BR112022007540A2 - Composições de irna de componente complementar c3 e métodos de uso das mesmas - Google Patents

Composições de irna de componente complementar c3 e métodos de uso das mesmas

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Publication number
BR112022007540A2
BR112022007540A2 BR112022007540A BR112022007540A BR112022007540A2 BR 112022007540 A2 BR112022007540 A2 BR 112022007540A2 BR 112022007540 A BR112022007540 A BR 112022007540A BR 112022007540 A BR112022007540 A BR 112022007540A BR 112022007540 A2 BR112022007540 A2 BR 112022007540A2
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Brazil
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methods
pemphigus
complementary
gene
irna compositions
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BR112022007540A
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English (en)
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Keating Mark
D Mcininch James
Yucius Kristina
Kaittanis Charalambos
Castoreno Adam
Solomon Sarah
Fishilevich Elane
K Schlegel Mark
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Alnylam Pharmaceuticals Inc
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Publication of BR112022007540A2 publication Critical patent/BR112022007540A2/pt

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • C12N2310/3125Methylphosphonates
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

Abstract

COMPOSIÇÕES DE IRNA DE COMPONENTE COMPLEMENTAR C3 E MÉTODOS DE USO DAS MESMAS. A presente invenção refere-se a agentes de RNAi, por exemplo, agentes de RNA de fita dupla (dsRNA), alvejando o gene C3 de componente complementar (C3). A invenção também se refere a métodos de uso de tais agentes de RNAi para inibir a expressão de um gene C3 e a métodos de prevenção e tratamento de um distúrbio associado a C3, por exemplo, doença por aglutinina fria (CAD), anemia hemolítica autoimune quente e hemoglobinúria paroxística noturna (PNH), nefrite lúpica (LN), penfigoide bolhoso, pênfigo, por exemplo, pênfigo vulgar (PV) e pênfigo foliáceo (PF) e C3 de glomerulopatia.
BR112022007540A 2019-10-22 2020-10-21 Composições de irna de componente complementar c3 e métodos de uso das mesmas BR112022007540A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962924210P 2019-10-22 2019-10-22
PCT/US2020/056563 WO2021081026A1 (en) 2019-10-22 2020-10-21 Complement component c3 irna compositions and methods of use thereof

Publications (1)

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BR112022007540A2 true BR112022007540A2 (pt) 2022-07-12

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022007540A BR112022007540A2 (pt) 2019-10-22 2020-10-21 Composições de irna de componente complementar c3 e métodos de uso das mesmas

Country Status (13)

Country Link
US (1) US20220364088A1 (pt)
EP (1) EP4048793A1 (pt)
JP (1) JP2022553348A (pt)
KR (1) KR20220084399A (pt)
CN (1) CN115176004A (pt)
AR (1) AR120267A1 (pt)
AU (1) AU2020369515A1 (pt)
BR (1) BR112022007540A2 (pt)
CA (1) CA3158320A1 (pt)
IL (1) IL292360A (pt)
MX (1) MX2022004726A (pt)
TW (1) TW202134435A (pt)
WO (1) WO2021081026A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6868561B2 (ja) 2015-01-28 2021-05-12 アレクシオン ファーマシューティカルズ, インコーポレイテッド アルカリホスファターゼ欠損を有する被験者を治療する方法
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2019190752A1 (en) 2018-03-30 2019-10-03 Alexion Pharmaceuticals, Inc. Manufacturing of glycoproteins
AU2022261922A1 (en) * 2021-04-20 2023-10-26 AstraZeneca Ireland Limited Compositions and methods for inhibiting complement component 3 expression
WO2023044370A2 (en) * 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
WO2023088455A1 (zh) * 2021-11-22 2023-05-25 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
WO2023186056A1 (zh) * 2022-04-02 2023-10-05 上海舶望制药有限公司 用于抑制补体成分c3蛋白表达的组合物和方法

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