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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English (en)
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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    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
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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 (enExample)
EP (1) EP4048807A1 (enExample)
JP (1) JP2022548320A (enExample)
CN (1) CN114391040A (enExample)
AU (1) AU2020355000A1 (enExample)
CA (1) CA3147641A1 (enExample)
WO (1) WO2021061707A1 (enExample)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020352552A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
JP2025526074A (ja) * 2022-08-11 2025-08-07 エピジェニック・セラピューティクス・プライベイト・リミテッド エピトープ編集標的のための方法およびその使用

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