IL315078A - RNAI constructs for inhibiting PNPLA3 expression and methods for using them - Google Patents

RNAI constructs for inhibiting PNPLA3 expression and methods for using them

Info

Publication number
IL315078A
IL315078A IL315078A IL31507824A IL315078A IL 315078 A IL315078 A IL 315078A IL 315078 A IL315078 A IL 315078A IL 31507824 A IL31507824 A IL 31507824A IL 315078 A IL315078 A IL 315078A
Authority
IL
Israel
Prior art keywords
methods
rnai constructs
pnpla3 expression
inhibiting pnpla3
inhibiting
Prior art date
Application number
IL315078A
Other languages
English (en)
Hebrew (he)
Inventor
Bryan Meade
Justin K Murray
Ingrid Rulifson
Oliver Homann
Original Assignee
Amgen Inc
Bryan Meade
Justin K Murray
Ingrid Rulifson
Oliver Homann
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Amgen Inc, Bryan Meade, Justin K Murray, Ingrid Rulifson, Oliver Homann filed Critical Amgen Inc
Publication of IL315078A publication Critical patent/IL315078A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • 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)
IL315078A 2022-03-23 2023-03-21 RNAI constructs for inhibiting PNPLA3 expression and methods for using them IL315078A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263322845P 2022-03-23 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 (1)

Publication Number Publication Date
IL315078A true IL315078A (en) 2024-10-01

Family

ID=88102183

Family Applications (1)

Application Number Title Priority Date Filing Date
IL315078A IL315078A (en) 2022-03-23 2023-03-21 RNAI constructs for inhibiting PNPLA3 expression and methods for using them

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

Also Published As

Publication number Publication date
CN118891364A (zh) 2024-11-01
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
JP2025512771A (ja) 2025-04-22
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

Similar Documents

Publication Publication Date Title
GB202019709D0 (en) Anti-Coronavirus antibodies and methods of use
IL288398A (en) rnai constructs to prevent scap expression and methods for using them
IL312348A (en) HIF-2ALPHA inhibitors and methods of using it
IL287973A (en) acss2 inhibitors and methods of their use
IL316619A (en) PI3Kα inhibitors and methods of using them
IL314320A (en) Compounds and methods of use
IL321357A (en) KIF18A inhibitors and their uses
IL292860B1 (en) EGFR allosteric inhibitors and methods of using them
IL318393A (en) Azaquinazoline compounds and methods of use
IL315078A (en) RNAI constructs for inhibiting PNPLA3 expression and methods for using them
SI4192814T1 (sl) Zaviralci transglutaminaz
IL317288A (en) Inhibitors of expression and/or function
IL318694A (en) Antibodies against CCR8 and methods of using them
IL317825A (en) RNA interference constructs and methods for inhibiting fam13a expression
IL314277A (en) Anti-B7-H3 compounds and methods of use
SI4192813T1 (sl) Zaviralci transglutaminaz
IL316643A (en) RNA interference constructs for inhibiting scap expression and methods for using them
IL300104A (en) Antibody specific for mucin-1 and methods of using it
IL321194A (en) RNA interference constructs for inhibiting TTR expression and methods for using them
IL311839A (en) RNA interference constructs to inhibit gpam expression and methods for using them
HK40091690A (en) Rnai constructs for inhibiting hsd17b13 expression and methods of use thereof
HK40115744A (en) Rnai constructs for inhibiting gpam expression and methods of use thereof
HK40129741A (en) Rnai constructs for inhibiting ttr expression and methods of use thereof
CA3276645A1 (en) Rnai constructs for inhibiting ttr expression and methods of use thereof
GB202119051D0 (en) Wingsail and method of use