JP2025512771A - ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法 - Google Patents

ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法 Download PDF

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JP2025512771A
JP2025512771A JP2024555941A JP2024555941A JP2025512771A JP 2025512771 A JP2025512771 A JP 2025512771A JP 2024555941 A JP2024555941 A JP 2024555941A JP 2024555941 A JP2024555941 A JP 2024555941A JP 2025512771 A JP2025512771 A JP 2025512771A
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Prior art keywords
rnai construct
pnpla3
seq
nucleotides
antisense strand
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JP2024555941A
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Japanese (ja)
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JP2025512771A5 (https=
Inventor
ブライアン ミード，
ジャスティン ケー． マリー，
イングリッド ルリフソン，
オリバー ホーマン，
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Amgen Inc
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Amgen Inc
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Publication of JP2025512771A publication Critical patent/JP2025512771A/ja
Publication of JP2025512771A5 publication Critical patent/JP2025512771A5/ja
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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01051—1-Acylglycerol-3-phosphate O-acyltransferase (2.3.1.51)
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01004—Phospholipase A2 (3.1.1.4)
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/0104—Orsellinate-depside hydrolase (3.1.1.40)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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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
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024555941A 2022-03-23 2023-03-21 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法 Pending JP2025512771A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263322845P 2022-03-23 2022-03-23
US63/322,845 2022-03-23
PCT/US2023/064765 WO2023183801A2 (en) 2022-03-23 2023-03-21 Rnai constructs for inhibiting pnpla3 expression and methods of use thereof

Publications (2)

Publication Number Publication Date
JP2025512771A true JP2025512771A (ja) 2025-04-22
JP2025512771A5 JP2025512771A5 (https=) 2026-03-30

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JP2024555941A Pending JP2025512771A (ja) 2022-03-23 2023-03-21 ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト及びその使用方法

Country Status (13)

Country Link
US (1) US20250207138A1 (https=)
EP (1) EP4496893A2 (https=)
JP (1) JP2025512771A (https=)
KR (1) KR20240162559A (https=)
CN (1) CN118891364A (https=)
AR (1) AR128877A1 (https=)
AU (1) AU2023237880A1 (https=)
CA (1) CA3254112A1 (https=)
CL (1) CL2024002802A1 (https=)
IL (1) IL315078A (https=)
MX (1) MX2024011595A (https=)
TW (1) TW202345869A (https=)
WO (1) WO2023183801A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025252063A1 (zh) * 2024-06-04 2025-12-11 正大天晴药业集团股份有限公司 靶向pnpla3的双链核糖核酸

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2976445A1 (en) * 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
TW202542311A (zh) * 2018-09-19 2025-11-01 美商Ionis製藥公司 Pnpla3表現之調節劑

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Publication number Publication date
CN118891364A (zh) 2024-11-01
IL315078A (en) 2024-10-01
MX2024011595A (es) 2024-11-08
US20250207138A1 (en) 2025-06-26
AU2023237880A1 (en) 2024-09-12
TW202345869A (zh) 2023-12-01
CL2024002802A1 (es) 2025-02-07
AR128877A1 (es) 2024-06-19
KR20240162559A (ko) 2024-11-15
EP4496893A2 (en) 2025-01-29
WO2023183801A2 (en) 2023-09-28
WO2023183801A3 (en) 2023-11-02
CA3254112A1 (en) 2023-09-28

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