AU2021320550B2 - Compositions and methods for inhibiting lpa expression - Google Patents

Compositions and methods for inhibiting lpa expression

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Publication number
AU2021320550B2
AU2021320550B2 AU2021320550A AU2021320550A AU2021320550B2 AU 2021320550 B2 AU2021320550 B2 AU 2021320550B2 AU 2021320550 A AU2021320550 A AU 2021320550A AU 2021320550 A AU2021320550 A AU 2021320550A AU 2021320550 B2 AU2021320550 B2 AU 2021320550B2
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Australia
Prior art keywords
lpa
oligonucleotide
galnac
seq
ome
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AU2021320550A
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AU2021320550A1 (en
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Marc Abrams
Bob Dale Brown
Henryk T. Dudek
Wen HAN
Anton Turanov
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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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
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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
    • C12N15/1137—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 enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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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)
  • Saccharide Compounds (AREA)
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AU2021320550A 2020-08-05 2021-08-05 Compositions and methods for inhibiting lpa expression Active AU2021320550B2 (en)

Priority Applications (1)

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AU2025252633A AU2025252633A1 (en) 2020-08-05 2025-10-17 Compositions and methods for inhibiting lpa expression

Applications Claiming Priority (5)

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US202063061676P 2020-08-05 2020-08-05
US63/061,676 2020-08-05
US202063074779P 2020-09-04 2020-09-04
US63/074,779 2020-09-04
PCT/US2021/071109 WO2022032288A1 (en) 2020-08-05 2021-08-05 Compositions and methods for inhibiting lpa expression

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AU2021320550B2 true AU2021320550B2 (en) 2025-11-06

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US (3) US12435336B2 (enExample)
EP (1) EP4192954A1 (enExample)
JP (3) JP7476422B2 (enExample)
KR (2) KR20250077604A (enExample)
CN (1) CN116323943A (enExample)
AU (2) AU2021320550B2 (enExample)
BR (1) BR112023001957A2 (enExample)
CA (1) CA3185348A1 (enExample)
IL (1) IL300338A (enExample)
MX (1) MX2023001538A (enExample)
TW (2) TWI886314B (enExample)
WO (1) WO2022032288A1 (enExample)

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CN122514601A (zh) * 2023-11-29 2026-08-04 美国圣因生物股份有限公司 靶向Lp(a)的小干扰RNA及其用途
CN120098993A (zh) * 2023-12-05 2025-06-06 广东东阳光药业股份有限公司 一种新型的双链siRNA、其缀合物及其用途
TW202546233A (zh) * 2024-02-05 2025-12-01 美商靖因藥業(上海)有限公司 用於抑制lp(a)或apoc3之表現的多核酸分子、醫藥組合物及其用途
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