IL300338A - Compounds and methods for inhibiting the expression of LPA - Google Patents

Compounds and methods for inhibiting the expression of LPA

Info

Publication number
IL300338A
IL300338A IL300338A IL30033823A IL300338A IL 300338 A IL300338 A IL 300338A IL 300338 A IL300338 A IL 300338A IL 30033823 A IL30033823 A IL 30033823A IL 300338 A IL300338 A IL 300338A
Authority
IL
Israel
Prior art keywords
seq
lpa
nucleotides
oligonucleotide
antisense strand
Prior art date
Application number
IL300338A
Other languages
English (en)
Hebrew (he)
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of IL300338A publication Critical patent/IL300338A/en

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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
    • 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
    • C12N15/1137—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21007—Plasmin (3.4.21.7), i.e. fibrinolysin
    • 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
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/32—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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/35—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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • 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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
IL300338A 2020-08-05 2021-08-05 Compounds and methods for inhibiting the expression of LPA IL300338A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063061676P 2020-08-05 2020-08-05
US202063074779P 2020-09-04 2020-09-04
PCT/US2021/071109 WO2022032288A1 (en) 2020-08-05 2021-08-05 Compositions and methods for inhibiting lpa expression

Publications (1)

Publication Number Publication Date
IL300338A true IL300338A (en) 2023-04-01

Family

ID=77519885

Family Applications (1)

Application Number Title Priority Date Filing Date
IL300338A IL300338A (en) 2020-08-05 2021-08-05 Compounds and methods for inhibiting the expression of LPA

Country Status (12)

Country Link
US (2) US12435336B2 (enExample)
EP (1) EP4192954A1 (enExample)
JP (2) JP7476422B2 (enExample)
KR (2) KR20250077604A (enExample)
CN (1) CN116323943A (enExample)
AU (2) AU2021320550B2 (enExample)
BR (1) BR112023001957A2 (enExample)
CA (1) CA3185348A1 (enExample)
IL (1) IL300338A (enExample)
MX (1) MX2023001538A (enExample)
TW (2) TW202535432A (enExample)
WO (1) WO2022032288A1 (enExample)

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EP4192954A1 (en) 2020-08-05 2023-06-14 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting lpa expression
EP4450624A4 (en) * 2021-12-16 2025-03-26 Tuojie Biotech (Shanghai) Co., Ltd. LPA-TARGETTING SIRNA AND CONJUGATE
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
CA3245064A1 (en) * 2022-04-15 2023-10-19 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF MODULATING THE ACTIVITY OF STRUCTURAL AND CYTOSKELETON-ASSOCIATED PROTEINS
CN120076811A (zh) * 2022-08-11 2025-05-30 靖因药业有限公司 用于抑制Lp(a)的表达的多核酸分子、药物组合物及其用途
CN115851723B (zh) * 2022-10-24 2023-10-03 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用
WO2024112865A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
KR20250099265A (ko) 2022-11-23 2025-07-01 일라이 릴리 앤드 캄파니 표적화 리간드-접합된 뉴클레오티드 포스포르아미다이트의 합성 방법
AU2023408776A1 (en) 2022-12-22 2025-07-10 Eli Lilly And Company Oligonucleotide fragments and methods of making rnai agents using the same
KR20250131826A (ko) 2023-01-13 2025-09-03 일라이 릴리 앤드 캄파니 치료 올리고뉴클레오티드-함유 제약 조성물 및 그를 사용한 투여 요법
WO2024197065A2 (en) * 2023-03-20 2024-09-26 Verve Therapeutics, Inc. In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease
CN118685405A (zh) * 2023-06-20 2024-09-24 施能康医药科技(苏州)有限公司 靶向脂蛋白a的核酸及其用途
WO2025064821A2 (en) 2023-09-21 2025-03-27 Ionis Pharmaceuticals, Inc. Compounds and methods for inhibiting lpa
WO2025082058A1 (zh) * 2023-10-16 2025-04-24 润佳(上海)医药技术有限公司 一种抑制LPA基因表达的dsRNA及其用途
CN120344664A (zh) * 2023-10-27 2025-07-18 北京安龙生物医药有限公司 靶向脂蛋白a的寡核苷酸及其用途
CN120020252A (zh) * 2023-11-17 2025-05-20 纳肽得(青岛)生物医药有限公司 抑制LPA表达的siRNA及其用途
WO2025117709A2 (en) * 2023-11-29 2025-06-05 Sanegene Bio Usa Inc. Small interfering rna targeting lp(a) and uses thereof
WO2025119212A1 (zh) * 2023-12-05 2025-06-12 广东东阳光药业股份有限公司 一种新型的双链siRNA、其缀合物及其用途
WO2025170868A1 (en) * 2024-02-05 2025-08-14 Sirius Therapeutics, Inc. Polynucleic acid molecules for inhibiting expression of lp(a) or apoc3, pharmaceutical compositions, and uses thereof

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Also Published As

Publication number Publication date
TW202535432A (zh) 2025-09-16
US20240344063A1 (en) 2024-10-17
BR112023001957A2 (pt) 2023-04-25
AU2021320550A1 (en) 2023-02-16
MX2023001538A (es) 2023-03-08
KR102812602B1 (ko) 2025-05-28
EP4192954A1 (en) 2023-06-14
WO2022032288A1 (en) 2022-02-10
JP2023536974A (ja) 2023-08-30
CA3185348A1 (en) 2022-02-10
KR20250077604A (ko) 2025-05-30
US20240401053A1 (en) 2024-12-05
AU2021320550B2 (en) 2025-11-06
US12435336B2 (en) 2025-10-07
AU2025252633A1 (en) 2025-11-27
JP2024081668A (ja) 2024-06-18
TWI886314B (zh) 2025-06-11
US12252691B2 (en) 2025-03-18
CN116323943A (zh) 2023-06-23
KR20230043912A (ko) 2023-03-31
TW202220673A (zh) 2022-06-01
JP7476422B2 (ja) 2024-04-30

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