CN118265785A - 靶向腺苷激酶的反义寡核苷酸 - Google Patents

靶向腺苷激酶的反义寡核苷酸 Download PDF

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Publication number
CN118265785A
CN118265785A CN202280062861.5A CN202280062861A CN118265785A CN 118265785 A CN118265785 A CN 118265785A CN 202280062861 A CN202280062861 A CN 202280062861A CN 118265785 A CN118265785 A CN 118265785A
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CN
China
Prior art keywords
seq
antisense oligonucleotide
adk
disease
lna
Prior art date
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Pending
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CN202280062861.5A
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English (en)
Chinese (zh)
Inventor
马库斯·萨卡里·考皮宁
吕克·佩德森
斯蒂娜·诺曼·汉森
亨里克·瓦尔德马尔·克利特高德
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Neuro Microribonucleic Acid Therapy Co ltd
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Neuro Microribonucleic Acid Therapy Co ltd
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Application filed by Neuro Microribonucleic Acid Therapy Co ltd filed Critical Neuro Microribonucleic Acid Therapy Co ltd
Publication of CN118265785A publication Critical patent/CN118265785A/zh
Pending 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
    • C12N15/1137—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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/0102—Adenosine kinase (2.7.1.20)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pain & Pain Management (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280062861.5A 2021-08-19 2022-08-19 靶向腺苷激酶的反义寡核苷酸 Pending CN118265785A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DKPA202170418 2021-08-19
DKPA202170418 2021-08-19
PCT/EP2022/073188 WO2023021184A1 (en) 2021-08-19 2022-08-19 Antisense oligonucleotides targeting adenosine kinase

Publications (1)

Publication Number Publication Date
CN118265785A true CN118265785A (zh) 2024-06-28

Family

ID=83280116

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280062861.5A Pending CN118265785A (zh) 2021-08-19 2022-08-19 靶向腺苷激酶的反义寡核苷酸

Country Status (7)

Country Link
US (1) US20250136990A1 (https=)
EP (1) EP4388096A1 (https=)
JP (1) JP2024531342A (https=)
CN (1) CN118265785A (https=)
AU (1) AU2022329462A1 (https=)
CA (1) CA3228833A1 (https=)
WO (1) WO2023021184A1 (https=)

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
CN1350542A (zh) 1999-03-18 2002-05-22 埃克西库恩公司 木－lna类似物
JP4768132B2 (ja) 1999-03-24 2011-09-07 エクシコン エ／エス ［２．２．１］ビシクロヌクレオシドの改良された製法
IL148916A0 (en) 1999-10-04 2002-09-12 Exiqon As Design of high affinity rnase h recruiting oligonucleotide
JP4413493B2 (ja) 2000-10-04 2010-02-10 サンタリス ファーマ アー／エス プリンｌｎａ類似体の改善された合成方法
AU2002328792A1 (en) 2001-07-12 2003-01-29 Santaris Pharma A/S Method for preparation of lna phosphoramidites
JP4476802B2 (ja) 2002-05-08 2010-06-09 サンタリス ファーマ アー／エス ロックト核酸誘導体の製造
US7481672B2 (en) 2005-07-21 2009-01-27 Rosemount Tank Radar Ab Dielectric connector, DC-insulating through-connection and electronic system
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
US20070203086A1 (en) * 2006-02-24 2007-08-30 Detlev Boison Adenosine therapy via interfering RNA
CA2648132C (en) 2006-04-03 2019-05-28 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
KR20160083876A (ko) 2013-11-14 2016-07-12 로슈 이노베이션 센터 코펜하겐 에이/에스 ApoB 안티센스 접합체 화합물
JP2024522272A (ja) * 2021-06-04 2024-06-13 ニューミルナ セラピューティクス エーピーエス アデノシンキナーゼを標的とするアンチセンスオリゴヌクレオチド

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Publication number Publication date
CA3228833A1 (en) 2023-02-23
AU2022329462A1 (en) 2024-03-28
WO2023021184A1 (en) 2023-02-23
US20250136990A1 (en) 2025-05-01
EP4388096A1 (en) 2024-06-26
JP2024531342A (ja) 2024-08-29

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