BR112022016324A2 - Composições de irna de apolipoproteína c3 (apoc3) e métodos de uso das mesmas - Google Patents

Composições de irna de apolipoproteína c3 (apoc3) e métodos de uso das mesmas

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Publication number
BR112022016324A2
BR112022016324A2 BR112022016324A BR112022016324A BR112022016324A2 BR 112022016324 A2 BR112022016324 A2 BR 112022016324A2 BR 112022016324 A BR112022016324 A BR 112022016324A BR 112022016324 A BR112022016324 A BR 112022016324A BR 112022016324 A2 BR112022016324 A2 BR 112022016324A2
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BR
Brazil
Prior art keywords
apoc3
methods
apolipoprotein
irna compositions
gene
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BR112022016324A
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English (en)
Inventor
Tremblay Frederic
D Bondurant Lucas
D Mcininch James
Castoreno Adam
K Schlegel Mark
Kaittanis Charalambos
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Alnylam Pharmaceuticals Inc
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Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of BR112022016324A2 publication Critical patent/BR112022016324A2/pt

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Abstract

COMPOSIÇÕES DE IRNA DE APOLIPOPROTEÍNA C3 (APOC3) E MÉTODOS DE USO DAS MESMAS. A presente invenção refere-se a agentes de RNAi, por exemplo, agentes de RNA de fita dupla (dsRNA), direcionado para o gene da apolipoproteína C3 (APOC3). A invenção também se refere a métodos de uso de tais agentes de RNAi para inibir a expressão de um gene de APOC3 e a métodos de prevenção e tratamento de um distúrbio associado à APOC3, por exemplo, hipertrigliceridemia, doença hepática gordurosa não alcoólica, esteato-hepatite não alcoólica, síndrome do ovário policístico, doença renal, obesidade, diabetes melito tipo 2 (resistência à insulina), hipertensão, arteroesclerose e pancreatite.
BR112022016324A 2020-02-18 2021-02-12 Composições de irna de apolipoproteína c3 (apoc3) e métodos de uso das mesmas BR112022016324A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062977875P 2020-02-18 2020-02-18
US202163144516P 2021-02-02 2021-02-02
PCT/US2021/017826 WO2021167841A1 (en) 2020-02-18 2021-02-12 Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112022016324A2 true BR112022016324A2 (pt) 2022-10-11

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Family Applications (1)

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Country Status (12)

Country Link
US (2) US11162103B2 (pt)
EP (1) EP4107265A1 (pt)
JP (1) JP2023514336A (pt)
KR (1) KR20220143106A (pt)
CN (1) CN115397989A (pt)
AU (1) AU2021224778A1 (pt)
BR (1) BR112022016324A2 (pt)
CA (1) CA3171654A1 (pt)
IL (1) IL295496A (pt)
MX (1) MX2022010052A (pt)
TW (1) TW202144571A (pt)
WO (1) WO2021167841A1 (pt)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230272393A1 (en) * 2021-12-01 2023-08-31 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating apoc3 expression
WO2024002093A1 (zh) * 2022-06-27 2024-01-04 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
CN116814621A (zh) * 2022-08-05 2023-09-29 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
CN116400082A (zh) * 2023-02-02 2023-07-07 广东省妇幼保健院 检测ApoC3蛋白含量的试剂在制备预测卵母细胞发育试剂中的应用
CN117563009A (zh) * 2023-09-01 2024-02-20 北京悦康科创医药科技股份有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物

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