KR20230171431A - Pnpla3 발현을 조절하기 위한 조성물 및 방법 - Google Patents

Pnpla3 발현을 조절하기 위한 조성물 및 방법 Download PDF

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KR20230171431A
KR20230171431A KR1020237034676A KR20237034676A KR20230171431A KR 20230171431 A KR20230171431 A KR 20230171431A KR 1020237034676 A KR1020237034676 A KR 1020237034676A KR 20237034676 A KR20237034676 A KR 20237034676A KR 20230171431 A KR20230171431 A KR 20230171431A
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seq
nos
dsirna
antisense
strand
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Korean (ko)
Inventor
우차브 삭세나
헨리크 티. 두데크
마크 에이브럼스
안톤 투라노브
밥 데일 브라운
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다이서나 파마수이티컬, 인크.
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Publication of KR20230171431A publication Critical patent/KR20230171431A/ko
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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
    • 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
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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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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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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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  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Liquid Crystal Substances (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020237034676A 2021-04-14 2022-04-13 Pnpla3 발현을 조절하기 위한 조성물 및 방법 Pending KR20230171431A (ko)

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 (1)

Publication Number Publication Date
KR20230171431A true KR20230171431A (ko) 2023-12-20

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Family Applications (1)

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KR1020237034676A Pending KR20230171431A (ko) 2021-04-14 2022-04-13 Pnpla3 발현을 조절하기 위한 조성물 및 방법

Country Status (13)

Country Link
US (2) US12084662B2 (https=)
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=)
WO (1) WO2022221430A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
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CA3209418A1 (en) 2021-04-14 2022-10-20 Utsav SAXENA Compositions and methods for modulating pnpla3 expression
WO2024112865A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
KR20250099265A (ko) 2022-11-23 2025-07-01 일라이 릴리 앤드 캄파니 표적화 리간드-접합된 뉴클레오티드 포스포르아미다이트의 합성 방법
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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Publication number Publication date
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
TWI857290B (zh) 2024-10-01
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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