CO2023014681A2 - Composiciones y métodos para inhibir la expresión del componente 3 del complemento - Google Patents

Composiciones y métodos para inhibir la expresión del componente 3 del complemento

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Publication number
CO2023014681A2
CO2023014681A2 CONC2023/0014681A CO2023014681A CO2023014681A2 CO 2023014681 A2 CO2023014681 A2 CO 2023014681A2 CO 2023014681 A CO2023014681 A CO 2023014681A CO 2023014681 A2 CO2023014681 A2 CO 2023014681A2
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Colombia
Prior art keywords
expression
methods
complement component
inhibiting
compositions
Prior art date
Application number
CONC2023/0014681A
Other languages
English (en)
Inventor
Melissa Lasaro
Susan Faas Mcknight
Henryk T Dudek
Jihye Park
Bob Dale Brown
Chengjung Lai
Sungkwon Kim
Original Assignee
Astrazeneca Ireland Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Astrazeneca Ireland Ltd filed Critical Astrazeneca Ireland Ltd
Publication of CO2023014681A2 publication Critical patent/CO2023014681A2/es

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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
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/35—Nature of the modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

En el presente documento se describen oligonucleótidos (p. ej., oligonucleótidos de iARN) que contienen hebras sentido y antisentido para actuar sobre el ARNm del componente 3 del complemento (C3). El oligonucleótido de iARN puede usarse para inhibir la expresión, los niveles y/o la actividad de C3 en una célula. Además, en el presente documento se describen métodos para usar un oligonucleótido (p. ej., un oligonucleótido de iARN) para la profilaxis o el tratamiento de una enfermedad, trastorno o afección mediados por la activación o desregulación de la vía del complemento.
CONC2023/0014681A 2021-04-20 2023-10-30 Composiciones y métodos para inhibir la expresión del componente 3 del complemento CO2023014681A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163177254P 2021-04-20 2021-04-20
PCT/US2022/025648 WO2022226127A1 (en) 2021-04-20 2022-04-20 Compositions and methods for inhibiting complement component 3 expression

Publications (1)

Publication Number Publication Date
CO2023014681A2 true CO2023014681A2 (es) 2024-01-25

Family

ID=83723122

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CONC2023/0014681A CO2023014681A2 (es) 2021-04-20 2023-10-30 Composiciones y métodos para inhibir la expresión del componente 3 del complemento

Country Status (10)

Country Link
EP (1) EP4326876A1 (es)
JP (1) JP2024515344A (es)
KR (1) KR20230173116A (es)
CN (1) CN117295819A (es)
AU (1) AU2022261922A1 (es)
BR (1) BR112023021703A2 (es)
CA (1) CA3177629A1 (es)
CO (1) CO2023014681A2 (es)
IL (1) IL307721A (es)
WO (1) WO2022226127A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202400193A (zh) 2022-06-24 2024-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110249053A (zh) * 2016-10-17 2019-09-17 阿佩利斯制药有限公司 用于c3抑制的组合疗法
BR112022007540A2 (pt) * 2019-10-22 2022-07-12 Alnylam Pharmaceuticals Inc Composições de irna de componente complementar c3 e métodos de uso das mesmas

Also Published As

Publication number Publication date
JP2024515344A (ja) 2024-04-09
CA3177629A1 (en) 2022-10-27
IL307721A (en) 2023-12-01
AU2022261922A1 (en) 2023-10-26
BR112023021703A2 (pt) 2024-01-30
EP4326876A1 (en) 2024-02-28
WO2022226127A1 (en) 2022-10-27
KR20230173116A (ko) 2023-12-26
CN117295819A (zh) 2023-12-26

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