BR112021008069A2 - Oligonucleotídeos antisense biespecíficos para exon skipping da distrofina - Google Patents

Oligonucleotídeos antisense biespecíficos para exon skipping da distrofina

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Publication number
BR112021008069A2
BR112021008069A2 BR112021008069A BR112021008069A BR112021008069A2 BR 112021008069 A2 BR112021008069 A2 BR 112021008069A2 BR 112021008069 A BR112021008069 A BR 112021008069A BR 112021008069 A BR112021008069 A BR 112021008069A BR 112021008069 A2 BR112021008069 A2 BR 112021008069A2
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BR
Brazil
Prior art keywords
antisense oligonucleotides
exon skipping
dystrophin exon
bispecific antisense
bispecific
Prior art date
Application number
BR112021008069A
Other languages
English (en)
Inventor
Christina Theodora Van Deutekom Judith
Ann DATSON Nicole
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Biomarin Tech Bv
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Publication date
Application filed by Biomarin Tech Bv filed Critical Biomarin Tech Bv
Publication of BR112021008069A2 publication Critical patent/BR112021008069A2/pt

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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2320/33—Alteration of splicing

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Abstract

oligonucleotídeos antisense biespecíficos para exon skipping da distrofina. a presente invenção fornece compostos de splice-switching com características melhoradas que aumentam a aplicabilidade clínica, preferencialmente, para o tratamento, melhora, prevenção e/ou retardo de distúrbios neuromusculares, mais especificamente, a dmd.
BR112021008069A 2018-11-02 2019-10-30 Oligonucleotídeos antisense biespecíficos para exon skipping da distrofina BR112021008069A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18204170 2018-11-02
PCT/EP2019/079714 WO2020089325A1 (en) 2018-11-02 2019-10-30 Bispecific antisense oligonucleotides for dystrophin exon skipping

Publications (1)

Publication Number Publication Date
BR112021008069A2 true BR112021008069A2 (pt) 2021-11-03

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BR112021008069A BR112021008069A2 (pt) 2018-11-02 2019-10-30 Oligonucleotídeos antisense biespecíficos para exon skipping da distrofina

Country Status (12)

Country Link
US (1) US20220025368A1 (pt)
EP (1) EP3874044A1 (pt)
JP (1) JP2022506219A (pt)
KR (1) KR20210091180A (pt)
CN (1) CN113286887A (pt)
AU (1) AU2019370937A1 (pt)
BR (1) BR112021008069A2 (pt)
CA (1) CA3118167A1 (pt)
IL (1) IL282818A (pt)
MX (1) MX2021004822A (pt)
SG (1) SG11202103938UA (pt)
WO (1) WO2020089325A1 (pt)

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CA3216839A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
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WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
TW202406557A (zh) * 2022-05-05 2024-02-16 美商拜奧馬林製藥公司 治療杜興氏肌肉失養症之方法

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Also Published As

Publication number Publication date
WO2020089325A1 (en) 2020-05-07
SG11202103938UA (en) 2021-05-28
CN113286887A (zh) 2021-08-20
AU2019370937A1 (en) 2021-05-27
EP3874044A1 (en) 2021-09-08
KR20210091180A (ko) 2021-07-21
JP2022506219A (ja) 2022-01-17
US20220025368A1 (en) 2022-01-27
MX2021004822A (es) 2021-07-06
CA3118167A1 (en) 2020-05-07
IL282818A (en) 2021-06-30

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