TWI857290B - 用於調節pnpla3表現之組合物及方法 - Google Patents

用於調節pnpla3表現之組合物及方法 Download PDF

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TWI857290B
TWI857290B TW111114009A TW111114009A TWI857290B TW I857290 B TWI857290 B TW I857290B TW 111114009 A TW111114009 A TW 111114009A TW 111114009 A TW111114009 A TW 111114009A TW I857290 B TWI857290 B TW I857290B
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pnpla3
nucleotides
oligonucleotide
oligonucleotides
expression
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TW111114009A
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TW202300645A (zh
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烏薩夫 薩克森納
亨立克 T 杜迪克
馬克 艾布蘭
安東 杜拉諾
鮑伯 戴爾 布朗
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美商戴瑟納製藥股份有限公司
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07H21/02Compounds 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
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    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
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    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides

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TW111114009A 2021-04-14 2022-04-13 用於調節pnpla3表現之組合物及方法 TWI857290B (zh)

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US202163174932P 2021-04-14 2021-04-14
US63/174,932 2021-04-14

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EP (1) EP4323519A1 (https=)
JP (1) JP2024514880A (https=)
KR (1) KR20230171431A (https=)
CN (1) CN117120613A (https=)
AR (1) AR125351A1 (https=)
AU (1) AU2022258459A1 (https=)
BR (1) BR112023021109A2 (https=)
CA (1) CA3209418A1 (https=)
IL (1) IL307625A (https=)
MX (1) MX2023012216A (https=)
TW (2) TWI857290B (https=)
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WO2024112865A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
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WO2024182446A2 (en) * 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof

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US12084662B2 (en) 2024-09-10
EP4323519A1 (en) 2024-02-21
CA3209418A1 (en) 2022-10-20
US20220364098A1 (en) 2022-11-17
TW202500748A (zh) 2025-01-01
US20240401059A1 (en) 2024-12-05
KR20230171431A (ko) 2023-12-20
BR112023021109A2 (pt) 2023-12-12
CN117120613A (zh) 2023-11-24
TW202300645A (zh) 2023-01-01
IL307625A (en) 2023-12-01
JP2024514880A (ja) 2024-04-03
WO2022221430A1 (en) 2022-10-20
AR125351A1 (es) 2023-07-12
MX2023012216A (es) 2023-10-26
AU2022258459A1 (en) 2023-09-14

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