CA3172117A1 - Compositions and methods for inhibiting angptl3 expression - Google Patents

Compositions and methods for inhibiting angptl3 expression Download PDF

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Publication number
CA3172117A1
CA3172117A1 CA3172117A CA3172117A CA3172117A1 CA 3172117 A1 CA3172117 A1 CA 3172117A1 CA 3172117 A CA3172117 A CA 3172117A CA 3172117 A CA3172117 A CA 3172117A CA 3172117 A1 CA3172117 A1 CA 3172117A1
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CA
Canada
Prior art keywords
oligonucleotide
angptl3
nucleotides
seq
length
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3172117A
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English (en)
French (fr)
Inventor
Bob D. Brown
Henryk T. Dudek
Utsav SAXENA
Marc Abrams
Anton Turanov
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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Filing date
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Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of CA3172117A1 publication Critical patent/CA3172117A1/en
Pending legal-status Critical Current

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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/10—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2310/00—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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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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
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CA3172117A 2020-03-18 2021-03-18 Compositions and methods for inhibiting angptl3 expression Pending CA3172117A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062991335P 2020-03-18 2020-03-18
US62/991,335 2020-03-18
PCT/US2021/022967 WO2021188795A1 (en) 2020-03-18 2021-03-18 Compositions and methods for inhibiting angptl3 expression

Publications (1)

Publication Number Publication Date
CA3172117A1 true CA3172117A1 (en) 2021-09-23

Family

ID=75478225

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3172117A Pending CA3172117A1 (en) 2020-03-18 2021-03-18 Compositions and methods for inhibiting angptl3 expression

Country Status (13)

Country Link
US (1) US20230287425A1 (enExample)
EP (1) EP4121536A1 (enExample)
JP (3) JP7398007B2 (enExample)
KR (1) KR20220156880A (enExample)
CN (1) CN116096889A (enExample)
AU (2) AU2021236674B2 (enExample)
BR (1) BR112022018667A2 (enExample)
CA (1) CA3172117A1 (enExample)
CL (1) CL2022002521A1 (enExample)
IL (1) IL296549A (enExample)
MX (1) MX2022011550A (enExample)
TW (2) TW202530410A (enExample)
WO (1) WO2021188795A1 (enExample)

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US20230383294A1 (en) * 2020-10-16 2023-11-30 Sanofi Novel rna compositions and methods for inhibiting angptl3
BR112023017737A2 (pt) 2021-03-04 2023-10-03 Alnylam Pharmaceuticals Inc Composições de irna de angiopoietina-semelhantes 3 (angptl3) e métodos de uso das mesmas
WO2023045994A1 (en) * 2021-09-23 2023-03-30 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of angiopoietin-like 3 (angptl3) protein
WO2023092102A1 (en) * 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof
EP4453210A1 (en) * 2021-12-22 2024-10-30 Regeneron Pharmaceuticals, Inc. Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors
JP2025512445A (ja) * 2022-04-15 2025-04-17 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｓｃａｐ活性を調節するための組成物及び方法
JP7812495B2 (ja) 2022-11-23 2026-02-09 イーライ リリー アンド カンパニー ４’－ホスフェート類似体ヌクレオチドホスホラミダイトを合成する方法
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Publication number Publication date
KR20220156880A (ko) 2022-11-28
TWI870563B (zh) 2025-01-21
MX2022011550A (es) 2023-01-04
IL296549A (en) 2022-11-01
JP2023518409A (ja) 2023-05-01
JP2024041748A (ja) 2024-03-27
TW202530410A (zh) 2025-08-01
WO2021188795A1 (en) 2021-09-23
US20230287425A1 (en) 2023-09-14
JP7749645B2 (ja) 2025-10-06
AU2021236674A1 (en) 2022-10-13
TW202200163A (zh) 2022-01-01
AU2021236674B2 (en) 2025-03-06
BR112022018667A2 (pt) 2022-11-29
JP7398007B2 (ja) 2023-12-13
CL2022002521A1 (es) 2023-05-19
EP4121536A1 (en) 2023-01-25
JP2026016378A (ja) 2026-02-03
CN116096889A (zh) 2023-05-09
AU2025203784A1 (en) 2025-06-19

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