WO2023069754A3 - Rnai constructs for inhibiting gpam expression and methods of use thereof - Google Patents

Rnai constructs for inhibiting gpam expression and methods of use thereof Download PDF

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Publication number
WO2023069754A3
WO2023069754A3 PCT/US2022/047491 US2022047491W WO2023069754A3 WO 2023069754 A3 WO2023069754 A3 WO 2023069754A3 US 2022047491 W US2022047491 W US 2022047491W WO 2023069754 A3 WO2023069754 A3 WO 2023069754A3
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Prior art keywords
gpam
methods
rnai constructs
expression
inhibiting
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PCT/US2022/047491
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French (fr)
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WO2023069754A2 (en
Inventor
Ingrid Rulifson
Bryan Meade
Jason C. LONG
Justin K. Murray
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Amgen Inc.
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Priority to AU2022370883A priority Critical patent/AU2022370883A1/en
Priority to KR1020247016487A priority patent/KR20240090496A/en
Priority to CA3235262A priority patent/CA3235262A1/en
Priority to IL311839A priority patent/IL311839A/en
Publication of WO2023069754A2 publication Critical patent/WO2023069754A2/en
Publication of WO2023069754A3 publication Critical patent/WO2023069754A3/en

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    • 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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
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    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
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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
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    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
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  • Genetics & Genomics (AREA)
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  • Biomedical Technology (AREA)
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  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The disclosure relates to RNAi constructs, such as siRNA, for reducing expression of the GPAM gene. Methods of using such RNAi constructs to treat or prevent liver disease, such as nonalcoholic fatty liver disease (NAFLD), are also described.
PCT/US2022/047491 2021-10-22 2022-10-21 Rnai constructs for inhibiting gpam expression and methods of use thereof WO2023069754A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2022370883A AU2022370883A1 (en) 2021-10-22 2022-10-21 Rnai constructs for inhibiting gpam expression and methods of use thereof
KR1020247016487A KR20240090496A (en) 2021-10-22 2022-10-21 RNAI constructs for suppressing GPAM expression and methods of using the same
CA3235262A CA3235262A1 (en) 2021-10-22 2022-10-21 Rnai constructs for inhibiting gpam expression and methods of use thereof
IL311839A IL311839A (en) 2021-10-22 2022-10-21 Rnai constructs for inhibiting gpam expression and methods of use thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163270813P 2021-10-22 2021-10-22
US63/270,813 2021-10-22

Publications (2)

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WO2023069754A2 WO2023069754A2 (en) 2023-04-27
WO2023069754A3 true WO2023069754A3 (en) 2023-05-25

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PCT/US2022/047491 WO2023069754A2 (en) 2021-10-22 2022-10-21 Rnai constructs for inhibiting gpam expression and methods of use thereof

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KR (1) KR20240090496A (en)
AU (1) AU2022370883A1 (en)
CA (1) CA3235262A1 (en)
IL (1) IL311839A (en)
WO (1) WO2023069754A2 (en)

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US20160271137A1 (en) * 2010-02-18 2016-09-22 The Trustees Of Princeton University Inhibitors of long and very long chain fatty acid metabolism as broad spectrum anti-virals

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Publication number Publication date
IL311839A (en) 2024-05-01
WO2023069754A2 (en) 2023-04-27
CA3235262A1 (en) 2023-04-27
KR20240090496A (en) 2024-06-21
AU2022370883A1 (en) 2024-04-18

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