CR20190572A - AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO - Google Patents

AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO

Info

Publication number
CR20190572A
CR20190572A CR20190572A CR20190572A CR20190572A CR 20190572 A CR20190572 A CR 20190572A CR 20190572 A CR20190572 A CR 20190572A CR 20190572 A CR20190572 A CR 20190572A CR 20190572 A CR20190572 A CR 20190572A
Authority
CR
Costa Rica
Prior art keywords
enac
alpha
rnai agents
methods
inhibiting expression
Prior art date
Application number
CR20190572A
Other languages
English (en)
Inventor
Rui Zhu
Anthony Nicholas
Zhen Li
Tao Pei
Erik W Bush
Original Assignee
Arrowhead Pharmaceuticals Inc
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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of CR20190572A publication Critical patent/CR20190572A/es

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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
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/38—Drugs for disorders of the endocrine system of the suprarenal hormones
    • A61P5/40—Mineralocorticosteroids, e.g. aldosterone; Drugs increasing or potentiating the activity of mineralocorticosteroids
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Endocrinology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se describen agentes de iARN, composiciones que incluyen agentes de iARN y métodos para la inhibición de un gen de alfa-ENaC (SCNN1A). Los agentes de iARN de alfa-ENaC y conjugados de agente de iARN divulgados en la presente inhiben la expresión del gen de alfa-ENaC. También se describen composiciones farmacéuticas que incluyen uno o más agentes de iARN de alfa-ENaC, opcionalmente con uno o más agentes terapéuticos adicionales. El suministro de los agentes de iARN de alfa-ENaC descritos a las células epiteliales, tales como células epiteliales pulmonares, in vivo, prevé la inhibición de la expresión del gen de alfa-ENaC y una reducción de la actividad de ENaC, lo que puede proporcionar un beneficio terapéutico a los sujetos, incluyendo sujetos humanos
CR20190572A 2017-07-06 2018-07-05 AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO CR20190572A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762529132P 2017-07-06 2017-07-06
US201862631683P 2018-02-17 2018-02-17
US201862679549P 2018-06-01 2018-06-01
PCT/US2018/040874 WO2019010274A1 (en) 2017-07-06 2018-07-05 RNAI AGENTS OF INHIBITION OF ALPHA-ENAC GENE EXPRESSION AND METHODS OF USE

Publications (1)

Publication Number Publication Date
CR20190572A true CR20190572A (es) 2020-05-23

Family

ID=64904123

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20190572A CR20190572A (es) 2017-07-06 2018-07-05 AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO

Country Status (22)

Country Link
US (3) US10590416B2 (es)
EP (1) EP3649240A4 (es)
JP (3) JP2020526192A (es)
KR (1) KR20200024793A (es)
CN (2) CN119662650A (es)
AU (1) AU2018297262A1 (es)
BR (1) BR112019023650A2 (es)
CA (1) CA3061752A1 (es)
CL (1) CL2020000019A1 (es)
CO (1) CO2019014671A2 (es)
CR (1) CR20190572A (es)
EC (1) ECSP20000655A (es)
IL (1) IL271843A (es)
JO (1) JOP20190263A1 (es)
MX (1) MX2019014800A (es)
PE (1) PE20200746A1 (es)
PH (1) PH12019502660A1 (es)
TN (1) TN2019000308A1 (es)
TW (1) TW201919654A (es)
UY (1) UY37800A (es)
WO (1) WO2019010274A1 (es)
ZA (1) ZA202100643B (es)

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TW202227627A (zh) * 2020-09-11 2022-07-16 美商愛羅海德製藥公司 用於抑制DUX4表現之RNAi藥劑、其組合物及使用方法
CN118202049A (zh) 2021-12-21 2024-06-14 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
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JP2025185070A (ja) 2025-12-18
US20220090077A1 (en) 2022-03-24
CN110832077A (zh) 2020-02-21
KR20200024793A (ko) 2020-03-09
EP3649240A4 (en) 2021-07-07
CL2020000019A1 (es) 2020-06-12
JOP20190263A1 (ar) 2019-11-12
ZA202100643B (en) 2023-03-29
US11214802B2 (en) 2022-01-04
PE20200746A1 (es) 2020-07-24
AU2018297262A1 (en) 2020-02-27
CA3061752A1 (en) 2019-01-10
TN2019000308A1 (en) 2021-05-07
UY37800A (es) 2019-01-02
EP3649240A1 (en) 2020-05-13
WO2019010274A1 (en) 2019-01-10
CN110832077B (zh) 2025-01-03
US20200299691A1 (en) 2020-09-24
ECSP20000655A (es) 2020-05-29
JP2023158192A (ja) 2023-10-26
CN119662650A (zh) 2025-03-21
CO2019014671A2 (es) 2020-01-17
US10590416B2 (en) 2020-03-17
PH12019502660A1 (en) 2020-06-08
JP2020526192A (ja) 2020-08-31
MX2019014800A (es) 2020-02-10
BR112019023650A2 (pt) 2020-06-02
IL271843A (en) 2020-02-27
TW201919654A (zh) 2019-06-01
US20190010494A1 (en) 2019-01-10

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