JP7877355B2 - Ｐｎｐｌａ３発現を調節するための組成物及び方法 - Google Patents

Ｐｎｐｌａ３発現を調節するための組成物及び方法

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Publication number
JP7877355B2
JP7877355B2 JP2023562909A JP2023562909A JP7877355B2 JP 7877355 B2 JP7877355 B2 JP 7877355B2 JP 2023562909 A JP2023562909 A JP 2023562909A JP 2023562909 A JP2023562909 A JP 2023562909A JP 7877355 B2 JP7877355 B2 JP 7877355B2
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strand
sequence
dsirna
antisense
sense
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JP2024514880A (ja
JP2024514880A5 (enExample
Inventor
ユトサフ サクセナ
ヘンリク ティー デュデック
マーク エイブラムス
アントン トゥラノフ
ボブ デール ブラウン
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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
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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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
    • C—CHEMISTRY; METALLURGY
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    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides

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  • Wood Science & Technology (AREA)
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  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Microbiology (AREA)
  • Epidemiology (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Liquid Crystal Substances (AREA)
JP2023562909A 2021-04-14 2022-04-13 Ｐｎｐｌａ３発現を調節するための組成物及び方法 Active JP7877355B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163174932P 2021-04-14 2021-04-14
US63/174,932 2021-04-14
PCT/US2022/024657 WO2022221430A1 (en) 2021-04-14 2022-04-13 Compositions and methods for modulating pnpla3 expression

Publications (3)

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JP2024514880A JP2024514880A (ja) 2024-04-03
JP2024514880A5 JP2024514880A5 (enExample) 2025-04-11
JP7877355B2 true JP7877355B2 (ja) 2026-06-22

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US (2) US12084662B2 (enExample)
EP (1) EP4323519A1 (enExample)
JP (1) JP7877355B2 (enExample)
KR (1) KR20230171431A (enExample)
CN (1) CN117120613A (enExample)
AR (1) AR125351A1 (enExample)
AU (1) AU2022258459A1 (enExample)
BR (1) BR112023021109A2 (enExample)
CA (1) CA3209418A1 (enExample)
IL (1) IL307625A (enExample)
MX (1) MX2023012216A (enExample)
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WO (1) WO2022221430A1 (enExample)

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KR20230171431A (ko) 2021-04-14 2023-12-20 다이서나 파마수이티컬, 인크. Pnpla3 발현을 조절하기 위한 조성물 및 방법
EP4622982A1 (en) 2022-11-23 2025-10-01 Eli Lilly and Company Methods of synthesizing a targeting ligand-conjugated nucleotide phosphoramidite
JP7812495B2 (ja) 2022-11-23 2026-02-09 イーライ リリー アンド カンパニー ４’－ホスフェート類似体ヌクレオチドホスホラミダイトを合成する方法
WO2024182446A2 (en) * 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
WO2026130520A1 (zh) * 2024-12-20 2026-06-25 正大天晴药业集团股份有限公司 靶向pnpla3的双链核糖核酸

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US12084662B2 (en) 2024-09-10
TW202500748A (zh) 2025-01-01
MX2023012216A (es) 2023-10-26
TW202300645A (zh) 2023-01-01
IL307625A (en) 2023-12-01
KR20230171431A (ko) 2023-12-20
AU2022258459A1 (en) 2023-09-14
US20220364098A1 (en) 2022-11-17
CN117120613A (zh) 2023-11-24
AR125351A1 (es) 2023-07-12
US20240401059A1 (en) 2024-12-05
EP4323519A1 (en) 2024-02-21
WO2022221430A1 (en) 2022-10-20
BR112023021109A2 (pt) 2023-12-12
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