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
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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
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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
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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
    • 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
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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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
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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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (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 (https=)
EP (1) EP4121536A1 (https=)
JP (3) JP7398007B2 (https=)
KR (1) KR20220156880A (https=)
CN (1) CN116096889A (https=)
AU (2) AU2021236674B2 (https=)
BR (1) BR112022018667A2 (https=)
CA (1) CA3172117A1 (https=)
CL (1) CL2022002521A1 (https=)
IL (1) IL296549A (https=)
MX (1) MX2022011550A (https=)
TW (2) TWI870563B (https=)
WO (1) WO2021188795A1 (https=)

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WO2022079222A1 (en) * 2020-10-16 2022-04-21 Sanofi Novel rna compositions and methods for inhibiting angptl3
JP2024508896A (ja) 2021-03-04 2024-02-28 アルナイラム ファーマシューティカルズ， インコーポレイテッド アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびその使用方法
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
US20230374522A1 (en) * 2022-04-15 2023-11-23 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating scap activity
KR20250099265A (ko) 2022-11-23 2025-07-01 일라이 릴리 앤드 캄파니 표적화 리간드-접합된 뉴클레오티드 포스포르아미다이트의 합성 방법
WO2024112865A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
KR20250124879A (ko) 2022-12-22 2025-08-20 일라이 릴리 앤드 캄파니 올리고뉴클레오티드 단편 및 이를 사용하여 rnai 작용제를 제조하는 방법
WO2024151772A2 (en) * 2023-01-11 2024-07-18 Sirius Therapeutics, Inc. Polynucleic acid molecules for inhibiting expression of angptl3, pharmaceutical compositions, and uses thereof
WO2024175550A1 (en) * 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
CN118599845A (zh) * 2023-06-27 2024-09-06 施能康医药科技(苏州)有限公司 靶向人血管生成素样蛋白3的核酸及其用途
WO2025096970A2 (en) * 2023-11-03 2025-05-08 Basecure Therapeutics Llc Modified oligonucleotides and methods of use thereof
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Also Published As

Publication number Publication date
KR20220156880A (ko) 2022-11-28
CL2022002521A1 (es) 2023-05-19
JP7398007B2 (ja) 2023-12-13
JP2023518409A (ja) 2023-05-01
JP7749645B2 (ja) 2025-10-06
BR112022018667A2 (pt) 2022-11-29
WO2021188795A1 (en) 2021-09-23
IL296549A (en) 2022-11-01
TWI870563B (zh) 2025-01-21
MX2022011550A (es) 2023-01-04
TW202200163A (zh) 2022-01-01
EP4121536A1 (en) 2023-01-25
AU2025203784A1 (en) 2025-06-19
TW202530410A (zh) 2025-08-01
AU2021236674B2 (en) 2025-03-06
JP2026016378A (ja) 2026-02-03
AU2021236674A1 (en) 2022-10-13
CN116096889A (zh) 2023-05-09
US20230287425A1 (en) 2023-09-14
JP2024041748A (ja) 2024-03-27

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