BR112022002889A2 - Método para tratar distrofia muscular tendo como alvo o gene lama1 - Google Patents

Método para tratar distrofia muscular tendo como alvo o gene lama1

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Publication number
BR112022002889A2
BR112022002889A2 BR112022002889A BR112022002889A BR112022002889A2 BR 112022002889 A2 BR112022002889 A2 BR 112022002889A2 BR 112022002889 A BR112022002889 A BR 112022002889A BR 112022002889 A BR112022002889 A BR 112022002889A BR 112022002889 A2 BR112022002889 A2 BR 112022002889A2
Authority
BR
Brazil
Prior art keywords
targeting
muscular dystrophy
seq
guide rna
lama1 gene
Prior art date
Application number
BR112022002889A
Other languages
English (en)
Inventor
Tetsuya Yamagata
Yuanbo Qin
Original Assignee
Modalis Therapeutics Corp
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 filed Critical Modalis Therapeutics Corp
Publication of BR112022002889A2 publication Critical patent/BR112022002889A2/pt

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    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/39—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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Abstract

método para tratar distrofia muscular tendo como alvo o gene lama1. a presente invenção tem como objetivo fornecer uma nova abordagem terapêutica para a distrofia muscular humana (particularmente mdc1a). a presente invenção fornece um polinucleotídeo compreendendo as seguintes sequências de bases: (a) uma sequência de base que codifica uma proteína de fusão de uma proteína efetora crispr deficiente de nuclease e um ativador de transcrição, e (b) uma sequência de base que codifica (i) um rna guia alvejando uma região contínua estabelecida na seq id no: 15, 20, 25, 50, 56 ou 61, (ii) um rna guia alvejando uma região contínua estabelecida na seq id no: 124, ou (iii) um rna guia alvejando uma região contínua apresentada em seq id no: 178, 193 ou 195, na região reguladora de expressão do gene lama1 humano.
BR112022002889A 2019-08-16 2020-08-14 Método para tratar distrofia muscular tendo como alvo o gene lama1 BR112022002889A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962887863P 2019-08-16 2019-08-16
US202063008059P 2020-04-10 2020-04-10
PCT/JP2020/030864 WO2021033635A1 (en) 2019-08-16 2020-08-14 Method for treating muscular dystrophy by targeting lama1 gene

Publications (1)

Publication Number Publication Date
BR112022002889A2 true BR112022002889A2 (pt) 2022-05-17

Family

ID=72292600

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022002889A BR112022002889A2 (pt) 2019-08-16 2020-08-14 Método para tratar distrofia muscular tendo como alvo o gene lama1

Country Status (12)

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US (1) US20230173036A1 (pt)
EP (1) EP4013874A1 (pt)
JP (1) JP2022544320A (pt)
KR (1) KR20220045013A (pt)
CN (1) CN114364802A (pt)
AU (1) AU2020334462A1 (pt)
BR (1) BR112022002889A2 (pt)
CA (1) CA3146751A1 (pt)
IL (1) IL290249A (pt)
MX (1) MX2022001859A (pt)
TW (1) TW202112797A (pt)
WO (1) WO2021033635A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022176859A1 (en) * 2021-02-16 2022-08-25 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting lama1 gene
WO2023129940A1 (en) * 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
US9015089B2 (en) * 2012-04-17 2015-04-21 The Mitre Corporation Identifying and forecasting shifts in the mood of social media users
TW201642837A (zh) 2015-03-24 2016-12-16 Kyowa Hakko Kirin Co Ltd 含有核酸之脂質奈米粒子
JP6926825B2 (ja) * 2017-08-25 2021-08-25 沖電気工業株式会社 通信装置、プログラムおよびオペレータ選択方法

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IL290249A (en) 2022-04-01
KR20220045013A (ko) 2022-04-12
EP4013874A1 (en) 2022-06-22
JP2022544320A (ja) 2022-10-17
CN114364802A (zh) 2022-04-15
TW202112797A (zh) 2021-04-01
WO2021033635A1 (en) 2021-02-25
MX2022001859A (es) 2022-03-11
CA3146751A1 (en) 2021-02-25
AU2020334462A1 (en) 2022-02-24
US20230173036A1 (en) 2023-06-08

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