CO2021006957A2 - Método para el tratamiento de la distrofia muscular mediante el gen de utrofina - Google Patents

Método para el tratamiento de la distrofia muscular mediante el gen de utrofina

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Publication number
CO2021006957A2
CO2021006957A2 CONC2021/0006957A CO2021006957A CO2021006957A2 CO 2021006957 A2 CO2021006957 A2 CO 2021006957A2 CO 2021006957 A CO2021006957 A CO 2021006957A CO 2021006957 A2 CO2021006957 A2 CO 2021006957A2
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Colombia
Prior art keywords
muscular dystrophy
treatment
region
utrophin gene
sequence encoding
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Application number
CONC2021/0006957A
Other languages
English (en)
Inventor
Eiji Yoshimi
Katsuro Yoshioka
Tetsuya Yamagata
Yuanbo Qin
Iain Robert Thompson
Nidhi Khanna
Original Assignee
Modalis Therapeutics Corp
Astellas Pharma Inc
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 Modalis Therapeutics Corp, Astellas Pharma Inc filed Critical Modalis Therapeutics Corp
Publication of CO2021006957A2 publication Critical patent/CO2021006957A2/es

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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
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Abstract

RESUMEN Polinucleótidos que comprenden las siguientes secuencias de bases: (a) una secuencia de bases que codifica una proteína de fusión de una proteína efectora CRISPR deficiente en nucleasas y un activador de la transcripción, y (b) una secuencia de bases que codifica un ARN guía dirigido a una región continua de 18 a 24 nucleótidos de longitud en una región establecida en SEQ ID NO: 104, 105, 135, 141, 153, 167 o 172 en la región reguladora de la expresión del gen de la utrofina humana Se espera que sean útiles para tratar o prevenir la distrofia muscular de DUCHENNE o la distrofia muscular de BECKER.
CONC2021/0006957A 2018-11-16 2021-05-26 Método para el tratamiento de la distrofia muscular mediante el gen de utrofina CO2021006957A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862768474P 2018-11-16 2018-11-16
US201962861039P 2019-06-13 2019-06-13
US201962931925P 2019-11-07 2019-11-07
PCT/JP2019/045716 WO2020101042A1 (en) 2018-11-16 2019-11-15 Method for treating muscular dystrophy by targeting utrophin gene

Publications (1)

Publication Number Publication Date
CO2021006957A2 true CO2021006957A2 (es) 2021-06-10

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CONC2021/0006957A CO2021006957A2 (es) 2018-11-16 2021-05-26 Método para el tratamiento de la distrofia muscular mediante el gen de utrofina

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US (1) US11473071B2 (es)
EP (1) EP3880255A1 (es)
JP (2) JP7069426B2 (es)
KR (1) KR20210093954A (es)
CN (1) CN113271982A (es)
AU (1) AU2019381460A1 (es)
BR (1) BR112021009481A2 (es)
CA (1) CA3119618A1 (es)
CO (1) CO2021006957A2 (es)
IL (1) IL283123A (es)
MX (1) MX2021005720A (es)
PH (1) PH12021551122A1 (es)
SG (1) SG11202105030VA (es)
TW (1) TW202033224A (es)
WO (1) WO2020101042A1 (es)

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WO2019204766A1 (en) 2018-04-19 2019-10-24 The Regents Of The University Of California Compositions and methods for gene editing
SG11202012228QA (en) * 2018-06-08 2021-01-28 Modalis Therapeutics Corp MODIFIED Cas9 PROTEIN AND USE THEREOF
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WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
EP4222263A1 (en) 2020-09-29 2023-08-09 Genethon Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof
KR102421411B1 (ko) 2021-10-29 2022-07-18 주식회사 케이에스비튜젠 옥시라세탐을 포함하는 근이영양증 예방 또는 치료용 조성물
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CN113271982A (zh) 2021-08-17
TW202033224A (zh) 2020-09-16
JP7069426B2 (ja) 2022-05-17
MX2021005720A (es) 2021-07-21
US11473071B2 (en) 2022-10-18
WO2020101042A1 (en) 2020-05-22
WO2020101042A8 (en) 2020-07-30
CA3119618A1 (en) 2020-05-22
JP2022115976A (ja) 2022-08-09
PH12021551122A1 (en) 2021-11-22
KR20210093954A (ko) 2021-07-28
BR112021009481A2 (pt) 2021-08-17
JP2022509560A (ja) 2022-01-20
IL283123A (en) 2021-06-30
SG11202105030VA (en) 2021-06-29
EP3880255A1 (en) 2021-09-22
AU2019381460A1 (en) 2021-06-17
US20210355464A1 (en) 2021-11-18

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