CA3034463C - ARNI CONSTRUCTIONS THAT INHIBIT ASGR1 EXPRESSION AND THEIR METHODS OF USE - Google Patents

ARNI CONSTRUCTIONS THAT INHIBIT ASGR1 EXPRESSION AND THEIR METHODS OF USE

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Publication number
CA3034463C
CA3034463C CA3034463A CA3034463A CA3034463C CA 3034463 C CA3034463 C CA 3034463C CA 3034463 A CA3034463 A CA 3034463A CA 3034463 A CA3034463 A CA 3034463A CA 3034463 C CA3034463 C CA 3034463C
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Prior art keywords
rnai construct
sense strand
seq
sequence
phos
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CA3034463A
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English (en)
French (fr)
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CA3034463A1 (en
Inventor
Michael OLLMANN
Yang Li
Jun Zhang
Kaustav Biswas
Oliver HOMANN
Leslie P. Miranda
Justin K. Murray
Bin Wu
Oh Kyu YOON
John Gordon Allen
Chawita Netirojjanakul
Yuan Cheng
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Amgen Inc
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1138Non-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 receptors or cell surface proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2851Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the lectin superfamily, e.g. CD23, CD72
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52Constant or Fc region; Isotype
    • C07K2317/526CH3 domain
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Obesity (AREA)
  • Vascular Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3034463A 2016-08-26 2017-08-25 ARNI CONSTRUCTIONS THAT INHIBIT ASGR1 EXPRESSION AND THEIR METHODS OF USE Active CA3034463C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662380216P 2016-08-26 2016-08-26
US62/380,216 2016-08-26
PCT/US2017/048757 WO2018039647A1 (en) 2016-08-26 2017-08-25 Rnai constructs for inhibiting asgr1 expression and methods of use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3277899 Division 2017-08-25

Publications (2)

Publication Number Publication Date
CA3034463A1 CA3034463A1 (en) 2018-03-01
CA3034463C true CA3034463C (en) 2025-09-16

Family

ID=59846644

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3034463A Active CA3034463C (en) 2016-08-26 2017-08-25 ARNI CONSTRUCTIONS THAT INHIBIT ASGR1 EXPRESSION AND THEIR METHODS OF USE

Country Status (10)

Country Link
US (2) US10870856B2 (cg-RX-API-DMAC7.html)
EP (1) EP3504333A1 (cg-RX-API-DMAC7.html)
JP (3) JP7082610B2 (cg-RX-API-DMAC7.html)
AR (1) AR110629A1 (cg-RX-API-DMAC7.html)
AU (2) AU2017315952B2 (cg-RX-API-DMAC7.html)
CA (1) CA3034463C (cg-RX-API-DMAC7.html)
MX (2) MX2019002271A (cg-RX-API-DMAC7.html)
TW (1) TW201823461A (cg-RX-API-DMAC7.html)
UY (1) UY37376A (cg-RX-API-DMAC7.html)
WO (1) WO2018039647A1 (cg-RX-API-DMAC7.html)

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WO2019079294A1 (en) * 2017-10-17 2019-04-25 Arrowhead Pharmaceuticals, Inc. RNAI-BASED AGENTS AND COMPOSITIONS FOR INHIBITING ASIALOGLYCOPROTEIN RECEPTOR 1 RECEPTOR 1
AU2018360697A1 (en) * 2017-11-01 2020-05-14 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
KR102798620B1 (ko) * 2018-06-07 2025-04-22 암젠 인크 단백질-폴리뉴클레오티드 접합체에 대한 검출 검정
EP3894554A1 (en) * 2018-12-10 2021-10-20 Amgen Inc. Chemically-modified rnai constructs and uses thereof
SG11202113112WA (en) * 2019-05-30 2021-12-30 Amgen Inc Rnai constructs for inhibiting scap expression and methods of use thereof
EP3990465A1 (en) 2019-06-25 2022-05-04 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
US20230314417A1 (en) 2020-03-23 2023-10-05 Amgen Inc. Monoclonal antibodies to chemically-modified nucleic acids and uses thereof
CN111705063B (zh) * 2020-06-02 2023-08-08 成都中科奥格生物科技有限公司 Asgr1突变基因及其在制备哺乳动物肝损伤敏感模型中的应用
WO2024046297A1 (zh) * 2022-09-02 2024-03-07 北京福元医药股份有限公司 抑制去唾液酸糖蛋白受体基因表达的siRNA、其缀合物和药物组合物及用途

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Also Published As

Publication number Publication date
US11732266B2 (en) 2023-08-22
JP2019524151A (ja) 2019-09-05
AU2017315952A1 (en) 2019-03-14
MX2024009873A (es) 2024-08-20
US20210108212A1 (en) 2021-04-15
CA3034463A1 (en) 2018-03-01
AR110629A1 (es) 2019-04-17
MX2019002271A (es) 2019-06-20
TW201823461A (zh) 2018-07-01
JP7082610B2 (ja) 2022-06-08
UY37376A (es) 2018-03-23
JP2024019733A (ja) 2024-02-09
JP7472180B2 (ja) 2024-04-22
AU2017315952B2 (en) 2023-11-30
JP2022060357A (ja) 2022-04-14
EP3504333A1 (en) 2019-07-03
AU2024201349A1 (en) 2024-04-11
US20190309306A1 (en) 2019-10-10
US10870856B2 (en) 2020-12-22
WO2018039647A1 (en) 2018-03-01

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