AU2021381363A1 - Compounds and methods for modulating angiotensinogen expression - Google Patents

Compounds and methods for modulating angiotensinogen expression Download PDF

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Publication number
AU2021381363A1
AU2021381363A1 AU2021381363A AU2021381363A AU2021381363A1 AU 2021381363 A1 AU2021381363 A1 AU 2021381363A1 AU 2021381363 A AU2021381363 A AU 2021381363A AU 2021381363 A AU2021381363 A AU 2021381363A AU 2021381363 A1 AU2021381363 A1 AU 2021381363A1
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Prior art keywords
oligomeric compound
modified
sugar moiety
certain embodiments
nucleosides
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AU2021381363A
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AU2021381363A9 (en
Inventor
Susan M. Freier
Adam Mullick
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of AU2021381363A1 publication Critical patent/AU2021381363A1/en
Publication of AU2021381363A9 publication Critical patent/AU2021381363A9/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • A—HUMAN NECESSITIES
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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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    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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AU2021381363A 2020-11-18 2021-11-18 Compounds and methods for modulating angiotensinogen expression Pending AU2021381363A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063115499P 2020-11-18 2020-11-18
US63/115,499 2020-11-18
US202163232109P 2021-08-11 2021-08-11
US63/232,109 2021-08-11
PCT/US2021/059896 WO2022109139A1 (en) 2020-11-18 2021-11-18 Compounds and methods for modulating angiotensinogen expression

Publications (2)

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AU2021381363A1 true AU2021381363A1 (en) 2023-06-15
AU2021381363A9 AU2021381363A9 (en) 2025-01-09

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AU2021381363A Pending AU2021381363A1 (en) 2020-11-18 2021-11-18 Compounds and methods for modulating angiotensinogen expression

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US (2) US11447521B2 (https=)
EP (2) EP4136092B1 (https=)
JP (1) JP7820374B2 (https=)
KR (1) KR20230108728A (https=)
CN (3) CN116615540B (https=)
AU (1) AU2021381363A1 (https=)
CA (1) CA3201661A1 (https=)
ES (1) ES2990026T3 (https=)
HU (1) HUE068561T2 (https=)
IL (1) IL302817A (https=)
LT (1) LT4136092T (https=)
MX (1) MX2023005736A (https=)
PL (1) PL4136092T3 (https=)
SI (1) SI4136092T1 (https=)
TW (1) TW202227101A (https=)
WO (1) WO2022109139A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4587573A2 (en) * 2022-09-16 2025-07-23 Sirnaomics, Inc. Products and compositions
WO2025097040A1 (en) * 2023-11-03 2025-05-08 Ionis Pharmaceuticals, Inc. Methods and compositions for reducing angiotensinogen

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