BR112022021136A2 - Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas - Google Patents

Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas

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Publication number
BR112022021136A2
BR112022021136A2 BR112022021136A BR112022021136A BR112022021136A2 BR 112022021136 A2 BR112022021136 A2 BR 112022021136A2 BR 112022021136 A BR112022021136 A BR 112022021136A BR 112022021136 A BR112022021136 A BR 112022021136A BR 112022021136 A2 BR112022021136 A2 BR 112022021136A2
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BR
Brazil
Prior art keywords
cfb
methods
complement factor
irna compositions
gene
Prior art date
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BR112022021136A
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English (en)
Inventor
D Mcininch James
Castoreno Adam
K Schlegel Mark
Fishilevich Elane
Yucius Kristina
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 BR112022021136A2 publication Critical patent/BR112022021136A2/pt

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    • 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
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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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    • C12N2310/3533—Halogen
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
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    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

COMPOSIÇÕES DE iRNA DE FATOR B DE COMPLEMENTO (CFB) E MÉTODOS DE USO DAS MESMAS. A presente invenção refere-se a agentes de RNAi, por exemplo, agentes de dsRNA, visando o gene do fator B de complemento (CFB). A invenção também se refere a métodos de uso de tais agentes de RNAi para inibir a expressão de um gene CFB e a métodos de tratamento ou prevenção de uma doença associada a CFB em um indivíduo.
BR112022021136A 2020-04-30 2021-04-29 Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas BR112022021136A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063017725P 2020-04-30 2020-04-30
US202063119009P 2020-11-30 2020-11-30
US202163157899P 2021-03-08 2021-03-08
PCT/US2021/029872 WO2021222549A1 (en) 2020-04-30 2021-04-29 Complement factor b (cfb) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112022021136A2 true BR112022021136A2 (pt) 2022-11-29

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Family Applications (1)

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Country Status (13)

Country Link
US (1) US20240018515A1 (pt)
EP (1) EP4142738A1 (pt)
JP (1) JP2023523790A (pt)
KR (1) KR20230017789A (pt)
CN (1) CN116096381A (pt)
AU (1) AU2021265813A1 (pt)
BR (1) BR112022021136A2 (pt)
CA (1) CA3181198A1 (pt)
CL (1) CL2022002951A1 (pt)
CO (1) CO2022016196A2 (pt)
IL (1) IL297680A (pt)
MX (1) MX2022013606A (pt)
WO (1) WO2021222549A1 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022336157A1 (en) 2021-09-02 2024-03-14 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法
WO2023129496A2 (en) * 2021-12-27 2023-07-06 Apellis Pharmaceuticals, Inc. Rnas for complement inhibition
TW202345866A (zh) * 2022-01-20 2023-12-01 美商艾力克森製藥公司 用於抑制補體因子ｂ的組成物及方法
CN116769780A (zh) * 2022-07-08 2023-09-19 北京福元医药股份有限公司 用于抑制补体因子B表达的siRNA、其缀合物和药物组合物及其用途
CN117136236B (zh) * 2022-09-29 2024-03-15 广州必贝特医药股份有限公司 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用

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