CL2022002951A1 - Composiciones de arni del factor b del complemento (cfb) y métodos para su uso. - Google Patents

Composiciones de arni del factor b del complemento (cfb) y métodos para su uso.

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Publication number
CL2022002951A1
CL2022002951A1 CL2022002951A CL2022002951A CL2022002951A1 CL 2022002951 A1 CL2022002951 A1 CL 2022002951A1 CL 2022002951 A CL2022002951 A CL 2022002951A CL 2022002951 A CL2022002951 A CL 2022002951A CL 2022002951 A1 CL2022002951 A1 CL 2022002951A1
Authority
CL
Chile
Prior art keywords
cfb
methods
complement factor
arni
compositions
Prior art date
Application number
CL2022002951A
Other languages
English (en)
Inventor
Castoreno Adam
Kaittanis Charalambos
K Schlegel Mark
D Mcininch James
Fishilevich Elane
Yucius Kristina
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Alnylam Pharmaceuticals Inc
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Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CL2022002951A1 publication Critical patent/CL2022002951A1/es

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    • 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
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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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
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Organic Chemistry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
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Abstract

La presente invención se refiere a agentes de ARNi, por ejemplo, agentes de ARNbc, dirigidos al gen del factor B del complemento (CFB). La invención también se refiere a métodos para usar dichos agentes de ARNi para inhibir la expresión de un gen CFB y a métodos para tratar o prevenir una enfermedad asociada a CFB en un sujeto.
CL2022002951A 2020-04-30 2022-10-25 Composiciones de arni del factor b del complemento (cfb) y métodos para su uso. CL2022002951A1 (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
CL2022002951A1 true CL2022002951A1 (es) 2023-06-23

Family

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CL2022002951A CL2022002951A1 (es) 2020-04-30 2022-10-25 Composiciones de arni del factor b del complemento (cfb) y métodos para su uso.

Country Status (13)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL311146A (en) 2021-09-02 2024-04-01 Silence Therapeutics Gmbh Nucleic acids to inhibit expression of complement factor B (CFB) in the cell
CA3234636A1 (en) * 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
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
WO2024008114A1 (zh) * 2022-07-08 2024-01-11 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
WO2024059873A2 (en) * 2022-09-16 2024-03-21 Sirnaomics, Inc. Products and compositions
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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