
reducing
snca
alpha
Prior art date
Application number
EA202091125A
Other languages
English (en)
Inventor
Холли Кордашевич
Приям Сингх
Сьюзан М. Фрайер
Трейси А. Коул
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.
Publication of EA202091125A1 publication Critical patent/EA202091125A1/ru

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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
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    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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Abstract

Предложены соединения, способы и фармацевтические композиции для снижения количества или активности мРНК SNCA в клетке или у животного, а также в некоторых случаях для снижения количества белка альфа-синуклеина в клетке или у животного. Такие соединения, способы и фармацевтические композиции пригодны для ослабления по меньшей мере одного симптома или признака нейродегенеративного заболевания. Такие симптомы и признаки включают двигательную дисфункцию, агрегацию альфа-синуклеина, нейродегенерацию, снижение когнитивных функций и деменцию. Такие нейродегенеративные заболевания включают болезнь Паркинсона, деменцию с тельцами Леви, болезнь диффузных телец Леви, истинную вегетативную недостаточность, множественную системную атрофию, нейронопатическую болезнь Гоше и болезнь Альцгеймера.
EA202091125A 2017-11-09 2018-11-09 Соединения и способы для снижения экспрессии snca EA202091125A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762584009P 2017-11-09 2017-11-09
PCT/US2018/060097 WO2019164562A2 (en) 2017-11-09 2018-11-09 Compounds and methods for reducing snca expression

Publications (1)

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EA202091125A1 true EA202091125A1 (ru) 2020-08-11

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US (2) US11230712B2 (ru)
EP (2) EP3707260A4 (ru)
JP (2) JP7167148B2 (ru)
KR (1) KR20200079304A (ru)
CN (2) CN111373043B (ru)
AR (1) AR113870A1 (ru)
AU (1) AU2018409843A1 (ru)
BR (1) BR112020007240A2 (ru)
CA (1) CA3078788A1 (ru)
CL (1) CL2020001066A1 (ru)
CO (1) CO2020004762A2 (ru)
CR (1) CR20200246A (ru)
EA (1) EA202091125A1 (ru)
IL (1) IL274394A (ru)
JO (1) JOP20200105A1 (ru)
MX (1) MX2020004761A (ru)
PH (1) PH12020550586A1 (ru)
SG (1) SG11202003102TA (ru)
TW (1) TWI809004B (ru)
UY (1) UY37967A (ru)
WO (1) WO2019164562A2 (ru)
ZA (1) ZA202002517B (ru)

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AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CA3056179A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
SG11202006526YA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
PE20240696A1 (es) * 2021-03-08 2024-04-10 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina
EP4342988A1 (en) * 2022-09-20 2024-03-27 Eberhard Karls Universität Tübingen, Medizinische Fakultät Compositions and methods for modification of the expression of a snca gene
US20240124513A1 (en) * 2022-09-23 2024-04-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing mecp2 expression
WO2024166019A1 (en) * 2023-02-08 2024-08-15 Biorchestra Co., Ltd. Sirna targeting alpha-synuclein and uses thereof

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