ZA202100643B - Rnai agents for inhibiting expression of alpha-enac and methods of use - Google Patents

Rnai agents for inhibiting expression of alpha-enac and methods of use

Info

Publication number
ZA202100643B
ZA202100643B ZA2021/00643A ZA202100643A ZA202100643B ZA 202100643 B ZA202100643 B ZA 202100643B ZA 2021/00643 A ZA2021/00643 A ZA 2021/00643A ZA 202100643 A ZA202100643 A ZA 202100643A ZA 202100643 B ZA202100643 B ZA 202100643B
Authority
ZA
South Africa
Prior art keywords
enac
alpha
methods
rnai agents
inhibiting expression
Prior art date
Application number
ZA2021/00643A
Other languages
English (en)
Inventor
Zhen Li
Rui Zhu
Tao Pei
Anthony Nicholas
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 ZA202100643B publication Critical patent/ZA202100643B/en

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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)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Endocrinology (AREA)
  • Diabetes (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)
ZA2021/00643A 2017-07-06 2021-01-29 Rnai agents for inhibiting expression of alpha-enac and methods of use ZA202100643B (en)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
ZA202100643B true ZA202100643B (en) 2023-03-29

Family

ID=64904123

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2021/00643A ZA202100643B (en) 2017-07-06 2021-01-29 Rnai agents for inhibiting expression of alpha-enac and methods of use

Country Status (22)

Country Link
US (3) US10590416B2 (https=)
EP (1) EP3649240A4 (https=)
JP (3) JP2020526192A (https=)
KR (1) KR20200024793A (https=)
CN (2) CN110832077B (https=)
AU (1) AU2018297262A1 (https=)
BR (1) BR112019023650A2 (https=)
CA (1) CA3061752A1 (https=)
CL (1) CL2020000019A1 (https=)
CO (1) CO2019014671A2 (https=)
CR (1) CR20190572A (https=)
EC (1) ECSP20000655A (https=)
IL (1) IL271843A (https=)
JO (1) JOP20190263A1 (https=)
MX (1) MX2019014800A (https=)
PE (1) PE20200746A1 (https=)
PH (1) PH12019502660A1 (https=)
TN (1) TN2019000308A1 (https=)
TW (1) TW201919654A (https=)
UY (1) UY37800A (https=)
WO (1) WO2019010274A1 (https=)
ZA (1) ZA202100643B (https=)

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TW202130809A (zh) * 2019-10-29 2021-08-16 美商愛羅海德製藥公司 用於抑制β-ENaC表現之RNAi藥劑、其組合物及使用方法
IL301187A (en) * 2020-09-11 2023-05-01 Arrowhead Pharmaceuticals Inc RNAi agents for suppressing DUX4 expression, preparations containing them and uses thereof
AU2022422864A1 (en) 2021-12-21 2024-07-04 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
JP2025524135A (ja) * 2022-07-27 2025-07-25 イー－セラピューティクス・ピーエルシー 核酸化合物
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WO2024188174A1 (zh) 2023-03-10 2024-09-19 苏州瑞博生物技术股份有限公司 一种药物组合物及其用途
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Also Published As

Publication number Publication date
EP3649240A4 (en) 2021-07-07
CN119662650A (zh) 2025-03-21
UY37800A (es) 2019-01-02
US20220090077A1 (en) 2022-03-24
PH12019502660A1 (en) 2020-06-08
TW201919654A (zh) 2019-06-01
JP2023158192A (ja) 2023-10-26
US11214802B2 (en) 2022-01-04
US10590416B2 (en) 2020-03-17
JOP20190263A1 (ar) 2019-11-12
CR20190572A (es) 2020-05-23
JP2020526192A (ja) 2020-08-31
EP3649240A1 (en) 2020-05-13
CN110832077B (zh) 2025-01-03
CO2019014671A2 (es) 2020-01-17
US20200299691A1 (en) 2020-09-24
PE20200746A1 (es) 2020-07-24
IL271843A (en) 2020-02-27
JP2025185070A (ja) 2025-12-18
CN110832077A (zh) 2020-02-21
WO2019010274A1 (en) 2019-01-10
TN2019000308A1 (en) 2021-05-07
CA3061752A1 (en) 2019-01-10
US20190010494A1 (en) 2019-01-10
MX2019014800A (es) 2020-02-10
CL2020000019A1 (es) 2020-06-12
AU2018297262A1 (en) 2020-02-27
ECSP20000655A (es) 2020-05-29
KR20200024793A (ko) 2020-03-09
BR112019023650A2 (pt) 2020-06-02

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