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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CA
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Prior art keywords
rnai construct
sense strand
seq
sequence
phos
Prior art date
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Application number
CA3034463A
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English (en)
French (fr)
Other versions
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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Application filed by Amgen Inc filed Critical Amgen Inc
Priority to CA3277899A priority Critical patent/CA3277899A1/en
Publication of CA3034463A1 publication Critical patent/CA3034463A1/en
Application granted granted Critical
Publication of CA3034463C publication Critical patent/CA3034463C/en
Active legal-status Critical Current
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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2851—Immunoglobulins [IG], 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52—Constant or Fc region; Isotype
    • C07K2317/526—CH3 domain
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide

Landscapes

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

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3277899A CA3277899A1 (en) 2016-08-26 2017-08-25 Rnai constructs for inhibiting asgr1 expression and methods of use thereof

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
CA3277899A Division CA3277899A1 (en) 2016-08-26 2017-08-25 Rnai constructs for inhibiting asgr1 expression and methods of use thereof

Publications (2)

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

Family

ID=59846644

Family Applications (2)

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
CA3277899A Pending CA3277899A1 (en) 2016-08-26 2017-08-25 Rnai constructs for inhibiting asgr1 expression and methods of use thereof

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3277899A Pending CA3277899A1 (en) 2016-08-26 2017-08-25 Rnai constructs for inhibiting asgr1 expression and methods of use thereof

Country Status (10)

Country Link
US (2) US10870856B2 (enExample)
EP (1) EP3504333A1 (enExample)
JP (4) JP7082610B2 (enExample)
AR (1) AR110629A1 (enExample)
AU (2) AU2017315952B2 (enExample)
CA (2) CA3034463C (enExample)
MX (2) MX2019002271A (enExample)
TW (1) TW201823461A (enExample)
UY (1) UY37376A (enExample)
WO (1) WO2018039647A1 (enExample)

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AU2016332900C1 (en) * 2015-09-29 2024-07-04 Amgen Inc. ASGR inhibitors
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CN111212909A (zh) * 2017-10-17 2020-05-29 箭头药业股份有限公司 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物
CA3078971A1 (en) * 2017-11-01 2019-05-09 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
SG11202105900UA (en) * 2018-12-10 2021-07-29 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
CA3143047A1 (en) 2019-06-25 2020-12-30 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
EP4126967A1 (en) 2020-03-23 2023-02-08 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
KR20240082367A (ko) * 2021-09-23 2024-06-10 아르네이 사이언시스 엘엘씨 치료학적 물질로서 고리형 구조의 올리고뉴클레오티드
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
WO2025231448A2 (en) * 2024-05-03 2025-11-06 University Of Massachusetts Oligonucleotides targeting myostatin (mstn)

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Also Published As

Publication number Publication date
JP7082610B2 (ja) 2022-06-08
JP2022060357A (ja) 2022-04-14
JP7472180B2 (ja) 2024-04-22
US11732266B2 (en) 2023-08-22
WO2018039647A1 (en) 2018-03-01
AU2024201349B2 (en) 2026-07-30
CA3277899A1 (en) 2026-03-02
US20210108212A1 (en) 2021-04-15
CA3034463A1 (en) 2018-03-01
JP2024019733A (ja) 2024-02-09
TW201823461A (zh) 2018-07-01
US20190309306A1 (en) 2019-10-10
AU2024201349A1 (en) 2024-04-11
JP2019524151A (ja) 2019-09-05
EP3504333A1 (en) 2019-07-03
AU2017315952A1 (en) 2019-03-14
MX2024009873A (es) 2024-08-20
AU2017315952B2 (en) 2023-11-30
AR110629A1 (es) 2019-04-17
JP2026083134A (ja) 2026-05-19
JP7873261B2 (ja) 2026-06-11
US10870856B2 (en) 2020-12-22
UY37376A (es) 2018-03-23
MX2019002271A (es) 2019-06-20

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