CA3147641A1 - 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
CA3147641A1
CA3147641A1 CA3147641A CA3147641A CA3147641A1 CA 3147641 A1 CA3147641 A1 CA 3147641A1 CA 3147641 A CA3147641 A CA 3147641A CA 3147641 A CA3147641 A CA 3147641A CA 3147641 A1 CA3147641 A1 CA 3147641A1
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Prior art keywords
apob
site
specific
disrupting agent
effector
Prior art date
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CA3147641A
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English (en)
French (fr)
Inventor
Rahul KARNIK
Vishwesh Ashok PATIL
Jeremiah D. FARELLI
Jesse Jerome SMITH
Can SARISOZEN
Adam Walter SCHEIDEGGER
Barbara Bennett
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Omega Therapeutics Inc
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Omega Therapeutics Inc
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Publication of CA3147641A1 publication Critical patent/CA3147641A1/en
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/595—Polyamides, e.g. nylon
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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CA3147641A 2019-09-23 2020-09-23 Compositions and methods for modulating apolipoprotein b (apob) gene expression Pending CA3147641A1 (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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CA3147641A1 true CA3147641A1 (en) 2021-04-01

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CA3147641A Pending CA3147641A1 (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)

Families Citing this family (2)

* 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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