MX2021011921A - Compuesto, metodo y composicion farmaceutica para modular la expresion del doble homeobox 4 (dux4). - Google Patents

Compuesto, metodo y composicion farmaceutica para modular la expresion del doble homeobox 4 (dux4).

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Publication number
MX2021011921A
MX2021011921A MX2021011921A MX2021011921A MX2021011921A MX 2021011921 A MX2021011921 A MX 2021011921A MX 2021011921 A MX2021011921 A MX 2021011921A MX 2021011921 A MX2021011921 A MX 2021011921A MX 2021011921 A MX2021011921 A MX 2021011921A
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MX
Mexico
Prior art keywords
dux4
compound
pharmaceutical composition
nucleic acid
dux4 expression
Prior art date
Application number
MX2021011921A
Other languages
English (en)
Inventor
Shinji Kumagai
Hiroyuki Furukawa
Tomo Araki
Takashi Yashiro
Takayuki Kanagawa
Chieko Okagaki
Original Assignee
Mitsubishi Tanabe Pharma Corp
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Publication date
Application filed by Mitsubishi Tanabe Pharma Corp filed Critical Mitsubishi Tanabe Pharma Corp
Publication of MX2021011921A publication Critical patent/MX2021011921A/es

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Abstract

Un objetivo de la presente invención es proporcionar un compuesto, un método y una composición farmacéutica para normalizar el doble homeobox 4 (DUX4) de un individuo en donde el gen del DUX4 se ha expresado de manera anormal; se proporciona un oligonucleótido modificado que consiste en 12 a 30 residuos; el oligonucleótido modificado incluye una secuencia de nucleobases que incluye por lo menos 8 de nucleobases contiguas y es complementaria con una porción de igual longitud en las posiciones 126 a 147, 232 a 248, 1306 a 1325 o 1472 a1495 de un extremo 5' de una secuencia de nucleobases de un ARNm maduro de DUX4 de SEQ ID NO: 1; la secuencia de nucleobases del oligonucleótido modificado tiene por lo menos 90% de complementariedad con la porción de igual longitud en la secuencia de nucleobases del ARNm maduro de DUX4 de SEQ ID NO: 1.
MX2021011921A 2019-03-29 2020-03-27 Compuesto, metodo y composicion farmaceutica para modular la expresion del doble homeobox 4 (dux4). MX2021011921A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2019067914 2019-03-29
PCT/JP2020/014307 WO2020203880A1 (ja) 2019-03-29 2020-03-27 Ｄｕｘ４の発現を調節するための化合物、方法及び医薬組成物

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MX2021011921A true MX2021011921A (es) 2021-10-26

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MX2021011921A MX2021011921A (es) 2019-03-29 2020-03-27 Compuesto, metodo y composicion farmaceutica para modular la expresion del doble homeobox 4 (dux4).

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US (1) US20220228140A1 (es)
EP (1) EP3950006A4 (es)
JP (1) JPWO2020203880A1 (es)
KR (1) KR20210142699A (es)
CN (1) CN113710283A (es)
AR (1) AR118513A1 (es)
AU (1) AU2020252710B2 (es)
BR (1) BR112021018793A2 (es)
CA (4) CA3198029A1 (es)
IL (1) IL286733A (es)
MX (1) MX2021011921A (es)
SG (1) SG11202110674XA (es)
TW (1) TW202104595A (es)
WO (1) WO2020203880A1 (es)

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EP3881851A4 (en) * 2018-11-12 2023-02-15 Mitsubishi Tanabe Pharma Corporation ALNA CROSS-LINKED ARTIFICIAL NUCLEIC ACID
JP2023537798A (ja) 2020-03-19 2023-09-06 アビディティー バイオサイエンシーズ，インク． 顔面肩甲上腕型筋ジストロフィーを処置するための組成物および方法
KR20230023612A (ko) 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제
KR20240055874A (ko) * 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
WO2024135721A1 (ja) * 2022-12-21 2024-06-27 田辺三菱製薬株式会社 製造中間体の製造方法、その製造中間体及びそれらを用いた架橋型人工核酸中間体の製造方法

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