JP2024501288A - 修飾tremの組成物及びその使用 - Google Patents

修飾tremの組成物及びその使用 Download PDF

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JP2024501288A
JP2024501288A JP2023538736A JP2023538736A JP2024501288A JP 2024501288 A JP2024501288 A JP 2024501288A JP 2023538736 A JP2023538736 A JP 2023538736A JP 2023538736 A JP2023538736 A JP 2023538736A JP 2024501288 A JP2024501288 A JP 2024501288A
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trem
binding moiety
certain embodiments
independently
asgpr
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JP2024501288A5 (enExample
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テオニー アナスタシアディス,
デイビッド チャールズ ドネール バトラー,
ニール クビサ,
チンイー リー,
ウェティー, アルマンド ガティアン エンガーヌー
グアンリアン ワン,
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フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー
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Publication of JP2024501288A publication Critical patent/JP2024501288A/ja
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    • CCHEMISTRY; METALLURGY
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    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • 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
    • C12N15/67General methods for enhancing the expression
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2023538736A 2020-12-23 2021-12-23 修飾tremの組成物及びその使用 Pending JP2024501288A (ja)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202063130381P 2020-12-23 2020-12-23
US202063130375P 2020-12-23 2020-12-23
US202063130377P 2020-12-23 2020-12-23
US202063130387P 2020-12-23 2020-12-23
US202063130374P 2020-12-23 2020-12-23
US202063130373P 2020-12-23 2020-12-23
US63/130,373 2020-12-23
US63/130,377 2020-12-23
US63/130,374 2020-12-23
US63/130,381 2020-12-23
US63/130,375 2020-12-23
US63/130,387 2020-12-23
PCT/US2021/065159 WO2022140702A1 (en) 2020-12-23 2021-12-23 Compositions of modified trems and uses thereof

Publications (2)

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JP2024501288A true JP2024501288A (ja) 2024-01-11
JP2024501288A5 JP2024501288A5 (enExample) 2025-01-07

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JP2023538736A Pending JP2024501288A (ja) 2020-12-23 2021-12-23 修飾tremの組成物及びその使用

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US (1) US20240175020A1 (enExample)
EP (1) EP4267732A1 (enExample)
JP (1) JP2024501288A (enExample)
KR (1) KR20230135585A (enExample)
AU (1) AU2021409740A1 (enExample)
BR (1) BR112023012377A2 (enExample)
CA (1) CA3206285A1 (enExample)
IL (1) IL303886A (enExample)
MX (1) MX2023007630A (enExample)
TW (1) TW202242108A (enExample)
WO (1) WO2022140702A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024538097A (ja) * 2021-10-13 2024-10-18 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー Trem組成物及び使用方法
US20250179492A1 (en) * 2022-06-22 2025-06-05 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
EP4695393A1 (en) * 2023-04-12 2026-02-18 Flagship Pioneering Innovations VI, LLC Modified trems, compositions, and related methods thereof
WO2025196508A1 (en) * 2024-03-22 2025-09-25 Takeda Pharmaceutical Company Limited Targeted delivery of therapeutic agents to hepatocytes

Citations (4)

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Publication number Priority date Publication date Assignee Title
WO2006045116A2 (en) * 2004-10-20 2006-04-27 The Scripps Research Institute In vivo site-specific incorporation of n-acetyl-galactosamine amino acids in eubacteria
WO2016055601A1 (en) * 2014-10-10 2016-04-14 F. Hoffmann-La Roche Ag Galnac phosphoramidites, nucleic acid conjugates thereof and their use
WO2019090154A1 (en) * 2017-11-02 2019-05-09 University Of Iowa Research Foundation Methods of rescuing stop codons via genetic reassignment with ace-trna
WO2020243560A1 (en) * 2019-05-31 2020-12-03 Flagship Pioneering, Inc. Uses of trem compositions to modulate trna pools

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