AU2020355000A1 - Compositions and methods for modulating apolipoprotein B (APOB) gene expression - Google Patents

Compositions and methods for modulating apolipoprotein B (APOB) gene expression Download PDF

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Publication number
AU2020355000A1
AU2020355000A1 AU2020355000A AU2020355000A AU2020355000A1 AU 2020355000 A1 AU2020355000 A1 AU 2020355000A1 AU 2020355000 A AU2020355000 A AU 2020355000A AU 2020355000 A AU2020355000 A AU 2020355000A AU 2020355000 A1 AU2020355000 A1 AU 2020355000A1
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Australia
Prior art keywords
apob
site
specific
disrupting agent
effector
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AU2020355000A
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Inventor
Barbara Bennett
Jeremiah D. FARELLI
Rahul KARNIK
Vishwesh Ashok PATIL
Can SARISOZEN
Adam Walter SCHEIDEGGER
Jesse Jerome SMITH
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Omega Therapeutics Inc
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Omega Therapeutics Inc
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2800/00—Nucleic acids vectors
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AU2020355000A 2019-09-23 2020-09-23 Compositions and methods for modulating apolipoprotein B (APOB) gene expression Pending AU2020355000A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962904325P 2019-09-23 2019-09-23
US62/904,325 2019-09-23
PCT/US2020/052119 WO2021061707A1 (en) 2019-09-23 2020-09-23 Compositions and methods for modulating apolipoprotein b (apob) gene expression

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AU2020355000A1 true AU2020355000A1 (en) 2022-03-17

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AU2020355000A Pending AU2020355000A1 (en) 2019-09-23 2020-09-23 Compositions and methods for modulating apolipoprotein B (APOB) gene expression

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US (1) US20220288237A1 (https=)
EP (1) EP4048807A1 (https=)
JP (1) JP2022548320A (https=)
CN (1) CN114391040A (https=)
AU (1) AU2020355000A1 (https=)
CA (1) CA3147641A1 (https=)
WO (1) WO2021061707A1 (https=)

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CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
CN119698469A (zh) * 2022-08-11 2025-03-25 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途

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