BR112021023488A2 - Oligonucleotídeos de gapmer modificados e métodos de uso - Google Patents

Oligonucleotídeos de gapmer modificados e métodos de uso

Info

Publication number
BR112021023488A2
BR112021023488A2 BR112021023488A BR112021023488A BR112021023488A2 BR 112021023488 A2 BR112021023488 A2 BR 112021023488A2 BR 112021023488 A BR112021023488 A BR 112021023488A BR 112021023488 A BR112021023488 A BR 112021023488A BR 112021023488 A2 BR112021023488 A2 BR 112021023488A2
Authority
BR
Brazil
Prior art keywords
methods
gapmer oligonucleotides
modified
modified gapmer
oligonucleotides
Prior art date
Application number
BR112021023488A
Other languages
English (en)
Portuguese (pt)
Inventor
Bernard Smith David
Jin Hong
Leonid Beigelman
K Pandey Rajendra
Kumar Rajwanshi Vivek
Original Assignee
Aligos Therapeutics 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 Aligos Therapeutics Inc filed Critical Aligos Therapeutics Inc
Publication of BR112021023488A2 publication Critical patent/BR112021023488A2/pt

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • 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
    • CCHEMISTRY; METALLURGY
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    • 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
    • C12N15/1131Non-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 viruses
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/333Modified A
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/336Modified G
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • CCHEMISTRY; METALLURGY
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112021023488A 2019-05-31 2020-05-29 Oligonucleotídeos de gapmer modificados e métodos de uso BR112021023488A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962855793P 2019-05-31 2019-05-31
US201962937760P 2019-11-19 2019-11-19
US201962943532P 2019-12-04 2019-12-04
PCT/US2020/035212 WO2020243490A2 (en) 2019-05-31 2020-05-29 Modified gapmer oligonucleotides and methods of use

Publications (1)

Publication Number Publication Date
BR112021023488A2 true BR112021023488A2 (pt) 2022-01-18

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BR112021023488A BR112021023488A2 (pt) 2019-05-31 2020-05-29 Oligonucleotídeos de gapmer modificados e métodos de uso

Country Status (15)

Country Link
US (1) US11466274B2 (https=)
EP (1) EP3976056A2 (https=)
JP (1) JP2022535735A (https=)
KR (1) KR20220024192A (https=)
CN (1) CN113905744A (https=)
AU (1) AU2020282828A1 (https=)
BR (1) BR112021023488A2 (https=)
CA (1) CA3141874A1 (https=)
CO (1) CO2021018034A2 (https=)
IL (1) IL288259A (https=)
MX (1) MX2021014206A (https=)
PE (1) PE20220574A1 (https=)
SG (1) SG11202112741XA (https=)
TW (1) TW202113079A (https=)
WO (1) WO2020243490A2 (https=)

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AU2021318943A1 (en) * 2020-07-27 2023-03-02 Aligos Therapeutics, Inc. HBV binding oligonucleotides and methods of use
TW202229554A (zh) * 2020-12-31 2022-08-01 中山大學附屬第一醫院 用於降低血管收縮素轉化酶2(ace2)之表現的寡核苷酸及其用於治療病毒感染的用途
US20250243491A1 (en) 2021-09-01 2025-07-31 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
CN118234862A (zh) 2021-09-08 2024-06-21 阿利戈斯治疗公司 经修饰的短干扰核酸分子(siNA)及其用途
TW202340468A (zh) 2022-01-10 2023-10-16 中國大陸商杭州浩博醫藥有限公司 B型肝炎病毒(hbv)表現之調節
KR20240158340A (ko) * 2022-03-17 2024-11-04 알리고스 테라퓨틱스 인코포레이티드 변형 갭머 올리고머 및 이의 사용 방법
WO2024182446A2 (en) 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
US20240352464A1 (en) 2023-03-07 2024-10-24 Aligos Therapeutics, Inc. Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
CN121175419A (zh) * 2023-04-14 2025-12-19 苏州炫景生物科技有限公司 抑制c3基因表达的化合物、药物组合物及其用途
US20250368996A1 (en) 2024-05-14 2025-12-04 Aligos Therapeutics, Inc. Modified antisense oligonucleotides for treating hepatitis b virus
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WO2020243490A3 (en) 2021-01-21
TW202113079A (zh) 2021-04-01
US20200385735A1 (en) 2020-12-10
CA3141874A1 (en) 2020-12-03
KR20220024192A (ko) 2022-03-03
IL288259A (en) 2022-01-01
SG11202112741XA (en) 2021-12-30
MX2021014206A (es) 2022-01-06
AU2020282828A1 (en) 2022-01-27
EP3976056A2 (en) 2022-04-06
JP2022535735A (ja) 2022-08-10
US11466274B2 (en) 2022-10-11
CO2021018034A2 (es) 2022-04-19
CN113905744A (zh) 2022-01-07
PE20220574A1 (es) 2022-04-20
WO2020243490A2 (en) 2020-12-03

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